In recent years, several new therapies approved by the US Food and Drug Administration for the treatment of acute myeloid leukemia (AML) have been used in the management of accelerated or blast-phase myeloproliferative neoplasms (MPN-AP/BP). However, due to a dearth of prospective data on the efficacy of these therapies in patients with MPN-AP/BP, there remains a lack of consensus regarding their use in this population.1
In a retrospective, multicenter cohort study published in Blood Advances, researchers aimed to address this gap by investigating outcomes among 202 patients with MPN-AP/BP who were diagnosed and treated in the current era of myeloid therapies.1
Study Findings
The results demonstrated a median overall survival (OS) of 0.86 years, with no significant differences observed by first-line treatment type. The most common frontline strategies were intensive chemotherapy, DNA methyltransferase inhibitor (DNMTi)-based regimens, and DNMTi plus venetoclax–based regimens.1
An analysis of 65 patients who went on to receive allogeneic hematopoietic stem cell transplant (allo-HSCT) revealed a median OS of 2.30 years from the time of transplant.
In an interview with Hematology Advisor, study co-author Evan Chen, MD, a medical oncologist at Dana-Farber Cancer Institute and instructor in medicine at Harvard Medical School in Boston, Massachusetts, described 2 main takeaways from the findings: “First, outcomes of patients with MPN-AP/BP remain poor despite intensive chemotherapy and more recently developed, less-intensive venetoclax-based combinations. Second, a bone marrow transplant remains necessary for the possibility of long-term survival in the current treatment era for these patients.”
Despite the poor outcomes observed in this patient population, the present study found that “only 14% of patients were enrolled in clinical trials, and the criteria for assessing response in these patients is heterogeneous,” noted American Society of Hematology (ASH) media expert Ruben A. Mesa, MD, president and executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center in Winston-Salem, North Carolina.