Safety and efficacy of ruxolitinib in an open-label, multicenter, single-arm phase 3b expanded-access study in patients with myelofibrosis: a snapshot of 1144 patients in the JUMP trial

Rákkutatás · Acute myeloid leukemia

Ruxolitinib is a potent JAK1/JAK2 inhibitor that has demonstrated superiority over placebo and best available therapy in the pivotal phase 3 COMFORT studies in patients with intermediate‑2‑or high‑risk MF.

The JUMP phase 3b expanded‑access trial evaluated the safety and efficacy of ruxolitinib in 1144 patients with myelofibrosis, including 163 intermediate‑1‑risk patients not represented in earlier phase 3 COMFORT studies. The primary endpoint was safety and tolerability; key efficacy outcomes included ≥50 % reduction in palpable spleen length and symptomatic improvement. At weeks 24 and 48, 56.9 % and 62.3 % of evaluable patients achieved a ≥ 50 % spleen reduction, with 23 % reaching a non‑palpable spleen by week 48. Clinically meaningful symptom relief was observed rapidly, with approximately half of all patients reporting significant improvements per patient‑reported outcomes. Safety profile mirrored that of COMFORT, with anemia (56.3 % all grades) and thrombocytopenia (42.2 % all grades) most common hematologic adverse events; only 3.2 % discontinued due to these events. Non‑hematologic adverse events were mostly grade 1/2 (diarrhea, pyrexia, fatigue, asthenia), and infections were low and generally mild. These results support the consistent safety and efficacy of ruxolitinib across all risk strata of myelofibrosis.

These data confirm that ruxolitinib remains a safe and effective therapy for myelofibrosis across all risk categories, including the intermediate‑1‑risk population that was not included in earlier pivotal trials.

Bizonyítékszint: Klinikai vizsgálat. Formális klinikai vizsgálati eredmény.

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