Mau, Lih‐Wen

Abstract Palliative care (PC) benefits patients undergoing hematopoietic stem cell transplantation (HSCT), but it remains underutilized. Although transplant physicians report concerns regarding how patients perceive PC, HSCT recipients’ perceptions about PC remain unaddressed. We conducted a multisite, cross-sectional survey of autologous and allogeneic HSCT recipients 3 to 12 months after transplant to assess their familiarity, knowledge, and perception of PC, as well as their unmet PC needs. We computed a composite score of patients’ perceptions of PC and used a generalized linear regression model to examine factors associated with these perceptions. We enrolled 69.6% (250/359) of potential participants (median age = 58.1; 63.1% autologous HSCT). Overall, 44.3.8% (109/249) reported limited knowledge about PC and 52% (127/245) endorsed familiarity with PC. Most patients felt hopeful (54%) and reassured (50%) when they heard the term PC; 83% saw referral as a sign their doctor cared about what was happening to them. In multivariate analyses, patients who were more knowledgeable about PC were more likely to have positive perceptions of PC (B = 7.54, standard error = 1.61, P < .001). Patients’ demographics, HSCT features, quality of life, and symptom burden were not significantly associated with perceptions of PC. HSCT recipients have positive perceptions of PC, though many have limited knowledge about its role. Patients who were more knowledgeable about PC were more likely to have positive perceptions of PC. These data do not support transplant physicians’ negative concerns about how patients perceive PC and underscore the need to further educate patients and transplant physicians about PC.

Abstract Background: Palliative care (PC) benefits patients with cancer and those undergoing hematopoietic stem cell transplantation (HSCT), but remains under-utilized in HSCT. While transplant physicians have substantial concerns regarding how patients perceive PC, studies examining HSCT recipients' perceptions of PC are lacking. Methods: Between 11/2020-4/2021, the CIBMTR Survey Research Group conducted a multi-site cross-sectional survey of adult autologous and allogeneic HSCT recipients at 11 transplant centers in the United States who were 3-12 months post-transplant to assess their experience, knowledge, and perceptions of PC. Using validated questionnaires, we assessed patients' familiarity with PC, knowledge and perception of PC, unmet PC needs, quality of life, and symptom burden. We computed a composite score of patients' perceptions of PC (mean = 48.8, SD = 12.2). We used a generalized linear regression model to examine factors associated with patients' perceptions of PC. Results: Of 359 potentially eligible patients, 250 (69.6%) enrolled in the study and 249 were eligible for analysis. Median participant age was 58.1 years (IQR 16.6). Most were white (85.9%), non-Hispanic or Latino (95.2%), and male (53.0%). Overall, 63.1% underwent autologous HSCT. The most common diagnoses were multiple myeloma (42.2%), lymphoma (22.1%), leukemia (16.9%), and MDS/MPN (12.4%). Overall, 44.2% (110/249) of patients reported limited knowledge about PC and 51.0% (127/249) endorsed being familiar with PC. Most patients stated that when they hear the term PC, they feel hopeful (54%), and reassured (50%) while a minority reported feeling scared (21%), stressed (22%), or depressed (15%) [Figure 1]. Most endorsed that when a PC referral is suggested for them, they think their doctor really cares about what is happening to them (83%), and only 7% of patients stated that when a PC referral is suggested, they think their doctor has given up on them [Figure 2]. In multivariate analyses, patients who were more knowledgeable about PC were more likely to have a positive perception of PC (B = 7.54, SE=1.61, P < 0.001) compared to those who had less knowledge about PC. Patients with postgraduate education were more likely to have a negative perception of PC (B = -5.55, SE=2.60, P = 0.034) compared to those with high school education or less. Patients' demographics, HSCT type, diagnosis, unmet PC needs, quality of life, and symptom burden were not statistically significantly associated with their perceptions of PC. Conclusions: A substantial proportion of HSCT recipients report limited knowledge and familiarity with PC. Most patients reported positive perceptions of PC, and those who were more knowledgeable about PC were more likely to have positive perceptions of PC. These data do not support transplant physicians' concerns about how patients perceive PC and underscore the need for future interventions to further educate patients about PC to overcome barriers to PC utilization in HSCT. Figure 1 Figure 1. Disclosures Burns: Astellas Pharma Inc.: Research Funding; Medac GmbH: Research Funding; Sanofi: Research Funding; OncoImmune: Research Funding; Fate: Research Funding; Bristol Meyers Squibb: Research Funding; PrioThera: Research Funding; Kyowa Kirin International: Research Funding. Murthy: CRISPR Therapeutics: Research Funding. LeBlanc: Amgen: Consultancy, Other: travel; Pfizer: Consultancy, Other: Advisory Board; AstraZeneca: Consultancy, Honoraria, Other: Advisory board, Research Funding; Flatiron: Consultancy, Other: Advisory board; Agios: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; BMS/Celgene: Consultancy, Honoraria, Other: Travel fees, Research Funding, Speakers Bureau; Daiichi-Sankyo: Consultancy, Honoraria, Other: Advisory board; AbbVie: Consultancy, Honoraria, Other: Advisory board; Travel fees, Speakers Bureau; Helsinn: Consultancy, Research Funding; Duke University: Research Funding; Jazz Pharmaceuticals: Research Funding; UpToDate: Patents & Royalties; NINR/NIH: Research Funding; CareVive: Consultancy, Other, Research Funding; Otsuka: Consultancy, Honoraria, Other; American Cancer Society: Research Funding; Seattle Genetics: Consultancy, Other: Advisory board, Research Funding; Astellas: Consultancy, Honoraria, Other: Advisory board; Heron: Consultancy, Honoraria, Other: advisory board.

