Report to Congressional Requesters

United States General Accounting Office

GAO

September 2001 ORGAN

TRANSPLANTS

Allocation Policies

Include Special

Protections for

Children

GAO-01-498
Page i GAO-01-498 Pediatric Organ Transplants

Letter 1

Results in Brief 2

Background 3

Demand for Organs for Pediatric Patients Continues to Outpace

Supply 6

Most Pediatric Organs Are Transplanted Into Adults 12

Pediatric Patients Generally Fare As Well As or Better Than Adult

Patients on Critical Measures 19

Allocation Policies Aim to Provide Protection for Pediatric Patients 25

Concluding Observations 27

Agency Comments 28

Appendix I Insurance Coverage for Immunosuppressive Drugs for

Children 30

Sources of Payment for Transplants 30

Medicare Coverage for Immunosuppressive Drugs 31

Medicaid Coverage for Immunosuppressive Drugs 32

Private Insurance Coverage for Immunosuppressive Drugs 33

Appendix II Causes of Death Most Likely to Result in Organ

Donation 35

Appendix III Distribution of Kidneys, Livers, and Hearts, 1994

Through 1999 37

Appendix IV GAO Contact and Staff Acknowledgments 39

Tables

Table 1: Mortality Rates in Children by Age for Deaths Most Likely

to Result in Organ Donation, 1989 and 1997 7

Table 2: Organ Recovery from Potential Pediatric Donors at 31

Organ Procurement Organizations, and Reasons for

Nonrecovery, 1997 and 1998 12

Table 3: Causes of Death Most Likely to Result in Organ Donation 36

Table 4: Distribution of Kidneys, 1994 through 1999 37

Contents
Page ii GAO-01-498 Pediatric Organ Transplants

Table 5: Distribution of Livers, 1994 through 1999 37

Table 6: Distribution of Hearts, 1994 through 1999 38

Figures

Figure 1: Pediatric and Adult Cadaveric Donors, 1991 Through 2000 8

Figure 2: Numbers of Pediatric and Adult Patients on Waiting Lists,

1991 Through 2000 10

Figure 3: Distribution of Pediatric Kidneys, Livers, and Hearts by

Age of Recipient, 1994 Through 1999 13

Figure 4: Distribution of Pediatric Kidneys by Age of Donor and

Recipient, 1994 Through 1999 14

Figure 5: Distribution of Pediatric Livers by Age of Donor and

Recipient, 1994 Through 1999 16

Figure 6: Distribution of Pediatric Hearts by Age of Donor and

Recipient, 1994 Through 1999 18

Figure 7: Median Waiting Times by Organ and Age 20

Figure 8: Deaths on the Waiting List, 2000 21

Figure 9: One-Year Survival Rates for Transplants Performed in

1999 23

Figure 10: Five-Year Survival Rates for Transplants Performed in

1994 and 1995 24

Figure 11: Expected Sources of Payment for Pediatric Kidney,

Liver, and Heart Transplants, 1997 Through 1999 31

Abbreviations

AOPO Association of Organ Procurement Organizations

EPSDT Early and Periodic Screening, Diagnostic and Treatment

Program

ESRD end-stage renal disease

FPL federal poverty level

HHS Department of Health and Human Services

HRSA Health Resources and Services Administration

OPTN Organ Procurement and Transplantation Network

UNOS United Network for Organ Sharing
Page 1 GAO-01-498 Pediatric Organ Transplants

September 28, 2001

Congressional Requesters

Organ transplantation can offer individuals with end stage organ disease

the opportunity for a healthy life. Organ transplants can be particularly

beneficial for children with organ failure. However, pediatric organ

transplants involve some special considerations.1

 Children with end stage

organ disease face different medical consequences than adults. Disease

progression can be faster in children, and their physical and mental

development may be affected if they do not receive an organ transplant

early in their illness.

With the scarcity of donor organs, children, as well as adults, may wait a

long time for a transplant or die while waiting. You raised concerns about

the supply of organs and the number of pediatric organs that are allocated

to adults. You also raised concerns about how children in need of an organ

transplant are faring in comparison with adults waiting for a transplant

and whether organ allocation policies sufficiently recognize the unique

needs of children. Specifically, you asked us to determine (1) the trends in

organ donation and demand among adults and children and the factors

that affect pediatric donation, (2) the extent to which pediatric organs are

transplanted into adults, (3) whether pediatric patients are disadvantaged

in terms of waiting times and survival, and (4) how the national organ

allocation policies compare for adult and pediatric patients.

In conducting this study, we interviewed officials and obtained documents

from the Department of Health and Human Services’ (HHS) Health

Resources and Services Administration (HRSA), which regulates and

provides oversight of the Organ Procurement and Transplantation

Network (OPTN). We also interviewed officials and obtained documents

on organ allocation policies and data on donation, transplant, survival, and

waiting times from the United Network for Organ Sharing (UNOS), the

organization that coordinates the OPTN for HHS. The OPTN develops

policies for organ allocation, maintains the waiting list, and tracks the

OPTN data on each patient. We also interviewed officials from the Health

Care Financing Administration (now the Centers for Medicare and

Medicaid Services) about coverage criteria for organ transplants and

 1

The pediatric population includes children aged 17 and younger.

United States General Accounting Office

Washington, DC 20548
Page 2 GAO-01-498 Pediatric Organ Transplants

medication therapy because of Medicare coverage for disabled children

and children with end stage renal disease. Additionally, we visited six

transplant centers that represented different geographic locations and

perform a high volume of pediatric organ transplants. At these centers, we

interviewed medical and other transplant services personnel and obtained

relevant documents about donation and allocation issues, survival

measures, and coverage for medication therapy. Representatives from the

local organ procurement organization attended meetings we held at four

centers, and we separately interviewed officials from two other organ

procurement organizations. We also obtained and analyzed data on the

causes of death most likely to result in organ donation from the Centers

for Disease Control and Prevention’s mortality and population database.

Because the overwhelming majority of the organs used in transplant

operations come from cadaveric donors, the discussion on donor organs in

this report is limited to cadaveric organs. We conducted our work from

March 2000 through September 2001 in accordance with generally

accepted government auditing standards.

Pediatric patients in need of an organ transplant continue to face a

shortage of donated organs. From 1991 through 2000, the number of

pediatric organ donors each year has remained relatively constant, despite

a drop in the number of potential donors. The number of adult donors has

increased 45 percent during the same period, in large part because donor

eligibility criteria have been expanded to include older donors and donors

with certain diseases that were not accepted in the past. Simultaneously,

the demand for organs for pediatric patients has grown substantially, with

the number of children on waiting lists for organ transplants more than

doubling. However, compared to adults, children account for a small

number of transplant candidates. Several factors can prevent the recovery

of organs from a potential donor, such as refusal by the family to give

consent for donation, failure by health professionals to identify potential

donors or approach families, and refusal by medical examiners and

coroners to release the body. Nonetheless, organs are recovered from a

higher proportion of potential pediatric donors than potential adult

donors.

Most pediatric organs are transplanted into adults because adults make up

the vast majority of patients waiting for an organ transplant and therefore

are more likely to be at a higher status on local organ waiting lists than

children. The degree to which pediatric organs are transplanted into adults

varies by organ. In particular, adult patients receive more pediatric

kidneys than pediatric patients do, partly because of the importance of

Results in Brief
Page 3 GAO-01-498 Pediatric Organ Transplants

tissue-type matching criteria in the allocation of kidneys. The picture

differs for livers and hearts, where organs from children aged 11 to 17 are

often transplanted into adults, because of size considerations, but organs

from children under 10 are usually transplanted into pediatric patients.

