Re-energising therapy options in sickle cell disease: the mitapivat phase 2 trial results

Affiliations

¹ St Jude Children's Research Hospital, Memphis, TN 38107, USA.
² St Jude Children's Research Hospital, Memphis, TN 38107, USA. Electronic address: jane.hankins@stjude.org.

Comment

Parul Rai et al. Lancet Haematol. 2025 Jan.

. 2025 Jan;12(1):e5-e6.

doi: 10.1016/S2352-3026(24)00378-8.

¹ St Jude Children's Research Hospital, Memphis, TN 38107, USA.
² St Jude Children's Research Hospital, Memphis, TN 38107, USA. Electronic address: jane.hankins@stjude.org.

No abstract available

Conflict of interest statement

PR receives consulting fees from Pfizer. JSH receives honoraria from UpToDate.

Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial.
Idowu M, Otieno L, Dumitriu B, Lobo CLC, Thein SL, Andemariam B, Nnodu OE, Inati A, Glaros AK, Bartolucci P, Colombatti R, Taher AT, Abboud MR, Darbari D, Ataga KI, Antmen AB, Kuo KHM, de Souza Medina S, Oluyadi A, Iyer V, Morris S, Yates AM, Shao H, Patil S, Urbstonaitis R, Zaidi AU, Gheuens S, Smith WR. Idowu M, et al. Lancet Haematol. 2025 Jan;12(1):e35-e44. doi: 10.1016/S2352-3026(24)00319-3. Epub 2024 Dec 4. Lancet Haematol. 2025. PMID: 39644907 Clinical Trial.