Kiladjian JJ, Gisslinger H, Passamonti F, Niederwieser D, Mendelson E, Sirulnik LA, Copley-Merriman K, Zhou X, Levy R, Knoops L, Cervantes F, Barbui T, Barosi G, Vannucchi A, Harrison C. Health-related quality of life (HRQoL) and symptom burden in patients (Pts) with myelofibrosis (MF) in the COMFORT-II study. Poster presented at the 17th Congress of the European Hematology Association; June 14, 2012. Amsterdam, the Netherlands. [abstract] Haematologica. 2012 Jul 16; 97(s1):154-5. Previously presented at the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO).

Background: Ruxolitinib has demonstrated rapid and durable reductions in splenomegaly and improved disease-related symptoms and QoL in 2 phase 3 studies (COMFORT-I and -II) in patients with primary MF (PMF), post-polycythemia vera-MF (PPV-MF), or post-essential thrombocythemia-MF (PET-MF) and has been approved in the US. The prevalence of individual symptoms among these patients has not been defined.

Aims: This analysis evaluated baseline HRQoL and symptoms among patients enrolled in COMFORT-II. Methods. COMFORT-II is a randomized, open-label, multicenter, phase 3 study comparing ruxolitinib with best available therapy. HRQoL and symptoms were assessed at baseline using the European Organisation for the Research and Treatment of Cancer QoL Questionnaire-Core 30 (EORTC QLQ-C30) and Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym); this analysis summarizes these scores for all patients, regardless of assigned treatment.

Results: In COMFORTII (N = 219), 52% of patients were aged > 65 years and 57% were male. By International Prognostic Scoring System criteria (Cervantes et al. Blood. 2009), 40% had intermediate-2-risk MF, and 60% had high-risk MF.Patients had a mean palpable spleen length of 15.2 cm (range, 5-37 cm) below the costal margin. Patients with PMF or PPV-MF had larger spleens (mean, 15.8 cm; 95% CI, 14.6-16.9 cm and 16.2 cm; 95% CI, 14.5-17.9 cm, respectively) than those with PET-MF (11.4 cm; 95% CI, 9.7-13.1 cm).The most frequent symptoms (reported as “quite a bit”or “very much”) were fatigue (54%), dyspnea (30%), insomnia (30%), pain (29%), night sweats (23%), and itching (21%), and there were differences in baseline symptoms across MF subtypes (Table, upper panel). Mean EORTC QLQ-C30 functioning, global health status/QoL, and symptom scores were comparable to or worse than those that have been reported for patients with acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and breast cancer (Table, lower panel).

Conclusions: This analysis shows that patients with MF experience severe disease-related symptoms and have diminished HRQoL similar to those with AML, but because patients with MF have a longer life expectancy (an average of 2.3 to 4 years for high- and intermediate-2-risk patients, respectively), they may suffer with a reduced QoL for many years.