Abstract Introduction: Allogeneic hematopoietic cell transplantation (alloHCT) is the only potentially curative treatment available for acute myelogenous leukemia (AML). It is a medically complicated and resource intensive procedure for which there are patient-, provider- and system-related factors that may impact its utilization. While Medicare coverage for alloHCT may help address a major financial barrier to access, there is still an unmet need due to other factors. This study intended to examine trends and factors associated with utilization of alloHCT and to estimate unmet need for alloHCT among Medicare beneficiaries with AML. Methods: This retrospective cohort study included all patients with a diagnosis of AML identified in the Medicare claims data from 2010 through 2016. Primary patient selection criteria included: primary or secondary diagnosis of AML, age limit (65-74), and continuous enrollment (for at least 180 days after AML diagnosis in Part A and Part B fee-for-service programs). To study trends in utilization, the transplant rates were calculated as the number of patients who received an alloHCT within 180 days and one year of diagnosis (numerator) divided by the total number of patients diagnosed with AML within each diagnosis year (denominator). Transplant rates within one year of diagnosis were further adjusted by patient characteristics, including age group, sex, race, residential region, and Elixhauser Comorbidity Index (ECI). A multivariable logistic regression was utilized to identify factors associated with the likelihood of receiving alloHCT within one year of diagnosis. Two approaches were applied to estimate unmet need for alloHCT. The first approach (Approach 1) used claims data to identify the potential need for alloHCT among patients who achieved complete remission for at least 90 days; patients who achieved 90-day remission but did not receive alloHCT at any time point were considered to have unmet need. In the second approach (Approach 2), the total number of patients diagnosed with AML in the claims data was run through the National Marrow Donor Program (NMDP) methodology , which takes estimates of risk level, response to treatment, comorbidity, and early mortality into consideration. Overall estimated need and unmet need from 2010-2015 and over different time periods were evaluated for both approaches. Results: Among the 5,974 patients diagnosed with AML from March 1, 2010 through June 30 th, 2016, 1226 patients (21%) received an alloHCT by the end of 2016. Trend analysis results suggest that utilization of alloHCT increased from 2010 to 2016 (P < 0.0001) (Figure 1). The likelihood of receiving alloHCT within one year of diagnosis was found to be associated with diagnosis year, age, race, geographic region, ECI, and population-level median household income (Table 1). Both approaches estimated that approximately 36% of the diagnosed patients were in need of alloHCT between 2010 and 2015. The overall unmet need was estimated as 59% and 43% based on the claims data approach and the NMDP methodology, respectively. Despite the differences in estimated unmet need between the two approaches, the unmet need for alloHCT was found to trend down over time (Figures 2 & 3). Discussion and Conclusions: Medicare coverage facilitates access to the health care system and receipt of health services when a need for treatment or care is recognized. Utilization of alloHCT has increased over time among Medicare beneficiaries with AML. However, there are persistent differences in utilization of alloHCT by age, race, geographic region, comorbidity, and socioeconomic status, indicating disparities in access to alloHCT among this population. The results of estimated need and unmet need for alloHCT may be affected by the lack of cytogenetics and molecular information in administrative claims. To minimize this limitation, we used NMDP methodology for validation and found a similar estimation of potential need for alloHCT and downward trend in unmet need. Although the unmet need for alloHCT for AML has improved over time, policy efforts, research, and continued education are needed to close the gap between the actual utilization of alloHCT and unmet need for this potentially curative treatment. Figure 1 Figure 1. Disclosures Saber: Govt. COI: Other.

