Primary Myelofibrosis

Rákkutatás · Myelofibrosis

Pacritinib, especially the 200 mg twice‑daily regimen, was more effective than best available therapy (including ruxolitinib) for reducing splenomegaly and symptoms in patients with myelofibrosis and thrombocytopenia.

Myelofibrosis is a hematologic malignancy characterized by splenomegaly and debilitating symptoms. Thrombocytopenia is a poor prognostic feature and limits use of Janus kinase 1 (JAK1)/Janus kinase 2 (JAK2) inhibitor ruxolitinib. The objective of this phase 3 randomized international multicenter study (PERSIST‑2) was to compare the efficacy and safety of the JAK2 inhibitor pacritinib with that of best available therapy (BAT, including ruxolitinib) in patients with myelofibrosis and platelet count ≤ 100 × 10⁹/L. Of 311 randomized patients (175 pacritinib, 136 BAT), pacritinib twice daily achieved a significantly higher 35 % or greater spleen volume reduction (SVR) and a greater 50 % or greater reduction in total symptom score (TSS) at week 24 compared to BAT. Pacritinib also improved hemoglobin and reduced transfusion burden. Grade 3–4 adverse events were most commonly thrombocytopenia and anemia, with discontinuation due to adverse events occurring in 14 % of the pacritinib once‑daily arm, 9 % of the twice‑daily arm, and 4 % of the BAT arm.

Patients with myelofibrosis and thrombocytopenia have limited treatment options; pacritinib offers a new therapeutic strategy that is effective even in those previously exposed to ruxolitinib.

Bizonyítékszint: Irányelv / elfogadott gyakorlat. Magas szintű klinikai elfogadottság.

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