Payer-Reported Unmet Need Burden Post Allogeneic Hematopoietic Cell Transplantation (HCT) Due to Poor Graft Function (PGF) and Characteristics of an Ideal Treatment for PGF

OBJECTIVES: Poor graft function (PGF) that may occur after allogeneic hematopoietic cell transplantation (HCT) does not respond well to current therapies. This research elucidates the areas of unmet need in managing PGF and the expected characteristics of an ideal treatment for PGF.

METHODS: Ten private and institutional payers in the United States were interviewed in 2022.

RESULTS: According to the interviewed payers, approximately 7% of patients post-HCT suffer from PGF. Most of them are treated with a cell-based therapy (e.g., CD34+ selected stem-cell boost), with some receiving drug therapy (e.g., G-CSF) as a supportive treatment. Approximately three-quarters of respondents are not satisfied with current treatments for managing PGF; only about one-third of PGF patients’ respond well to these therapies. Top four areas of unmet need in managing PGF include (which were cited by at least half of the respondents), lack of effective targeted therapies for PGF; more-efficacious treatments for controlling refractory thrombocytopenia or pancytopenia or neutropenia; combination therapy (i.e., key therapy + supporting treatment) use is not always utilized at appropriate intervals in pre-HCT, HCT, and post-HCT phases for better response rate; and the lack of standardized diagnostic criteria for PGF diagnosis. The expectations from an ideal treatment for PGF include, shortens time to recovery post HCT; prevents spread of acute or chronic Graft-versus-host disease (GvHD); reduces the possibility of infections after transplants; reduces chances of readmission to hospitals; and increases the overall survival in patients with primary cancer disease. Some respondents also mention the need for a prophylactic drug to prevent the occurrence of PGF.

CONCLUSIONS: No effective and targeted therapies are available to manage PGF, and thereby exists the high need for promising therapeutic approach(es) to promote hematopoietic recovery post-HCT, to prevent PGF, as well as to effectively treat PGF.