Sickle cell anemia

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Are you looking for a regimen but can't find it here? It is possible that we've moved it to the historical regimens page. For placebo or observational studies in this condition, please visit this page. If you still can't find it, please let us know so we can add it!
*We have moved How I Treat articles to a dedicated page.
Last updated on 2024-12-19:5 regimens on this page
5 variants on this page
=Guidelines=
Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.
==ASH==
*2021: Kanter et al. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation link to PMC article EuroPMC
*2020: Chou et al. American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support link to PMC article EuroPMC
*2020: DeBaun et al. American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults link to PMC article EuroPMC
*2020: Brandow et al. American Society of Hematology 2020 guidelines for sickle cell disease: management of acute and chronic pain link to PMC article EuroPMC
*2019: Liem et al. American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease link to PMC article EuroPMC
==BSH==
*2021: Oteng-Ntim et al. Management of sickle cell disease in pregnancy. A British Society for Haematology Guideline EuroPMC
==NHLBI==
*2014: Evidence-Based Management of Sickle Cell Disease: Expert Panel Report, 2014 link to PMC article EuroPMC
=All lines of therapy=
==Crizanlizumab monotherapy==
===Regimen===
FDA-recommended dose
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
Ataga et al. 2016 (SUSTAIN)	2013-2015 	Randomized Phase 2 (E-RT-esc)	1. Placebo 	Lower annual rate of crises (primary endpoint)Median annual rate of crises: 1.63 vs 2.98
Ataga et al. 2016 (SUSTAIN)	2013-2015 	Randomized Phase 2 (E-RT-esc)	2. Crizanlizumab; 2.5 mg/kg	Not reported
====Supportive therapy====
*Crizanlizumab (Adakveo) 5 mg/kg IV once on day 1
14-day cycle for 26 cycles (1 year)
===References===
# SUSTAIN: Ataga KI, Kutlar A, Kanter J, Liles D, Cancado R, Friedrisch J, Guthrie TH, Knight-Madden J, Alvarez OA, Gordeuk VR, Gualandro S, Colella MP, Smith WR, Rollins SA, Stocker JW, Rother RP. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017 Feb 2;376(5):429-439. Epub 2016 Dec 3. link to original article link to PMC article dosing details in abstract have been reviewed by our editors EuroPMC NCT01895361
==Exagamglogene autotemcel monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence
Frangoul et al. 2024 (CLIMB SCD-121)	2018-11-27 to not reported	Non-randomized phase 3 (RT)
====Targeted therapy====
*Exagamglogene autotemcel (Casgevy)
One course
===References===
#CLIMB SCD-121: Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, Wall D, Liem RI, Telfer P, Shah AJ, Cavazzana M, Corbacioglu S, Rondelli D, Meisel R, Dedeken L, Lobitz S, de Montalembert M, Steinberg MH, Walters MC, Eckrich MJ, Imren S, Bower L, Simard C, Zhou W, Xuan F, Morrow PK, Hobbs WE, Grupp SA; CLIMB SCD-121 Study Group. Exagamglogene Autotemcel for Severe Sickle Cell Disease. N Engl J Med. 2024 May 9;390(18):1649-1662. Epub 2024 Apr 24. link to original article EuroPMC NCT03745287
==Hydroxyurea monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator	Comparative Efficacy
Charache et al. 1995 (MSH) 	1992-1994 	Phase 3 (E-esc)	Placebo 	Lower annual rate of crises
Wang et al. 2011 (BABY HUG)	2003-2009 	Phase 3 (E-esc)	Placebo 	Did not meet co-primary endpoints of splenic and renal function
====Chemotherapy====
*Hydroxyurea (Hydrea) 15 mg/kg/day PO
Continued indefinitely
====Dose and schedule modifications====
Doses were subsequently adjusted, see articles for details.
