Health Care Reimbursement, Service Utilization, and Outcomes among Medicare Beneficiaries with Multiple Myeloma Receiving Autologous Hematopoietic Cell Transplantation in Inpatient and Outpatient Settings.

Autologous hematopoietic stem cell transplantation (auto-HCT) is a complex procedure that can be performed in both inpatient (IP) and outpatient (OP) care settings. We examined reimbursement, service utilization, and patient financial responsibility among Medicare beneficiaries with multiple myeloma who underwent auto-HCT in the IP and OP settings using a merged dataset of the Center for International Blood and Marrow Transplant Research observational database and Centers for Medicare & Medicaid Services Medicare administrative claims data. Selection criteria included first auto-HCT, time from diagnosis to auto-HCT <18 months, and continuous enrollment in Medicare Parts A and B for 30 days before HCT index claims and 100 days post-HCT or until death. Total reimbursement and patient responsibility were adjusted for patient and disease characteristics using a weighted generalized linear model. The final cohort comprised 1640 patients, 1445 (88%) who received IP-HCT and 195 (12%) who received OP-HCT. The adjusted total mean reimbursement was higher for IP-HCT compared with OP-HCT ($82,368 [95% CI, $77,643 to $87,381] versus $46,824 [95% CI, $43,567-$50,325]; P < .0001). Adjusted total mean patient responsibility was $4736 for IP-HCT (95% CI, $4731 to $5133) and $6944 for OP-HCT (95% CI, $6296 to $7658) (P < .0001). Within 100 days post-HCT, 107 of the 195 OP-HCT recipients (55%) had at least 1 subsequent admission, compared with 348 of the 1445 IP-HCT recipients (24%). Reimbursement, service utilization, and financial responsibility varied by HCT setting. As the number of Medicare beneficiaries who undergo auto-HCT increases, coverage policy needs to consider how location of services leads to variations in the financial burden for both hospital systems and patients.

Information Needs for Treatment Decision-making of Hematopoietic Cell Transplant Patients 65 Years or Older and Caregivers.

Hematopoietic cell transplantation (HCT) is a complex and potentially life-threatening treatment option for patients with hematologic malignant and non-malignant diseases. Advances have made HCT a potentially curative treatment option for patients 65 years of age and older (older patients), and patient education resources should be adapted to meet their needs. To better understand the information needs of older patients and their caregivers for HCT treatment decision-making, the National Marrow Donor Program® (NMDP)/Be The Match® conducted a qualitative comprehensive needs assessment. Focus groups, offered in person or by phone, were conducted with older HCT patients and primary caregivers of older HCT patients at three transplant centers in the USA that were selected based on the number of older adults treated and geographic diversity. The one-hour, semi-structured discussions were recorded and transcribed verbatim. The analysis was performed with the NVivo 10 software for identification of conceptual themes. Five telephone and six in person focus groups of patients (n = 35) and caregivers (n = 10) were conducted. Themes that emerged included the following: (1) the need for tailored resources with age-specific recovery expectations; (2) the need for the right amount of information at the right times; and (3) the benefit of peer support. Effective patient education supports learning and treatment decision-making. As HCT increasingly becomes a treatment option for older patients, tailored educational resources are needed. These focus group results can inform and guide the development of new educational resources for older adults with hematologic diseases considering and planning for HCT.

Addressing Knowledge Gaps in Acute Myeloid Leukemia to Improve Referral for Hematopoietic Cell Transplantation Consultation.