Adult organs are also transplanted into children, but in much smaller

numbers because of size and matching criteria.

Pediatric patients appear to be faring as well as or better than adult

patients, both while on the waiting list and after transplantation. On key

measures such as time on the waiting list, deaths while waiting for a

transplant, and post-transplant survival, children do as well as or better

than adults, with some exceptions for very young patients and heart

transplant patients. Pediatric patients generally wait fewer days on

average than adults for transplants, have lower death rates on the waiting

list, and have equivalent or better rates for 1- and 5-year post-transplant

survival.

Allocation policies for kidneys, livers, and hearts provide a number of

protections for children awaiting transplants. The organ transplant

community has recognized the distinctive needs of children waiting for a

transplant, and the OPTN has revised organ allocation policies over the

past decade to consider the pediatric patient. The priority a child receives

takes into account differences between children and adults in the

progression and treatment of end stage organ disease, with the policies

differing for each organ. For example, the kidney policy strives to reduce

waiting time for pediatric patients, in part because of the difficulties they

experience with dialysis, and pediatric liver patients now receive priority

for livers from pediatric donors because research has shown that pediatric

livers will improve pediatric patients’ chances of survival after transplant.

HHS and UNOS provided technical comments on a draft of the report

which we incorporated, where appropriate.

Transplants are performed for organs such as kidney, liver, heart,

intestine, pancreas, heart-lung, and kidney-pancreas. However, the kidney,

liver, and heart are the most commonly transplanted organs. In 2000,

doctors performed 13,333 kidney, 4,950 liver, and 2,197 heart transplants.2

 2

Approximately 39 percent of the kidney transplants and 7 percent of the liver transplants

were from living donors.

Background
Page 4 GAO-01-498 Pediatric Organ Transplants

Of these, children made up 617 of the kidney recipients, 569 of the liver

recipients, and 274 of the heart recipients. Organ transplants were

performed at 261 centers, which had one or more specific organ transplant

programs, in 1998. Some of these centers accept both adults and children,

and others are for children only.3

 In 1998, pediatric kidney transplants

were performed at 129 of the 241 centers that performed kidney

transplants; pediatric liver transplants were performed at 77 of the 116

centers that transplanted livers; and pediatric heart transplants were

performed at 54 of the 134 centers that transplanted hearts.

In 1984, Congress enacted the National Organ Transplant Act (P.L. 98-507),

which requires HHS to establish the OPTN. In 1986, HHS awarded the

OPTN contract to UNOS, which operates the network under HRSA’s

oversight. The OPTN develops national transplantation policy, maintains

the list of patients waiting for transplants, and fosters efforts to increase

the nation’s organ supply.4

 OPTN members include all transplant centers,

organ procurement organizations, and tissue-typing laboratories.

Only a small fraction of those who die are considered for organ donation.

Most cadaveric organs derive from donors who have been pronounced

brain-dead as a result of a motor vehicle collision, stroke, violence,

suicide, or severe head injury.

When an organ becomes available, staff from the local organ procurement

organization typically identify potential recipients from the OPTN

computerized waiting list. Patients are ranked on the OPTN waiting list

according to points assigned on the basis of time waiting, medical

urgency,5

 organ size, and the quality of the tissue-type match between the

donor and the potential recipient, as determined by antigen matching.6

 The

criteria that determine the order of candidates on the list are applied or

 3

No count is maintained of the number of programs that are specifically for pediatric

patients.

4

HHS issued final regulations governing the operation of the OPTN, which became effective

on March 16, 2000. Because of concern over geographic disparities in organ allocation, the

Final Rule authorizes continuous evaluation and revision of organ allocation policies to

achieve an equitable national allocation system.

5

Medical urgency is measured differently for different organs and may include such factors

as life expectancy and intensity of current treatment.

6

Tissue type is determined by identification of six human-leukocyte-associated antigens.

Organ Donation and

Allocation
Page 5 GAO-01-498 Pediatric Organ Transplants

defined differently for each type of organ and for pediatric versus adult

patients. With certain limitations, organs from pediatric donors can be

transplanted into adults, and vice versa. The UNOS computer matches

each patient in the OPTN database against a donor’s characteristics and

then generates a different ranked list of potential recipients for each

transplantable organ from the donor. Organs are generally allocated first

to patients waiting in the local organ procurement organization’s service

area, with priority based on a patient’s severity of illness. If a matching

recipient is not found locally, the organ is offered regionally and then

nationally.7

 Organ allocation policies are revised from time to time to

reflect advancements in medical science and technology.

Title XXI of the Children’s Health Act of 2000 (P.L. 106-310, October 17,

2000) requires the OPTN to recognize the differences in organ

transplantation needs between children and adults and adopt criteria,

policies, and procedures that address the unique health care needs of

children. In addition, the OPTN is to carry out studies and demonstration

projects for improving procedures for organ procurement and allocation,

including projects to examine and to increase transplantation among

populations with special needs, such as children and racial or ethnic

minority groups. Finally, the act requires the Secretary of HHS to conduct

a study and make recommendations regarding the (1) special growth and

developmental issues that children have before and after transplant; (2)

extent of denials by medical examiners and coroners to allow donation of

organs; (3) other special health and transplantation needs of children; and

(4) costs of the immunosuppressive drugs that children must take after

receiving a transplant and the extent of their coverage by health plans and

insurers. (For a discussion of children’s access to these necessary

medications, see app. I.) The Secretary must report to the Congress by

December 31, 2001.

 7

The OPTN has divided the country into 11 regions for allocating organs. Under certain

circumstances, regional or national sharing may occur from the outset when medically

appropriate or when interregional sharing arrangements exist. For example, regional

sharing is allowed for the highest-priority liver patients.

Children’s Health Act of

2000
Page 6 GAO-01-498 Pediatric Organ Transplants

Pediatric patients in need of an organ transplant continue to face a

shortage of donated organs. From 1991 through 2000, the number of

pediatric organ donors each year has remained relatively constant, even

though the number of potential pediatric donors decreased. The number of

adult donors has increased significantly during the same period, in large

part because donor eligibility criteria have been expanded to include older

donors and donors with certain diseases that were not accepted in the

past. Simultaneously, the demand for organs for pediatric patients has

grown substantially, with the number of children on waiting lists for organ

transplants more than doubling. However, compared to adults, children

account for a small number of transplant candidates. Several factors can

prevent the recovery of organs from a potential donor. Refusal by the

family to give consent for donation is the primary reason for nonrecovery

of an organ, but failure by health professionals to identify potential donors

or approach families and refusal by medical examiners and coroners to

release the body also account for significant losses of transplantable

organs. Nonetheless, organs are recovered from a higher proportion of

potential pediatric donors than potential adult donors.

The number of pediatric donors has held relatively steady despite a drop

in the number of potential donors. Our analysis of 1989 through 1997

mortality data for children showed a 20-percent decline in deaths of the

kinds that are most likely to result in organ donation, such as those

resulting from head trauma, motor vehicle collisions, and violence. (See

app. II for a complete list of these causes of death.) Mortality for potential

donors up to age 19 years declined from 24,069 deaths in 1989 to 19,327 in

1997, the latest data available at the time of our analysis (see table 1).

