NATIONAL HEMOPHILIA FOUNDATION
  for all bleeding disorders

Board of Directors
Officers
Mark Skinner, Presrdent

Washlngton, D C
American Insurance Assoclatlon

Glenn Pierce, PhD, MD, President Elect
Selective Genetics
Rancho Santa Fe, Callfornla

Calvin Price. Treasure,
Mldvale, Utah
ShideyWilson, Secretary
Ramsey, Mlnnesota

Direclors
Roger Ailes
FOX News
NewYork. NewYork

Camlyne Arnold, ScD

Brooklme, Massachusetts
Unlverslty of Massachusetts

Nikki J. Beneke, PT
Dallas, Texas

Bob Beran
evildent, Inc
Great Falls, Vlrglnla

Ross Btuner
San Paolo IMI Asset Management
NewYork. NewYork

Edward Burke
Gentlva Health Servlces
Palm Harbor, Florlda

Michael Coyne, MD

Plttsfleld, Massachusetts
Berkshire Medlcal Center

Orlv Dahan

TagRag
Beverly H~lls, Callfornla

hula Elbirt, MD
Mount Sinal Medical Center
NewYork, NewYork

Steven Faust,
Atlanta, Georgla
Fred Ferguson

PR Newswire
NewYork, NewYork
Elizabeth Fung, MSW, PhD
Children's Memorlal Hospltal
Chicago, llllnols
Keith Hoots, MD
Gulf States Hernophllla Center
Houston, Texas
Edward Jones

Tulsa, Oklahoma
Team One Realtors

Gregory Kerfoot
Sears, Roebuck and Co
Napervllle, llllnols

Jordan Lune, MD
Loyola Unlverslty Medical Center
Wheaton, llllnols

Carlos Ruiz
Mlaml VA Medlcal Center
Mlaml Lakes, Florlda

Gina Shreve, PhD
Wayne State Unlverslty
Ann Arbor, Mlchlgan

Jan van Eys, PhD, MD
Vanderbllt Unwerslty School
of Medicme
Nashville, Tennessee

AndrewVoegtle
Appralsal Research Corporatlon
Brlghton. Mlchlgan

Stephen E. Bajardi
Executlve Dlrector and CEO

January 10,2002

Jo Anne B. Bamhart
Commissioner
Social Security Administration
P.O. Box 17703
Baltimore, MD 21235-7703

RE: RIN 0960-AD47

Dear Commissioner Bamhart:

The National Hemophilia Foundation, hereinafter referred to as NHF, is a not-for-
profit organization dedicated to improving the quality of life for all individuals
with hemophilia and other bleeding disorders. NHF wishes to provide written
comment to your proposed revisions to the medical criteria for evaluating
eligibility for federal disability assistance for patients with hemophilia and von
Willebrand disease (vWD), which were published in the November 27,2001
Federal Register.

While the advent of factor replacement products and comprehensive care models
for the treatment of hemophilia and vWD patients have increased quality of life
and reduced reliance on public disability assistance for many individuals, both
adults and children, the continued importance of access to disability assistance
cannot and should not be underestimated. We commend the Social Security
Administration (SSA) for its past efforts to recognize the changes in the standard
of care for the treatment of individuals with bleeding disorders and support the
efforts of the SSA to reorder and regroup hematological disorders apart from
malignant neoplastic diseases. While we commend the SSA for its commitment to
updating its medical criteria in light of advances in the treatment of hemophilia,
vWD and other diseases, we are concerned that these revisions are based on older
listings that were not consistent and did not reflect the standard of care for persons
with hemophilia. Thusly, many of the provisions in the proposed rule continue to
reflect a poor understanding of the current standard of care for these disorders, and
continue to exhibit inconsistencies. Furthermore, we are also deeply concerned

     116 West 32nd Street. 1 Ith Floor
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www.hemophilia.org . info8hemophilia.org

about changes to the listings that could significantly reduce the ability of children with
severe hemophilia to obtain disability benefits.

