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Decitabine With or Without Ruxolitinib Shows Benefit in Advanced Phases of MPN

When initiated early in the course of the disease, decitabine with or without ruxolitinib has shown clinical benefit in patients with advanced phases of myeloproliferative neoplasms (MPNs; Acta Haematol. 2021;144[1]:48-57).

“Treatment options are limited for patients with advanced forms of [MPN] including blast-phase disease (MPN-BP). Decitabine has frequently been deployed but its efficacy and safety profile are not well described in this population,” wrote Selena Zhou, MD, Icahn School of Medicine at Mount Sinai, New York, and colleagues.

Using data for 42 patients (16 with MPN-BP, 14 with MPN accelerated-phase [MPN-AP], and 12 with myelofibrosis with high-risk features [MF-HR]) treated with decitabine alone or in combination with ruxolitinib, Dr Zhou et al carried out a retrospective review.

According to the review, the median overall survival (OS) for patients with MPN-BP patients was 2.6 months, and 6.7 months for those given ≥2 cycles of decitabine. The patients with MPN-BP and a poor performance status who required hospitalization at the point of decitabine initiation had a dismal prognosis.

Dr Zhou et al reported that the median OS was not reached after a median follow-up of 12.4 months for patients with MPN-AP and 38.7 months for patients with MF-HR patients; 1 and 2 patients in these cohorts were alive at 60 months, respectively.

Furthermore, the likelihood of spleen length being reduced and transfusion independence within 12 months of decitabine initiation was 28.6 and 23.5%, respectively.

Compared with decitabine monotherapy, decitabine plus ruxolitinib improved OS (21 months and 12.9 months, respectively).

“Decitabine, alone or in combination with ruxolitinib, appears to have clinical benefit for patients with advanced phases of MPN when initiated early in the disease course prior to the development of MPN-BP,” Dr Zhou and co-investigators concluded.—Hina Porcelli

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