BACKGROUND: Outcomes after hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) are better when HCT is performed during first complete remission (CR1). This study aimed to identify and address knowledge gaps that affect the timely referral of patients for HCT consultation. METHODS: A mixed-methods educational needs assessment included a national survey and focus groups consisting of hematologists/oncologists. An educational intervention of 3 webinars addressed identified knowledge gaps. RESULTS: A total of 150 hematologists/oncologists were recruited for the survey, of whom 20 participated in focus groups. Physicians in practice 0 to 10 years were 4.2 times more likely to refer for HCT consultation in CR1 than those with >10 years in practice (P=.0027). Physicians seeing ≤10 patients with AML in the past year were 3.7 times more likely to refer for HCT consultation in CR1 than those seeing >10 patients (P=.0028). Knowledge gaps included (1) improper classification of molecular/cytogenetic results for risk stratification, (2) lack of understanding that disease stage impacts outcomes, and (3) use of chronologic age alone for referral decision-making. Combined attendance for the webinars was 1,098 clinicians; >74% of participants indicated that they would apply the knowledge they gained in clinical practice. Trends were observed toward improvement in identifying favorable-risk AML, from 48% to 60% (n=85; P=.12); improvement in identifying 2 poor-risk cytogenetic/molecular abnormalities, with the percentage of respondents indicating chromosome 7 deletion increasing from 51% to 70% (n=53; P=.05) and that of respondents indicating TP53 mutation increasing from 42% to 62% (n=62; P=.03); and improvement in identifying which patients with AML aged >60 years were most likely to benefit from HCT based on cytogenetic/molecular features, with the percentage of correct responses increasing from 66% to 81% (n=62; P=.07). CONCLUSIONS: The webinars met the educational needs of learners and improved knowledge gaps. This study provided novel insights into the learning needs of clinicians who care for patients with AML and a roadmap for future educational interventions.

Caregiver availability and patient access to hematopoietic cell transplantation: social worker perspectives inform practice.

PURPOSE: Hematopoietic cell transplantation (HCT) often involves a long hospitalization and recovery period, with patients generally required to have a caregiver. This study aimed to identify transplant center (TC) requirements for a caregiver, describe challenges that impact caregiver availability, and identify potential solutions. METHODS: An exploratory sequential mixed-methods approach was used. Qualitative data was obtained from focus groups of TC social workers in the United States (US) (three focus groups; n = 15 total participants). Results informed the development of a national, web-based survey that was administered to the primary social worker contact at TCs in the National Marrow Donor Program (NMDP)/Be The Match Network (n = 133). RESULTS: Respondents included social workers from adult (n = 47) and pediatric (n = 19) TCs (response rate = 49%). The majority (89%) of both adult and pediatric TCs required a caregiver for a patient to proceed to transplant, but requirements varied in length of time, formality, transplant type, and HCT setting. Regardless of transplant type or patient population, social workers identified loss of caregiver income as the greatest challenge to caregiver availability, with the most common solution being allowing patients to have multiple caregivers throughout the transplant course. DISCUSSION: Caregiver availability is an important concern for patients considering and receiving HCT, and may be a barrier proceeding to HCT when a caregiver is unavailable. Results from this study highlight caregiver availability barriers and solutions of TCs across the US. These results can inform TCs about other center experiences with caregiver availability and identify potential practice changes for individual TCs.

Access to Linguistically Appropriate Information for Blood and Marrow Transplant Patients: Results from Transplant Center Staff Survey.

Blood or marrow transplant (BMT) is a potentially curative treatment for numerous cancers and non-malignant disorders. BMT is a resource-intense treatment process, requiring patients to comprehend difficult health information and navigate a complex healthcare system. Linguistic and cultural barriers create additional challenges for patients with limited English proficiency (LEP) who may need translated information and interpretive services to make an informed decision about treatment. To identify information needs and gaps in language services for BMT patients with LEP, the National Marrow Donor Program® (NMDP)/Be The Match® administered a cross-sectional, web-based survey to 139 transplant centers (TCs) across the United States (U.S.). The survey yielded a 59% response rate. Findings show a significant need for translated patient education materials, especially in Spanish, Chinese, and Arabic, and practice gaps in the use of appropriate interpreters. Nearly one third of respondents indicated using family and friends to interpret for patients. The inability to locate educational resources in a specific language, lack of available bilingual staff, lack of a formal, centralized tracking system, and outdated tracking systems also pose significant barriers to meeting the language needs of BMT patients with LEP.

Translation of Clinical Research into Practice: An Impact Assessment of the Results from the Blood and Marrow Transplant Clinical Trials Network Protocol 0201 on Unrelated Graft Source Utilization.

