Preventing stroke among children with sickle cell anemia: An analysis of strategies that involve transcranial Doppler testing and chronic transfusion

Pediatrics

Volumn 120, Issue 4, 2007, Pages

  Mazumdar, Maitreyi ^a,b   Heeney, Matthew M ^a,c   Sox, Colin M ^d   Lieu, Tracy A ^a,d  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b Harvard Pediatric Health Services Research Fellowship Program   (United States)

^c DANA FARBER CANCER INSTITUTE   (United States)

^d HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Health services research; Sickle cell anemia; Stroke; Transcranial Doppler; Transfusions

Indexed keywords

IRON CHELATING AGENT;

ARTICLE; BLOOD TRANSFUSION; CHILD; CLINICAL DECISION MAKING; COHORT ANALYSIS; DOPPLER ECHOGRAPHY; EARLY DIAGNOSIS; HIGH RISK PATIENT; HUMAN; IRON CHELATION; LIFE EXPECTANCY; MAJOR CLINICAL STUDY; PATIENT COMPLIANCE; PERIODIC MEDICAL EXAMINATION; PRIORITY JOURNAL; RISK BENEFIT ANALYSIS; SCREENING TEST; SENSITIVITY ANALYSIS; SICKLE CELL ANEMIA; STROKE;

ANEMIA, SICKLE CELL; CEREBROVASCULAR ACCIDENT; CEREBROVASCULAR CIRCULATION; CHILD; CHILD, PRESCHOOL; DECISION SUPPORT TECHNIQUES; ECHOENCEPHALOGRAPHY; ERYTHROCYTE TRANSFUSION; HUMANS; IRON CHELATING AGENTS; LIFE EXPECTANCY; MARKOV CHAINS; MASS SCREENING; PRACTICE GUIDELINES; ULTRASONOGRAPHY, DOPPLER;

EID: 35148900984     PISSN: 00314005     EISSN: 02105721     Source Type: Journal    
DOI: 10.1542/peds.2006-2002     Document Type: Article

References (45)

