bluuebunny/crossref_metadata_2025 huggingface dataset:
Effect of 9–11 on U.S. national cancer clinical trials accrual and lack of effect of National Cancer Institute budget increases
Authors: Bleyer A.
Published: March 2020

6551

Background: During the past decade, a variety of initiatives have been implemented to improve the accrual of cancer patients on clinical trials. In the U.S., these have included comprehensive reviews and recommendations by the two most recent National Cancer Institute (NCI) administrations, reorganization of the clinical trials infrastructure at the NCI, and campaigns by the NCI Cooperative Groups and their Coalition. During the past six years, additional funds were allocated to this effort as part of the doubling of the NCI budget. The impact of these efforts on national cancer treatment clinical trials was evaluated, with emphasis on age groups. Methods: Accrual data from NCI-sponsored treatment trials conducted between 1997 and 2006 were obtained from the NCI Cancer Therapy Evaluation Program. Entries were analyzed by patient age, gender, race, type of cancer treated, and calendar year of trial entry. Results: Overall, national cancer treatment trial entries declined after 9–11–2001 and in 2003 reached the lowest levels since 1997. As of 2005 accrual recovered to pre 9–11 levels only in 15–29 and >60 year-olds, with the former demonstrating the greatest gain ( Table ). Entries among <15 and 30–49 year- olds declined steadily since 1997 with no evidence for recovery as of 2005 ( Table ). Overall, the estimated proportion of the nation's cancer patients entered onto national treatment trials remains below 3%. Conclusions: Despite continued national and local efforts to increase the participation of cancer patients on clinical trials, accompanied by significant increases in the NIH and NCI budgets, there is little evidence of a beneficial impact. The effect of 9–11 has yet to be overcome, except in young and elderly adults, in whom specific, targeted initiatives appear to have been successful. The latter approaches may be useful to apply to other age groups, particularly in view of the recent cuts in the cooperative group budgets and current mandated decreases in study accruals.

No significant financial relationships to disclose.

[Table: see text]
Quantitative evaluation of heterogeneities in treatment effects and baseline risks among hospitals from a phase III surgical trial of gastric cancer
Authors: Kurokawa Y. , Yamaguchi T. , Yoshimura K. , Yamamoto S. , Nakamura K. , Sasako M. , Fukuda H.
Published: March 2020

6553

Background: Although the JCOG9501 phase III trial demonstrated that standard D2 plus extended lymphadenectomy (D3) could not improve the survival in cT2b-T4 gastric cancer (ASCO2006 Abs#LBA4015), it is important to examine the variation in the treatment effects for evaluating the generalizability, as written in international statistical guideline (ICH-E9). In addition, investigating the ranges of baseline risks among hospitals gives important information for the standardization of cancer treatment. Methods: Mixed effect models were applied to survival and surgical outcome data for 521 (D2: 263, D3: 258) gastric cancer patients from 23 Japanese hospitals, which were the leading institutions of gastric cancer surgery in Japan. After adjustment with 10 clinicopathological factors, the variation in the treatment effects and the baseline risks among hospitals were estimated. If heterogeneity was identified, we further estimated the Spearman’s correlation coefficients between the variation and hospital characteristics. Results: In the treatment effects, the ranges of the hazard ratio of overall survival (OS) and recurrence-free survival (RFS) for D3 compared with D2 were 1.05–1.48 and 1.03–1.77, respectively. The ranges of baseline risks for D2 were shown in Table . The correlation coefficients between hospital volume of gastrectomy and the heterogeneity in operation time, blood loss and postoperative complication were -0.31 (p=0.15), −0.40 (p=0.06), −0.52 (p=0.01), respectively. Any institutional characteristics showed little correlation with OS or RFS. Conclusions: The final result of JCOG9501 seemed to be generalizable to the target populations, although some degree of heterogeneity of treatment effects existed. The hospitals entered in this trial had some variation also in the baseline risks for D2. The heterogeneities of some surgical outcomes may be correlated with hospital volume of gastrectomy.

No significant financial relationships to disclose.

[Table: see text]
Willingness to pay for new hormone-refractory prostate cancer (HRPC) therapies: Experience from Ontario hospitals
Authors: Milliken D. , Sawka C. , Trudeau M. E.
Published: March 2020

6559

Background: The New Drug Funding Program (NDFP) is an IV-based government funded provincial formulary that reimburses over 90 Ontario hospitals for new and expensive anti-cancer and supportive care drugs. An expert committee considers clinical and economic evidence, including a hierarchy of benefits and evidence to inform decisions. There can be extended unpredictable delays from the time evidence is available to funding, during which hospitals decide whether to pay for therapies. This report assesses hospitals’ willingness to pay for HRPC therapies prior to a funding decision. Methods: Patterns of uptake for docetaxel (D) and zoledronic acid (Z) were analyzed from the NDFP database and compared to publication dates, hierarchy of benefits and evidence, and costs for these new HRPC therapies. Results: Analysis of NDFP data for patterns of uptake revealed an apparent cost shift from hospitals to the NDFP for D but not for Z once funding was in place. This cost shift demonstrated that despite a higher cost, shorter time from publication to funding and equivalent levels of evidence that hospitals paid for D but not for Z. The only factor that favored payment for D over Z was that D ranked higher than Z in the NDFP hierarchy of benefits: Survival was the primary outcome for D versus prevention of skeletal related events (SRE) for Z. Publication of provincial practice guidelines supporting these therapies occurred after NDFP funding was in place and would not have impacted hospitals’ decisions to pay. Despite immediate uptake of Z once NDFP funding was introduced, hospitals did not absorb the cost when funding was temporarily suspended for a 5 month period. Conclusions: Survival benefit appeared to be the primary factor impacting hospitals’ willingness to pay for unfunded HRPC therapies. This observation is consistent with the ranking of clinical benefits used to inform NDFP formulary decisions. The higher cost of D did not appear to impact hospitals’ willingness to pay.