Barriers and facilitators to adoption of results of clinical trials are substantial and poorly understood. We sought to examine whether the results of the randomized, multicenter Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0201 study comparing peripheral blood (PB) with bone marrow (BM) stem cells for unrelated donor (URD) hematopoietic cell transplantation (HCT) changed practice from PB to BM graft utilization and explored factors that impact graft selection and translation of research results into practice. The difference between use of URD BM and PB in the 2 years before and after publication of results in 2012 was examined using observational data collected by the Center for Blood and Marrow Transplant Research. A web-based survey of transplant physicians was conducted to understand the change in physician-reported personal and center preferred URD graft. No significant change in use of BM versus PB grafts occurred after 2012. Both BMT CTN participating and nonparticipating centers continued to use PB. Ninety-two percent of respondents were aware of the study results; 18% reported a change in personal and 16% reported a change in their center's practice of requesting BM instead of PB for URD HCT. Patient characteristics and the perception that engaging local champions to increase the evidence uptake were factors associated with personal or center change in practice. Despite awareness of the trial results, fewer than one-fifth of HCT physicians reported practice change in response to the BMT CTN 0201 results. Observational data confirmed no discernible change in practice.

Burnout, Moral Distress, Work-Life Balance, and Career Satisfaction among Hematopoietic Cell Transplantation Professionals.

A projected shortage of hematopoietic cell transplantation (HCT) health professionals was identified as a major issue during the National Marrow Donor Program/Be The Match System Capacity Initiative. Work-related distress and work-life balance were noted to be potential barriers to recruitment/retention. This study examined these barriers and their association with career satisfaction across HCT disciplines. A cross-sectional, 90-item, web-based survey was administered to advanced practice providers, nurses, physicians, pharmacists, and social workers in 2015. Participants were recruited from membership lists of 6 professional groups. Burnout (measured with the Maslach Burnout Inventory subscales of emotional exhaustion and depersonalization) and moral distress (measured by Moral Distress Scale-Revised) were examined to identify work-related distress. Additional questions addressed demographics, work-life balance, and career satisfaction. Of 5759 HCT providers who received an individualized invitation to participate, 914 (16%) responded; 627 additional participants responded to an open link survey. Significant differences in demographic and practice characteristics existed across disciplines (P < .05). The prevalence of burnout differed across disciplines (P < .05) with an overall prevalence of 40%. Over one-half of pharmacists had burnout, whereas social workers had the lowest prevalence at less than one-third. Moral distress scores ranged from 0 to 336 and varied by discipline (P < .05); pharmacists had the highest mean score (62.9 ± 34.8) and social workers the lowest (42.7 ± 24.4). In multivariate and univariate analyses, variables contributing to burnout varied by discipline; however, moral distress was a significant contributing factor for all providers. Those with burnout were more likely to report inadequate work-life balance and a low level of career satisfaction; however, overall there was a high level of career satisfaction across disciplines. Burnout, moral distress, and inadequate work-life balance existed at a variable rate in all HCT disciplines, yet career satisfaction was high. These results suggest specific areas to address in the work environment for HCT health professionals, especially the need for relief of moral distress and a greater degree of personal time. As the creation of healthy work environments is increasingly emphasized to improve quality care and decrease costs, these findings should be used by HCT leadership to develop interventions that mitigate work-related distress and in turn foster recruitment and retention of HCT providers.

What do transplant physicians think about palliative care? A national survey study.

BACKGROUND: Despite its established benefits, palliative care (PC) is rarely utilized for hematopoietic stem cell transplant (HSCT) patients. We sought to examine transplant physicians' perceptions of PC. METHODS: We conducted a cross-sectional survey of transplant physicians recruited from the American-Society-for-Blood-and-Marrow-Transplantation. Using a 28-item questionnaire adapted from prior studies, we examined physicians' access to PC services, and perceptions of PC. We computed a composite score of physicians' attitudes about PC (mean = 16.9, SD = 3.37) and explored predictors of attitudes using a linear mixed model. RESULTS: 277/1005 (28%) of eligible physicians completed the questionnaire. The majority (76%) stated that they trust PC clinicians to care for their patients, but 40% felt that PC clinicians do not have enough understanding to counsel HSCT patients about their treatments. Most endorsed that when patients hear the term PC, they feel scared (82%) and anxious (76%). Nearly half (46%) reported that the service name 'palliative care' is a barrier to utilization. Female sex (ß = 0.85, P = .024), having <10 years of clinical practice (ß = 1.39, P = .004), and perceived quality of PC services (ß = 0.60, P < .001) were all associated with a more positive attitude towards PC. Physicians with a higher sense of ownership over their patients' PC issues (ß = -0.36, P < .001) were more likely to have a negative attitude towards PC. CONCLUSIONS: The majority of transplant physicians trust PC, but have substantial concerns about PC clinicians' knowledge about HSCT and patients' perception of the term 'palliative care'. Interventions are needed to promote collaboration, improve perceptions, and enhance integration of PC for HSCT recipients.

