Sickle cell anemia - historical

Back to Top
The purpose of this page is to provide references to regimens that are obsolete, outdated, or of historical interest only. Is there a regimen missing from this list? See the main sickle cell anemia page for current regimens.
Last updated on 2024-12-19:2 regimens on this page
2 variants on this page
 
=All lines of therapy=
==Hydroxyurea & Voxelotor==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
Vichinsky et al. 2019 (HOPE)	2017-01 to 2018-05 	Phase 3 (E-esc)	1. Placebo 	Better hemoglobin response (primary endpoint)
Vichinsky et al. 2019 (HOPE)	2017-01 to 2018-05 	Phase 3 (E-esc)	2. Voxelotor; 900 mg/day	Not reported
Note: no specific dose of hydroxyurea is specified, just that the dose had to be stable for at least 3 months prior to trial enrollment. Approximately two-thirds of the patients were noted to be on hydroxyurea at baseline.
====Chemotherapy====
*Hydroxyurea (Hydrea) (dose not specified)
====Targeted therapy====
*Voxelotor (Oxbryta) 1500 mg PO once per day
Continued indefinitely
===References===
# HOPE: Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. Epub 2019 Jun 14. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT03036813
## Update: Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. Epub 2021 Apr 7. link to original article EuroPMC
==Voxelotor monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
Vichinsky et al. 2019 (HOPE)	2017-01 to 2018-05 	Phase 3 (E-esc)	1. Placebo 	Better hemoglobin response (primary endpoint)
Vichinsky et al. 2019 (HOPE)	2017-01 to 2018-05 	Phase 3 (E-esc)	2. Voxelotor; 900 mg/day	Not reported
====Supportive therapy====
*Voxelotor (Oxbryta) 1500 mg PO once per day
Continued indefinitely
===References===
# HOPE: Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. Epub 2019 Jun 14. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT03036813
## Update: Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. Epub 2021 Apr 7. link to original article EuroPMC
Category:Sickle cell anemia regimens
Category:Disease-specific pages
Category:Hemolytic process
Category:Inherited hemoglobinopathies
Category:Historical regimens