No significant financial relationships to disclose.

[Table: see text]
Primary care physician willingness to provide follow-up care for adult cancer survivors
Authors: Del Giudice M. , Verma S. , Piliotis E. , Harvey B. J. , Grunfeld E.
Published: March 2020

6562

Background: Routine follow-up of adult cancer survivors is an important clinical and health service issue. Due to a lack of evidence suggesting advantages of long-term follow-up care in oncology clinics, there is increasing support for the locus of this care to be provided by primary care physicians (PCPs). However, views from PCPs on this issue remain to be studied. Methods: A mail survey of a random sample of community based PCPs in Canada, stratified by region and proximity to urban centres was conducted. Views on routine follow-up of adult cancer survivors and modalities to facilitate PCPs in providing this care were determined. Results: 330 PCPs responded (adjusted response rate 52%). PCPs are willing to assume exclusive responsibility for routine follow-up care approximately 2.5 (95 % Confidence Interval [CI], 2.3–2.7;) years after active treatment of colorectal and prostate cancers; 2.8 (95 % CI, 2.5–3.1) years for breast cancer and 3.2 years (95% CI, 2.9–3.5) years for lymphoma. Respondents ranked care shared between cancer specialists and PCPs as their preferred strategy of follow-up, followed equally by exclusive care by cancer specialists or by PCPs. PCPs considered the most useful modalities to assist them in assuming exclusive responsibility for follow-up care were: (1) a patient-specific letter from the specialist; (2) printed guidelines; (3) expedited routes of re-referral; and (4) expedited access to investigations for suspected recurrence. The least useful modalities were: (1) pamphlets; (2) more office staff; and (3) websites. The majority of PCPs recognized that specialist clinics are overcrowded (55.6 %) and that PCPs are better placed to provide psychosocial support (79.8 %). However, they believed that specialist follow-up is important so that patients remain in the cancer centre system (67.2 %) and that patients expect follow-up care by cancer specialists (71.7%). Variations in responses were observed according to current practices and proximity to urban centres. Conclusions: With appropriate information and support in place, PCPs are willing to assume exclusive responsibility for the follow-up care of prevalent adult cancer survivors. Insights gained from this survey may help guide strategies in providing optimal care to cancer survivors.

No significant financial relationships to disclose.
Application of quality indicators in breast cancer care using linked national registry and administrative claims data
Authors: Meguerditchian A. , Stewart A. , Watroba N. , Roistacher J. , Cropp M. , Ko C. , Edge S. B.
Published: March 2020

6565

Background: The National Quality Forum (NQF) recently approved quality measures for cancer care. These are intended for application through cancer registries. Variably complete cancer registry data on outpatient care may limit application of these measures. Administrative claims matched to registries may provide more complete data. This study tested the ability to match claims with the National Cancer Data Base (NCDB) registry and to apply NQF breast cancer measures in a limited pilot in one region. Methods: Claims data from one regional payer in Western New York (WNY) were matched with registry data from the 3 WNY hospitals participating in the NCDB for 2001–2003. NQF measures for hormone therapy (HT), radiation (RT) with breast conserving surgery (BCS) and chemotherapy (CT) were applied independently using registry and claims data. Results: 480 women with this payer had claims for breast cancer surgery at the NCDB hospitals and 439 (91%) matched NCDB based on birth date, date of treatment and reporting hospital. Among these, 17 had duplicate or incomplete records. Among 422 analytic cases the median age was 54 and stage was 0 –9%; I –49%; II –33%; III –7%; IV –2%. Claims identified CT, HT and RT in substantially more cases than identified by the registry ( Table 1 ). Claims also provided information not collected by registry including specific drugs used and duration of therapy. Conclusions: Matching NCDB with payer claims is possible based on limited identifiers, and quality measures can be applied using claims. Registry data in WNY NCDB hospitals did not capture some RT, HT and CT identified by claims. Though in aggregate the registries in WNY shows substantially lower treatment rates than national NCDB averages, this study demonstrates that claims enhance registry data. Linkage of claims with NCDB may be a robust tool to apply quality measures of cancer care.

No significant financial relationships to disclose.

