Perspectives on drug development in chronic myelomonocytic leukemia: changing the paradigm.

Autor: Hunter AM; Department of Hematology and Medical Oncology, Winship Cancer Institute of Emory University, Atlanta, GA., Patnaik MM; Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN., Itzykson R; Université Paris Cité, Génomes, Biologie Cellulaire et Thérapeutique U944, INSERM, Centre National de la Recherche Scientifique, Paris, France.; Département Hématologie et Immunologie, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France., Mesa R; Hematology and Medical Oncology, Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, NC., Karanes C; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD., Li Y; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD., de Claro RA; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD., Norsworthy KJ; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD., Theoret M; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.; Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD., Pulte E; Office of Oncologic Diseases, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD., Padron E; Hematologic Malignancies. H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL.
Jazyk: angličtina
Zdroj: Blood [Blood] 2024 Nov 07; Vol. 144 (19), pp. 1987-1992.
DOI: 10.1182/blood.2024025648
Abstrakt: Abstract: Drug development for chronic myelomonocytic leukemia (CMML) has failed to parallel the recent success observed in related myeloid neoplasms. To address these shortcomings, the US Food and Drug Administration (FDA) held a "Mini-symposium on CMML: Current State of the Art and Trial Design" in September 2023. This symposium brought together a panel of key FDA regulators and academic experts in CMML drug development to discuss challenges and provide perspectives on future drug development for this disease. The panel explored unique challenges that underlie the lack of therapeutic advances in CMML to date and discussed relevant topics such as clinical trial design, study end points, and key regulatory considerations. This article summarizes the key points of discussion from this symposium to facilitate advancements in the field.
Databáze: MEDLINE