Proposed 7.OOE-How Do We Evaluate Episodic Hematological Disorders? Under this
new section, an individual with hemophilia would be required to have bleeding episodes
occurring at least 3 times in a consecutive 12-month period. These events can include
"pain crises, hospitalization, treatment with parenteral antimicrobial medication, bleeding
episodes and thromboses." Furthermore, the new section requires a respite of at least one
month between health events to ensure that the agency is evaluating separate episodes. In
the most severe form of the disorder, patients may suffer from fi-equent bleeding episodes
and often these episodes can occur without a known injury. Therefore, requiring a
month-long lapse between bleeding events to ensure that the agency is evaluating
separate episodes may fail to recognize the course and severity of this disorder in a
particular individual. A series of three separate, discrete bleeding episodes in a
consecutive year may be less lifethreatening or disabling than a continuous, serious bleed.
Also, the listing contends that a bleed must occur despite prophylactic treatment. While
NHF does encourage the prophylactic treatment for patients with hemophilia (MASAC
Recommendation #117, attached),prophylaxis is not a universal treatment and therefore
should be removed from the SSA listing.

Proposed 7.OOG(3)(a)-Disorders of Hemostasis. This new section indicates that the
agency will document "the frequency, severity, and treatment of bleeding episodes'' as
part of its effort to determine eligibility for disability assistance; however, the provision
fails to define terms such as "frequency" or "severity." How does the agency reconcile
the use of the term "frequent" with its requirement that a month transpire between
bleeding episodes to determine if a bleeding episode is a new versus a recurrent bleed?
How will the agency define "severe" and what documentation will be necessary? The
Centers for Disease Control and Prevention's planning workbook on hemophilia indicates
that bleeds in several key anatomical sites should always be viewed as potentially
lifethreatening. These include bleeding into the head, neck or tongue, spinal cord or
digestive tract. While bleeding into the head accounts is a leading cause of premature
death in hemophilia patients, the symptoms of bleeding into the head, if present at all, can
be diffuse, such as fatigue, headache and neck pain. Head bleeds may also occur
spontaneously and may not be linked to either major or minor trauma to the head. We
would urge the agency to use great caution in developing documentation criteria to
accompany this section.

Proposed 7.00G(3)(c). NHF supports the agency's proposal to consider such
complications as the development of inhibitors against clotting factor, intrusiveness of
treatment, and joint deformity in its criteria for considering eligibility for disability. NHF
also encourages the agency to consider other complications such as bleeding-related
nerve compression, which could lead to loss of cognitive ability as well as mobility and
motility. The SSA should consider the confluence of multiple morbidities in individuals
with hemophilia who are also HIV and/or HCV positive, The SSA should also be aware
of other more rare complications such as the hemophilic pseudotumor, which if
undiagnosed or untreated can lead to neuropathy, bone necrosis, and if located in the

large muscle masses of the pelvis may lead to erosion into the bowel. While the
prevalence of this complication has decreased over the years due to the availability of
adequate factor treatment, pseudotumors continue to be considered a significant
complication of hemophilia, especially in HIV-positive individuals with hemophilia.
NHF also encourages the agency to consider the complications of treatment, such as the
development of hepatitis after the use of plasma-derived products. While advances in
technology have significantly reduced the risk for contracting viral infection from
plasma-derived products, the risk for contracting blood-borne viruses still exists. Indeed,
the recent shortage of recombinant product has led to an increased reliance on plasma-
derived products, further increasing the risk of contracting hepatitis. While vaccines
currently exist for hepatitis A and B, no vaccine exists for hepatitis C, and hepatitis C
infection represents a growing problem in the hemophilia community [This implies that
hepatitis C infections continue to occur; in reality, there have been no new infections in
over 10 years from plasma-derived products]. Furthermore, hemophilia patients are at
risk for contracting other infectious diseases through the use of plasma-based products.
While donor deferral programs are currently being implemented and the exact risk for
contracting Creutzfeldt-Jakob Disease from blood products is unknown, hemophilia
patients may be at risk for developing this lethal disorder through the use of plasma-
derived replacement factor. Persons with hemophilia are also at risk for developing
parvoviruses, which cannot be "killed" using current methods for killing viruses. While
very common in the general population, parvoviruses may pose a more significant health
risk for those hemophilia patients with immune systems compromised by HIV or HCV
infection. Finally, the SSA needs to consider disability caused by the use of essential
medications such as HAART, interferon, ribavirin and certain narcotics.