[Table: see text]
Understanding differences in the receipt of chemotherapy between African-American (AA) and white (W) patients with stage III colon cancer
Authors: Polite B. N. , Huskey B. , McKee M. , Dignam J. J.
Published: March 2020

6568

Background: Even when stage is controlled for, AA are more likely to die from colon cancer than are W. Previous research suggests that AA are less likely to receive adjuvant chemotherapy for Stage III colon cancer than W. This study examines the differences in the receipt of chemotherapy for stage III colon cancer and the reasons behind those differences. Methods: The records of patients diagnosed with and/or receiving their first-course of treatment for Stage III colon cancer at the University of Chicago between 1995–2004 were examined. Specifically, patient charts were audited to determine whether the patients had received chemotherapy, and if not, the documented reasons for the non-receipt of therapy. In the case of incomplete records, the patients’ outside physicians were contacted to ascertain the chemotherapy history. Chemotherapy information was unavailable for only 13 patients (5 AA and 8 W). Results: A total of 186 patients (110 AA and 76 W) were diagnosed with stage III colon cancer at the University of Chicago between 1995–2004. No significant differences were seen with respect to age, sex or tumor location between AA and W. In total, 65% of AA versus 82% of W received chemotherapy (OR 0.43; 95% CI: 0.20–0.86). AA were more likely to not undergo chemotherapy because of comorbidities (OR 3.80; 95%CI 1.35–10.50). Those pts not receiving therapy because of comorbidities had a poorer overall survival than those who received therapy (HR 5.9; 95%CI 3.4–10.3). This effect held for both AA and W pts (p=0.65 for race and comorbidity interaction). Among the 146 pts (86AA, 63W) for whom it is known that chemotherapy was recommended, AA were over 9 times as likely to have a documented refusal (OR 9.5; 95% CI 1.19–75.4). These findings were robust to adjustments for age, sex, insurance status and marital status. Conclusions: AA were significantly less likely than W to receive chemotherapy for stage III colon cancer. The reasons for the difference include both refusal of therapy and the presence of comorbidities that the treating physicians felt were a contraindication to therapy. Future research should be directed at better understanding the reasons behind the higher refusal rates for AA patients.

No significant financial relationships to disclose.
Provider feedback improves reporting on quality measures: National profile reports for adjuvant chemotherapy for stage III colon cancer
Authors: Stewart A. K. , Gay E. G. , Patel-Parekh L. , Winchester D. P. , Edge S. B. , Ko C. Y.
Published: March 2020

6572

Background: The National Quality Forum recently announced accountability measures including use of chemotherapy (ACT) with Stage III colon cancer. This study examines the impact of performance reporting using cancer registry data to assess the quality of care for Stage III colon cancer patients using the Commission on Cancer's (CoC) Cancer Program Practice Profile Reports. Cancer registries are the primary source for measurement but adjuvant treatment may be incomplete. Methods: In January 2005 the CoC provided reports to 1,337 hospitals using registry data on over 80,000 Stage III colon cancer patients diagnosed from 1998–2003. Each hospital received a weighted performance rate (PR) and comparison to hospitals of similar types, nationally and regionally. Hospitals could review and correct missing or inaccurate data to provide an updated profile. Results: The initial overall hospital-level PR was 66.2%, hospitals in the top quartile had PRs =80.1%. After 24 months 603 hospitals had corrected data. The mean PR was 74.9%, an increase of 8.7%. The top quartile PR was =89.7%. The PRs for hospitals in the top quartile increased on average by 4.3%, and for hospitals outside this group by 22.7%. The proportion of hospitals with PRs =90% increased from 7.9% to 23.9%. Conclusions: With over two-fifths of hospitals correcting data, the concordance rate with ACT in Stage III colon cancer has significantly increased compared to initial registry data. Reporting quality data and allowing auditing and correction on key quality indicators corrects the data gap in cancer registry out-patient treatment data. Exposure to profile reports can spur local providers to promote better communication with centralized data repositories, significantly contributing to the assessment of care provided to cancer patients.

No significant financial relationships to disclose.
Utility of photography to confirm skin lesion response assessed using a severity weighted assessment tool (SWAT) in cutaneous T-cell lymphoma (CTCL)
Authors: Mann B. S. , Johnson J. R. , He K. , Sridhara R. , Farrell A. T. , Dagher R. , Justice R. , Pazdur R.
Published: March 2020

6579

Background: In Oct 2006, FDA approved vorinostat for treatment of cutaneous manifestations of CTCL–based on response rate and duration of response observed in CTCL skin lesions. Response was determined by change in SWAT score at follow up compared to the baseline score. SWAT Score: sum of % Body Surface Area weighted by factor 1, 2, or 4 for patch (no elevation from normal skin), plaque (< 5 mm elevation) and tumor (= 5 mm elevation), respectively. All responses but one were partial. A partial response required = 50% reduction in whole body SWAT score lasting = 4 weeks. FDA also reviewed serial photographs of all patients (standard whole body views and close-ups of skin lesions), submitted as supportive evidence. Methods: An expert external reviewer, blind to SWAT scores, adjudicated responses by examining photographs and response determinations by photographs and by SWAT scores were compared. Results: Based on SWAT scores, skin lesions in 18 of 61 stage = IIB CTCL patients responded to vorinostat; there were 17 partial and 1 complete responses. Blinded reviewer adjudicated 4 photographs not evaluable (reasons: blurred image, color or light problem, wrong label), easily confirmed 7 responses, and called 7 responses equivocal. Responses seemed easier to determine in skin lesions with tumors or ulcers. Predominantly patch or plaque lesions were harder to evaluate: patches due to poor color reproduction and plaques due to inability to palpate. A response from plaque to patch morphology decreases SWAT score 50%, but the change may not manifest adequately in a photograph. Small sample size did not allow a reliable statistical analysis for correlation between different lesion types and ability to confirm objective responses (based on SWAT scores) by photography. Conclusions: Photography allows a limited evaluation of skin lesions in CTCL: inability to measure lesion thickness in photographs limits direct confirmation of SWAT scores. Photography complements SWAT evaluation: in half the cases in the vorinostat pivotal trial, a response by SWAT score was easily confirmed by the reviewer blind to SWAT scores. Further experience and technological advances in photography may reduce the number of not evaluable photographs.

