Heath-related Quality of Life in Double Umbilical Cord Blood vs. Haploidentical Marrow Transplantation: a QOL Analysis Report of BMT CTN 1101

INTRODUCTION: The Blood and Marrow Transplant Clinical Trials Network study 1101 (BMT CTN 1101, NCT01597778) was a multicenter phase III randomized trial comparing the clinical outcomes and quality of life (QOL) of patients with hematological malignancies receiving double umbilical cord blood transplants (dUCBT) or HLA‐haploidentical bone marrow transplants (haplo‐BM) after reduced intensity conditioning. Five‐year follow up found no significant differences in progression free survival (PFS) or overall survival (OS) between the two cohorts. The impact of alternative donor source on QOL, however, is unknown. METHODS: English and Spanish speaking patients completed the Functional Assessment of Cancer Therapy‐General (FACT‐G), SF‐36, EQ‐5D, and Global QOL patient‐reported outcome (PRO) assessments pre‐transplant and at 12 and 24 months post‐transplant. We compared longitudinal QOL measures between dUCBT and haplo‐BMT and investigated the association of QOL and clinical outcomes using an inverse probability weighted‐independent estimating equations (IPW‐IEE) method accounting for missingness and baseline variables. RESULTS: We found no significant differences in any of the QOL scores between the treatment arms at pre‐transplant, 12, and 24 months. Pre‐transplant scores were the only significant predictors of post‐transplant QOL scores. Relapse and Grade III‐IV acute graft‐versus‐host disease (GVHD) were associated with significant declines in mean FACT‐BMT and SF‐36 physical component scores. Chronic GVHD was associated with a decline in mean EQ‐5D utility scores. We found no significant association between pre‐transplant QOL scores and OS or PFS. CONCLUSIONS: Donor type did not impact post‐transplant QOL. Pre‐transplant QOL scores and clinical events of GVHD and relapse were the only predictors of post‐transplant QOL. QOL was not associated with survival in either treatment arm. PROs may be a valuable tool in pre‐transplant risk assessment strategies to improve QOL outcomes.