===References===
# MSH: Charache S, Terrin ML, Moore RD, Dover GJ, Barton FB, Eckert SV, McMahon RP, Bonds DR; Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. N Engl J Med. 1995 May 18;332(20):1317-22. link to original article dosing details in abstract have been reviewed by our editors EuroPMC
## Update: Steinberg MH, Barton F, Castro O, Pegelow CH, Ballas SK, Kutlar A, Orringer E, Bellevue R, Olivieri N, Eckman J, Varma M, Ramirez G, Adler B, Smith W, Carlos T, Ataga K, DeCastro L, Bigelow C, Saunthararajah Y, Telfer M, Vichinsky E, Claster S, Shurin S, Bridges K, Waclawiw M, Bonds D, Terrin M. Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment. JAMA. 2003 Apr 2;289(13):1645-51. Erratum in: JAMA. 2003 Aug 13;290(6):756. link to original article EuroPMC
## HRQoL analysis: Ballas SK, Barton FB, Waclawiw MA, Swerdlow P, Eckman JR, Pegelow CH, Koshy M, Barton BA, Bonds DR. Hydroxyurea and sickle cell anemia: effect on quality of life. Health Qual Life Outcomes. 2006 Aug 31;4:59. link to original article link to PMC article EuroPMC
#BABY HUG: Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, Rana S, Thornburg CD, Rogers ZR, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik SA, Howard TH, Wynn LW, Kutlar A, Armstrong FD, Files BA, Goldsmith JC, Waclawiw MA, Huang X, Thompson BW; BABY HUG investigators. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011 May 14;377(9778):1663-72. link to original article link to PMC article EuroPMC NCT00006400
#NOHARM MTD: John CC, Opoka RO, Latham TS, Hume HA, Nabaggala C, Kasirye P, Ndugwa CM, Lane A, Ware RE. Hydroxyurea Dose Escalation for Sickle Cell Anemia in Sub-Saharan Africa. N Engl J Med. 2020 Jun 25;382(26):2524-2533. link to original article EuroPMC NCT03128515
==L-glutamine monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
Niihara et al. 2018 (GLUSCC09-01)	2010-2013 	Phase 3 (E-switch-ooc)	Placebo +/- Hydroxyurea	Higher reduction in the number of sickle cell crises through week 48 (primary endpoint)Median rate: 3 vs 4
====Supportive therapy====
*L-glutamine (Endari) 300 mg/kg PO twice per day 
48-week course
===References===
# GLUSCC09-01: Niihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP; Investigators of the Phase 3 Trial of l-Glutamine in Sickle Cell Disease. A phase 3 trial of l-glutamine in sickle cell disease. N Engl J Med. 2018 Jul 19;379(3):226-235. link to original article dosing details in abstract have been reviewed by our editors EuroPMC NCT01179217
==RBC transfusions==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator 	Comparative Efficacy
DeBaun et al. 2014 (SIT)	2004-2010 	Phase 3 (E-esc)	Observation	Seems to have fewer cerebral infarcts (primary endpoint)
====Supportive therapy====
*RBC transfusions
===References===
# SIT: DeBaun MR, Gordon M, McKinstry RC, Noetzel MJ, White DA, Sarnaik SA, Meier ER, Howard TH, Majumdar S, Inusa BP, Telfer PT, Kirby-Allen M, McCavit TL, Kamdem A, Airewele G, Woods GM, Berman B, Panepinto JA, Fuh BR, Kwiatkowski JL, King AA, Fixler JM, Rhodes MM, Thompson AA, Heiny ME, Redding-Lallinger RC, Kirkham FJ, Dixon N, Gonzalez CE, Kalinyak KA, Quinn CT, Strouse JJ, Miller JP, Lehmann H, Kraut MA, Ball WS Jr, Hirtz D, Casella JF. Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia. N Engl J Med. 2014 Aug 21;371(8):699-710. link to original article link to PMC article EuroPMC NCT00072761
Category:Sickle cell anemia regimens
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