No significant financial relationships to disclose.
Patterns of care among veterans with colon cancer: Insights from California SEER, Medicare and VA-linked data
Authors: Hynes D. M. , Tarlov E. , Perrin R. , Zhang Q. , Bennett C. , Ferreira M. , Lee T. , Benson A.
Published: March 2020

6586

Background: U.S. veterans have been shown to be a vulnerable population with high cancer rates, yet practice pattern information is lacking. Linkage of cancer registry, Medicare, and VA data provides a more complete view of health status and healthcare received among veterans with cancer. Methods: As part of a national study, a retrospective cohort of incident colon cancer patients from the California Cancer Registry data, who were = 66 years old and eligible to use VA and Medicare between 1999 and 2001, were followed for three years through 2004. We examined practice patterns for stages I-IV colon cancer, specifically, surgery and use of chemotherapy, and trends in one-year mortality using descriptive and multivariate regression models. Results: Among 633 veterans with colon cancer, 93% were male; 16% were African American; 28% were diagnosed at a VA facility compared to 72% at a Medicare facility, and 553 (87%) had colectomy. One year mortality rate was 29%. Among the 553 colectomy patients, prevalence of chemotherapy use, according to stage was 8%, 27%, 61% and 52% for stages I-IV, respectively (p-value <0.0001). Regression analyses of factors affecting whether chemotherapy was received revealed that Stage III patients were most likely to receive adjuvant chemotherapy (Odds Ratio (OR) 19.80, 95% Confidence Interval (CI): 9.68–40.50). However stage II and stage IV patients were also highly likely to receive chemotherapy following surgery (OR: 4.41 CI: 2.18–8.91 and OR: 13.21; CI: 6.01–29, respectively). Patients =76 years and those =86 years were less likely to receive chemotherapy following surgery (OR: 0.61, CI: 0.40–0.94 and OR: 0.25, CI: 0.094–0.64, respectively) compared to patients 66–75 years. Patients with Charlson Comorbidity Score of 2 or greater were less likely to receive chemotherapy (OR: 0.49; CI: 0.29–0.84). Treating facility (VA or Medicare), did not affect chemotherapy use. Conclusions: Among veterans with colon cancer in California, older patients were less likely to receive chemotherapy even when stage and comorbidity status were considered. Efforts focused on improving guideline consistent adjuvant chemotherapy use among older veterans with stage III colon cancer across treating facilities may yield the greatest benefit.

No significant financial relationships to disclose.
Ethnic neutropenia among women of European, African, and Caribbean backgrounds
Authors: Grann V. R. , Bowman N. , Wei Y. , Horwitz M. , Joseph C. , Abdul K. , Abdul K. , Barlatier H. , Sandoval R. , Jacobson J. , Hershman D.
Published: March 2020

6587

Background: Black women are less likely than white women to receive complete chemotherapy for cancer. One reason for interruption of chemotherapy may be a patient's low white blood count (WBC) or absolute neutrophil count (ANC). In studies of white blood cell count (WBC) and race, white individuals’ WBC has been considered the standard, and 25–40% of non-Hispanic black individuals have been described as having “benign ethnic neutropenia.”. We therefore studied the prevalence and severity of ethnic neutropenia among women in six ethnic groups and of the association between neutropenia and serum cytokine and C-reactive protein (CRP) levels and neutrophil elastase (ELA2) polymorphisms. Methods: Following IRB approval, each participant provided a blood sample that was tested for WBC with differentials, cytokines and CRP levels, and ELA2 polymorphisms. We compared median WBC and ANC using Kruskal-Wallis tests. We used Fisher's exact tests to analyze association of severe neutropenia with ethnicity, cytokine and CRP levels, and ELA2 polymorphisms. Results: We enrolled 263 women 20–70 years old who had no cancer diagnosis.. As the table shows, median WBC and ANC differed by group; Dominicans had significantly higher median WBCs and ANCs than all other groups. Of 12 women with severe neutropenia (ANC = 1,500), none was Dominican or European-American. Subjects with a common upregulating ELA2 promoter polymorphism (C-199A) had lower ANC counts than other subjects. No associations were found between ANC counts and either cytokines or CRP levels. Conclusions: Median WBC and ANC and severe neutropenia were associated with ethnicity. Mild neutropenia is considered clinically benign, but because severe neutropenia may result in decreased doses or delays in chemotherapeutic treatment for malignancies, ethnic neutropenia and its role in treatment decisions requires further investigation.

No significant financial relationships to disclose.

