Health‐related quality of life in reduced‐intensity hematopoietic cell transplantation based on donor availability in patients aged 50–75 with advanced myelodysplastic syndrome: BMT CTN 1102

Abstract

For myelodysplastic syndrome (MDS), allogeneic hematopoietic cell transplantation (alloHCT) is the only available curative therapy. The Blood and Marrow Transplant Clinical Trials Network study 1102 (BMT CTN 1102, NCT02016781) was a multicenter, biologic assignment trial based on matched donor availability in adults aged 50-75 with higher risk de novo MDS who were candidates for reduced-intensity conditioning (RIC) alloHCT. The primary analysis showed that those who received alloHCT had a survival benefit, but whether this is at the cost of worse quality of life (QOL) has not been described in detail. English-and Spanish-speaking trial participants completed the Functional Assessment of Cancer Therapy-General (FACT-G), the SF-36, and the EQ-5D, at enrollment, every 6 months until 24 months, and 36 months. We compared patient-reported outcome (PRO) scores between study arms using an inverse probability weighted-independent estimating equation (IPW-IEE) model.

Unlocking Insights

“…The researchers concluded that the survival advantage for patients who underwent alloHCT did not come at the cost of worse quality of life. 12 The results also showed baseline QOL was a consistent predictor of survival outcomes and subsequent QOL outcomes. Two studies from the Center for International Blood and Marrow Transplant Research (CIBMTR) also showed PROs can be an indicator of post-HCT outcomes and QOL.…”

“…Patients who underwent HCT, as well as those who did not, had similar PRO trajectories. The researchers concluded that the survival advantage for patients who underwent alloHCT did not come at the cost of worse quality of life 12…”

Cost-Effectiveness of Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation for Older Patients With High-Risk Myelodysplastic Syndrome: Analysis of BMT CTN 1102

“…To account for missing surveys and to develop an approach to extrapolate results across the time period of interest, we generated an inverse probability weighted independent estimating equations (IPW-IEE) model. Using the IPW-IEE model similar to a recent analysis of health-related QOL for BMT 1102 participants, 3 we generated monthly utility values for individuals from survey results.…”

“…16 Of note, an evaluation of trial participants completing several different PRO measures found no significant differences in QOL between patients in the donor and no-donor arms across several physical, social, and emotional well-being domains, and an overall trend of improvement in QOL among transplant patients after 6 months. 3 Although the clinical results of BMT 1102 were similar for older persons who are typically covered by commercial insurance (those age 50-64 years), the economic evidence is less compelling. The projected survival benefit in this population (3.4 life-years) is high compared with other common treatments for advanced cancer, including hematologic malignancies, in this age group.…”

“…1 Importantly, allogeneic hematopoietic cell transplantation (alloHCT) did not come at the cost of worse quality of life (QOL). 3 Although the trial showed HCT to be a viable option for older patients with MDS, patient selection remains highly complex, transplant is costly, and age-related comorbidities in older adults necessitate a comprehensive assessment of transplant outcomes including mortality, morbidity, and patient-reported outcomes. Accordingly, we conducted an economic analysis alongside the BMT CTN 1102 trial to determine the cost-effectiveness of RIC HCT versus non-HCT therapy among older adults with high-risk MDS.…”