Proposed 7.03B-Category of Impairments, Disorders of Hemostasis. The NHF's
concerns with the language of 7.03B were previously stated in our comments to section
7.00E.

Proposed 7.03C-Category of Impairments, Disorders of Hemostasis. NHF is dismayed
with the provisions in section 7.03C that outline the eligibility criteria for vWD, and
would ask that the agency hold patients with this disease to the same criteria, i.e. number
of bleeding episodes per 12-month period, as in hemophilia. vWD is reported to be the
most common bleeding disorder in humans. Although the disease affects both sexes,
women with vWD are at higher risk for complications such as anemia, because of
menses. Section 7.03C states that a hospitalization of 24 hours or more, occurring at least
3 times in a consecutive 12-month period is necessary for consideration of disability for a
patient with vWD. NHF opposes in the strongest terms this criteria as it sets a higher
standard for individuals with vWD for disability assistance than it does for any other
hematological disorder. vWD is classified into three major types: I, 11, and 111. Type I1
vWD is subdivided into four different subtypes. Correct typing of vWD is necessary to
ensure that the proper treatment regimen is followed. People with type I vWD are treated
with the synthetic agent desmopressin, either as an injectible or in nasal spray form. In
the case of surgery, trauma or other serious bleeding episodes, a factor VI11 concentrate
with a high concentration of von Willebrand factor is often required. Persons with type
IIA, IIM, and IIN are also treated with desmopressin. Individuals with the more severe

.

forms of the disorder, IIB and 111 or those non-responsive to desmopressin are treated
with factor VI11 concentrate that contains a higher molecular weight multimers of von
Willebrand factor. Because of the increased risk of HIV and hepatitis transmission,
cyroprecipitate is not recommend except in the direst of emergencies. The standard of
care for individuals with vWD, as with hemophilia, does not require routine
hospitalization. Furthermore, the SSA recommendation goes against shifts in our health
care delivery system away from in-patient care to treatment in an outpatient facility or at
home.

Proposed Listing 107.03-Disorders of Hemostasis (in children). SSA proposes to
utilize the similar criteria for hemophilia and vWD in children that it will use in adults
(see. 7.03B and C). The SSA contends that these changes will bring clarity to the issue of
children's disability. Certainly, the previous listing was confusing both to parents and to
SSA staff, resulting in variability from state to state in disability determinations. While
we support the intention of bringing clarity to this issue, we strongly object to proposed
changes that would specify the level of joint deformity required in children with
hemophilia, which is not a requirement for adults, using the criteria in listing 101.02, the
listing for juvenile rheumatoid arthritis (JRA). The etiology, pathophysiology and
treatment for JRA and hemophilia-related joint disease are vastly different. JRA is an
autoimmune disease characterized by swelling and stiffness and limitation of movements
in the joints. In addition, in JRA high fever and skin rash are often present. JRA is
diagnosed through sero-testing for rheumatoid factor and antinuclear antibodies.
Treatment regimen for JRA includes heavy use of non-steroidal anti-inflammatory drugs
(NSAIDS), corticosteroids and other disease-modifying anti-rheumatic drugs.
Hemophilia is predominantly an inherited disorder, although acquired hemophilia does
exist. The breakdown in synovium in the joints of persons with hemophilia is due to
repeated bleeding into that joint, whether from minor or major trauma or from
spontaneous bleeding. While physical therapy is recommended as a treatment for both
disorders, the most common treatments for minor hemarthroses include infusion of
factor, immobilization and elevation, compression and ice therapy. The use of many
analgesics commonly used to treat JRA, including aspirin and NSAIDS, are
contraindicated because of the risk of potential bleeding due to inhibition of platelet
function. In more serious cases, surgical intervention may be warranted.