[Table: see text]
Influence of health insurance status on breast cancer diagnosis and treatment delay: A safety net hospital's experience
Authors: Vakkalanka B. K. , Ruda K. , O'Brien T.
Published: March 2020

6592

Background: Given barriers to access, uninsured women with breast cancer might experience delays in diagnosis and treatment when compared to insured women. The influence of insurance status on breast cancer outcome was addressed at a single institution whose mission statement requires that all pts be treated the same, regardless of their ability to pay Methods: Records of all pts with breast cancer seen from 1/00- 12/04 at a safety net hospital were reviewed. Insurance status was defined at the time of the first abnormal mammogram. Cases were divided into Group A (private insurance), B (medicaid or medicare) or C (uninsured). Time to diagnosis (TDx) was defined as the interval from abnormal mammogram to pathologic diagnosis of breast cancer. Time to treatment (TRx) was defined as time from pathologic diagnosis to definitive surgical therapy. Results: 560 cases were reviewed. Group A: 48%, B: 33%, C: 19%. Median age= 57 (GrpA=53, B= 67, C= 55). Race Caucasian 59%; AA 34%; other 7%. Stage (0, I, II, III and IV, in %, respectively): Grp A= 14.6, 42.9, 31.5, 7.1 and 3.9; Grp B= 13.1, 46, 22.7, 12.5 and 5.7; Grp C = 13.9, 33.7, 32.7, 11.9 and 7.9. No significant diff between groups, although there was a trend towards later (III/IV) pts in grps B and C than A (18.2% and 19.8% vs 10.9%, respectively). Mean TDx: Grp A= 50.6 days, B= 68.1d and C=52.8d; p= 0.006 when Group B was compared to the other two groups but there was no difference between uninsured and private groups. Mean TRx: 43.8d, 48.6d and 47.8 days, respectively, for groups A, B and C, without any statistical difference between any of the groups. Conclusions: A 5-year review of breast cancer cases at a large safety net hospital did not reveal any difference in the time to diagnosis or initiation of treatment for insured vs. uninsured pts. Uninsured and medicaid/medicare pts tended to present with later stage disease, perhaps due to access problems. A statistically significant delay in diagnosis was noted in the medicaid/medicare group but it is unlikely that this difference (18 days) was clinically important. Because uninsured pts are provided care at greatly discounted rates at our institution, these results may differ from those at other institutions. Further studies addressing this issue are warranted.

No significant financial relationships to disclose.
Solid tumor cancer patients treated with zoledronic acid experienced reduced skeletal related events as compared to untreated patients in clinical practice claims database
Authors: Hatoum H. T. , Lin S. , Smith M. R. , Barghout V. , Koukouras K. , Lipton A.
Published: March 2020

6596

Background: Patients (pts) with bone metastasis are at high risk for skeletal related events (SREs), leading to increased morbidity, mortality, and decreased QOL. Methods: To study the impact of zoledronic acid on SRE rates, follow-up duration (from 1st bone metastasis diagnosis to last claim) in treated pts as compared to the experience of non bisphosphonate-treated pts (untreated), and to investigate relative impact of zoledronic acid (ZA) frequency of administration using data from PharMetrics integrated claims database of 80 health plans across the U.S, from Jan 02 to Oct. 05. Results: There were 1,518 ZA treated-pts and 3308 untreated-pts, who met study inclusion criteria of having breast, prostate, or lung cancers, with confirmed bone metastasis and SRE experience. At baseline, breast cancer pts (N=1,799) had lowest Charlson Co-morbidity Index (CCI); lung cancer pts (N=2,413) had highest CCI (p<0.05). Prostate cancer pts (N=622) were significantly older (p<0.05). The average monthly ZA prescription rate (Rx) was 0.73 with average total Rxs of 7.82 over the follow-up duration. ZA use reduced monthly SRE rate by 30% from 0.41 ± 0.4 in untreated pts to 0.29 ± 0.3 in treated pts (p<0.001) and prolonged follow-up durations in months from 9.4 ± 9.8 in untreated to 12.2 ± 9.0 in treated pts (p<0.001). Regression models of SRE rate or follow-up duration as the dependent variable investigating the impact of ZA prescribing patterns indicated that pts who followed ZA label dose of 4 mg every q3, q4 experienced the lowest SRE rate (p<0.001) and had longer follow-up duration (p<0.005) as compared untreated pts. ZA use almost doubled time to 1st SRE from 101±165 days in untreated pts, to 185±210 days in treated pts (p<0.0001). ZA also delayed time between 1st to 2nd SRE from 85 days in untreated to 111 days in treated pts (p<0.05). Conclusion: Zoledronic acid significantly reduced SRE rate in cancer pts with bone metastasis by 30%, increased follow-up duration by almost 3 months, doubled time to 1st SRE, delayed time to 2nd SRE, with its label dose of 4 mg every 3 to 4 weeks achieving the best outcome.

No significant financial relationships to disclose.
Concordance of study objectives with efficacy findings in metastatic breast cancer (MBC) treatment trials
Authors: Frame D. , Ross S. D.
Published: March 2020

6600

Background: Despite ongoing development of new agents, overall survival in MBC remains discouraging. We reviewed large randomized controlled trials (RCTs) published since 2000 to compare stated objectives with efficacy endpoints. Methods: Systematic review of RCTs of systemic antineoplastic treatment in advanced / metastatic breast cancer, published 2000–2006 in English. Studies enrolling fewer than 100 patients or performed only at sites outside North America and Europe were excluded. Study success was characterized with regard to the primary endpoint as described in prospective power calculations. Results: 68 RCTs, enrolling 24,956 patients, met inclusion criteria. The 68 RCTs comprised 83 efficacy comparisons due to multiple arms and/or more than one primary endpoint. Frequency of success was similar by geographic location and presence of industry sponsorship, but higher for noninferiority comparisons ( Table 1 ). 5 of 13 non-inferiority studies reported unplanned superiority findings for the experimental arm, while 11 of 39 studies failing to meet planned superiority endpoints made statements regarding equivalence or superiority to control treatments. For 10 of 20 successful superiority studies, reported results did not match/exceed planned between-group differences, though the comparisons reached statistical significance: 4 such studies substantially exceeded planned enrollment (2 of which recalculated sample size after reviewing interim data), while reasons for attaining superiority in 6 cases were unclear. Of the remaining 10 superiority comparisons, 4 reached significance due to underperforming control arms (enabling PFS/TTP differences of <2 months to establish superiority), 2 exceeded point estimates, and 4 exceeded estimated between- group ratios. Conclusions: Slippage from prospective study goals, even in large randomized trials, may contribute to a disconnect between the profusion of research activity and disappointing efficacy gains in MBC.