Demand for Organs

for Pediatric Patients

Continues to Outpace

Supply

Number of Pediatric Organ

Donors Has Remained

Relatively Constant
Page 7 GAO-01-498 Pediatric Organ Transplants

Table 1: Mortality Rates in Children by Age for Deaths Most Likely to Result in

Organ Donation, 1989 and 1997

1989 1997

Age, years

Number of

deaths Mortality ratea

Number of

deaths Mortality ratea

Under 1 6,604 163 3,958 102

1-4 2,032 14 1,566 10

5-9 1,712 10 1,328 7

10-14 2,242 13 2,143 11

15-19 11,479 63 10,332 54

Total deaths 24,069 19,327

a

The Centers for Disease Control and Prevention calculates death rates for infants (aged less than 1

year) as the number of deaths per 100,000 live births. For other ages, the mortality rate is based on

the number of deaths per 100,000 population.

Source: Centers for Disease Control and Prevention, “Compressed Mortality File, CDC Wonder on

the Web” (http://wonder.cdc.gov), Apr. 13, 2000.

OPTN data show that from 1991 through 2000, while the number of

pediatric donors remained relatively constant, the number of adult donors

increased 45 percent (see fig. 1).8

 The large increase in the number of adult

donors is primarily due to changes in the criteria for accepting organs

from a donor. At one time, organs were accepted only from someone who

had been declared brain-dead and was relatively young and free from

diseases that could affect organ quality. However, because of the

continuing shortage of transplantable organs, transplant professionals

have gradually expanded the criteria for acceptable organs. Older

individuals and persons with certain medical conditions who previously

would have been excluded from donating organs can now be donors.9

From 1991 through 2000, the number of cadaveric donors aged 50 to 64

increased 108 percent, and the number of cadaveric donors aged 65 or

older increased 272 percent.

 8

Throughout this report, we use OPTN data that were current as of the date cited. These

data are subject to change based on future data submissions or corrections.

9

Individuals not routinely considered for organ donation include diabetics and those with

systemic infections or abnormal organ functions.
Page 8 GAO-01-498 Pediatric Organ Transplants

Figure 1: Pediatric and Adult Cadaveric Donors, 1991 Through 2000

Source: OPTN, June 4, 2001.

The number of children waiting for a transplant has increased over time,

but not as much as for adults (see fig. 2). OPTN data show that the number

of pediatric patients awaiting transplants increased from 1,010 in 1991 to

2,299 in 2000, a 128-percent increase. The number of adults on the waiting

list has increased even faster, from 23,709 in 1991 to 77,047 in 2000, a 225-

percent increase. These increases have been spurred by advances in

Demand for Pediatric

Transplants Has More

Than Doubled Over the

Past Decade

Adults aged 65 years or older

Adults aged 18-64 years

Children aged 17 years or younger

1991 1992 1993 1994 1995 1996 1997 1998 1999 2000

Number of donors

0

1000

2000

3000

4000

5000

6000

4526 4520

5984

 4861

5100

 5361 5418 5478

5798 5822
Page 9 GAO-01-498 Pediatric Organ Transplants

medical science and technology, which have made transplantation a more

acceptable medical procedure; improvements in immunosuppressive

medications, which have increased survival rates; and an increase in the

incidence of certain diseases that lead to end stage organ failure. Despite

these increases, the proportion of patients awaiting transplant who are

children has remained fairly constant from 1991 through 2000, at between

3 and 4 percent overall.
Page 10 GAO-01-498 Pediatric Organ Transplants

Figure 2: Numbers of Pediatric and Adult Patients on Waiting Lists, 1991 Through 2000

Source: OPTN, June 4, 2001.

Number on waiting list

77,047

1,010

23,709

 1,206

28,209

 1,338

32,056

 1,409

 36,275

 1,589

 42,348

1,744

48,386

1,889

54,817

2,122

 62,301

2,159

 69,951

 2,299

1991 1992 1993 1994 1995 1996 1997 1998 1999 2000

Adults

Children aged 17 years or younger

0

10,000

20,000

30,000

40,000

50,000

60,000

70,000

80,000
Page 11 GAO-01-498 Pediatric Organ Transplants

Several factors can prevent the recovery of organs from potential pediatric

and adult donors and thus contribute to the continuing shortage of

transplantable organs for both children and adults. For example, for many

potential donors, families refuse to give consent for organ donation. For

others, health care professionals may fail to offer the families the

opportunity to donate.10 Further, some medical examiners and coroners

believe that the need to preserve forensic evidence in certain types of

cases, such as suspected child abuse and sudden infant death syndrome,

makes it impossible for them to allow organ donation to proceed.

The Association of Organ Procurement Organizations (AOPO) recently

conducted a study at 31 organ procurement organizations on the reasons

why potential adult and pediatric donors do not become organ donors.

The study found that consent was not given for 39 percent of potential

donors and only 41 percent of suitable individuals actually become organ

donors.11

AOPO provided us with the survey data from the referral, request, and

organ recovery processes for the pediatric patients. As our analysis shows

in table 2, of the 2,420 potential pediatric donors, organs were recovered in

1,230 cases, or about 51 percent of pediatric cases, a rate higher than the

overall donation rate. Family refusal (25 percent) was the most common

obstacle to organ recovery, but this occurred less frequently for potential

pediatric donors than for the entire group of potential donors.

 10Centers for Medicare and Medicaid Services regulations require hospitals that participate

in Medicare and Medicaid to refer all deaths and imminent deaths to organ procurement

organizations as potential donors.

11S. Conrad, L. Brigham, E. Sheehy, Association of Organ Procurement Organizations

(AOPO) Death Record Review Study. Paper presented at AOPO Briefing of Federal

Officials; Nov. 15, 2000, Rockville, Md.

Several Factors Can Inhibit

Both Pediatric and Adult

Organ Donation
Page 12 GAO-01-498 Pediatric Organ Transplants

Table 2: Organ Recovery from Potential Pediatric Donors at 31 Organ Procurement

Organizations, and Reasons for Nonrecovery, 1997 and 1998

Number Percentage of total

Total potential pediatric donors 2,420

Recovery of at least one organ 1,230 51

No organ recovery 1,190 49

Reason for nonrecoverya

No referral to organ procurement organization 291b 12

Referred to organ procurement organization

but family not approached

153c 6

Referred to organ procurement organization

but family refused

597 25

Consent obtained but organs not recovered 136d6

a

Numbers do not total to 1,190 because of missing information for 13 records.

b

Hospital staff ruled out 123 cases; families denied consent in 97 cases; medical examiners or

coroners refused referral in 14 cases; and 57 cases were not referred for other reasons.

c

The potential donor was determined to be medically unsuitable in 60 cases; medical examiner and

coroner refusals occurred in 38 cases; cardiac arrest occurred before the request could be made in

18 cases; no placement could be made for the organ prior to the request in 5 cases; and the request

was not made for other reasons in 32 cases.

d

The organs were determined to be medically unsuitable in 47 cases; cardiac arrest occurred prior to

recovery in 36 cases; unsuccessful placement occurred in 21 cases; medical examiner or coroner

denials occurred in 18 cases; and the organs were not recovered for other reasons in 14 cases.

Source: GAO analysis of data from the AOPO Death Record Review Project.

Most pediatric organs are transplanted into adults because adults make up

the vast majority of patients waiting for an organ transplant and therefore

are more likely to be at a higher status on local organ waiting lists than

children. However, the degree to which pediatric organs are transplanted

into adults varies by organ. In particular, adult patients receive more

pediatric kidneys than pediatric patients do, partly because of the

importance of tissue-type matching criteria in the allocation of kidneys. 12

While most pediatric kidneys are transplanted into adults, adult kidneys

are sometimes transplanted into children. The situation is different for

livers and hearts, where organ size is an important determinant of

suitability. Livers and hearts from children under 10 are usually

transplanted into pediatric patients, whereas those from children aged 11

 12Although first priority is given to patients for whom a donor kidney is a perfect match,

antigen-matching criteria are becoming less important because of improvements in

immunosuppressive therapies.