I hope that these comments will be useful to the SSA in revising its criteria for disability.
NHF would welcome the opportunity work with the agency to provide you with expert
knowledge on hemophilia and other bleeding disorders and we look forward to further
discussions on the proposed listing.

Sincerelv.

Mark W. Skinner
President

/ /'

Eomd ot Directon

omom
M.k Skinnr Restdent
Amermn Insurance Assaclation
Washington. D C.

NATIONAL HEMOPHILIA FOUNDATION

for all bleeding disorders

MASAC Recommendation #117

Gkm b, PhD. MD, Presdent Elect
Selenlw Genetlcs MASAC RECOMMENDATION CONCERNING PROPHYLAXIS
Rancho Santa Fe. Callfornla
Calvin Rim, Tleasurer (PROPHYLACTIC ADMINISTRATION OF CLOTTING FACTOR
Midvale. Utah
shiiwihon, Secretary CONCENTRATE TO PREVENT BLEEDING)
Ramsey, Mlnnesota

DinC(0n

FOX News
Newbrk, NewYork Scientific Advisory Council (MASAC) on May 19,2001, and adopted
CwdyMImOld sd)

Uniwrslty of Massachusetts by the NHF Board of Directors on June 9,2001.
Brooklme, Massachusetts

RwAik The following recommendations were approved by the Medical and

Nikki J. Beneko, PT
Dalbs.Texas In view of the demonstrated benefits of prophylaxis begun at a young age in persons with

eWdent. Inc.

Bobm

Great Falls. Virginla
Ron BNW for individuals with severe hemophilia A and B (factor Vm or factor IX 4 %).

San Fado IMI Asset Ma~gernent
NewYork. NewYork

hemophilia A and B, MASAC reconmiends that prophylaxis be considered optimal therapy

Edward BurLe
Genti Health Servlces
Palm Harbor, Florlda

Michd Coyno. MD
Berkshire Medical Centel
Pinsfield. Massachusens

Tag Rag
DdyDdmn

Beverly Hlls. Callfornla

hula Elbirt MD
Mount %ai Medlcal Center
NewYork. NewYcik

Atlanta. Georgta
Shm huat

wF=lw=n
PA Newswlre
NewYork, NewYork

Elh.b.m Funs MSW, PhD
Chtldren's Memorlal Hospltal
Chmgo. llllnas

Kaith Hoots, MD
Gulf States Hemophiia Center
Houston, Texas
Edward Jones
Team One Realtors
Tulsa. Okbhcma

Gngor).Knfoo(
Sears. Roebuck and Co.
Napernlle. llllnas

Jordan Lurk, MD
Lwola Unlversltv Medical Center
Wheaton. Illmots

Wor Rub
Mtarnl VA Med~cal Center
Miaml Lakes, Flwtda

Wayne State Uniwrslty
Gina shnn. PhD

Ann Arbor, Mlchgan
Jan vm Eyr. PhD, MD

of Medicine
Vande~tult Unnrerslty school

Nashnlle. Tennessee

Appraisal Rsearch Corporation
Bnghton.,MIchlgan
~taphm%. bjardi
Executwe Director and CEO

AndnwVoeptlr

Prophylactic therapy should be instituted early (prior to the onset of fkquent bleeding), with
the aim of keeping the trough FVIII or FIX level above 1% between doses. This can usually
be accomplished by giving 25-40 FVIII units/kg three times per week or every other day, or
40- 100 FIX unitskg twice yekly.

