Interest is growing in economic and comparative effectiveness analyses, with increasing emphasis on optimizing healthcare resources and costs. Limited information is available on the economic aspects of hematopoietic cell transplantation (HCT). We review contemporary literature on the costs and cost-effectiveness of HCT in the United States and worldwide. Published studies confirm the high costs associated with HCT, although the reported costs are highly variable, related to the differing methodologies used across studies. We examine the challenges in reviewing costs and cost-effectiveness across studies specific to HCTand highlight factors identified as associated with higher costs of HCT. We also discuss opportunities for future research in this area.

Survivorship Care Plans (SCPs) may facilitate long-term care for cancer survivors, but their effectiveness has not been established in hematopoietic cell transplantation recipients. We evaluated the impact of individualized SCPs on patient-reported outcomes among transplant survivors. Adult (≥18 years at transplant) survivors who were 1-5 years post transplantation, proficient in English, and without relapse or secondary cancers were eligible for this multicenter randomized trial. SCPs were developed based on risk-factors and treatment exposures using patient data routinely submitted by transplant centers to the Center for International Blood and Marrow Transplant Research and published guidelines for long-term follow up of transplant survivors. Phone surveys assessing patient-reported outcomes were conducted at baseline and at 6 months. The primary end point was confidence in survivorship information, and secondary end points included cancer and treatment distress, knowledge of transplant exposures, health care utilization, and health-related quality of life. Of 495 patients enrolled, 458 completed a baseline survey and were randomized (care plan=231, standard care=227); 200 (87%) and 199 (88%) completed the 6-month assessments, respectively. Patients’ characteristics were similar in the two arms. Participants on the care plan arm reported significantly lower distress scores at 6 months and an increase in the Mental Component Summary quality of life score assessed by the Short Form 12 (SF-12) instrument. No effect was observed on the end point of confidence in survivorship information or other secondary outcomes. Provision of individualized SCPs generated using registry data was associated with reduced distress and improved mental domain of quality of life among 1-5 year hematopoietic cell transplantation survivors. Trial registered at clinicaltrials.gov 02200133 .

Social determinants of health, including poverty, contribute significantly to health outcomes in the United States, yet their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood-poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research (CIBMTR) database. We assembled two pediatric cohorts who received a first, allogeneic HCT from 2006-2015 at age ≤18 years; including 2053 children with malignant disease and 1696 children with non-malignant disease. Neighborhood-poverty exposure was defined a priori per U.S. Census definition as living in a high-poverty ZIP code (>=20% of persons below 100% Federal Poverty Level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS) defined as time from HCT until death from any cause. Secondary outcomes included relapse and transplant-related mortality (TRM) in malignant disease, acute and chronic GVHD, and infection in the first 100 days post-HCT. Among children transplanted for non-malignant disease, neighborhood-poverty was not associated with any HCT outcome. Among children transplanted for malignant disease, neighborhood-poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplant outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared to those with private insurance. These data suggest opportunities for future investigation of household-level poverty exposures on HCT outcomes in pediatric malignant disease to inform care delivery interventions.

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 (US) aged 50-64 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 2007-2011 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 (GLM) 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 (ECI) and 60-day pre-diagnosis 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, a total of 985 patients met inclusion criteria (774 [79%] receiving chemotherapy alone and 211 [21%] alloHCT). Adjusted mean one-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 following 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 health care costs and utilization with chemotherapy alone and with alloHCT. Our analysis informs health care providers, policy makers and payers so they can better understand treatment costs and utilization for privately-insured patients age 50-64 with AML.

Purpose: Variation in Medicaid policies among states may lead to differences in coverage for complex treatments. This article uses hematopoietic cell transplantation (HCT), an established treatment for patients with hematologic cancers, as a case study to highlight state variation in Medicaid coverage of complex oncology treatments. Methods:Information on HCT coverage benefits for 2012 was collected from state Medicaid Web sites and was compared with recommended HCT benefits developed by multiple stakeholders. Coverage was reviewed for five categories: one, transplantation procedure; two, donor search; three, prescriptions; four, clinical trials; and five, patient food, lodging, and transportation.Coverage was coded on a three-point scale for each category for each state. States were ranked by the number of variables for which they met recommended benefits criteria (maximum rank score, 5).Results: Detailed information on Medicaid coverage was available for 47 states. No state provided the recommended coverage benefits in all five categories. Prescription coverage most often met the recommended criteria, whereas only a small number of states provided clinical trial coverage for HCT. There was substantial variation in Medicaid coverage for HCT by state. Conclusion:Findings highlight substantial variation in Medicaid coverage for HCT by state, which may increase disparities in access for already medically underserved populations.