Difference between revisions of "Sickle cell anemia"
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|rowspan=2|[https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481200/ Ataga et al. 2016 (SUSTAIN)] | |rowspan=2|[https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481200/ Ataga et al. 2016 (SUSTAIN)] | ||
| − | |rowspan=2 style="background-color:#1a9851"|Randomized Phase II (E-RT) | + | |rowspan=2 style="background-color:#1a9851"|Randomized Phase II (E-RT-esc) |
|Placebo | |Placebo | ||
|style="background-color:#1a9850"|Superior annual rate of crises | |style="background-color:#1a9850"|Superior annual rate of crises | ||
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|[https://www.nejm.org/doi/full/10.1056/NEJM199505183322001 Charache et al. 1995 (MSH)] | |[https://www.nejm.org/doi/full/10.1056/NEJM199505183322001 Charache et al. 1995 (MSH)] | ||
| − | |style="background-color:#1a9851"|Phase III (E) | + | |style="background-color:#1a9851"|Phase III (E-esc) |
|Placebo | |Placebo | ||
|style="background-color:#1a9850"|Superior annual rate of crises | |style="background-color:#1a9850"|Superior annual rate of crises | ||
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|- | |- | ||
|[https://www.nejm.org/doi/full/10.1056/NEJMoa1715971 Niihara et al. 2018 (GLUSCC09-01)] | |[https://www.nejm.org/doi/full/10.1056/NEJMoa1715971 Niihara et al. 2018 (GLUSCC09-01)] | ||
| − | |style="background-color:#1a9851"|Phase III (E) | + | |style="background-color:#1a9851"|Phase III (E-switch-ooc) |
|Placebo +/- Hydroxyurea | |Placebo +/- Hydroxyurea | ||
|style="background-color:#1a9850"|Superior reduction in the number of sickle cell crises through week 48 | |style="background-color:#1a9850"|Superior reduction in the number of sickle cell crises through week 48 | ||
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!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]] | !style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]] | ||
|- | |- | ||
| − | |[https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4195437/ DeBaun et al. 2014] | + | |[https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4195437/ DeBaun et al. 2014 (SIT)] |
| − | |style="background-color:#1a9851"|Phase III (E) | + | |style="background-color:#1a9851"|Phase III (E-esc) |
|Observation | |Observation | ||
|style="background-color:#91cf60"|Seems to have fewer cerebral infarcts | |style="background-color:#91cf60"|Seems to have fewer cerebral infarcts | ||
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===References=== | ===References=== | ||
| − | # 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. [https://www.nejm.org/doi/full/10.1056/NEJMoa1401731 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4195437/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/25140956 PubMed] | + | # '''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. [https://www.nejm.org/doi/full/10.1056/NEJMoa1401731 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4195437/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/25140956 PubMed] |
[[Category:Sickle cell anemia regimens]] | [[Category:Sickle cell anemia regimens]] | ||
Revision as of 02:03, 11 December 2019
| Section editor | |
|---|---|
 |
Benjamin Tillman, MD Vanderbilt University Nashville, TN |
6 regimens on this page
6 variants on this page
|
Guidelines
ASH
- 2019: Liem et al. American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease
NHLBI
All lines of therapy
Crizanlizumab monotherapy
| back to top |
Regimen
| FDA-recommended dose |
| Study | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|
| Ataga et al. 2016 (SUSTAIN) | Randomized Phase II (E-RT-esc) | Placebo | Superior annual rate of crises |
| Crizanlizumab; 2.5 mg/kg | Not reported |
Chemotherapy
- 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 | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|
| Charache et al. 1995 (MSH) | 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
- 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
L-glutamine monotherapy
| back to top |
Regimen
| Study | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|
| Niihara et al. 2018 (GLUSCC09-01) | 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
RBC transfusions
| back to top |
Regimen
| Study | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|
| DeBaun et al. 2014 (SIT) | 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