634. Myeloproliferative Syndromes: Clinical: Clinical Trials in Polycythemia Vera

Watch Session

John Mascarenhas, Brian Higgins, Doreen Anders, et al.

Authors Conclusion from the Abstract: Idasanutlin showed relevant clinical activity in pts with HU-resistant/intolerant PV and also in a subset of pts with prior rux tx. However, the low-grade gastrointestinal toxicity profile of idasanutlin was not effectively mitigated with antiemetic prophylaxis and led to frequent discontinuations. These results iterate the importance of tolerability of novel therapies administered over the long term for pts with PV.

Ghaith Abu-Zeinah, Spencer Krichevsky, Tatiana Cruz, et al.

Authors Conclusion from the Abstract: Our results support the early use of rIFN-a as a safe, disease-modifying treatment of rigorously defined PV to delay and potentially prevent PPVMF and prolong overall survival of PV pts.

Heinz Gisslinger, Christoph Klade, Pencho Georgiev, et al.

Authors Conclusion from the Abstract: In a randomized controlled setting, ropeg treatment effectively controlled hematocrit and minimized the occurrence of thromboembolic events in patients with PV. Disease progression was very rare during long-term treatment with ropeg and this possible change in the disease's natural history appears to be related to deep and durable molecular responses selectively achieved with ropeg.

Marina Kremyanskaya, Yelena Ginzburg, Andrew T. Kuykendall, et al.

Authors Conclusion from the Abstract: The current results indicate that PTG-300 is an effective agent for the treatment of PV, reversing iron deficiency and eliminating the need for TP in PV patients. Elimination of TP requirements for 7 months in TP-dependent PV patients is significant and unexpected. The effect of PTG-300 on PV-related symptoms is also being evaluated. Continued patient enrollment will enable more definitive conclusions regarding the efficacy and safety of hepcidin mimetic PTG-300 in PV patients with high TP requirements. PTG-300 looks very promising in eliminating the therapeutic phlebotomies in both low and high-risk patients.

Rafael Daltro De Oliveira, Juliette Soret-Dulphy, Lin-Pierre Zhao, et al.

Authors Conclusion from the Abstract: Overall, our study demonstrates that IFN discontinuation represents a safe strategy in MPN patients who achieved CHR, and particularly in patients with a driver VAF lower than 10% at time of discontinuation. Importantly, relapsed patients did not develop IFN resistance. Our data contributes to set the frame for future clinical trials evaluating the possibility of IFN treatment holidays in good responding MPN patients.

Brady L. Stein, Kamal Patel, Robyn M. Scherber, et al.

Authors Conclusion from the Abstract: In this analysis from REVEAL, the largest prospective and contemporary cohort of pts with PV in the US, the estimated 4-y mortality was >10%, a finding that is surprising given the mean age at enrollment was only ~66 y. Approximately one-third of the deaths were due to thrombotic complications; in the 6 months prior to death, more than a quarter of pts had elevated HCT or uncontrolled myeloproliferation. Compared with pts alive at study completion, pts who had died had higher-risk disease, and higher rates of comorbid conditions. The high rate of respiratory disorders observed in the deceased population, both as comorbidities and causes of death, has not been well characterized in other PV mortality studies and warrants further investigation.