OBJECTIVES: The phase III RISE UP trial evaluated the safety and efficacy of mitapivat in patients with SCD. Mitapivat was found to be well tolerated and improved clinical markers of SCD, however it is unclear how these findings will influence real-world use of mitapivat. This study investigated providers’ perceptions of mitapivat for SCD, the RISE trial’s findings, and prescribing behavior.

METHODS: US hematologists and oncologists attended an in-person forum in April 2024 where they responded to questions their clinical practice. Not all participants answered every question. Data were analyzed using descriptive statistics.

RESULTS: Sixty-five physicians participated. Providers (n=59) reported that in the last 3 months, they had 1-3 (27%), 4-6 (14%), 7-9 (7%), 10 or more (3%) unique patients with SCD referred to their practice and 27% did not manage SCD. Sixty-three providers personally managed patients with SCD or β-thalassemia in longitudinal care in both outpatient and inpatient settings (41%), in the outpatient setting (11%), in the inpatient setting (10%), and in the emergency care setting (5%) while 21% reported other physicians manage those patients and 13% refer them elsewhere. Providers (n=63) reported that efficacy (38%), payer approval (37%), patient’s quality of life (33%), and therapy availability (30%) were the factors most influential on their SCD prescribing behavior, when asked to select three factors. After reviewing the RISE UP data, providers (n=63) reported they were very likely (59%) or somewhat likely (17%) to prescribe mitapivat with 2% reporting they were not very likely to prescribe mitapivat if it was FDA approved.

CONCLUSIONS: This study demonstrated that efficacy, payer approval, patient quality of life, and therapy availability influence providers’ therapeutic decisions. Physicians exposed to mitipivat data were overwhelmingly likely to consider its incorporation into SCD treatment strategy.