title: Progressive burden of myelofibrosis in untreated patients: assessment of patient-reported outcomes in patients randomized to placebo in the COMFORT-I study.
abstract: Patient-reported outcomes (PROs) and spleen size in patients not receiving therapy (N=154) in COMFORT-I, a randomized, double-blind study of the JAK1/JAK2 inhibitor ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis were evaluated. Baseline PROs indicated considerable disease burden. European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 scores, modified Myelofibrosis Symptom Assessment Form v2.0 Total Symptom Score, and Patient Reported Outcome Measurement Information System Fatigue scores worsened from baseline through week 24. At weeks 4 and 24, 18.3 and 40.2% of patients evaluated their condition as having worsened from baseline on the Patient Global Impression of Change questionnaire. Spleen volume and palpable length increased in most patients. These results demonstrate the progressive and debilitating effects of myelofibrosis. The consequences of delayed intervention should be assessed in the management of patients with myelofibrosis and treatment should be considered as clinically indicated for symptomatic relief or splenomegaly control.
authors.affiliation: Mayo Clinic, Scottsdale, AZ 85259, USA. mesa.ruben@mayo.edu
authors.forename: Ruben A
authors.initials: RA
authors.lastname: Mesa
authors.forename: Alan
authors.initials: A
authors.lastname: Shields
authors.forename: Thomas
authors.initials: T
authors.lastname: Hare
authors.forename: Susan
authors.initials: S
authors.lastname: Erickson-Viitanen
authors.forename: William
authors.initials: W
authors.lastname: Sun
authors.forename: Nicholas J
authors.initials: NJ
authors.lastname: Sarlis
authors.forename: Victor
authors.initials: V
authors.lastname: Sandor
authors.forename: Richard S
authors.initials: RS
authors.lastname: Levy
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D017326:Clinical Trials, Phase III as Topic; D018450:Disease Progression; D004311:Double-Blind Method; D006801:Humans; D053612:Janus Kinases / Q000037:antagonists & inhibitors; D008875:Middle Aged; D009570:Nitriles; D017063:Outcome Assessment, Health Care / Q000379:methods*; D010919:Placebos; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000473:pathology; D011720:Pyrazoles / Q000627:therapeutic use*; D011743:Pyrimidines; D016032:Randomized Controlled Trials as Topic; D013154:Spleen / Q000187:drug effects / Q000473:pathology; D011795:Surveys and Questionnaires; D013997:Time Factors
chemical_list: D009570:Nitriles; D010919:Placebos; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D053612:Janus Kinases