Healthcare Costs and Utilization for Patients Age 50 to 64 Years with Acute Myeloid Leukemia Treated with Chemotherapy or with Chemotherapy and Allogeneic Hematopoietic Cell Transplantation.

The primary aim of this study was to describe healthcare costs and utilization during the first year after a diagnosis of acute myeloid leukemia (AML) for privately insured non-Medicare patients in the United States aged 50 to 64 years who were treated with either chemotherapy or chemotherapy and allogeneic hematopoietic cell transplantation (alloHCT). MarketScan (Truven Health Analytics) adjudicated total payments for inpatient, outpatient, and prescription drug claims from 2007 to 2011 were used to estimate costs from the health system perspective. Stabilized inverse propensity score weights were constructed using logistic regression to account for differential selection of alloHCT over chemotherapy. Weighted generalized linear models adjusted costs and utilization (hospitalizations, inpatient days, and outpatient visit-days) for differences in age, sex, diagnosis year, region, insurance plan type, Elixhauser Comorbidity Index), and 60-day prediagnosis costs. Because mortality data were not available, models could not be adjusted for survival times. Among 29,915 patients with a primary diagnosis of AML, 985 patients met inclusion criteria (774 [79%] receiving chemotherapy alone and 211 [21%] alloHCT). Adjusted mean 1-year costs were $280,788 for chemotherapy and $544,178 for alloHCT. Patients receiving chemotherapy alone had a mean of 4 hospitalizations, 52.9 inpatient days, and 52.4 outpatient visits in the year after AML diagnosis; patients receiving alloHCT had 5 hospitalizations, 92.5 inpatient days, and 74.5 outpatient visits. Treating AML in the first year after diagnosis incurs substantial healthcare costs and utilization with chemotherapy alone and with alloHCT. Our analysis informs healthcare providers, policymakers, and payers so they can better understand treatment costs and utilization for privately insured patients aged 50 to 64 with AML.

Administrative Claims Data for Economic Analyses in Hematopoietic Cell Transplantation: Challenges and Opportunities.

There is an increasing need for the development of approaches to measure quality, costs, and resource utilization patterns among allogeneic hematopoietic cell transplantation (HCT) patients. Administrative claims data provide an opportunity to examine service utilization and costs, particularly from the payer's perspective. However, because administrative claims data are primarily designed for reimbursement purposes, challenges arise when using it for research. We use a case study with data derived from the 2007 to 2011 Truven Health MarketScan Research database to discuss opportunities and challenges for the use of administrative claims data to examine the costs and service utilization of allogeneic HCT and chemotherapy alone for patients with acute myeloid leukemia (AML). Starting with a cohort of 29,915 potentially eligible patients with a diagnosis of AML, we were able to identify 211 patients treated with HCT and 774 treated with chemotherapy alone where we were sufficiently confident of the diagnosis and treatment path to allow analysis. Administrative claims data provide an avenue to meet the need for health care costs, resource utilization, and outcome information. However, when using these data, a balance between clinical knowledge and applied methods is critical to identifying a valid study cohort and accurate measures of costs and resource utilization.

Patient housing barriers to hematopoietic cell transplantation: results from a mixed-methods study of transplant center social workers.

