Information de reference pour ce titreAccession Number: | 01445489-201208000-00043.
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Author: | Strouse, John J. MD, PhD 1,2,*; Heeney, Matthew M. MD 3,4
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Institution: | (1)Division of Pediatric Hematology, Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland (2)Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland (3)Division of Hematology/Oncology, Department of Medicine, Children's Hospital Boston, Boston, Massachusetts (4)Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts (*)Division of Pediatric Hematology, 720 Rutland Avenue, Ross 1125, Baltimore, MD 21205.
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Title: | |
Source: | Pediatric Blood & Cancer. 59(2):365-371, August 2012.
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Abstract: | Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children. Pediatr Blood Cancer 2012;59:365-371. (C) 2012 Wiley Periodicals, Inc.
Copyright (C) 2012 John Wiley & Sons, Inc.
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Author Keywords: | children; efficacy; hydroxyurea; sickle cell disease.
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References: | 1. Charache S, Barton FB, Moore RD, et al. Hydroxyurea and sickle cell anemia. Clinical utility of a myelosuppressive "switching" agent. The multicenter study of hydroxyurea in sickle cell anemia. Medicine (Baltimore), 1996; 75: 300- 326.
2. Kato GJ, Gladwin MT, Steinberg MH. Deconstructing sickle cell disease: Reappraisal of the role of hemolysis in the development of clinical subphenotypes. Blood Rev, 2007; 21: 37- 47.
3. Benkerrou M, Delarche C, Brahimi L, et al. Hydroxyurea corrects the dysregulated l-selectin expression and increased H2O2 production of polymorphonuclear neutrophils from patients with sickle cell anemia. Blood, 2002; 99: 2297- 2303.
4. Gladwin MT, Shelhamer JH, Ognibene FP, et al. Nitric oxide donor properties of hydroxyurea in patients with sickle cell disease. Br J Haematol, 2002; 116: 436- 444.
5. Fetal hemoglobin induction. In: National Institutes of Health National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources; The management of sickle cell disease: NIH publication no. 02-2117. Bethesda, MD: 2002; p 161- 166.
6. Segal J, Strouse J, Beach M, et al. Hydroxyurea for the treatment of sickle cell disease evidence report (publication no. 08-e007). Washington, D.C.: Agency for Healthcare Research and Quality; 2008.
7. Strouse JJ, Lanzkron S, Beach MC, et al. Hydroxyurea for sickle cell disease: A systematic review for efficacy and toxicity in children. Pediatrics, 2008; 122: 1332- 1342.
8. Brawley OW, Cornelius LJ, Edwards LR, et al. National Institutes of Health consensus development conference statement: Hydroxyurea treatment for sickle cell disease. Ann Intern Med, 2008; 148: 932- 938.
9. Wang WC, Ware RE, Miller ST, et al. Hydroxycarbamide in very young children with sickle-cell anaemia: A multicentre, randomised, controlled trial (BABY HUG). Lancet, 2011; 377: 1663- 1672.
10. Wang W, Brugnara C, Snyder C, et al. The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: Results of the multi-centre CHAMPS trial. Br J Haematol, 2011; 152: 771- 776.
11. Ware RE, Helms RW. Stroke with transfusions changing to hydroxyurea (switch): A phase 3 randomized clinical trial for treatment of children with sickle cell anemia, previous stroke, and iron overload. ASH Ann Meeting Abstr, 2010; 116: 844.
12. Hankins JS, Helton KJ, McCarville MB, et al. Preservation of spleen and brain function in children with sickle cell anemia treated with hydroxyurea. Pediatr Blood Cancer, 2008; 50: 293- 297.
13. Lefevre N, Dufour D, Gulbis B, et al. Use of hydroxyurea in prevention of stroke in children with sickle cell disease. Blood, 2008; 111: 963- 964.
14. Greenway A, Ware RE, Thornburg CD. Long-term results using hydroxyurea/phlebotomy for reducing secondary stroke risk in children with sickle cell anemia and iron overload. Am J Hematol, 2011; 86: 357- 361.
15. Gordeuk VR, Campbell A, Rana S, et al. Relationship of erythropoietin, fetal hemoglobin, and hydroxyurea treatment to tricuspid regurgitation velocity in children with sickle cell disease. Blood, 114: 4639- 4644.
16. Lobo C, Hankins JS, Moura P, et al. Hydroxyurea therapy reduces mortality among children with sickle cell disease. ASH Ann Meet Abstr, 2010; 116: 843.
17. Voskaridou E, Christoulas D, Bilalis A, et al. The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: Results of a 17-year, single-center trial (LASHS). Blood, 2010; 115: 2354- 2363.
18. Berthaut I, Guignedoux G, Kirsch-Noir F, et al. Influence of sickle cell disease and treatment with hydroxyurea on sperm parameters and fertility of human males. Haematologica, 2008; 93: 988- 993.
19. Lukusa AK, Vermylen C, Vanabelle B, et al. Bone marrow transplantation or hydroxyurea for sickle cell anemia: Long-term effects on semen variables and hormone profiles. Pediatr Hematol Oncol, 2009; 26: 186- 194.
20. Lukusa AK, Vermylen C. Use of hydroxyurea from childhood to adult age in sickle cell disease: Semen analysis. Haematologica, 2008; 93: e67 discussion e68.