Most Pediatric Organs

Are Transplanted Into

Adults
Page 13 GAO-01-498 Pediatric Organ Transplants

to 17 are usually transplanted into adults. Figure 3 shows the distribution

of pediatric kidneys, livers, and hearts to pediatric and adult recipients.

(See app. III for a detailed listing of the distribution of kidneys, livers, and

hearts by age of donor and recipient.) Pediatric livers and hearts that are

given to adults have sometimes been refused beforehand for a pediatric

patient by the patient’s physician for various medical or logistical

reasons.13 Adult organs are also transplanted into children, but in much

smaller numbers.

Figure 3: Distribution of Pediatric Kidneys, Livers, and Hearts by Age of Recipient, 1994 Through 1999

Source: OPTN, June 17, 2000.

Although the majority of pediatric kidneys are transplanted into adults,

some adult kidneys are transplanted into children. From 1994 through

1999, adult donors provided 81 percent of the kidneys procured and

pediatric donors provided 19 percent (see table 4 in app. III). Of the adult

kidneys, 4 percent were transplanted into children. Of the pediatric

kidneys, 93 percent were transplanted into adults. Figure 4 shows the

 13Comparable refusal data are not available for kidney transplants.

Most Pediatric Kidneys Are

Transplanted Into Adults,

While Most Pediatric

Patients Receive Kidneys

From Adults

93

 7

63

37

Percentage into adults

Percentage into children

Kidneys Livers Hearts

Percentage

61

39
Page 14 GAO-01-498 Pediatric Organ Transplants

distribution of pediatric kidneys by age of donor and recipient. During that

period, 32 percent of the kidneys given to pediatric recipients came from

children, and 68 percent came from adults.

Figure 4: Distribution of Pediatric Kidneys by Age of Donor and Recipient, 1994 Through 1999

Note: Percentages may exceed 100 due to rounding.

Source: OPTN, June 17, 2000.

Kidneys from pediatric donors are most often transplanted into adults

because children make up only a small portion of the kidney waiting list14

and because of the importance of antigen matching as a ranking factor for

this organ. Also, the matching criteria for kidneys generally do not include

the size (weight and height) of the donor and recipient. When kidneys

 14At the end of 1998, 648 children were on the waiting list, compared to 41,744 adults.

2 2 3

94

Age of donor

0-5

Age of donor

6-10

Age of donor

11-17

1 2 3

94

2 2 6

90

Age of recipient (years)

0-5

6-10

11-17

18+

Percentage
Page 15 GAO-01-498 Pediatric Organ Transplants

from small children are given to adults, they are typically transplanted en

bloc, meaning that both kidneys are transplanted into the recipient.

Transplant center representatives told us that adult kidneys are often

preferred for children because of the larger kidney mass. If complications

occur, the larger kidney is more apt to continue functioning than a small,

pediatric kidney.

For liver transplants, the sizes of the donor and the recipient are factors

that are considered to obtain an organ of compatible size. From 1994

through 1999, adult donors provided 78 percent of the livers procured and

pediatric donors provided 22 percent (see table 5 in app. III). Of the adult

livers, 4 percent were transplanted into children. Of the pediatric livers, 63

percent were transplanted into adults, but this varied greatly by age of the

donor. Most livers (81 percent) from donors aged 5 years or younger went

to recipients in the same age group, and 4 percent went to adults. For the

6- to 10-year-old donors, 47 percent of the livers went to adult recipients,

and for the 11- to 17-year-old donors, 89 percent of the livers went to adult

recipients. Figure 5 shows the distribution of pediatric livers by age of

donor and recipient from 1994 through 1999. During that period, 72

percent of the livers given to pediatric recipients came from children, and

28 percent came from adults.

Liver Transplants Present a

More Varied Picture, but

Most Children Receive

Pediatric Livers
Page 16 GAO-01-498 Pediatric Organ Transplants

Figure 5: Distribution of Pediatric Livers by Age of Donor and Recipient, 1994 Through 1999

Note: Percentages may exceed 100 due to rounding.

Source: OPTN, June 17, 2000.

Unlike kidneys and hearts, livers can be reduced in size or split to

accommodate the size of the recipient. A reduced-size liver from an adult

donor can be transplanted into a pediatric patient. A split liver can yield a

portion for an adult and a portion for a child.15 However, the number of

livers that are either reduced or split is small. From 1994 through 1999,

fewer than 2 percent of donor livers were reduced for transplantation, and

 15The liver consists of a right and left lobe. A reduced-size liver graft consists of the left

lobe of an adult liver, with the right lobe discarded. In split-liver transplantation, the left

lobe of an adult liver is transplanted into a child, and the right lobe is transplanted into an

adult.

4

11

4

81

Age of donor

0-5

Age of donor

6-10

Age of donor

11-17

5

2

4

89

25

15

13

47

Age of recipient (years)

0-5

6-10

11-17

18+

Percentage
Page 17 GAO-01-498 Pediatric Organ Transplants

about 1 percent were split for transplantation. Although using reduced or

split livers can provide a needed transplant for children, initial studies

found that survival rates were lower for pediatric recipients of these types

of liver transplants.16 However, a recent OPTN analysis of 1997-99

transplants has shown similar 1-year survival rates for whole and split-liver

transplants.

Sometimes an organ from a pediatric donor is transplanted into an adult

even though there is a higher-ranking pediatric patient waiting. This only

occurs if the transplant center refuses the organ for the higher-ranked

patient. According to OPTN data, 1,122 liver transplants occurred during

1997 and 1998 in which an adult recipient received a pediatric organ. Of

these, 222 livers were each refused for at least one potential pediatric

recipient who was ranked higher on the waiting list than the adult

recipient. The most common reasons for refusing the pediatric liver for a

pediatric patient involved administrative reasons (e.g., medical judgment,

transportation, logistics, and distance concerns) (33 percent), donor size

and/or weight (26 percent), and poor donor quality (18 percent).17

From 1994 through 1999, adult donors provided 75 percent of the hearts

procured and pediatric donors provided 25 percent (see table 6 in app. III).

Of the adult hearts, 3 percent were transplanted into children. Of the

pediatric hearts, 39 percent were transplanted into adults, but this varied

greatly by age of donor. For heart transplants, organ size is critically

important both to proper functioning and to proper fit into the chest

cavity. Hearts from small children, aged 5 years or younger, are therefore

likely to be transplanted into children of the same age group. Of the hearts

recovered from donors aged 5 years and younger, 93 percent were

transplanted into recipients in the same age group, and 1 percent went to

adults. During the same period, adults received about 24 percent of the

hearts from donors aged 6 through 10 years, and 89 percent from donors

aged 11 through 17 years. Figure 6 shows the distribution of pediatric

hearts by age of donor and recipient. During that period, 83 percent of the

 16For the period 1990 through 1996, the rate of graft survival for children at 1 year for

whole-liver grafts was 70.9 percent, whereas the rates for reduced-liver grafts and split-liver

grafts were 61.1 and 60.3 percent, respectively (N. Scott Adzick and Michael L. Nance,

“Pediatric Surgery, Second of Two Parts,” New England Journal of Medicine, Vol. 342, No.