PURPOSE: Hematopoietic cell transplantation (HCT) is performed in select centers in the United States (U.S.), and patients are often required to temporarily relocate to receive care. The purpose of this study was to identify housing barriers impacting access to HCT and potential solutions. METHODS: A mixed-methods primary study of HCT social workers was conducted to learn about patient housing challenges and solutions in place that help address those barriers. Three telephone focus groups were conducted with adult and pediatric transplant social workers (n = 15). Focus group results informed the design of a national survey. The online survey was e-mailed to a primary social worker contact at 133 adult and pediatric transplant centers in the U.S. Transplant centers were classified based on the patient population cared for by the social worker. RESULTS: The survey response rate was 49%. Among adult programs (n = 45), 93% of centers had patients that had to relocate closer to the transplant center to proceed with HCT. The most common type of housing option offered was discounted hotel rates. Among pediatric programs (n = 20), 90% of centers had patients that had to relocate closer to the transplant center to proceed with HCT. Ronald McDonald House was the most common option available. CONCLUSIONS: This study is the first to explore housing challenges faced by patients undergoing HCT in the U.S. from the perspective of social workers and to highlight solutions that centers use. Transplant centers will benefit from this knowledge by learning about options for addressing housing barriers for their patients.

National Survey of Hematopoietic Cell Transplantation Center Personnel, Infrastructure, and Models of Care Delivery.

Hematopoietic cell transplantation (HCT) is a complex procedure that requires availability of adequate infrastructure, personnel, and resources at transplantation centers. We conducted a national survey of transplantation centers in the United States to obtain data on their personnel, infrastructure, and care delivery models. A 42-item web-based survey was administered to medical directors of transplantation centers in the United States that reported any allogeneic HCT to the Center for International Blood and Marrow Transplant Research in 2011. The response rate for the survey was 79% for adult programs (85 of 108 centers) and 82% for pediatric programs (54 of 66 centers). For describing results, we categorized centers into groups with similar volumes based on 2010 total HCT activity (adult centers, 9 categories; pediatric centers, 6 categories). We observed considerable variation in available resources, infrastructure, personnel, and care delivery models among adult and pediatric transplantation centers. Characteristics varied substantially among centers with comparable transplantation volumes. Transplantation centers may find these data helpful in assessing their present capacity and use them to evaluate potential resource needs for personnel, infrastructure, and care delivery and in planning for growth.

Cost-effectiveness of diabetes pay-for-performance incentive designs.

BACKGROUND: Taiwan's National Health Insurance (NHI) Program implemented a diabetes pay-for-performance program (P4P) based on process-of-care measures in 2001. In late 2006, that P4P program was revised to also include achievement of intermediate health outcomes. OBJECTIVES: This study examined to what extent these 2 P4P incentive designs have been cost-effective and what the difference in effect may have been. RESEARCH DESIGN AND METHOD: Analyzing data using 3 population-based longitudinal databases (NHI's P4P dataset, NHI's claims database, and Taiwan's death registry), we compared costs and effectiveness between P4P and non-P4P diabetes patient groups in each phase. Propensity score matching was used to match comparable control groups for intervention groups. Outcomes included life-years, quality-adjusted life-years (QALYs), program intervention costs, cost-savings, and incremental cost-effectiveness ratios. RESULTS: QALYs for P4P patients and non-P4P patients were 2.08 and 1.99 in phase 1 and 2.08 and 2.02 in phase 2. The average incremental intervention costs per QALYs was TWD$335,546 in phase 1 and TWD$298,606 in phase 2. The average incremental all-cause medical costs saved by the P4P program per QALYs were TWD$602,167 in phase 1 and TWD$661,163 in phase 2. The findings indicated that both P4P programs were cost-effective and the resulting return on investment was 1.8:1 in phase 1 and 2.0:1 in phase 2. CONCLUSIONS: We conclude that the diabetes P4P program in both phases enabled the long-term cost-effective use of resources and cost-savings regardless of whether a bonus for intermediate outcome improvement was added to a process-based P4P incentive design.

Return to school practices after hematopoietic cell transplantation: a survey of transplant centers in the United States.