e18862 Background: Hematopoietic cell transplantation (HCT) is a medically complicated, expensive and resource intensive medical technology. High health care costs are usually associated with high Out-of-Pocket (OOP) costs. In this population-based study, using deidentified administrative claims data from OptumLabs Data Warehouse, we describe total and patient level costs for one-year pre and post HCT and describe the characteristics of those with top 25% cost. Methods: Patients who had undergone an autologous or allogeneic HCT in 2015 to 2019 and had continuous health plan coverage for one year prior and post the index date-the date of the first claim for HCT, were included. We examined total costs, patient paid (OOP) and plan paid costs for one year before and after HCT. We also examined inpatient, outpatient, and pharmacy costs for the one year before and after the HCT. Logistic regression models examined factors including age, gender, race/ ethnicity, Charlson comorbidity index, geographic region and costs in the year prior to HCT (baseline costs) for their association with high total costs and high OOP costs (top 25%). Results: A total of 3,346 patients (2,344 commercial plan (CPE) and 1,002 under Medicare Advantage plan (MAPE) enrollees) were included in the study. Median one year post HCT costs for CPE was $ 612,517 (IQR 413,348- 960,456) and for MAPE was $ 521,000 (IQR 347,388-736,685). Median one year post HCT OOP costs were $ 5,407 (IQR 1,584 -10,000) in CPE and $7,199 (IQR 3,485 -16,396) in MAPE group. 625 subjects in the CPE and 253 subjects in MAPE group had overall costs in the top 25%. Median baseline costs were $482,107 vs. $846,943 in the low vs. high cost group in CPE and $278,656 vs. $343,633 in MAPE. There was a low correlation between OOP costs and overall costs (r = 0.17 for CPE and 0.15 for MAPE; p < 0.05). Younger age (OR for age 0.98; p < 0.001) and higher baseline costs (OR1.89; p < 0.001) predicted top 25% total costs post HCT in the CPE group. Not being African American (OR for African American 0.5; p = 0.02) and higher baseline costs (OR 1.07; p < 0.001) predicted top 25% total costs post HCT in MAPE group. Age (OR 0.97; p < 0.001), not being African American (OR 0.5; p = 0.001) and high baseline costs (OR 1.44; p < 0.001) also predicted higher OOP costs in the CPE group. The only factor that was associated with lower likelihood of OOP costs in top 25% in MAPE group was being Hispanic (OR 0.6; p = 0.004). Conclusions: HCT is an expensive treatment modality. High costs prior to HCT are associated with high post HCT costs both for patients on commercial and Medicare Advantage plan. The magnitude of correlation between patient level and total costs was low, likely due to differences in benefit plan. A careful assessment of benefits and costs of HCT is needed for providing high-value care and allow optimum allocation of the finite resources while minimizing patient level financial burden.

Abstract Introduction: Allogeneic stem cell transplant is an intensive procedure, offered in a limited number of medical centres. We sought to describe how sociodemographic variables impacted access to transplant across the United States, and if disadvantaged populations had inferior access to transplant. Methods: Data from the Surveillance, Epidemiology and End Results Program (SEER) and the Center for International Blood and Marrow Transplant Research (CIBMTR) was integrated to determine the rate of unrelated donor transplantation between 2000 and 2010 in each of the 612 counties included in the SEER registry. Patients under the age of 65 with AML, ALL, and MDS were included, and the analysis was restricted to unrelated donors due to limited availability of ZIP code in CIBMTR data. New incident cases were determined from SEER, and the number of transplants was determined from CIBMTR. The transplant rate was calculated (transplants performed divided by incident cases) for each county. County attributes (percent minority, rural/urban status, percentage below the poverty line, and median family size) were obtained from US Census data. Poisson regression was used to describe how county attributes impacted transplant rates. Transplant rates were calculated separately for AML, White residents, and pediatric ALL. Results: 3147 patients were identified in the CIBMTR dataset that met inclusion criteria. The estimated ZIP code completeness was 75%. There were 30,468 new incident diagnoses of ALL, AML, and MDS. For AML, patients from rural areas (less than 20,000 residents) and patients from areas with higher poverty levels had lower transplant rates (Table 1). Minority status and family size did not impact transplant rate. In regression models, higher levels of poverty remained associated with lower transplant rates, while rurality did not (Table 2). The results were similar among White residents. In contrast, in pediatric ALL, no county attributes (poverty, rurality, percent minority, and family size) were significantly associated with a difference in transplant rate (Table 1). However, numbers of transplants were smaller, limiting power. Discussion: Patients with AML from disadvantaged areas had lower rates of unrelated donor transplant. While patients from disadvantaged areas were also more likely to be non-White, and non-White Americans are less likely to have an available unrelated donor, this difference was also seen in White Americans from disadvantaged areas. This suggests the lower transplant rate is due impaired access to transplant. Poverty rate was the most important predictor of transplant rate. The results of this study suggest that improving access to transplant in disadvantaged populations should be a priority for health care administrators. Based on these results, approximately 2500 Americans do not undergo allogeneic transplant annually due to inferior access associated with higher poverty rates. Table 1 Univariate Analysis Table 1. Univariate Analysis Table 2 Acute Myeloid Leukemia, Regression Model * Metropolitan = county > 50,000 people, micropolitan = county > 20,000, rural county < 20,000. Table 2. Acute Myeloid Leukemia, Regression Model. / * Metropolitan = county > 50,000 people, micropolitan = county > 20,000, rural county < 20,000. Disclosures Hahn: Novartis: Equity Ownership; NIH: Research Funding.