23 (2000), pp. 1726-32).

17Poor donor quality can result from problems with a donor’s medical condition, such as

hypertension, cardiac arrest, evidence of infection, or diabetes.

Heart Transplants Are

Highly Dependent on

Organ Size
Page 18 GAO-01-498 Pediatric Organ Transplants

hearts given to pediatric recipients came from children, and 17 percent

came from adults.

Figure 6: Distribution of Pediatric Hearts by Age of Donor and Recipient, 1994 Through 1999

Note: Percentages may exceed 100 due to rounding.

Source: OPTN, June 17, 2000.

OPTN data indicate that 664 heart transplants occurred during 1997 and

1998 in which a pediatric organ was transplanted into an adult. Of these,

75 hearts were each refused for at least one pediatric patient who was

ranked higher on the waiting list than the adult recipient. In these

instances, the most common refusal reasons were donor quality (17

percent), donor size and/or weight (17 percent), administrative reasons (14

percent), and abnormal echocardiogram (14 percent).

1 1

5

93

Age of donor

0-5

Age of donor

6-10

Age of donor

11-17

1 2

8

89

Age of recipient (years)

0-5

6-10

11-17

18+

Percentage

19

22 35

24
Page 19 GAO-01-498 Pediatric Organ Transplants

Although the patterns vary by organ and present a complex picture,

pediatric patients appear to be faring as well as or better than adult

patients, both while on the waiting list and after transplantation. Data from

the OPTN and HHS on four key measures—time on the waiting list, deaths

while waiting for a transplant, and 1- and 5-year post-transplant survival—

show that children appear to fare as well as or better than adults, with

some exceptions for very young patients and heart transplant patients.

Other measures of importance for pediatric patients, such as growth and

development, are not routinely part of the current OPTN data collection.

Pediatric patients wait fewer days on average than adults for transplants.

With the exception of infants under 1 year of age and heart transplant

patients, death rates for pediatric patients on the waiting list are lower

than those for adults. Again with the exception of infants under 1 year old,

post-transplant survival rates for children generally appear to be

equivalent to or better than those for adult patients at the 1- and 5-year

post-transplant points. However, because the number of pediatric patients

is small, variation across time by even a few pediatric patients on any of

these measures could result in relatively large changes in the percentages.

We report on the most current data available.

In general, pediatric patients wait fewer days than adults for transplants

(see fig. 7). Adults are likely to wait about twice as long as children for a

kidney transplant. For patients added to the waiting list for a transplant in

1997, the median waiting time for pediatric kidney recipients ranged from

389 days for 6- to 10-year-olds to 548 days for 11- to 17-year-olds, while for

adults the range was from 1,044 days for 18- to 34-year-olds to 1,150 days

for 50- to 64-year-olds. For livers and hearts, the median waiting time for

adult candidates was two to three times as long as it was for children. For

livers, median waiting times for patients added to the waiting list in 1999

ranged across age subgroups from 182 to 318 days for children through

age 10. For children aged 11 to 17 years, however, the waiting time was

similar to waiting times for adults. Candidates aged 11 through 17 years

waited 746 days, whereas adult waiting times ranged across age subgroups

from 636 to 795 days. Across all age groups, waiting times for hearts were

much shorter than they were for kidneys and livers because survival is

lower without a transplant. Among heart transplant candidates added to

the waiting list in 2000, median waiting times for children ranged from 52

to 86 days and for adults from 137 to 242 days across the different age

subgroups.

Pediatric Patients

Generally Fare As

Well As or Better

Than Adult Patients

on Critical Measures

Pediatric Patients

Generally Spend Less Time

on the Waiting List Than

Adults
Page 20 GAO-01-498 Pediatric Organ Transplants

Figure 7: Median Waiting Times by Organ and Age

a

Median waiting time for kidney patients under 1 year of age was not calculated owing to the small

number of patients.

Note: Data are for patients added to the waiting list for a transplant for a kidney in 1997, a liver in

1999, or a heart in 2000.

Source: OPTN, June 8, 2001.

The death rates for pediatric patients on the waiting list vary considerably

by organ, with pediatric patients having slightly lower rates than adults for

kidneys and livers, but higher rates than adults for hearts (see fig. 8). In

Pediatric Deaths While

Waiting Vary by Organ

0

100

200

300

400

500

600

700

800

900

1,000

1,100

1,200

Median waiting time (days) by age group

Kidney (1997) Liver (1999) Heart (2000) a

<1

1-5

6-10

11-17

18-34

35-40

50-64

65+

496

 389

548

 1044

 1103

 1150 1136

182

 205

318

 746

 636

758 744

795

52

86

60 67

137

222

 242

184

Age group (years)
Page 21 GAO-01-498 Pediatric Organ Transplants

2000, death rates for children waiting for a kidney transplant ranged from

0 to 92 per 1,000 patient risk years (i.e., years on the waiting list), whereas

for adults they ranged from 36 to 104. Infants under 1 year old who were

awaiting liver or heart transplants had considerably higher death rates

than other pediatric or adult age groups; however, pediatric patients aged

1 year or older waiting for a liver transplant had lower death rates than

adults. For patients waiting for a heart transplant, pediatric patients of all

age groups had higher death rates than did adults.

Figure 8: Deaths on the Waiting List, 2000

a

Patient-years at risk is calculated as the number of deaths for every 1,000 patient years on the

waiting list, based on the actual time waiting for each patient listed.

b

Of 16 kidney patients under age 1, none died while on the waiting list.

Source: OPTN, June 8, 2001.

0

50

100

150

200

250

300

350

400

450

Annual death rate (per 1,000 patient risk yearsa)

Age group (years)

Kidney Liver Heart 

<1

1-5

6-10

11-17

18-34

35-40

50-64

65+

0b

 92.2

52.8

22

35.5

60.2

171.2

71.4 67.5

54

102.8 94.1

441.1

292.2

201.2 201.6

166.6

107.8

141.3

180

103.7

78.9

119.6

147
Page 22 GAO-01-498 Pediatric Organ Transplants

With the exception of infants under 1 year old, post-transplant survival

rates (i.e., the percentage of patients alive at 1 and 5 years after transplant)

for children generally appear to be as good as or better than those for

adults (see figs. 9 and 10). In general, 1-year survival rates vary more by

type of organ than they do by age group, with kidney transplant recipients

having the highest survival rates and heart transplant patients having the

lowest survival rates. Overall, survival rates for children at 5 years after

transplant are better than adult survival for kidneys and livers. Children 5

years old and younger have lower 5-year survival rates for heart

transplants.

Pediatric Survival After

Transplant Is Generally As

Good As or Better Than

That for Adults
Page 23 GAO-01-498 Pediatric Organ Transplants

Figure 9: One-Year Survival Rates for Transplants Performed in 1999

a

Survival rates for infants under 1 year of age for kidney transplants could not be calculated because

of an insufficient number of patients.

Source: OPTN, June 8, 2001.

Kidneya Liver Heart

0

20

40

60

80

100

<1

1-5

6-10

11-17

18-34

35-40

50-64

65+

Age group (years)

Percentage of patients alive 1 year after transplant

82.7

94.9

92

97.9 97.6 96.7

93.1

88.6

85 86.1 87.5 88.4

92.5

88.8

84.9

77.6

81.5 81.5

90.5

84.2 82.8

89

82.9
Page 24 GAO-01-498 Pediatric Organ Transplants

Figure 10: Five-Year Survival Rates for Transplants Performed in 1994 and 1995

a

Survival rates for infants under 1 year of age for kidney transplants could not be calculated because

of an insufficient number of patients.

