Association Between Transfusion Status, Hemoglobin Levels, and Patient‐Reported Outcomes in Myelofibrosis: A Post Hoc Clinical Trial Analysis

BACKGROUND: Quality of life and symptom burden of patients with myelofibrosis are well recognized and compounded in those with anemia; however, the effects of transfusion burden or anemia severity on quality of life have not been comprehensively characterized. This post hoc descriptive analysis explored the association between transfusion status or hemoglobin improvement and patient-reported outcomes (PROs). METHODS: The analysis used pooled populations across treatment arms from 3 clinical trials (SIMPLIFY-1, SIMPLIFY-2, MOMENTUM); sample sizes for each PRO measure were dependent on the trials in which they were administered. RESULTS: At both baseline and week 24, transfusion independence was associated with umerically greater mean SF-36v2 and EORTC QLQ-C30 scores than transfusion dependence; in the subgroup that was transfusion dependent at baseline, those who achieved transfusion independence at week 24 had greater PRO improvements than those who remained reliant on transfusions. Regardless of transfusion status, patients who achieved a hemoglobin improvement ≥ 1, ≥ 1.5, or ≥ 2 g/dL from baseline also had clinically meaningful improvements in quality of life (assessed via mean EQ-5D-5L or SF-36v2 scores) and symptoms (assessed via PGIC or MPN-SAF/MFSAF Total Symptom Score) at week 24 compared with those who did not. CONCLUSIONS: Collectively, these results provide preliminary insights into the associations of transfusion status and anemia severity with quality of life in myelofibrosis; as current PRO measures do not directly evaluate the relationship between symptoms such as fatigue and anemia, development of new measures to more comprehensively capture the patient experience for those with anemia in myelofibrosis may be warranted. TRIAL REGISTRATION: NCT01969838, NCT02101268, NCT04173494.