David P. Steensma, MD, on Myeloid Neoplasms: Results From a First-in-Human Trial of a Splicing Modulator
2019 ASH Annual Meeting & Exposition
David P. Steensma, MD, of Dana-Farber Cancer Institute, discusses early study findings on H3B-8800, which decreased the need for red blood cell or platelet transfusion in 14% of patients. This splicing modulator, used in the trial to treat patients with hematologic malignancies, also showed safety, dose-dependent target engagement, and a predictable pharmacokinetic profile (Abstract 673).
Ilaria Iacobucci, PhD, of St. Jude Children’s Research Hospital, discusses her work to more accurately define mutation subtypes in acute myeloid leukemia and myelodysplastic syndromes, as well as the implications for diagnosis, prognosis, and treatment (Abstract LBA-4 ).
Patrick A. Brown, MD, of Johns Hopkins University, discusses phase III findings from a Children’s Oncology Group Study showing that blinatumomab was superior to chemotherapy in terms of efficacy and tolerability for young patients as a post-reinduction therapy in the setting of high- and intermediate-risk first relapse of B-cell acute lymphoblastic leukemia (Abstract LBA-1).
Loretta J. Nastoupil, MD, of The University of Texas MD Anderson Cancer Center, discusses phase II study findings that showed obinutuzumab in combination with lenalidomide for patients with previously untreated, high tumor burden follicular lymphoma was associated with improved outcomes (Abstract 125).
C. Ola Landgren, MD, PhD, of Memorial Sloan Kettering Cancer Center, discusses phase II study findings that showed an 83% negative rate of minimal residual disease in newly diagnosed multiple myeloma treated weekly with 8 cycles of the quadruplet regimen of carfilzomib/lenalidomide/dexamethasone/daratumumab, without autologous stem cell transplant (Abstract 862).
Jerald P. Radich, MD, of the Fred Hutchinson Cancer Research Center, discusses a gene-expression model that distinguishes patients with chronic myeloid leukemia who achieved a deep molecular response from those with a poor response to treatment. This work could yield new therapeutic targets that could potentially turn a poor responder into a good responder who might even achieve treatment-free remission (Abstract 665).
