Update on clinical trials in MPN
Claire Harrison, Nicolaas Schaap, Alessandro Vannucchi, Jean-Jacques Kiladjian, et al.
Conclusion: FEDR provided clinically meaningful reductions in splenomegaly and symptom burden in pts with MF for whom RUX had failed. Importantly, data were markedly similar in a cohort of pts who met stringent criteria for prior RUX failure, and efficacy was confirmed in the sensitivity analysis. Safety in this heavily pretreated population with advanced disease was consistent with prior reports.
Natalia Curto-Garcia, Joanna Baxter, Erica Harris, Mary Francis McMullin, et al.
Conclusion: This analysis shows that as well as having clinical and symptom responses to Rux, PV patients may also have molecular responses. Molecular responses also occurred in patients on the BAT arm including the only complete molecular response. Previous studies have suggested that molecular responses can be slow for patients with PV and further time points from our study will be assessed. Importantly we show for the first time that molecular responses may correlate with reduced risk of thrombosis, this finding has the potential to change the therapeutic paradigm in PV.
Frank Stegelmann, Steffen Koschmieder, Susanne Isfort, Andreas Hochhaus, et al.
Conclusion: Combination treatment of RUX and POM is safe and feasible in pts with poor-risk MF and resulted in an objective anemia response rate of 18% with RUX plus low-dose POM in co1. Notably, 40% of pts showed a long-lasting stabilization of their disease with sustained improvement of Hb and quality of life. Updated results on more than 70 pts will be presented.
Trine Alma Knudsen, Dennis Lund Hansen, Lucas Frans Ocias, Ole Weis Bjerrum, et al.
Conclusion: At 36 months the clinicohematological ORR was higher in patients with ET, PV and Pre-MF treated with HU (p=0.02). There was no significant difference in PMR rate but the JAK2V617F reduction was higher for r-IFNα. Treatment discontinuation for any reason and for toxicity was higher in patients treated with r-IFNα compared with HU (p<0.05).
Juliette Soret-Dulphy, Nabih Maslah, Christine Dosquet, Laetitia Vercellino, et al.
Conclusion: This study demonstrates that mPV can be distinguished from PV and ET based on RCM measurement even when bone marrow biopsy is not performed. Our findings may explain why masked PV presents some characteristics of ET (thrombocytosis) and PV (low EPO, increased RCM, relative microcytosis), but is a distinct entity (hemodilution even in the absence of splenomegaly, intermediate JAK2 mutant allelic burden between ET and PV). These results also suggest that mPV probably needs to be managed like PV, since these patients present a true erythrocytosis.
Mohamad Jawhar, Juliana Schwaab, Iván Álvarez-Twose, Khalid Shoumariyeh, et al.
Conclusion: We conclude that the WHO classification remains the pivotal diagnostic and prognostic tool for subtyping of SM into indolent SM and AdvSM. The MARS is a WHO-independent and complementary tool for the heterogeneous cohort of patients with AdvSM by defining three risk groups based on a five-parameter prognostic score which may improve upfront treatment stratification for these rare hematologic neoplasms.