21. Ballas SK, McCarthy WF, Guo N, et al. Exposure to hydroxyurea and pregnancy outcomes in patients with sickle cell anemia. J Natl Med Assoc, 2009; 101: 1046- 1051.
22. Flanagan JM, Howard TA, Mortier N, et al. Assessment of genotoxicity associated with hydroxyurea therapy in children with sickle cell anemia. Mutat Res, 2010; 698: 38- 42.
23. McGann PT, Ware RE. Hydroxyurea for sickle cell anemia: What have we learned and what questions still remain? Curr Opin Hematol, 2011; 18: 158- 165.
24. Platt OS, Orkin SH, Dover G, et al. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J Clin Invest, 1984; 74: 652- 656.
25. Heeney MM, Ware RE. Hydroxyurea for children with sickle cell disease. Pediatr Clin North Am, 2008; 55: 483- 501.
26. Ware RE. How I use hydroxyurea to treat young patients with sickle cell anemia. Blood, 2010; 115: 5300- 5311.
27. Mueller BU. When should hydroxyurea be used for children with sickle cell disease? Pediatrics, 2008; 122: 1365- 1366.
28. Ryan KM, Heeney MM. Use of hydroxyurea in pediatric patients with sickle cell disease. Boston: 2004; (Accessed June 1, 2011, at http://www.nepscc.org/NewFiles/H...- ouverture dans une nouvelle fenêtre.).
29. Lane PA, Buchanan GR, Hutter JJ, et al., Sickle cell disease in children and adolescents: Diagnosis, guidelines for comprehensive care, and care paths and protocols for management of acute and chronic complications. 2001. (Accessed June 1, 2011, at www.dshs.state.tx.us/newborn/pdf/sedona02.pdf.).
30. Kinney TR, Helms RW, O'Branski EE, et al. Safety of hydroxyurea in children with sickle cell anemia: Results of the HUG-KIDS study, a phase I/II trial. Pediatric hydroxyurea group. Blood, 1999; 94: 1550- 1554.
31. Wang WC, Wynn LW, Rogers ZR, et al. A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia. J Pediatr, 2001; 139: 790- 796.
32. Thornburg CD, Dixon N, Burgett S, et al. Efficacy of hydroxyurea to prevent organ damage in young children with sickle cell anemia. Blood, 2007; 110: 3386.
33. Zimmerman SA, Schultz WH, Davis JS, et al. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood, 2004; 103: 2039- 2045.
34. Zimmerman SA, Schultz WH, Burgett S, et al. Hydroxyurea therapy lowers transcranial doppler flow velocities in children with sickle cell anemia. Blood, 2007; 110: 1043- 1047.
35. Heeney MM, Whorton MR, Howard TA, et al. Chemical and functional analysis of hydroxyurea oral solutions. J Pediatr Hematol Oncol, 2004; 26: 179- 184.
36. Harrod VL, Howard T, Abboud MR, et al. Chemical and functional analysis of generic hydroxyurea formulations. Pediatr Hematol Oncol, 2008; 25: 423- 429.
37. Bridges KR, Barabino GD, Brugnara C, et al. A multiparameter analysis of sickle erythrocytes in patients undergoing hydroxyurea therapy. Blood, 1996; 88: 4701- 4710.
38. Charache S, Dover GJ, Moore RD, et al. Hydroxyurea: Effects on hemoglobin F production in patients with sickle cell anemia. Blood, 1992; 79: 2555- 2565.
39. Ware RE, Zimmerman SA, Schultz WH. Hydroxyurea as an alternative to blood transfusions for the prevention of recurrent stroke in children with sickle cell disease. Blood, 1999; 94: 3022- 3026.
40. Ware RE, Zimmerman SA, Sylvestre PB, et al. Prevention of secondary stroke and resolution of transfusional iron overload in children with sickle cell anemia using hydroxyurea and phlebotomy. J Pediatr, 2004; 145: 346- 352.
41. Becker MH, Maiman LA. Sociobehavioral determinants of compliance with health and medical care recommendations. Med Care, 1975; 13: 10- 24.
42. Candrilli SD, O'Brien SH, Ware RE, et al. Hydroxyurea adherence and associated outcomes among Medicaid enrollees with sickle cell disease. Am J Hematol, 2011; 86: 273- 277.
43. Bunn HF, Nathan DG, Dover GJ, et al. Pulmonary hypertension and nitric oxide depletion in sickle cell disease. Blood, 2010; 116: 687- 692.
44. McGann PT, Howard TA, Flanagan JM, et al. Chromosome damage and repair in children with sickle cell anaemia and long-term hydroxycarbamide exposure. Br J Haematol, 2011; 154: 134- 140.
45. Ware RE, Aygun B. Advances in the use of hydroxyurea. Hematology Am Soc Hematol Educ Program, 2009; 62- 69.
46. Weatherall DJ. The inherited diseases of hemoglobin are an emerging global health burden. Blood, 2010; 115: 4331- 4336.
47. Quinn CT, Rogers ZR, McCavit TL, et al. Improved survival of children and adolescents with sickle cell disease. Blood, 2010; 115: 3447- 3452.
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Language: | English.
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Document Type: | Review: Special Series - Sickle Cell Disease: The First 100 Years.
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Journal Subset: | Clinical Medicine.
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ISSN: | 1545-5009
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DOI Number: | https://dx.doi.org/10.1002/pbc.2...- ouverture dans une nouvelle fenêtre
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Annotation(s) | |
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