Open AccessPatient‐reported Effects of Fedratinib, an Oral, Selective Inhibitor of Janus Kinase 2, on Myelofibrosis‐related Symptoms and Health‐related Quality of Life in the Randomized, Placebo‐controlled, Phase III JAKARTA Trial
Author(s) -
Mesa Ruben A.,
Schaap Nicolaas,
Vannucchi Alessandro M.,
Kiladjian JeanJacques,
Passamonti Francesco,
Zweegman Sonja,
Talpaz Moshe,
Verstovsek Srdan,
Rose Shelonitda,
Abraham Pranav,
LordBessen Jennifer,
Tang Derek,
Guo Shien,
Ye Xiaomei,
Harrison Claire N.
Publication year - 2021
Publication title -
hemasphere
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.677
H-Index - 11
ISSN - 2572-9241
DOI - 10.1097/hs9.0000000000000553
Subject(s) - medicine , placebo , randomized controlled trial , quality of life (healthcare) , janus kinase inhibitor , population , brief pain inventory , myelofibrosis , physical therapy , surgery , janus kinase , chronic pain , bone marrow , alternative medicine , nursing , environmental health , pathology , cytokine
Patients with myelofibrosis (MF) experience an array of symptoms that impair health‐related quality of life (HRQoL). Fedratinib, an oral, selective Janus‐kinase 2 (JAK2) inhibitor, was investigated in the randomized, placebo‐controlled, phase III JAKARTA study in adult patients with intermediate‐ or high‐risk JAK‐inhibitor‐naïve MF. The effect of fedratinib 400 mg/d on patient‐reported MF symptoms and HRQoL in JAKARTA was assessed. Participants completed the modified Myelofibrosis Symptom Assessment Form (MFSAF v2.0), which evaluates 6 key MF symptoms (night sweats, early satiety, pruritus, pain under ribs on the left side, abdominal discomfort, bone/muscle pain). The modified MFSAF v2.0 was completed during the first 6 treatment cycles and at end of cycle 6 (EOC6). Symptom response was a ≥50% improvement from baseline in total symptom score (TSS). Overall HRQoL was assessed by EQ‐5D‐3L health utility index (HUI) score. The MFSAF‐evaluable population comprised 91/96 patients randomized to fedratinib 400 mg and 85/96 patients randomized to placebo. Mean baseline TSS was 17.6 and 14.7 for fedratinib and placebo, respectively, and mean EQ‐5D‐3L HUI was 0.70 and 0.72. Fedratinib elicited statistically significant and clinically meaningful improvements in TSS from baseline versus placebo at all postbaseline visits. Symptom response rates at EOC6 were 40.4% with fedratinib and 8.6% with placebo (OR 7.0 [95% CI, 2.9‐16.9]; P < 0.001), and a significantly higher proportion of fedratinib‐treated patients achieved clinically meaningful improvement from baseline on the EQ‐5D‐3L HUI at EOC6 (23.2% versus 6.5%; P = 0.002). Fedratinib provided clinically meaningful improvements in MF symptoms and overall HRQoL versus placebo in patients with JAK‐inhibitor‐naïve MF.