To understand transplant center recommendations on return-to-school timing and related support for hematopoietic cell transplant (HCT) survivors, we conducted a two-phase, cross-sectional, web-based survey: In Phase I, medical directors of pediatric HCT centers from the National Marrow Donor Program/ Be The Match Registry were asked regarding the availability of a return to school standardized operating procedure (SOP). In Phase II, HCT physician members of the Pediatric Transplantation and Cellular Therapy Consortium were approached to study inter-physician practice variability regarding return to school post-HCT, factors affecting their decision-making, and support provided by HCT centers for return to school. Out of 46 respondents in Phase I (55% response rate), 28 (61%) reported having a SOP. Wide variations in recommendations were noted in 12 received SOPs. In Phase II, 122 physicians (60 centers) responded (30.6% response rate). The majority (60%) recommended autologous HCT recipients return to school within 6 months post-HCT but 65% recommended allogeneic HCT recipients return to school after 6 months or once off immunosuppression. Our findings indicate a lack of consensus within and across HCT centers regarding recommended return to school timing and underscore need for a guideline to standardize this process to ensure patient safety and re-integration into school.

What do patients think about palliative care? A national survey of hematopoietic stem cell transplant recipients.

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.

Return-to-School Practices for Pediatric Hematopoietic Cell Transplantation Recipients during the COVID-19 Pandemic.

Although organizations such as Centers for Disease Control and Prevention and American Academy of Pediatrics have published guidelines favoring the resumption of in-person schooling during the coronavirus disease 19 (COVID-19) pandemic, there is no specific guidance on hematopoietic cell transplantation (HCT) recipients' safe return to school. We conducted a cross-sectional survey of pediatric HCT physician members of the Pediatric Transplantation and Cellular Therapy Consortium practicing in the United States to describe current return-to-school practices during the COVID-19 pandemic for HCT recipients. A total of 122 respondents (response rate, 30.6%) from 60 transplant centers in 32 US states completed the survey. Most of the respondents (76%) recommended that HCT recipients consider a remote or hybrid school option at this time if possible. If not possible, the respondents recommended a return to in-person school if the patient is at least 12 months post-transplantation or off immune suppression, while taking school safety measures and local COVID-19 cases into account. These results provide valuable guidance for the HCT community, patients, and caregivers on important topics to consider while making return-to-school decisions.

Trends in Allogeneic Hematopoietic Cell Transplantation Utilization and Estimated Unmet Need Among Medicare Beneficiaries with Acute Myelogenous Leukemia.

Allogeneic hematopoietic cell transplantation (alloHCT) is a resource-intensive procedure and the sole potentially curative treatment available for patients with acute myelogenous leukemia (AML). Although Medicare coverage may help address a major financial barrier to accessing alloHCT, there remains an unmet need for alloHCT owing to sociodemographic disparities. This study examined trends and factors associated with the utilization of alloHCT and the estimated unmet need for alloHCT among Medicare beneficiaries with AML. This retrospective cohort study included patients (age 65 to 74 years) with a diagnosis of AML identified in Medicare claims data from 2010 through 2016. To study trends in utilization, transplantation rates were calculated as the number of patients who underwent alloHCT within 180 days and 1 year of diagnosis (numerator) divided by the total number of patients with AML within each diagnosis year (denominator). A multivariable logistic regression was used to identify factors associated with the likelihood of undergoing alloHCT within 1 year of diagnosis. Two approaches were applied to estimate the unmet need for alloHCT. The first approach used claims data to identify the potential need for alloHCT among patients who achieved complete remission for at least 90 days. The second approach used established National Marrow Donor Program (NMDP) methodology, which considers estimates of risk level, response to treatment, comorbidity, and early mortality, to identify the potential and unmet need for alloHCT. The overall estimated need and unmet need from 2010 to 2015 and over different time periods were evaluated for both approaches. The alloHCT rate within 180 days of diagnosis increased from 8% in 2010 to 15.8% in 2016 (P < .001), and the 1-year alloHCT rate also increased over time, from 11.9% in 2010 to 20.0% in 2015 (P < .001). The likelihood of undergoing alloHCT within 1 year of diagnosis was associated with diagnosis year, age, race, geographic region, Elixhauser Comorbidity Index, and population-level median household income. Between 2010 and 2015, the claims data approach estimated a lower potential need for alloHCT compared with the NMDP methodology estimate (27% versus 36%); both approaches estimated that 43% to 44% of patients with a potential need for alloHCT had an unmet treatment need. Despite the differences in estimated potential need between the 2 approaches, both showed a sustained unmet need but with a downward trend over time. Our data show that utilization of alloHCT has increased over time among Medicare beneficiaries with AML. Two approaches of need analysis were conducted for validation of estimated need and unmet need for alloHCT using claim-identified remission status, given the lack of cytogenetics and molecular information in claims data. Both approaches to estimating the unmet need for alloHCT found a downward trend over time; however, there are differences in utilization of alloHCT by age, race, geographic region, comorbidity, and socioeconomic status, indicating disparities in access to alloHCT among Medicare beneficiaries with AML. This suggests the need for policy efforts, research, and continued education to improve access to alloHCT and to close the gap between the actual utilization of alloHCT and the unmet need.