Source: OPTN, June 16, 2001.

0

20

40

60

80

100

<1

1-5

6-10

11-17

18-34

35-40

50-64

65+

Age group (years)

Kidneya Liver (1995) Heart

 91.7

97

93.3 92.2

 86.1

76.1

61.5

84.7

79.3

93

87.3

82.1

78.5

 73.6

67.7

53.7

57.7

67.7

70.6 72.1 72.3 71.9 70.8

Percentage of patients alive 5 years after transplant
Page 25 GAO-01-498 Pediatric Organ Transplants

Organ allocation policies provide a number of protections for children

awaiting transplants. The organ transplant community has recognized the

distinctive needs of children waiting for a transplant, and the OPTN has

revised organ allocation policies over time to consider the pediatric

patient. The priority a child receives takes into account differences

between children and adults in the progression and treatment of end stage

organ disease. Prolonged waiting times can be more harmful for children

than for adults because disease progression in children can be faster and

their growth and development can be compromised without timely

transplantation. The policies differ for each organ. For example, waiting

time requirements for kidney transplants are less stringent for pediatric

patients than for adult patients because of the unique problems children

experience with end stage renal disease, including difficulties with

dialysis. For livers, research showing better survival for pediatric patients

who received a pediatric liver led to a policy change giving priority for

pediatric livers to pediatric patients. For hearts, medical urgency status is

determined differently for pediatric patients because pretransplant

treatments appropriate for adults, such as heart assist devices, cannot

always be used for children who are waiting for transplants.

Current kidney allocation policy provides several protections for pediatric

kidney patients because of the unique problems they experience in

association with end stage renal disease. These problems include dialysis

difficulties and disruption of growth and development due to renal failure.

Early transplantation can avoid or ameliorate many of the effects of end

stage renal disease experienced by pediatric patients.

One advantage the allocation policy gives to pediatric patients concerns

waiting time, one factor in determining priority for obtaining a transplant.

Waiting time for children is measured from when they are placed on the

waiting list, whereas, since changes to the adult kidney allocation policy in

January 1998, waiting time for adults begins when they reach a certain

stage of disease. Therefore pediatric patients can begin moving up in

priority on the waiting list at an earlier point in their disease progression

than can adult patients.

In addition, pediatric patients receive higher priority for kidney allocation

at the time of listing and until they reach 18 years of age, based on their

age at listing. The criteria for granting this priority were first implemented

by the OPTN in 1990 and have been altered several times, most recently in

November 1998. Kidney transplant candidates less than 11 years of age at

Allocation Policies

Aim to Provide

Protection for

Pediatric Patients

Kidney Allocation Policy

Geared to Reduce Waiting

Time for Pediatric Patients
Page 26 GAO-01-498 Pediatric Organ Transplants

listing are assigned four additional points, and candidates aged 11 through

17 years are assigned three additional points.18

Another advantage was introduced by the OPTN in November 1998. It

provides that patients who are less than 18 years old at listing, and have

not received a transplant within a specified amount of time, must be the

first in line to receive available kidneys, except for those that must be

allocated to a patient with a perfect antigen match, to a patient needing a

kidney plus a nonrenal organ, or to a patient whose immune system makes

it difficult to receive organs. These specified times are within 6 months of

listing for candidates up to and including 5 years of age, 12 months for

those from 6 to 10 years, and 18 months for those from 11 to 17 years.

The liver allocation policy for pediatric patients has been revised several

times since 1994 to address conditions and challenges unique to pediatric

patients. Children with chronic liver disease may deteriorate rapidly and

unpredictably. Their growth and development may also be affected. The

policy revisions redefine medical urgency criteria, focus on disease

progression in children, and recognize factors distinctive to pediatric liver

candidates.

In June 2000, the OPTN approved a policy to give pediatric liver transplant

patients preference over adult patients for livers from pediatric donors.

Prior to the implementation of this change, the age of the donor was not a

factor. Now, a pediatric liver is offered to a pediatric patient before an

adult patient with the same medical urgency within the same organ

distribution area. If no local matches occur in a given medical urgency

category, the pediatric liver will be offered to a pediatric patient before an

adult patient with the same medical urgency at the regional level. This

change was made in response to the finding that pediatric liver transplant

recipients have higher survival rates and better graft survival19 if they are

transplanted with a pediatric liver rather than an adult liver. A study

showed that pediatric patients receiving livers from pediatric donors

during 1992 through 1997 had a 3-year graft survival rate of 81 percent,

 18According to current allocation policies, patients are ranked on the OPTN waiting list

according to points assigned on the basis of time waiting and medical urgency.

19Graft survival refers to the length of time the transplanted organ continues to function.

Liver Allocation Policy

Seeks to Provide Pediatric

Patients With Pediatric

Organs
Page 27 GAO-01-498 Pediatric Organ Transplants

compared to 63 percent for children receiving an adult liver. Adults,

however, had similar 3-year graft survival rates regardless of donor age.20

The OPTN policy also provides an advantage for pediatric patients with

chronic liver failure. The policy places these patients at the highest

medical urgency level when their condition worsens, a provision that is

not in place for adult patients with chronic liver failure. Moving pediatric

patients to the highest category provides the advantage of access to

donated organs locally and regionally before all patients in lower

categories.

The heart allocation criteria have also been revised recently to reflect

differences in treatment and progression of heart disease between

children and adults. Before these revisions, the use of certain mechanical

assist devices or other monitoring and treatment therapies was required

for any patient to be included in the highest medical urgency categories.

However, because some of these devices and therapies are generally not

used with pediatric patients, the OPTN removed this requirement for

pediatric patients in January 1999. The OPTN implemented two further

revisions in May 2000. One change allows pediatric patients on the waiting

list for a heart to retain their medical urgency status when they turn 18

rather than being subject to adult criteria. Another revision gives priority

to pediatric heart transplant candidates, within each medical urgency

category, for hearts recovered from 11- to 17-year-old donors.

Children constitute a small proportion of patients in need of an organ

transplant, but organ allocation policies have been designed to provide

this vulnerable population with some special protections. Our examination

of transplantation patterns across age groups and recent data on waiting

times and death and survival rates indicates that pediatric patients do not

appear to be at a disadvantage in the competition for scarce organs. These

data show comparable or better outcomes for pediatric patients even

before the most recent policy changes, such as the change to prioritize

pediatric livers for pediatric recipients.

 20Sue V. McDiarmid, Darcy B. Davis, Eric B. Edwards, “Improved Graft Survival of Pediatric

Liver Recipients Transplanted With Pediatric-Aged Liver Donors,” Transplantation, Vol.

70, No. 9 (Nov. 15, 2000), pp. 1283-91. Data from this study were presented to OPTN for

consideration prior to publication.

Heart Allocation Policy

Gives Some Priority to

Pediatric Patients

Concluding

Observations
Page 28 GAO-01-498 Pediatric Organ Transplants

We provided HHS with the opportunity to comment on a draft of this

report. HHS provided technical comments, which we have incorporated

where appropriate. We also provided a draft of the report to UNOS, and it

provided technical comments, which we have incorporated where

appropriate.

As agreed with your offices, unless you publicly announce the contents of

this report earlier, we plan no further distribution of it until 30 days from

the date of this letter. We will then send copies to others who are

interested and make copies available to others who request them. If you or

your staffs have any questions about this report, please call me at (202)

512-7119. Another contact and key contributors to this report are listed in

appendix IV.

