Sickle cell anemia

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Section editors
	Shruti Chaturvedi, MD, MSCI Baltimore, MD LinkedIn		Benjamin Tillman, MD Nashville, TN

6 regimens on this page 6 variants on this page

Guidelines

NHLBI

• 2014: Evidence-Based Management of Sickle Cell Disease: Expert Panel Report, 2014

All lines of therapy

Hydroxyurea monotherapy

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Regimen

Study	Evidence	Comparator	Efficacy
Charache et al. 1995 (MSH)	Phase III	Placebo	Superior annual rate of crises

Chemotherapy

• Hydroxyurea (Hydrea) 15 mg/kg/day PO

Doses were subsequently adjusted, see article for details.

References

1. Charache S, Terrin ML, Moore RD, Dover GJ, Barton FB, Eckert SV, McMahon RP, Bonds DR. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the multicenter Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med. 1995 May 18;332(20):1317-22. link to original article contains protocol PubMed
   1. 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 PubMed
2. QoL 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 PubMed

L-glutamine monotherapy

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Regimen

Study	Evidence	Comparator	Efficacy
No publication	Phase III	Placebo	Superior reduction in the number of sickle cell crises through week 48

Note: this trial is not available in published form; instructions here are from the ClinicalTrials.gov page.

Therapy

• L-glutamine (Endari) 0.3 g/kg PO BID

48-week course

References

1. ClinicalTrials.gov

RBC transfusions

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Regimen

Study	Evidence	Comparator	Efficacy
DeBaun et al. 2014	Phase III	Observation	Seems to have fewer cerebral infarcts

To be completed

References

1. 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 PubMed