Enhancing Administrative Claims Data: Feasibility, Validation and Application of Linking Medicare Claims Data and National Marrow Donor Program Search Data.

PURPOSE: Administrative claims data provide real-world service utilization of acute myeloid leukemia (AML) treatment, but lacks insight into treatment delays or barriers. The National Marrow Donor Program (NMDP)/Be The Match Search (Search) data contains information on donor search, but lacks information on treatment received if allogeneic hematopoietic cell transplant (HCT) is not performed. We hypothesized that linking these two data sets would create a rich resource to define factors associated with receiving HCT that could not be evaluated with either data set alone. METHODS: A subset of 2010-2016 Medicare administrative claims data was linked with Search data. A total of 5,351 patients with AML age 65-74 years (HCT = 607, no HCT = 4,744) were identified using Medicare. These patients were then linked to 93,800 records with a donor search between 2009 and 2016. Patient date of birth, sex, disease, ZIP code, transplant center/hospital, and diagnosis date were used for matching. Exploratory analysis was conducted to identify predictors associated with receiving HCT for patients with AML who received a search. RESULTS: The data sets were successfully linked, showing high sensitivity and specificity. The final cohort included 5,085 patients with AML (HCT = 533, no HCT = 4,552). Of 97 patients who received HCT without a matched search, more than 85% received a related donor HCT. Of those not receiving HCT, 609 had a matched NMDP search and 3,943 did not have a matched NMDP search. Multivariate analysis showed time to search, age, diagnosis year, race/ethnicity, and neighborhood education status associated with receiving HCT. CONCLUSION: Methods herein demonstrate the feasibility of linking Search and Medicare data. Similar methods may be applied to answer critical questions regarding barriers to HCT, thereby identifying areas to improve access to care.

Adding Centralized Electronic Patient-Reported Outcome Data Collection to an Established International Clinical Outcomes Registry.

The importance of patient-reported outcomes (PROs) in cellular therapies, including hematopoietic cell transplantation (HCT) is highlighted in this study. Longitudinal collection of PROs in a registry is recommended for several reasons, yet to date, PROs are not routinely collected from HCT patients to augment clinical registry data. The aim of this study was to determine the feasibility of electronic PRO data collection by a national clinical outcomes registry, by assessing differences between who does and does not report PROs. We conducted a cross-sectional pilot collection of PROs from HCT recipients after treatment using computer-adapted tests from the Patient-Reported Outcome Measurement Information System (PROMIS). We implemented centralized data collection through the Center for International Blood and Marrow Transplant Research (CIBMTR) among patients who underwent HCT for myelodysplastic syndromes (MDS), were at least 6 months post-HCT, and spoke English or Spanish. The main objective was identifying patient, disease, and transplant-related differences associated with completion of electronic PROs. Patients were excluded from analysis if they were determined to be ineligible (deceased, did not speak English or Spanish, refused to be contacted by the CIBMTR). A total of 163 patients were contacted and potentially eligible to participate; of these, 92 (56%) enrolled and 89 (55%) completed the PRO assessment. The most frequent reason for incomplete surveys was inability to contact patients (n = 88), followed by declining to participate in the study (n = 37). There were no sociodemographic or age differences between those who completed the PRO survey (n = 89) and eligible nonresponders (n = 155). Patient scores were within 3 points of the US average of 50 for all symptoms and functioning except physical functioning. Responders and nonresponders did not exhibit meaningfully different sociodemographic characteristics. Difficulty contacting patients posed the greatest barrier and also provided the greatest opportunity for improvement. Once enrolled, survey completion was high. These results support standardizing centralized PRO data collection through the CIBMTR registry.