ASH 2017 Top 10 Abstracts

The American Society of Hematology’s annual meeting provides an excellent opportunity to discover recent developments in the field. The staff of Cleveland Clinic Cancer Center’s Department of Hematology and Medical Oncology finds these 10 abstracts the most compelling, clinically relevant and potentially transformative to the practice:

Late-Breaking Abstract No. 2

Venetoclax Plus Rituximab Is Superior to Bendamustine Plus Rituximab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia — Results from Pre-Planned Interim Analysis of the Randomized Phase 3 Murano Study

This study details the first randomized trial to show a clear progression-free and overall survival benefit to venetoclax (V) instead of bendamustine (B) when used in combination with rituximab (R) for relapsed CLL. Tolerability was favorable with only one event of clinical tumor lysis syndrome in each arm. High rates of minimal residual disease (MRD) negative state were achieved (83.5% in VR vs 23.1% with BR). These data are the best example of the superiority of targeted therapy for CLL when compared head-to-head against conventional chemotherapy.

Late-Breaking Abstract No. 4

Phase 3 Randomized Study of Daratumumab Plus Bortezomib, Melphalan and Prednisone (D-VMP) Versus Bortezomib, Melphalan and Prednisone (VMP) in Newly Diagnosed Multiple Myeloma (NDMM) Patients (Pts) Ineligible for Transplant (ALCYONE)

In a randomized phase 3 trial of 706 patients with multiple myeloma who were ineligible for high-dose chemotherapy and autologous transplantation, addition of daratumumab to VMP doubled the odds of achieving progression-free survival compared to VMP alone (72% vs 50% at 18 months). This increase in progression-free survival was attributed to more patients achieving deeper responses as evidenced by a higher rate of MRD negativity in the D-VMP arm.

Late-Breaking Abstract No. 6

A Randomized, Open-Label, Blinded Outcome Assessment Trial Evaluating the Efficacy and Safety of LMWH/Edoxaban Versus Dalteparin for Venous Thromboembolism Associated with Cancer: Hokusai VTE-Cancer Study

This study demonstrated that an oral FXa inhibitor, edoxaban, was noninferior to the low-molecular-weight heparin dalteparin in terms of efficacy and safety in cancer patients with thrombosis.

Abstract 2

Clinical Activity in a Phase 1 Study of Blu-285, a Potent, Highly-Selective Inhibitor of KIT D816V in Advanced Systemic Mastocytosis (AdvSM)

In this study, the authors use a KIT inhibitor in 30 heavily pretreated patients with systemic mastocytosis, and show that the majority of patients have reductions in serum tryptase levels, spleen size and D816 allele burden, along with regression in skin lesions.

Abstract 15

Phase 2, Multiple-Dose Study of Anti-FcRn Antibody, Rozanolixizumab (UCB7665), in Patients with Primary Immune Thrombocytopenia: Interim Analysis

This study is the first report of a new therapy, anti-FcRn antibodies, in patients with primary persistent or chronic immune thrombocytopenia (ITP). These antibodies prevent recycling of IgG immune complexes and reduce autoantibody levels. Clinically significant improvements in the platelet count occurred in 40 to 50 percent of generally extensively pretreated ITP patients.

Abstract 160

A Personalized Prediction Model to Risk-Stratify Patients with Myelodysplastic Syndromes (MDS)

In this study, Cleveland Clinic’s Aziz Nazha, MD, applies machine learning to 925 patients with myelodysplastic syndromes, incorporating both clinical and molecular factors. His system is able to predict survival at any time during a patient’s disease course and regardless of therapy more accurately than any other existing prognostic system. Learn more here.

Abstract 603

Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia A

This study demonstrated persistent and prolonged responses to Bmn 270, an adenovirus 5-FVIII gene therapy, with marked increases in the FVIII level, significant reductions in bleeding and minimal liver inflammation.

Abstract 625

Anticoagulation Therapy in Selected Cancer Patients at Risk of Recurrence of Venous Thromboembolism: Results of the Select-D Pilot Trial

This study demonstrated that the oral FXa inhibitor rivaroxaban was equivalent to dalteparin in terms of prevention of recurrent events in cancer patients with thrombosis. Rivaroxaban was also associated with an increased risk of clinically relevant non-major bleeding.

Abstract 725

Ivosidenib (AG-120) in Mutant IDH1 AML and Advanced Hematologic Malignancies: Results of a Phase 1 Dose Escalation and Expansion Study

In this study of 258 patients with mutated IDH1, 125 of whom had relapsed or refractory acute myeloid leukemia, the overall response rate was 30.4 percent, and response duration was 6.5 months. The drug was well tolerated. This is the first study to target this molecular mutation seen in approximately 6 to 10 percent of AML patients.

Abstract 740

Durable Clinical Responses in Heavily Pretreated Patients with Relapsed/Refractory Multiple Myeloma: Updated Results from a Multicenter Study of bb2121 Anti-BCMA CAR T Cell Therapy

A multi-center phase I study of chimeric antigen receptor (CAR) T-cell therapy targeted towards B-cell maturation antigen (BCMA) showed overall response rate of 89 percent in 21 patients with heavily treated relapsed/refractory multiple myeloma. Other preclinical and clinical studies presented at the meeting also reported significant activity of BCMA CAR-T infusions in relapsed/refractory myeloma.