Janet Heinrich

Director, Health Care—Public Health Issues

Agency Comments
Page 29 GAO-01-498 Pediatric Organ Transplants

List of Requesters

The Honorable John D. Dingell

Ranking Minority Member

Committee on Energy and Commerce

The Honorable Sherrod Brown

The Honorable William J. Coyne

The Honorable Diana L. DeGette

The Honorable Frank R. Mascara

The Honorable John E. Peterson

The Honorable Henry A. Waxman

House of Representatives
Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Page 30 GAO-01-498 Pediatric Organ Transplants

Coverage for immunosuppressive medications may be extended to

children under Medicare, Medicaid, and private insurance. Pediatric

patients may also gain access to prescription drug coverage through

special state insurance programs for children. However, both adults and

children may have difficulty in obtaining and retaining insurance coverage

for the expensive immunosuppressive medications1

 necessary for survival

following transplantation. Further, gaps in coverage may occur during a

transition from one type of insurance to another. For example, if a parent

loses Medicaid eligibility, a child’s eligibility status could also be affected.

In addition, coverage problems can arise for both Medicaid- and privateinsurance-covered pediatric patients when they reach adulthood.

Transplant recipients covered by Medicaid as children may become

ineligible for continued coverage if they are able to obtain employment as

they reach adulthood. Children covered by private insurance under a

parent’s policy may be unable to afford coverage, given their expensive

preexisting medical condition, when they grow too old to be covered by a

parent’s policy. Data on the costs of immunosuppressive medications,

actual payments, and patient cost-sharing by the various insurers are not

readily available, so the level of the coverage cannot be specified with

certainty.

The proportion of transplant patients covered by different insurance

programs can be used to derive an indication of coverage for

immunosuppressive medications. Data from the Organ Procurement and

Transplantation Network (OPTN) on the expected sources of payment for

the pediatric transplants performed from 1997 through 1999 may serve as a

general estimate of the share of immunosuppressive medications for

children paid for by Medicare, Medicaid, and private insurance. OPTN data

show that 4,835 transplants were performed on children up to age 17 from

1997 to 1999. Of these, 2,775 transplants were for livers and hearts, and

2,060 were for kidneys. As figure 11 shows, private insurance paid for

almost half of the pediatric transplants for these three organs performed

from 1997 through 1999, while Medicaid paid for 25 percent and Medicare

paid for 14 percent of these transplants. For the same period, Medicare

paid for an estimated 30 percent of pediatric kidney transplants because of

 1

In 1999, the average annual charges for immunosuppressive medications for kidney, liver,

and heart transplant recipients ranged from $11,400 to $13,600 (Richard H. Hauboldt, Cost

Implications of Human Organ and Tissue Transplantations, An Update: 1999

(Brookfield, Wisc.: Milliman & Robertson, Inc., 1999)).

Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Sources of Payment

for Transplants
Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Page 31 GAO-01-498 Pediatric Organ Transplants

its special coverage for kidney patients under the End-Stage Renal Disease

(ESRD) program.

Figure 11: Expected Sources of Payment for Pediatric Kidney, Liver, and Heart

Transplants, 1997 Through 1999

Source: OPTN, April 23, 2000.

Medicare coverage for transplants and the associated medications is

provided to children either under a special entitlement to the Medicare

program created by the Congress for those diagnosed with ESRD or by

virtue of a parent’s enrollment as an eligible Medicare beneficiary.

The Medicare program has special entitlement rules for patients with

ESRD, the stage of kidney impairment that is considered irreversible and

requires either regular dialysis or a kidney transplant to maintain life. To

be eligible for Medicare entitlement as an ESRD patient, the patient

generally must have been on dialysis for 3 months and must be (1) entitled

to a monthly insurance benefit under title II of the Social Security Act (or

an annuity under the Railroad Retirement Act), (2) fully or currently

insured under Social Security, or (3) the spouse or dependent child of a

Medicare Coverage

for

Immunosuppressive

Drugs

47.1%

6.1%

8.4%

14%

24.5%

Other

Not reported

Medicare

Medicaid

Private insurance
Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Page 32 GAO-01-498 Pediatric Organ Transplants

person who meets at least the first 2 requirements.2 Currently, ESRD

patients’ entitlement to Medicare—and thus coverage for

immunosuppressive medications—ends 36 months after a transplant is

performed.3

In contrast, individuals who are eligible for Medicare under other

entitlement rules—that is, age 65 or disabled, and eligible for Social

Security or Railroad Retirement benefits—currently receive unlimited

coverage for immunosuppressive drug medications for the life of the

transplant under Part B.4

 Originally, Medicare limited immunosuppressive

drug coverage to 1 year. However, the Omnibus Budget Reconciliation Act

of 1993 (P.L. 103-66) expanded this coverage with a series of annual 6-

month increases beginning in 1995. As a result, by 1998, Medicare patients

received immunosuppressive medication coverage for 36 months after a

transplant operation. In 1999, the Medicare, Medicaid, and SCHIP

Balanced Budget Refinement Act of 1999 (P.L. 106-113) extended this

immunosuppressive drug coverage benefit for an additional 8 months.

Most recently, the Medicare, Medicaid and SCHIP Benefits Improvement

and Protection Act of 2000 (P.L. 106-554) eliminated all time limits for

immunosuppressive drug coverage under Part B of Medicare.

Medicaid is a joint federal/state entitlement that annually finances health

care coverage for more than 40 million low-income individuals, over half

of whom are children. Medicaid coverage for children is comprehensive,

offering a wide range of medical services and mandating coverage based

upon family income in relation to the federal poverty level (FPL). Federal

law requires states to cover children up to age 6 from families with

incomes up to 133 percent of the FPL, and children ages 6 to 15 for

incomes up to 100 percent of the FPL. Medicaid benefits are particularly

important for children because of Medicaid’s Early and Periodic

Screening, Diagnostic, and Treatment (EPSDT) Program. EPSDT, which is

 2

For ESRD participants, Medicare pays secondary to employer-sponsored group health

plans for the first 30 months.

3

An ESRD patient may be eligible for immunosuppressive drug coverage if, after 36 months,

the patient is otherwise entitled to Medicare based on age and/or disability.

4

Medicare insurance consists of two parts, Part A, which covers in-patient hospital

expenses, and Part B, which covers other medical expenses including physician, outpatient

hospital, laboratory, and other services such as immunosuppressive drug therapy.

Beneficiaries must pay a premium for Part B coverage, currently $50 per month, and are

also responsible for Part B deductibles, coinsurance, and copayments.

Medicaid Coverage

for

Immunosuppressive

Drugs
Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Page 33 GAO-01-498 Pediatric Organ Transplants

mandatory for categorically needy children, provides comprehensive,

periodic evaluations of health and developmental history, as well as vision,

hearing, and dental screening services to most Medicaid-eligible children.5

Under EPSDT, states are required to cover any service or item that is

medically necessary to correct or ameliorate a condition detected through

an EPSDT screening, regardless of whether the service is otherwise

covered under a state Medicaid program. This would include

immunosuppressive drugs.6

Private insurance, such as employer-sponsored health plans, generally

covers all aspects of organ transplants, including follow-up care and

necessary medications. Information is not readily available on private

insurance coverage specifically for immunosuppressive medications.

