Association between transfusion status, Hb levels, and PROs in MF: Pooled post hoc analysis of three phase III trials

Results from a pooled post hoc analysis of the phase III SIMPLIFY-1 (NCT01969838; n = 432), SIMPLIFY-2 (NCT02101268; n = 156), and MOMENTUM (NCT04173494; n = 195) momelotinib trials, exploring associations between transfusion status, hemoglobin improvement, and PROs in adults with primary or secondary myelofibrosis, were published in Cancer Medicine by Mesa et al. Sample sizes for each PRO measure varied based on the trial in which they were administered. 

Key data: At baseline and Week 24, TI was associated with numerically better SF-36v2 and EORTC QLQ-C30 scores than TD or TR. Among patients who were transfusion-dependent at baseline, those achieving TI at Week 24 had greater PRO improvements than those who remained transfusion-dependent; in the pooled SIMPLIFY trials, numerically greater improvements in all assessed SF-36v2 domains excluding vitality were observed, including physical functioning (4.8 vs 1.2), mental health (3.5 vs 1.5), and role-emotional (5.0 vs −0.4). Regardless of transfusion status, patients with anemia (hemoglobin <10 g/dL) at baseline who achieved hemoglobin improvement of ≥1, ≥1.5, or ≥2 g/dL had clinically meaningful improvements in HRQoL (EQ-5D-5L and SF-36v2 scores) and symptoms (PGIC and MPN-SAF/MFSAF Total Symptom Score) at Week 24 vs those who did not. 

Key learning: Achieving TI or hemoglobin improvement is associated with clinically meaningful improvements in HRQoL and symptom burden in MF, supporting the consideration of anemia-related benefit alongside traditional disease endpoints in clinical assessment. 

Abbreviations: EORTC QLQ-C30, European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–Core 30; EQ-5D-5L, EuroQol 5-Dimension 5-Level; Hb, hemoglobin; HRQoL, health-related quality of life; MF, myelofibrosis; MFSAF, Myelofibrosis Symptom Assessment Form; MPN-SAF, Myeloproliferative Neoplasm Symptom Assessment Form; PGIC, Patient Global Impression of Change; PRO, patient-reported outcome; SF-36v2, 36-Item Short Form Survey, version 2; TD, transfusion dependence; TI, transfusion independence; TR, transfusion requirement.