No significant financial relationships to disclose.

[Table: see text]
Lack of effect of prophylactic hematopoeitic colony stimulating factors in cancer patients and stem cell transplant recipients on short term survival: A meta-analysis of randomized trials
Authors: Sung L. , Nathan P. C. , Alibhai S. M. , Tomlinson G. A. , Beyene J.
Published: March 2020

6605

Background: Previous randomized trials and meta-analyses have failed to determine with certainty whether prophylactic hematopoietic colony-stimulating factors (CSFs) influence survival in adults and children receiving cancer chemotherapy or undergoing stem cell transplantation (SCT). The primary objective was to determine whether prophylactic CSFs improve short-term overall survival. Secondary objectives were to determine whether prophylactic CSFs affected infection-related mortality, documented infections, microbiologically documented infections or febrile neutropenia. Methods: We performed electronic searches of OVID Medline from 1966 to November 2006; EMBASE from 1980 to November 2006; and the Cochrane Central Register of Controlled Trials until the second quarter, 2006. We included studies if there was randomization between CSFs and placebo/no therapy; CSFs were given concurrently with or following initiation of chemotherapy in the prophylactic setting; and chemotherapy/conditioning regimen or supportive care were not planned to be systematically different between study arms. From 4,019 reviewed titles and abstracts, 145 studies were included. Independent extraction was performed by two observers using a standardized data collection sheet. Results: Prophylactic CSFs did not affect short-term overall survival, with a relative risk (RR) of 1.00 (95% confidence interval [CI] 0.99 to 1.01; P=.9). This result was consistent across different patient populations (including patient age and underlying disease) and study characteristics. The effect of prophylactic CSFs on infection-related mortality was less clear (RR 0.83, 95% CI 0.67 to 1.03; P=.09). Intervention did reduce documented infections (rate ratio 0.85, 95% CI 0.78 to 0.92; P<.00001), microbiologically documented infections (rate ratio 0.85, 95% CI 0.75 to 0.95; P=.004) and febrile neutropenia (rate ratio 0.70, 95% CI 0.62 to 0.79; P<.00001). Conclusions: Prophylactic CSFs do not improve short-term overall survival but are associated with decreased rates of infection. Decision-making around this intervention should be based upon further exploration of costs, preferences and quality of life.

No significant financial relationships to disclose.
Clinical variables are associated with treatment satisfaction in patients with breast, lung, and colorectal cancer
Authors: Trask P. C. , Tellefsen C. , Espindle D. , Getter C. , Yost K. , Hsu M.
Published: March 2020

6608

Background: The association between clinical variables (e.g. disease stage/type, side effects, ECOG performance status (PS)) and patient-reported treatment satisfaction has received little attention, despite the potential implications for treatment adherence and decision-making. We examined the relation between clinical variables and treatment satisfaction in a U.S. sample of patients with breast (BC), lung (LC), or colorectal (CRC) cancer using the Cancer Treatment Satisfaction Questionnaire (CTSQ), a recently validated 16-item measure designed to assess expectations of therapy (ET), feelings about side effects (FSE), and satisfaction with therapy (SWT). Methods: Cancer stage, line of therapy, physician-reported ECOG PS, presence vs. absence of medication side effects, and perceived change in cancer over the last two weeks were obtained on patients. These clinical variables were examined in relation to the ET, FSE, and SWT scales of the CTSQ. Higher scale scores indicated better outcomes (e.g., better satisfaction). Group means were compared using one-way ANOVAs; trends of scale scores across clinical variables were also examined. Results: Participants were 349 patients with early and late stage BC (n=137), CRC (n=121), or LC (n=91) who had ≥ 1 cycles of chemo-, bio-, or hormonal therapy in the last 6 months. In all cancer types, FSE differed by presence of side effects (BC<.03; CRC<.01; LC<.02). In BC patients, FSE differed by stage, and ET differed significantly depending on ECOG PS (p<.006) and perceived change (p<.05). For CRC patients, ET significantly differed by stage (p<.05) and perceived change (p<.02); and SWT by presence of side effects (p<.01). SWT also differed by perceived change (p<.01) for LC patients. Most trends were in the expected direction. Conclusions: These results indicate that patient-reported satisfaction with therapy is correlated with clinical variables. On average, patients who perceive less improvement in their cancer, have side effects, and poorer ECOG PS are less satisfied with their current cancer therapy. Unknown is the impact of this relation on adherence and decision-making. Recent changes in healthcare suggests that treatment satisfaction may become an increasingly important measure of clinical outcome.