However, according to a 1998 national survey of employer-sponsored

health plans, nearly all employers that offer health benefits include

benefits for outpatient prescription drugs.7

 In addition, a Kaiser Family

Foundation survey of employer health benefits found that 96 percent of all

firms with conventional fee-for-service plans and 99 percent of those with

managed care plans cover prescription drugs.8

 5

The categorically needy includes low-income children; pregnant women; aged, blind, or

disabled people meeting Supplemental Security Income program requirements; and

individuals who are eligible to receive federally assisted income maintenance payments.

The EPSDT benefit is optional for the medically needy population, who generally have too

much income to quality for Medicaid, but have “spent down” their income by incurring

medical and/or remedial care expenses. See 42 USC 1396(a)(10)(C). If a state chooses to

provide one EPSDT service, it must provide all EPSDT services to medically needy

individuals under age 21. The medically needy comprise individuals whose income,

resources, or both exceed the levels for the categorically needy, but who cannot afford to

pay their medical bills.

6

The Institute of Medicine reported that kidney, heart, and liver transplants are covered

services under Medicaid in nearly all states. See Organ Procurement and Transplantation,

“Assessing Current Policies and the Potential Impact of the DHHS Final Rule,”

(Washington, D.C.: National Academy Press, 1999). Generally, once a transplant is

approved, immunosuppressive drugs and medications to maintain the organ are also

approved.

7

Mercer/Foster Higgins, National Survey of Employer-Sponsored Health Plans 1998, a

survey of all U.S. employers with 10 or more employees.

8

The Kaiser Family Foundation and Health Research and Educational Trust, Employer

Health Benefits, Annual Survey, 1999 (Menlo Park, Calif., and Chicago, Ill., 1999).

Private Insurance

Coverage for

Immunosuppressive

Drugs
Appendix I: Insurance Coverage for

Immunosuppressive Drugs for Children

Page 34 GAO-01-498 Pediatric Organ Transplants

Privately insured organ transplant patients most likely will incur additional

expenses for medications, however, such as out-of-pocket expenses for

deductibles and copayments, because of limits on coverage. A recent

survey of employers with 1,000 or more employees on strategies to control

prescription drug expenditures found that 6 percent of employers cap

annual benefits and 10 percent are considering doing so.9

 The study also

found that 41 percent of employers limit the quantities of prescription

drugs and 7 percent are considering it. Moreover, 40 percent of employers

now require higher copayments than previously, and 39 percent are

considering it.

 9

Hewitt Associates, Health Care Expectations: Future Strategy and Direction, 2001

(Lincolnshire, Ill.: 2001).
Appendix II: Causes of Death Most Likely to

Result in Organ Donation

Page 35 GAO-01-498 Pediatric Organ Transplants

The causes and circumstances of death that could reasonably result in a

declaration of brain death and from which organ donation might be

possible are listed in table 3.1 We used the International Classification of

Diseases, 9th Revision, Clinical Modification (ICD-9-CM) codes to classify

deaths by causes and circumstances.2

 1

See Organ Procurement Organizations: Alternatives Being Developed to More

Accurately Assess Performance (GAO/HEHS-98-26, Nov. 26, 1997), where we developed

this list.

2

Medical staff use ICD-9-CM codes at the time of death to indicate cause of death on the

death certificate.

Appendix II: Causes of Death Most Likely to

Result in Organ Donation
Appendix II: Causes of Death Most Likely to

Result in Organ Donation

Page 36 GAO-01-498 Pediatric Organ Transplants

Table 3: Causes of Death Most Likely to Result in Organ Donation

ICD-9 CM Code Description

430-438 Cerebrovascular disease

798.0 Sudden infant death syndrome

E810-E825aMotor vehicle accident

E830 Accident of watercraft causing submersion

E832 Other accidental submersion or drowning in water transport accident

E850-E858 Accidental poisoning by drugs, medicinal substances, and biologicals

E910-E913 Accidental submersion, suffocation, and other foreign bodies

E920 Accidents caused by cutting and piercing instruments or objects

E922 Accidents caused by firearm missile

E930-E950.5 Drugs, medicinal and biological substances causing adverse effects in therapeutic use,

suicidal and self-inflicted poisoning by solid or liquid substances

E953-E955.4, E956, E958.5 Suicide

E962.0 Assault by poisoning

E963 Assault by hanging and strangulation

E964 Assault by drowning

E965-E965.4 Assault by firearms and explosives

E966 Assault by cutting and piercing instrument

E970 Injury due to legal intervention by firearms

E974 Injury due to legal intervention by cutting and piercing instrument

E980.0-E980.5 Poisoning, undetermined whether accidentally or purposely inflicted

E983 Hanging, strangulation, or suffocation undetermined whether accidentally or purposely

inflicted

E984 Drowning, undetermined whether accidentally or purposely inflicted

E985-E985.4 Injury by firearms, undetermined whether accidentally or purposely inflicted

E986 Injury by cutting, piercing instruments, undetermined whether accidentally or purposely

inflicted

a

E codes permit the classification of environmental events, circumstances, and conditions as the

cause of injury, poisoning, and other adverse effects.
Appendix III: Distribution of Kidneys, Livers,

and Hearts, 1994 Through 1999

Page 37 GAO-01-498 Pediatric Organ Transplants

The following tables show the distribution of kidneys, livers, and hearts

procured from all donors from 1994 to 1999, by age of donor and recipient.

Table 4: Distribution of Kidneys, 1994 through 1999

Recipients

Donors

0-5

years

6-10

years

11-17

years

Total

children

Total

adults, 18

and over

Total

recipients

0-5 years 27 23 47 97 1,412 1,509

6-10 years 29 38 99 166 1,479 1,645

11-17 years 55 88 227 370 5,477 5,847

Total children 111 149 373 633 8,368 9,001

Total adults, 18

and over 168 272 902 1,342 36,449 37,791

Total 279 421 1,275 1,975 44,817 46,792

Source: OPTN, June 17, 2000.

Table 5: Distribution of Livers, 1994 through 1999

Recipients

Donors

0-5

years

6-10

years

11-17

years

Total

children

Total

adults, 18

and over

Total

recipients

0-5 years 1,052 148 51 1,251 55 1,306

6-10 years 215 124 107 446 399 845

11-17 years 151 53 149 353 2,982 3,335

Total children 1,418 325 307 2,050 3,436 5,486

Total, adults, 18

and over 326 128 341 795 18,128 18,923

Total 1,744 453 648 2,845 21,564 24,409

 Source: OPTN, June 17, 2000.

Appendix III: Distribution of Kidneys, Livers,

and Hearts, 1994 Through 1999
Appendix III: Distribution of Kidneys, Livers,

and Hearts, 1994 Through 1999

Page 38 GAO-01-498 Pediatric Organ Transplants

Table 6: Distribution of Hearts, 1994 through 1999

Recipients

Donors

0-5

years

6-10

years

11-17

years

Total

children

Total

adults, 18

and over

Total

recipients

0-5 years 752 44 3 799 9 808

6-10 years 66 118 76 260 81 341

11-17 years 11 44 180 235 1,959 2,194

Total children 829 206 259 1,294 2,049 3,343

Total adults, 18

and over 8 20 236 264 9,978 10,242

Total 837 226 495 1,558 12,027 13,585

Source: OPTN, June 17, 2000.
Appendix IV: GAO Contact and Staff

Acknowledgments

Page 39 GAO-01-498 Pediatric Organ Transplants

Marcia Crosse, (202) 512-3407

In addition to the above, Donna Bulvin, Charles Davenport, Roy Hogberg,

Behn Miller, and Roseanne Price made key contributions to this report.

Appendix IV: GAO Contact and Staff

Acknowledgments

GAO Contact

Acknowledgments

(201043)

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