Preliminary safety and efficacy of navitoclax plus ruxolitinib in JAK‑inhibitor‑naïve patients with myelofibrosis: REFINE phase 2 study

Cancer research · Myelofibrosis

Navitoclax plus ruxolitinib has a tolerable safety profile and provides clinically meaningful improvements for JAK‑inhibitor‑naïve patients with myelofibrosis.

This phase 2, multicentre, open‑label REFINE study evaluated the safety and efficacy of combining the BCL‑XL/BCL‑2 inhibitor navitoclax with the JAK‑1/2 inhibitor ruxolitinib in patients with primary or secondary myelofibrosis who were naïve to JAK‑inhibitor therapy. In 32 enrolled patients, 63 % achieved a spleen volume reduction of ≥35 % at week 24, and 41 % achieved a ≥50 % reduction in total symptom score (TSS 50) at week 24. Bone marrow fibrosis improved by ≥1 grade in 48 % of evaluable patients. No treatment‑related deaths or bleeding events were reported; the safety profile was comparable to that previously reported for each agent alone. These data suggest that navitoclax plus ruxolitinib is well tolerated and provides clinically meaningful disease‑modifying activity in JAK‑inhibitor‑naïve patients with myelofibrosis.

Myelofibrosis is a rare, progressive myeloproliferative neoplasm that can cause splenomegaly, cytopenias, and debilitating symptoms. Although ruxolitinib improves spleen size and symptoms, it does not alter disease progression and many patients eventually progress. By combining ruxolitinib with navitoclax, the study demonstrates an increase in response rates for spleen volume, symptoms, and bone‑marrow fibrosis, with an acceptable safety profile. These findings indicate a potential disease‑modifying treatment strategy for patients who have not yet received JAK‑inhibitor therapy.

Evidence level: Számítógépes vagy elméleti. Modellből vagy adatbányászatból származó jel.

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