It is also recommended that individuals on prophylaxis have regular follow-up visits to
evaluate joint status, to document any complications, and to record any bleeding episodes
that occur during prophylaxis.

As always, a carefbl analysis of health risks and benefits must be performed by consumers
and their health care providers. As is the case with all recommendations, MASAC will
periodically reexamine this recommendation as new data emerge.

or engage in thepractice

or local treatment center before pursuing any course of treatment.

CHAPTERS: Please distribute to your membership.

PHYSICIANS: Please distribute to physicians in your area who treat persons with

hemophilia.

Founded 1948

    116 West 32id Street. 1 Ith Floor
        New York, NY loo01
(800) 42-HAND1 * (212) 328-3700 . fax (212) 328-3777

www.hemophilia.org - info@hemophilia.org

Appendix to MASAC Recommendation 117:

The National Hemophilia Foundation encourages persons with hemophilia and their families to
consider these recommendations within the context of comprehensive care, which emphasizes patient
and family involvement in the decision-making process, based on a thorough discussion of the risks
and benefits - medical, social, psychological, and economic - of such care. Thus, in considering
prophylaxis, the NHF encourages persons with hemophilia and their families to examine the following
issues with their medical team:

A. For parents of young children (age 1-2 years) with severe hemophilia who are being

considered for prophylaxis, the following issues should be considered:
1. Need for fiequent clotting factor infusion versus joint damage and other morbidities

2. Potential quality of life and psychological implications.
3. Potential costs and reimbursement implications.
4. Requirement for fiequent venous access, often necessitating use of a central venous

5. Potential complications of such central venous access devices (see MASAC

6. Possibility of other benefits and complications not yet identified.

associated with hemophilic bleeding.

access device (such as a surgically implanted port).

Recommendation ## 1 15 regarding central venous access devices).

B. Recombinant factor products are the most appropriate choice for prophylaxis because of

markedly reduced risk of blood-borne infection.

C. If prophylaxis is being considered for an older child, adolescent, or adult, the following

issues should be considered:
1. History of consumer's clotting factor usage and how prophylaxis would compare to

2. Current joint damage and what can reasonably be expected fiom prophylaxis.
3. Those considerations listed under A. above.

that usage.

D. If prophylaxis is selected as the therapeutic regimen of choice, the responsibilities and

potential risks and benefits for the individual and the family need to be clearly delineated.
The following points should be included in discussions held with patients and families:
1. Frequency of inhsion.
2. Potential quality of life and psychological implications.
3. Standard care for the central lines, if utilized, that the family must follow.
4. Frequency of comprehensive follow-up.
5. Statement of indications to alter or discontinue the protocol.
6. Occurrences that prompt an immediate physician or nurse contact.
7. Essential need for periodic follow-up education.
8. A statement of continued risk of bleeding with trauma.

'. 9. An acknowledgement that there may be current benefits and risks not yet identified.

After a thorough discussion with their medical team, persons with hemophilia and their families
should decide which therapy is appropriate for themhheir child. This decision should be evaluated

periodically, particularly in light of emerging data and changes in bleeding and clotting factor
usage.

References:

1. Braclanan HH, Eickhoff HJ, Oldenburg J et al.: Long-term therapy and on-demand

treatment of children and adolescents with severe hemophilia A: 12 years of experience.
Haemostasis 1992;22:25 1-8.

2. Nilsson IM, Bemtorp E, Liifqvist T, Petterson H.: Twenty-five years experience of

prophylactic treatment in severe haemophilia A and B. Journal of Internal Medicine

1992;232:25-32.

3. Petrini P, Lindvall N, Egberg N, Blomback M: Prophylaxis with factor concentrates in

preventing hemophilic arthropathy. American Journal of Pediatric Hematologv and
Oncology 1991;12:280-287.

4. Carlsson M, Bemtrop E, Bjorlanan S, Lindvall K: Pharmacokinetic dosing in prophylactic

treatment of hemophilia A. European J. Haemtology 1993;3 1 :247-252.

i