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).
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).
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 ).
The ASCO Post
Mhairi Copland, PhD, MB BChir, of the University of Glasgow, discusses results of a study on the combination of ponatinib and fludarabine, cytarabine, idarubicin, and G-CSF for patients with blast phase chronic myeloid leukemia, a rare complication with a poor outcome (Abstract 497).
Mark Bustoros, MD, of Dana-Farber Cancer Institute, discusses phase II study results showing that the combination of ixazomib, lenalidomide, and dexamethasone is effective in patients with high-risk smoldering disease, with a high response rate, convenient schedule, and manageable toxicity. Longer follow-up for disease outcome is ongoing (Abstract 580).
Saad Z. Usmani, MD, of the Levine Cancer Institute, discusses phase III study findings suggesting that the combination of carfilzomib/dexamethasone/daratumumab represents an efficacious new regimen for patients with relapsed or refractory disease, including those refractory to lenalidomide (Abstract LBA-6).
