MPN-086: Patient-Reported Outcome (PRO) Data from Patients with Lower-Risk Myelofibrosis (MF) Enrolled in the Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)

Abstract

Context: MF is a chronic myeloproliferative neoplasm (MPN) that can be accompanied by substantial symptom burden, diminished quality-of-life (QoL), and reduced survival. Objective: To report PROs in patients with lower-risk MF at time of enrollment in the MOST study. Design: MOST is a prospective, longitudinal, multicenter, noninterventional, observational study ( NCT02953704 ). Patients: Eligible adults had low-risk MF, using DIPSS risk categorization, or intermediate-1 risk by age alone (INT-1 age alone). Context: MF is a chronic myeloproliferative neoplasm (MPN) that can be accompanied by substantial symptom burden, diminished quality-of-life (QoL), and reduced survival. Objective: To report PROs in patients with lower-risk MF at time of enrollment in the MOST study. Design: MOST is a prospective, longitudinal, multicenter, noninterventional, observational study ( NCT02953704 ). Patients: Eligible adults had low-risk MF, using DIPSS risk categorization, or intermediate-1 risk by age alone (INT-1 age alone). Main outcome measures: PROs were assessed during usual-care visits every 6 months over 36 months of observation. Patient-reported symptom burden was assessed using the MPN-SAF; TSS was calculated. Health-related QoL was evaluated using the EORTC QLQ-C30 v3.0. Data were summarized descriptively; correlations between PROs and treatment modalities were not assessed. Results: Of 233 pts with lower-risk MF enrolled in MOST between November 29, 2016 and March 29, 2019, 125 with ≥1 PRO assessment at enrollment were included in this analysis (median age, 68 years; median duration from MF diagnosis to enrollment, 2.1 years; INT-1 age alone risk, 58%; full/part-time employment at diagnosis and enrollment, 46% and 33%, respectively). 124 patients completed the MPN-SAF at enrollment. Mean (SD) TSS was 18.1 (15.1) and was higher in women vs men (20.4 [15.8] vs 16.0 [14.2], respectively); 36% of pts had TSS ≥ 20. Fatigue was the highest mean (SD) individual symptom score (3.9 [2.7]) and the most commonly reported severe symptom (score ≥ 7; 22%). 123 patients completed the EORTC QLQ-C30 at enrollment. The highest mean (SD) symptom scale score was fatigue (30.2 [24.0]). The mean (SD) global health status/QoL score was 71.4 (21.0); functional scores ranged from 76.5 (21.3) for emotional functioning to 83.1 (16.1) for physical functioning. Conclusions: Despite having anticipated longer survival, patients with lower-risk MF reported high symptom burden and reduced QoL at the time of enrollment in MOST. Fatigue was the most common and most severe symptom. Future analyses from MOST will enhance understanding of symptom burden in patients with MF and its impact on QoL.