Patients with myelofibrosis who received ruxolitinib (Jakafi) therapy experienced “meaningful reductions in symptom burden” and improvements in quality of life in a double-blind, placebo-controlled phase III trial published in the Journal of Clinical Oncology.
The COMFORT-I (Controlled Myelofibrosis Study With Oral JAK Inhibitor Treatment–I) study enrolled 309 patients who were diagnosed with primary myelofibrosis, postpolycythemia vera myelofibrosis, or postessential thrombocythemia myelofibrosis, had palpable splenomegaly, and “were resistant or refractory to, intolerant of, or not candidates for available therapy (in the investigator’s opinion).” Patients randomly assigned to ruxolitinib had improvements in individual myelofibrosis-related symptoms, whereas those receiving placebo had worsening (P < .001).
The study also found that the modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 correlated well with established patient-reported outcome assessments and “was sensitive to differentiating responses in the placebo and ruxolitinib arms over time,” according to investigators. Every night from baseline through week 24, patients used the modified MFSAFv2.0 to rate the following myelofibrosis symptoms: night sweats, pruritus/itching, abdominal discomfort, pain under the ribs (left side), early satiety, bone/muscle pain, and inactivity.
The degree of spleen volume reduction with ruxolitinib correlated with improvements in several patient-reported outcomes—Total Symptom Score (TSS), Patient Global Impression of Change (PGIC), Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale, and the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) Global Health Status/Quality of Life. “Ruxolitinib-treated patients who achieved a ≥ 35% reduction in spleen volume experienced the greatest improvements in these [patient-reported outcomes],” the authors stated.
“Although there was a trend for greater improvements in [patient-reported outcomes] with greater spleen volume reductions with ruxolitinib, even patients with modest changes in spleen size or symptom scores demonstrated improvements in symptom burden and [quality of life], whereas patients receiving placebo continued to worsen by these same measures,” the authors concluded. ■
Mesa RA, et al: J Clin Oncol 31:1285-1292, 2013.