Long-term results using hydroxyurea/phlebotomy for reducing secondary stroke risk in children with sickle cell anemia and iron overload

American Journal of Hematology

Volumn 86, Issue 4, 2011, Pages 357-361

  Greenway, Anthea ^a   Ware, Russell E ^b   Thornburg, Courtney D ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

FERRITIN; HYDROXYUREA;

ADOLESCENT; ADULT; ARTICLE; CHELATION THERAPY; FERRITIN BLOOD LEVEL; FOLLOW UP; HUMAN; IRON OVERLOAD; MAJOR CLINICAL STUDY; MALE; NEUROIMAGING; PATIENT COMPLIANCE; PHLEBOTOMY; PRIORITY JOURNAL; SICKLE CELL ANEMIA; STROKE;

ADOLESCENT; ADULT; ANEMIA, SICKLE CELL; ANTISICKLING AGENTS; BLOOD TRANSFUSION; CHILD; COHORT STUDIES; COMBINED MODALITY THERAPY; FOLLOW-UP STUDIES; HUMANS; HYDROXYUREA; IRON OVERLOAD; MALE; MEDICAL RECORDS; PHLEBOTOMY; RECURRENCE; RETROSPECTIVE STUDIES; RISK; STROKE; TIME FACTORS; YOUNG ADULT;

EID: 79953065611     PISSN: 03618609     EISSN: 10968652     Source Type: Journal    
DOI: 10.1002/ajh.21986     Document Type: Article

References (33)

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. Russell MO, Goldberg HI, Hodson A, et al. Effect of transfusion therapy on arteriographic abnormalities and on recurrence of stroke in sickle cell disease. Blood 1984; 63: 162-169.
3. Powars D, Wilson B, Imbus C, et al. The natural history of stroke in sickle cell disease. Am J Med 1978; 65: 461-471.
4. Powars D, Imbus C. Cerebral vascular accidents in sickle cell anemia. Tex Rep Biol Med 1980; 40: 293-304.
5. Scothorn DJ, Price C, Schwartz D, et al. Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke. J Pediatr 2002; 140: 348-354.
6. Pegelow CH, Adams RJ, McKie V, et al. Risk of recurrent stroke in patients with sickle cell disease treated with erythrocyte transfusions. J Pediatr 1995; 126: 896-899.
7. Rana S, Houston PE, Surana N, et al. Discontinuation of long-term transfusion therapy in patients with sickle cell disease and stroke. J Pediatr 1997; 131: 757-760.
8. 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.
9. Wilimas J, Goff JR, Anderson HRJr, et al. Efficacy of transfusion therapy for one to two years in patients with sickle cell disease and cerebrovascular accidents. J Pediatr 1980; 96: 205-208.
10. 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.
11. Adamkiewicz TV, Abboud MR, Paley C, et al. Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injury. Blood 2009; 114: 4632-4638.
12. Steinberg MH, McCarthy WF, Castro O, et al. The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up. Am J Hematol 2010; 85: 403-408.
13. 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.
14. 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.
15. Hankins JS, Ware RE, Rogers ZR, et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: The HUSOFT extension study. Blood 2005; 106: 2269-2275.
16. Ware RE. How I use hydroxyurea to treat young patients with sickle cell anemia. Blood 2010.
17. 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.
18. 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.
19. 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.
20. Schmugge M, Frischknecht H, Yonekawa Y, et al. Stroke in hemoglobin (SD) sickle cell disease with moyamoya: Successful hydroxyurea treatment after cerebrovascular bypass surgery. Blood 2001; 97: 2165-2167.
21. 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.
22. 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; author reply 964.
23. Brousse V, Hertz-Pannier L, Consigny Y, et al. Does regular blood transfusion prevent progression of cerebrovascular lesions in children with sickle cell disease? Ann Hematol 2009; 88: 785-788.
24. Hulbert ML, McKinstry RC, Lacey JL, et al. Silent cerebral infarcts occur despite regular blood transfusion therapy after first stroke in children with sickle cell disease. Blood 2011; 117: 772-779.
25. 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.
26. Ballas SK. Iron overload is a determinant of morbidity and mortality in adult patients with sickle cell disease. Semin Hematol 2001; 38: 30-36.
27. Fung EB, Harmatz P, Milet M, et al. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload. Am J Hematol 2007; 82: 255-265.
28. Payne KA, Rofail D, Baladi JF, et al. Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Ther 2008; 25: 725-742.
29. Rofail D, Abetz L, Viala M, et al. Satisfaction and adherence in patients with iron overload receiving iron chelation therapy as assessed by a newly developed patient instrument. Value Health 2009; 12: 109-117.
30. Raphael JL, Bernhardt MB, Mahoney DH, Mueller BU. Oral iron chelation and the treatment of iron overload in a pediatric hematology center. Pediatr Blood Cancer 2009; 52: 616-620.
31. Mednick LM, Braunstein J, Neufeld E. Oral chelation: Should it be used with young children? Pediatr Blood Cancer 2010; 55: 603-605.
32. Flanagan JM, Steward S, Hankins JS, et al. Microarray analysis of liver gene expression in iron overloaded patients with sickle cell anemia and beta-thalassemia. Am J Hematol 2009; 84: 328-334.
33. Karam LB, Disco D, Jackson SM, et al. Liver biopsy results in patients with sickle cell disease on chronic transfusions: Poor correlation with ferritin levels. Pediatr Blood Cancer 2008; 50: 62-65.