Transfusion dependence is a “key” patient-centered outcome in myelofibrosis, according to a post hoc analysis of the SIMPLIFY-1 and SIMPLIFY-2 trials.
Ruben Mesa, MD, of the Wake Forest University School of Medicine and colleagues conducted the post hoc analysis and presented their findings during the 2023 American Society of Clinical Oncology Annual Meeting.
They conducted the exploratory post hoc analysis of the phase III trials to characterize the relationship between transfusion burden and patient-reported outcomes. Both trials evaluated momelotinib in patients with myelofibrosis.
The pooled analysis set included patients from both arms of the intent-to-treat populations in SIMPLIFY-1 and SIMPLIFY-2. In the SIMPLIFY-1 trial, patients were naïve to Janus kinase (JAK) inhibitor therapy and received momelotinib or ruxolitinib (n=432). In the SIMPLIFY-2 trial, patients who had been exposed to JAK inhibitors received momelotinib or the best available therapy, which was ruxolitinib for 88% (n=156).
Dr. Mesa and colleagues defined transfusion independence as no transfusions and hemoglobin ≥8 g/dL in 12 weeks. They defined transfusion dependence as at least four units transfused or hemoglobin <8 g/dL in eight weeks. Patients who did not meet criteria for transfusion dependence or transfusion independence were defined as “transfusion requiring.” The patient-reported outcome assessment included measurement domains for physical functioning, physical roles, general health, vitality, social functioning, and mental health. Norm-based scores ranged from 11.6 to 70.4 based on the domain.
In the pooled set of 588 patients, 503 (85.5%) had norm-based scores available at baseline. The mean norm-based scores were lower than the general population mean, according to Dr. Mesa and colleagues.
They found that the mean norm-based scores in all domains at baseline and at week 24 were lower in patients who were transfusion dependent than in those who were transfusion independent. Among the 150 patients who were transfusion dependent at baseline, 75 (50%) remained transfusion dependent, while 40 (26.6%) became transfusion independent, and 21 (14%) became transfusion requiring. There was no data available for the remaining 14 patients.
Patients who became transfusion independent “experienced greater improvement” in most domains than patients who remained transfusion dependent, according to Dr. Mesa and colleagues.
Furthermore, patients with transfusion dependence had “lower functioning” and health-related quality of life across domains than those who were transfusion independent, “suggesting that transfusion dependence has detrimental effects on multiple aspects” of quality of life, the study’s authors wrote.
“These analyses highlight transfusion dependence as a key [patient]-centered outcome and support further research into the impact of transfusions on [quality of life] in [myelofibrosis],” Dr. Mesa and colleagues concluded.
Mesa RA, Palandri F, Verstovsek S, et al. Impact of transfusion burden on health-related quality of life (HRQOL) and functioning in patients (pts) with myelofibrosis (MF): Post hoc analysis of SIMPLIFY-1 (S1) and -2 (S2). Abstract #7066. Presented at the 2023 American Society of Clinical Oncology Annual Meeting; June 2-6, 2023; Chicago, Illinois.