The treatment combination demonstrated an overall response rate of 44% and median overall survival of 9.5 months.
A phase 2 study assessing ruxolitinib and decitabine in patients with myeloproliferative neoplasms (MPNs) demonstrated that the treatment combination is well tolerated and elicits favorable overall survival (OS), according to the results published in Blood Advances.
These findings indicate the combination could be a viable treatment option for the high-risk group of patients.
Throughout September 2016 and March 2017, 25 patients were enrolled in the study. Follow-up showed that the treatment combination resulted in a median OS of 9.5 months (95% CI, 4.3-12.0) and the overall response rate was 44% (95% CI, 24.4%-65.1%), although this was not indicative of improved survival, the researchers noted.
According to the researchers, the survival data compare favorably with historical data showing a median OS of 3 to 5 months for conventional acute myeloid leukemia induction chemotherapy for these patients.
These patients included 15 in the blast phase (MPN-BP) of disease and 10 patients in the accelerated phase (MPN-AP) of disease, the latter of which typically has particularly poor outcomes.
“MPN-AP/BP is a genetically complex, treatment-resistant, and debilitating disease in dire need of improved interventions,” wrote the researchers, adding, “Outside of clinical trial options, this therapeutic approach should be considered in those patients with leukemic evolution of chronic MPN and may serve as a potential bridge to [hematopoietic stem cell transplantation (HCT)] in a subset of patient, which remains the only curative option.”
There were 2 patients in the current study who had incomplete platelet recovery and 9 who achieved a partial response. At the time of response assessment, 8 of the partial responders had a blast count of < 5% in the peripheral blood. There were no patients who achieved a complete response, and responses were not evaluable in 7 patients.
Assessment also showed that patients had a median reduction in spleen size of 70.5%.
The median number of cycles to best response was 4 cycles, which was also the median number of cycles administered for both groups of patients. Treatment cycles consisted of ruxolitinib 25 mg twice daily for the induction cycle followed by 10 mg twice daily for subsequent cycles in combination with decitabine 20 mg/m2 for 5 consecutive days in a 28-day cycle.
The most common treatment emergent adverse events of all grades experienced with the treatment combination included fatigue (32%), febrile neutropenia (28%), pneumonia (28%), diarrhea (28%), abdominal pain (24%), and edema (24%).
Since enrolling in the study, all patients have discontinued treatment, with reasons including adverse events (36%), disease progression (28%), physician decision (8%), HCT (8%), study disclosure (8%), patient withdrawal (4%), and unknown (8%). Nineteen patients died during follow-up.
“The combination of ruxolitinib and decitabine in MPN-AP/BP is a viable low-intensity, ambulatory regimen that reduces spleen size with a median OS that is particularly encouraging given multiple historical reports,” the authors concluded. “Outside of clinical trial options, this therapeutic approach should be considered in those patients with leukemic evolution of chronic MPN and may serve as a potential treatment bridge to HCT in a subset of patient, which remains the only curative option.”
Reference
Mascarenhas J, Rampal R, Kosiorek H, et al. Phase 2 study of ruxolitinib and decitabine in patients with myeloproliferative neoplasm in accelerated and blast phase. Blood Adv. 2020;4(20):5246-5256.
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
January 23rd 2024Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
December 26th 2023A recent study found that uptake of disease-modifying therapies (DMTs) has been low among patients with sickle cell disease, suggesting that more interventions that consider individual patient characteristics are needed to improve adoption.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More