Novel therapies and targets in MPN
LINKS TO ABSTRACTS:
Presentation ID p420-1
Presenter: Marina Kremyanskaya, New York, United States
The study authors conclude that the current results indicate that rusfertide is an effective agent for the treatment of Polycythemia vera (PV), reversing iron deficiency and eliminating the need for periodic therapeutic phlebotomy (PT) in PV patients. Elimination of TP requirements for 7 months in TP-dependent PV patients is significant and unexpected. The effect of rusfertide on PV-related symptoms is also being evaluated. Continued patient enrollment will enable more definitive conclusions regarding the efficacy and safety of rusfertide in PV patients. Rusfertide looks very promising in maintaining hematocrit <45% and in eliminating the therapeutic phlebotomies in both low and high-risk PV patients.
Presentation ID p420-2
Presenter: Andreas Reiter, Germany
The study authors conclude that the PATHFINDER registrational study interim analysis showed avapritinib 200 mg QD starting dose induced rapid, durable responses which deepened over treatment regardless of AdvSM subtype or prior therapy and improved symptoms in patients with AdvSM. Avapritinib was generally well tolerated with few patients discontinuing treatment due to AEs.
Presentation ID p420-3
Presenter: Ruben Mesa, United States
The study authors conclude that these new analyses suggest JAKi naïve patients receiving MMB who maintain or achieve transfusion independence (TI) at W24 have favorable overall survival compared to momelotinib non-responders, with a similar trend observed in S2. These findings are consistent with anemia and transfusion dependency being key predictors of shortened overall survival in myelofibrosis and suggest that TI response at W24 may become a surrogate for clinical benefit, supporting the clinical relevance of momelotinib's differentiated pro-erythropoietic ACVR1 inhibition.
Presentation ID p420-4
(S203) OVERALL AND PROGRESSION-FREE SURVIVAL IN PATIENTS TREATED WITH FEDRATINIB AS FIRST-LINE MYELOFIBROSIS (MF) THERAPY AND AFTER PRIOR RUXOLITINIB (RUX): RESULTS FROM THE JAKARTA AND JAKARTA2 TRIALS
Presenter: Claire Harrison, LONDON, United Kingdom
The study authors conclude that fedratinib significantly improved progression-free survival vs placebo as 1L MF Tx. Separation of progression-free and overall survival curves between fedratinib and placebo in JAKARTA, despite fedratinib Tx in the placebo arm after Tx crossover, suggests patients may derive greater benefit from early fedratinib use. For patients in JAKARTA2 treated with fedratinib after prior ruxolitinib, median progression-free and overall survival and 1-year survival rate compare favorably with outcomes after ruxolitinib discontinuation from a recent analysis of similar patients with myelofibrosis included in large healthcare databases (6.0 mo, 11.1 mo, and 47%, respectively) (Mascarenhas, 2020). These survival outcomes were greatly impacted by the fedratinib clinical hold. The ongoing FREEDOM and FREEDOM2 trials will further assess survival with fedratinib in patients with MF.
Presentation ID p420-5
Presenter: Jie Jin, China
The study authors conclude that Jaktinib is generally well-tolerated and safe in myelofibrosis patients. Reductions in spleen volume and constitutional symptom burden, and improvements in RBC transfusion dependency and hemoglobin levels were observed. The rate of spleen volume reduction was higher in the 100mg BID arm than the 200mg QD arm.