Response to ruxolitinib in patients with intermediate-1-, intermediate-2-, and high-risk myelofibrosis: results of the UK ROBUST Trial.
Myelofibrosis is characterized by splenomegaly and debilitating constitutional symptoms that negatively impact patients' quality of life. ROBUST, a UK, open-label, phase II study, evaluated the safety and efficacy of ruxolitinib in patients with myelofibrosis (N = 48), including intermediate-1 risk patients. The primary composite endpoint was the proportion of patients achieving treatment success [≥ 50% reduction in palpable spleen length and/or a ≥ 50% decrease in Myelofibrosis Symptom Assessment Form Total Symptom Score (MF-SAF TSS)] at 48 weeks. This was the first time that efficacy of ruxolitinib in myelofibrosis has been evaluated based on these criteria and the first time the MF-SAF was used in a population of patients solely from the United Kingdom. Overall, 50% of patients and 57% of intermediate-1 risk patients, achieved treatment success; reductions in spleen length and symptoms were observed in all risk groups. The majority of patients (66.7%) experienced ≥ 50% reductions from baseline in spleen length at any time. Improvements in MF-SAF TSS were seen in 80.0%, 72.7%, and 72.2% of intermediate-1, intermediate-2, and high-risk patients, respectively. Consistent with other studies of ruxolitinib, the most common haematological adverse events were anaemia and thrombocytopenia. Results indicate that most patients with myelofibrosis, including intermediate-1 risk patients, may benefit from ruxolitinib treatment.
['Mead|Adam J|AJ|', 'Milojkovic|Dragana|D|', 'Knapper|Steven|S|', 'Garg|Mamta|M|', 'Chacko|Joseph|J|', 'Farquharson|Mira|M|', 'Yin|John|J|', 'Ali|Sahra|S|', 'Clark|Richard E|RE|', 'Andrews|Chris|C|', 'Dawson|Meryem Ktiouet|MK|', 'Harrison|Claire|C|']
D000328:Adult D000368:Aged D000369:Aged, 80 and over D006801:Humans D008875:Middle Aged D009570:Nitriles D055728:Primary Myelofibrosis D011720:Pyrazoles D011743:Pyrimidines D012307:Risk Factors
ruxolitinib myelofibrosis symptoms Myelofibrosis Symptom Assessment Form Janus kinase inhibitor