1. Ohene-Frempong K, Weiner SJ, Sleeper LA, et al. Cerebrovascular accidents in sickle cell disease: rates and risk factors. Blood. 1998;91:288-294
2. Adams RJ, McKie VC, Hsu L, et al. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med. 1998;339:5-11
3. Adams R, McKie V, Nichols F, et al. The use of transcranial ultrasonography to predict stroke in sickle cell disease. N Engl J Med. 1992;326:605-610
4. Adams RJ, McKie VC, Carl EM, et al. Long-term stroke risk in children with sickle cell disease screened with transcranial Doppler. Ann Neurol. 1997;42:699-704
5. National Institutes of Health, National Library of Medicine. Clinical alert: periodic transfusions lower stroke risk in children with sickle cell anemia. Available at: www.nlm.nih.gov/databases/alerts/sickle97.html. Accessed October 5, 2005
6. Vichinsky EP. Current issues with blood transfusions in sickle cell disease. Semin Hematol. 2001;38(1 suppl 1):14-22
7. Lane PA, Buchanan GE, Ware RE. Variable approaches to therapeutic options for children with sickle cell disease (SCD): a practice survey of the American Society of pediatric Hematology/Oncology (ASPH/O) [abstract]. Pediatr Res. 2002;51(4 pt 2):238A
8. Fullerton HJ, Gardner M, Adams RJ, Lo LC, Johnston SC. Obstacles to primary stroke prevention in children with sickle cell disease. Neurology. 2006;67:1098-1099
9. American Academy of Pediatrics, Section on Hematology/Oncology Committee on Genetics. Health supervision for children with sickle cell disease. Pediatrics. 2002;109:526-535
10. Sox C. Health supervision for children with sickle cell disease. Pediatrics. 2003;111:710-711
11. Sloan MA, Alexandrov AV, Tegeler CH, et al. Assessment: transcranial Doppler ultrasonography - report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology. Neurology. 2004;62:1468-1481
12. National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources. The Management of Sickle Cell Disease. 4th ed. Bethesda, MD: National Institutes of Health; 2002
13. Quinn CT, Rogers ZR, Buchanan GR. Survival of children with sickle cell disease. Blood. 2004;103:4023-4027
14. Wilimas J, Goff JR, Anderson HR Jr, Langston JW, Thompson E. Efficacy of transfusion therapy for one to two years in patients with sickle cell disease and cerebrovascular accidents. J Pediatr. 1980;96:205-208
15. Rana S, Houston PE, Surana N, Shalaby-Rana EI, Castro OL. Discontinuation of long-term transfusion therapy in patients with sickle cell disease and stroke. J Pediatr. 1997;131:757-760
16. Wang WC, Kovnar EH, Tonkin IL, et al. High risk of recurrent stroke after discontinuance of five to twelve years of transfusion therapy in patients with sickle cell disease. J Pediatr. 1991;118:377-382
17. Adams RJ, Brambilla D. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med. 2005;353:2769-2778
18. Gold MR. Cost-effectiveness in Health and Medicine. New York, NY: Oxford University Press; 1996
19. Goldstein LB, Adams R, Alberts MJ, et al. Primary prevention of ischemic stroke: a guideline from the American Heart Association/American Stroke Association Stroke Council: cosponsored by the Atherosclerotic Peripheral Vascular Disease Interdisciplinary Working Group; Cardiovascular Nursing Council; Clinical Cardiology Council; Nutrition, Physical Activity, and Metabolism Council; and the Quality of Care and Outcomes Research Interdisciplinary Working Group: the American Academy of Neurology affirms the value of this guideline [published correction appears in Stroke. 2007;38:207]. Stroke. 2006;37:1583-1633
20. New England Pediatric Sickle Cell Consortium. Prevention and treatment of stroke for pediatric patients with sickle cell disease. Available at: www.nepscc.org/Completed%20CPGs/CVA%20CPG%20NEPSCC%20Dec03.pdf. Accessed November 14, 2005
21. Kuntz K, Weinstein MC. Modelling in economic evaluation. In: Drummond MF, McGuire A, eds. Economic Evaluation in Health Care: Merging Theory With Practice. New York, NY: Oxford University Press; 2001:141-171
22. Arias E. United States Life Tables, 2002. Hyattsville, MD: National Center for Health Statistics; 2004
23. Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle cell disease: life expectancy and risk factors for early death. N Engl J Med. 1994;330:1639-1644
24. Powars D, Wilson B, Imbus C, Pegelow C, Allen J. The natural history of stroke in sickle cell disease. Am J Med. 1978;65:461-471
25. Balkaran B, Char G, Morris JS, Thomas PW, Serjeant BE, Serjeant GR. Stroke in a cohort of patients with homozygous sickle cell disease. J Pediatr. 1992;120:360-366
26. Neonato MG, Guilloud-Bataille M, Beauvais P, et al. Acute clinical events in 299 homozygous sickle cell patients living in France. French Study Group on Sickle Cell Disease. Eur J Haematol. 2000;65:155-164
27. Adams RJ, Brambilla DJ, Granger S, et al. Stroke and conversion to high risk in children screened with transcranial Doppler ultrasound during the STOP study. Blood. 2004;103:3689-3694
28. Lee MT, Piomelli S, Granger S, et al. Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results. Blood. 2006;108:847-852
29. Nietert PJ, Abboud MR, Silverstein MD, Jackson SM. Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis. Blood. 2000;95:3057-3064
30. Schneider AT, Kissela B, Woo D, et al. Ischemic stroke subtypes: a population-based study of incidence rates among blacks and whites. Stroke. 2004;35:1552-1556
31. Wayne AS, Kevy SV, Nathan DG. Transfusion management of sickle cell disease. Blood. 1993;81:1109-1123
32. DiMatteo MR, Giordani PJ, Lepper HS, Croghan TW. Patient adherence and medical treatment outcomes: a meta-analysis. Med Care. 2002;40:794-811
33. Ballas SK. Iron overload is a determinant of morbidity and mortality in adult patients with sickle cell disease. Semin Hematol. 2001;38(1 suppl 1):30-36
34. Vichinsky EP, Earles A, Johnson RA, Hoag MS, Williams A, Lubin B. Alloimmunization in sickle cell anemia and transfusion of racially unmatched blood. N Engl J Med. 1990;322:1617-1621
35. Fullerton HJ, Adams RJ, Zhao S, Johnston SC. Declining stroke rates in Californian children with sickle cell disease. Blood. 2004;104:336-339
36. Barakat LP, Lutz M, Smith-Whitley K, Ohene-Frempong K. Is treatment adherence associated with better quality of life in children with sickle cell disease? Qual Life Res. 2005;14:407-414
37. Steen RG, Helton KJ, Horwitz EM, et al. Improved cerebrovascular patency following therapy in patients with sickle cell disease: initial results in 4 patients who received HLA-identical hematopoietic stem cell allografts. Ann Neurol. 2001;49:222-229
38. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996;335:369-376
39. Walters MC, Storb R, Patience M, et al. Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report - multicenter investigation of bone marrow transplantation for sickle cell disease. Blood. 2000;95:1918-1924
40. Gulbis B, Haberman D, Dufour D, et al. Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience. Blood. 2005;105:2685-2690
41. 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
42. 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
43. Sumoza A, de Bisotti R, Sumoza D, Fairbanks V. Hydroxyurea (HU) for prevention of recurrent stroke in sickle cell anemia (SCA). Am J Hematol. 2002;71:161-165
44. Kirkham FJ, DeBaun MR. Stroke in children with sickle cell disease. Curr Treat Options Neurol. 2004;6:357-375
45. National Institutes of Health, ClinicalTrials.gov. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Available at: www.clinicaltrials.gov/ct/show/NCT00122980. Accessed December 21, 2006