No significant financial relationships to disclose.
Utility of the of the M. D. Anderson Symptom Assessment Inventory (MDASI) for symptom evaluation during chemoradiation (CRT) in patients with gastrointestinal malignancies
Authors: Janjan N. A. , Wang X. S. , Mendoza T. , Zhang Y. , Das P. , Crane C. , Delclos M. , Krishnan S. , Cleeland C.
Published: March 2020

6613

Background: Symptom assessment has become an important therapeutic endpoint in clinical trials. Measures that evaluate symptoms during CRT are needed for evaluation of clinical benefit in developing therapeutics and in monitoring quality improvement in clinical practice. Methods: 330 patients with gastrointestinal malignancies (Liver/Biliary:44; Colon:45; Rectal:197; Gastric:44) were evaluated during CRT with the self-reported MDASI-GI. All patients received 45–56Gy of radiation (1.8Gy/fraction); 78% received between 45–50.4Gy. In all cases fluoropyrimidine (oral or intravenous) was given concurrently during radiation. Lowess curves were used to describe the severity and pattern of each symptom evaluated. ANOVA was used to compare the mean severity of symptoms by tumor type. Follow-up evaluations were performed with a t-test using a Bonferroni corrected level of significance [p< 0.002]. Results: Relationships among symptoms were examined using 1,020 symptom measurements (Liver/Biliary:141; Colon:132; Rectal:613; Gastric:134 observations). Lowess curves showed almost parallel steep increases in pain and poor appetite between weeks 2–4 of CRT. Fatigue was most profound in Liver/Biliary and Rectal cancers. As expected, no significant differences in dyspnea were found among these cancers. CRT for Rectal cancer produced the worst symptom burden with significantly more pain, skin pain, and diarrhea. Consistent with clinical findings, poor appetite, nausea and vomiting were statistically more common during CRT for Gastric cancer. Conclusions: The MDASI is a brief and easy to use symptom measure in the clinical setting and is sensitive to treatment-related symptom changes during CRT in abdominopelvic malignancies.

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Medicare 2005 demonstration project: Patient reporting of nausea symptoms and its impact on improving quality of patient care
Authors: Mueller B. , Page R. D.
Published: March 2020

6615

Background: In 2005, the Centers of Medicare and Medicaid (CMS) conducted a one-year Oncology Demonstration Project (ODP) establishing 12 G-codes for participants to provide and document specific services related to (1) pain control, (2) fatigue reduction, (3) and minimization of nausea and vomiting. Methods: This is a retrospective study involving a chart review and G-code reporting to determine the effectiveness of the 2005 ODP to improve the quality of patient care. There were 133 patients that reported level 3 or 4 nausea and vomiting symptoms (per G-codes) during the administration of the 2005 ODP. Analysis of the 133 patient charts attempted to identify if the 2005 ODP helped to improve three areas of antiemetic care: (1) increase awareness of patients suffering from severe nausea and vomiting, (2) improve antiemetic interventions once the nausea and vomiting symptoms of the patient had been assessed, (3) and improve the ability for subsequent antiemetic changes once a previous antiemetic intervention was unsuccessful. Data were analyzed comparing Medicare ODP vs. a non-medicare reporting group using Chi-square analysis. Results: The 292 cases of reported level 3 or 4 nausea and vomiting symptoms received a proper antiemetic assessment 57% of the time. Also, when a patient received an assessment for a reported level 3 or 4 nausea and vomiting symptom, an intervention occurred 74.5% of the time. This 74.5% was statistically insignificant (P=0.3283) when compared to the 70% of antiemetic interventions that occurred when a nausea and vomiting assessment was made without the reporting mechanism of the 2005 ODP. And finally, there was statistical insignificance (P=0.2389) when comparing the change of antiemetic care, with and without the administration of the 2005 ODP (89% vs. 85%, respectively), once a previous antiemetic intervention was unsuccessful. Conclusions: Based upon the three criteria of improving patient outcomes of nausea and vomiting, the 2005 ODP and the reporting of G-codes did not aid the ability of oncologists to administer better quality of patient care in the area of antiemetic management.

No significant financial relationships to disclose.
Relationship between compliance with imatinib mesylate and medical costs for patients with CML and GIST
Authors: Halpern R. , Barghout V. , Williams D.
Published: March 2020

6618

Background: Imatinib mesylate, an oral tyrosine kinase inhibitor for treatment of chronic myeloid leukemia (CML) and gastrointestinal stromal tumors (GIST), has been found both effective and cost-effective. This study examines the relationship between imatinib compliance and health care costs for patients (pts) with CML and GIST. Methods: This retrospective study used claims data from a large national US health plan. Pts had =2 imatinib claims from 6/1/01–3/31/05; =1-year follow-up after the 1st imatinib claim; and diagnosis of CML (ICD9 205.1x) or GIST (ICD9 159.0, 159.8, 159.9 or evaluation of claims and attribution of GIST by independent hematologist/oncologist). Compliance was measured with a medication possession ratio ((days of imatinib during follow-up / days of follow- up)*100). Compliance categories were good, =90%; medium, 70–89.9%; poor, <70%. Total follow-up medical and health care (medical + pharmacy) costs were measured and compared across compliance categories; t-tests compared mean costs. Results: 374 CML and 91 GIST pts had mean age of 52.1 ± 14.0 years. 274 (59%) pts were male. 234 (50%) of pts had 12–24 months follow-up; 231 (50%) had 25–57 months. Mean and median compliance across all pts were 69.4% and 79.7%, respectively. 122 (33%) CML and 32 (35%) GIST pts had good compliance; 149 (40%) CML and 42 (46%) GIST pts had poor compliance. Mean medical costs across all pts were lower and less variable with good compliance ($22,882 ± 22,791) than with medium ($40,366 ± 68,186), p=0.007) and poor ($104,961 ± 190,559), p<0.001). Similarly, mean medical costs for CML pts with good, medium, and poor compliance were $21,456 (±22,127), $41,537 (±72,238, p=0.008), and $117,004 (±211,292, p<0.001), respectively. Mean medical costs for GIST pts were: good=$28,318 (±24,781); medium=$33,270 (±35,356, p=0.584); and poor=$62,235 (±68,751, p<0.001). Mean total health care costs were 21.7% lower for pts with good compliance relative to those with poor. Conclusions: Good imatinib compliance was associated with significantly lower medical costs. Mean total medical costs were 78% lower with good compliance relative to poor. Compliance is an important treatment issue for both clinical and medical cost outcomes.

