Ropeginterferon alfa‑2b‑njft treatment in essential thrombocythemia across different driver mutations: results from a North American, single‑arm, multicentre study (EXCEED‑ET)

Rákkutatás · Acute myeloid leukemia

Ropeg showed efficacy and substantial molecular responses with good overall tolerability across a broad ET population.

Ropeginterferon alfa‑2b‑njft (ropeg) is a mono‑PEGylated interferon‑α that has shown efficacy and safety in hydroxyurea‑intolerant/resistant essential thrombocythemia (ET) in phase 3 SURPASS‑ET. The EXCEED‑ET study evaluated ropeg in North American patients with ET, both treatment‑naïve and hydroxyurea‑pretreated. In this single‑arm, multicentre, phase 2b trial, 91 patients were enrolled. Durable modified European LeukemiaNet (ELN) responses at Months 10 and 13 were achieved in 60.2 % (54/90) of patients. Median time to hematologic response was 8.4 weeks. Molecular responses at Month 13 were 35.0 % for JAK2V617F, 16.0 % for CALR, and 25.0 % for MPL mutations. Treatment‑related adverse events were mild to moderate, with no grade 5 events and a 1.1 % rate of grade 4 hepatitis. Ropeg demonstrated substantial efficacy and tolerability across a broad ET population.

This study confirms that ropeg, a novel interferon‑α therapy, is effective and well tolerated in North American patients with essential thrombocythemia, including both treatment‑naïve and hydroxyurea‑pretreated individuals, and achieves meaningful molecular responses across all major driver mutations.

Bizonyítékszint: Sejtvonalas. Laboratóriumi sejtekben vizsgálták.

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