Late-Breaking Abstracts Session

Marie Scully, MD1*, Spero R Cataland, MD2, Flora Peyvandi, MD, PhD3, Paul Coppo4*, et al.

The authors of the study conclude that:

Results of the Phase 3 HERCULES study confirm that treatment with caplacizumab reduces the time to platelet count response, resulting in faster resolution of aTTP. Treatment with caplacizumab also resulted in a highly clinically meaningful reduction in aTTP-related death, recurrence of aTTP, or a major thromboembolic event during study drug treatment. The relapses after stop of study drug in patients with ADAMTS13 activity <10% suggest that treatment should be continued until complete resolution of the underlying disease. Caplacizumab has a favorable safety profile, with mucocutaneous bleeding the most frequently reported AE. Caplacizumab, through rapid blocking of vWF-mediated platelet aggregation, represents a novel treatment option for patients with aTTP. (clinicaltrials.gov: NCT02553317)

John F Seymour, MBBS, PhD1, Thomas J. Kipps, MD2, Barbara F. Eichhorst, MD3*, Peter Hillmen, et al.

The authors of the study conclude that:

The primary analysis of MURANO, the first Phase 3 study of V in R/R CLL, shows a profound improvement in PFS vs standard BR chemoimmunotherapy, with consistent effects in all-risk subsets. Key secondary endpoints, including OS, ORR and CR rate, also showed consistent improvements with remarkable rates of peripheral blood MRD– that exceed those previously attained in treatment of R/R CLL. This enhanced disease control was achieved in a multinational setting with an acceptable safety profile, without significant TLS, demonstrating that treatment with VR resulted in outcomes superior to that of BR for pts with R/R CLL.

Christine Bellanné-Chantelot, PharmD, PhD1,2*, Caroline Marty, PhD1*, Barbara Schmaltz-Panneau, PhD1*, Odile Fenneteau, MD3*, et al.

The authors of the study conclude that:

This study thus identifies a novel pathological pathway implicating the co-translational process of protein targeting. This new genetic subtype which represents the second cause of CN in the French registry (prevalence 6.9%), is characterized by a promyelocytic maturation arrest with dysgranulopoiesis leading to a profound neutropenia, with a poor response to G-CSF, and in few patients, is associated with severe neurodevelopmental delay and exocrine pancreatic insufficiency.

Maria-Victoria Mateos, MD, PhD1, Meletios A. Dimopoulos2, Michele Cavo, MD3*, Kenshi Suzuki4*, et al.

The authors of the study conclude that:

The combination of D with VMP in transplant ineligible NDMM pts doubled the PFS (HR 0.50), which was driven by more pts achieving deep responses, including significantly higher ≥CR rate and tripling of the MRD-negativity rate. No new safety signals were observed when combining D with VMP. Three phase 3 studies have now demonstrated a consistent doubling of PFS and more than threefold increase in MRD-negativity rate when combining D with SOC regimens. These results support the use of a D-based combination, D-VMP, in transplant ineligible NDMM.

Mojca Jongen-Lavrencic, MD, PhD1, Tim Grob, MD1*, Francois G. Kavelaars1*, Adil S.A. Al Hinai1*, et al.

The authors of the study conclude that:

In an unprecedentedly large prospective study including training and validation cohorts, targeted NGS MRD detection is established as a powerful and independent predictor for relapse and survival. NGS MRD is applicable in virtually all newly diagnosed adults with AML while persistent CHIP-related mutations lack prognostic value.

Gary E. Raskob, PhD1, Nick Van Es, MD2*, Peter Verhamme, MD, PhD3*, Marc Carrier, MD4, et al.

The authors of the study conclude that:

Oral edoxaban for up to 12 months is noninferior to subcutaneous dalteparin for the treatment of cancer-associated VTE.