No significant financial relationships to disclose.
Patterns of care regarding adjuvant hormonal agents and treatment of bone health in postmenopausal women with breast cancer in community and academic centers before and after the availability of ATAC (Adjuvant Tamoxifen, Armidex or Combination) study
Authors: Tummala M. K. , Wajahath M. , Kotlarewsky M. , Aggarwal A. , Muller D. , Liu M. C. , McGuire W. P. , Ottaviano Y.
Published: March 2020

6621

Background: Results from the ATAC trial comparing Tamoxifen to the Aromatase inhibitors (AIs) anastrozole in PM women with early stage breast cancer were initially presented in San Antonio, Texas, in December 2001. ASCO issued guidelines for the adjuvant use of AIs in 2002, updated in 2003. We compared patterns of usage of adjuvant hormonal agents and bone health management before and after availability of the ATAC data in community versus academic centers. Methods: We conducted a retrospective analysis of 432 patients between 1999–2005 from group practices affiliated with two large community hospitals and one academic center. Data were collected from tumor registries regarding demographics, first-line hormonal agent choice, and use of bone density studies, vitamin D/calcium supplements and bisphosphonates. Results: Demographics were identical in both groups before and after January 2002. Before 2002, 96% of the patients were prescribed Tamoxifen in both community and academic centers. After the initial presentation of the ATAC data, 55.08% (65/118) of patients from the community centers versus only 17.11% (19/111) from the academic center were prescribed AIs (p=0.0001). Of the 84 patients who received an AI after January 2002, similar proportions of patients had baseline bone density scans (38.5% community vs. 36.8% academic; p=0.89) and follow up annual/biannual scans (33 % vs. 32%; p=0.85). In addition, similar proportions of patients on AIs were prescribed calcium/vitamin D supplements (47.4% vs. 52.6%; p=0.69) and bisphosphonates (36.8% vs. 21.05%; p=0.20) in community and academic centers, respectively. Conclusions: Community oncologists adopted AIs into clinical practice sooner than academic physicians on the basis of unpublished clinical trial results, even before ASCO published guidelines. Although patients on AIs are deemed to be at higher risk for bone fractures, fewer than 40% were evaluated with baseline or surveillance bone density scans in both community and academic practices. Similar proportions of patients received calcium/vitamin D supplements or bisphosphonates among centers.

No significant financial relationships to disclose.
The implementation of a rapid response team at Memorial Sloan-Kettering Cancer Center: A preliminary report
Authors: Klotz A. D. , Thattassery E. , Graham D.
Published: March 2020

6627

Background: The implementation of a rapid response team (RRT) has become a popular patient safety initiative at many health care institutions during the past decade. To date, there are no studies evaluating the utilization or impact of a rapid response team in a population of cancer patients. We report on our experience implementing such a team at Memorial Sloan-Kettering Cancer Center. Methods: A consecutive cohort of all consults performed by the RRT between July 1st and December 31th, 2006 had the following variables recorded: primary cancer diagnosis, reason for consult, RRT intervention. Outcomes measured were survival to discharge and 30 day survival. Hospital mortality and hospital codes were compared to historical data from 2005 and were adjusted for hospital census. Results: There were 195 consults requested between July 1st and December 31st, 2006. The majority of these patients were on the medical service (124 vs 66 on surgical service). The most common primary malignancies for which the RRT was called arose from the gastrointestinal tract (30.2%), thoracic cavity (12.8%), urologic tract (9.3%), and breast (7.6%). Most consults were requested for compromised respiratory function (n=76), cardiovascular (n=72, 34 hypotension, 23 tachycardia), and neurologic derangements (n=13). 33 patients (16.9%) required transfer to an alternate level of care such as telemetry or the intensive care unit. 121 patients (65%) seen by the RRT survived to discharge however 92 (47%) died within 30 days of an RRT consult. Hospital codes decreased 21% (55 vs 70, p<0.10) and hospital mortality decreased 22% (3.43 deaths per 1,000 discharges vs 4.43 deaths per 1,000 discharges) following implementation of the RRT although these results did not achieve statistical significance. Conclusions: The Rapid Response Team at MSKCC assists in the acute management of hospitalized cancer patients. Many of these patients are critically-ill and are near the end of their oncologic course. Further studies are necessary to better characterize the impact of this resource and determine how it can be utilized most effectively.

No significant financial relationships to disclose.