Difference between revisions of "Sickle cell anemia"
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## '''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. [http://jama.jamanetwork.com/article.aspx?articleid=196300 link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/12672732 PubMed] | ## '''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. [http://jama.jamanetwork.com/article.aspx?articleid=196300 link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/12672732 PubMed] | ||
## '''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. [http://hqlo.biomedcentral.com/articles/10.1186/1477-7525-4-59 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1569824/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/16942629 PubMed] | ## '''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. [http://hqlo.biomedcentral.com/articles/10.1186/1477-7525-4-59 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1569824/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/16942629 PubMed] | ||
− | #'''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. [https://doi.org/10.1056/nejmoa2000146 link to original article] [https://pubmed.ncbi.nlm.nih.gov/32579813/ PubMed] | + | #'''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. [https://doi.org/10.1056/nejmoa2000146 link to original article] [https://pubmed.ncbi.nlm.nih.gov/32579813/ PubMed] NCT03128515 |
==Hydroxyurea & Voxelotor {{#subobject:4eugac|Regimen=1}}== | ==Hydroxyurea & Voxelotor {{#subobject:4eugac|Regimen=1}}== |
Revision as of 12:18, 19 May 2021
Section editor | |
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Benjamin Tillman, MD Vanderbilt University Nashville, TN |
6 regimens on this page
6 variants on this page
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Guidelines
ASH
- 2020: Brandow et al American Society of Hematology 2020 guidelines for sickle cell disease: management of acute and chronic pain
- 2019: Liem et al. American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease
"How I Treat"
- 2019: Stenger et al. How I treat sickle cell disease with hematopoietic cell transplantation
NHLBI
All lines of therapy
Crizanlizumab monotherapy
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Regimen
FDA-recommended dose |
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Ataga et al. 2016 (SUSTAIN) | 2013-2015 | Randomized Phase II (E-RT-esc) | Placebo | Superior annual rate of crises |
Crizanlizumab; 2.5 mg/kg | Not reported |
Targeted 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 contains protocol PubMed
Hydroxyurea monotherapy
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Charache et al. 1995 (MSH) | 1992-1994 | Phase III (E-esc) | Placebo | Superior annual rate of crises |
Chemotherapy
- Hydroxyurea (Hydrea) 15 mg/kg/day PO
Doses were subsequently adjusted, see article 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 contains protocol PubMed
- 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
- 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
- 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 PubMed NCT03128515
Hydroxyurea & Voxelotor
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Vichinsky et al. 2019 (HOPE) | 2017-2018 | Phase III (E-esc) | 1. Placebo | Superior hemoglobin response |
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.
Supportive therapy
- Hydroxyurea (Hydrea)
- Voxelotor (Oxbryta) 1500 mg PO once per day
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 contains protocol PubMed 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 Apr 7:S2352-3026(21)00059-4. Epub ahead of print. link to original article PubMed
L-glutamine monotherapy
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Niihara et al. 2018 (GLUSCC09-01) | 2010-2013 | Phase III (E-switch-ooc) | Placebo +/- Hydroxyurea | Superior reduction in the number of sickle cell crises through week 48 |
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 contains protocol PubMed NCT01179217
RBC transfusions
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
DeBaun et al. 2014 (SIT) | 2004-2010 | Phase III (E-esc) | Observation | Seems to have fewer cerebral infarcts |
To be completed
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 PubMed NCT00072761
Voxelotor monotherapy
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|---|
Vichinsky et al. 2019 (HOPE) | 2017-2018 | Phase III (E-esc) | 1. Placebo | Superior hemoglobin response |
2. Voxelotor; 900 mg/day | Not reported |
Supportive therapy
- Voxelotor (Oxbryta) 1500 mg PO once per day
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 contains protocol PubMed 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 Apr 7:S2352-3026(21)00059-4. Epub ahead of print. link to original article PubMed