title: Long-term results using hydroxyurea/phlebotomy for reducing secondary stroke risk in children with sickle cell anemia and iron overload.
abstract: Children with sickle cell anemia (SCA) and a primary overt stroke are at high risk of recurrent (secondary) stroke. Chronic transfusion therapy dramatically reduces but does not eliminate this high risk, and inevitably results in transfusion-related hemosiderosis. We previously reported the use of hydroxyurea/phlebotomy as an alternative to transfusions to reduce the risk of secondary stroke and improve management of iron overload in 35 children with SCA. To report long-term results, we retrospectively reviewed clinical and laboratory data through October 2008. With a median of 5.6 years and total of 219 patient-years of follow-up, 10 of 35 patients (29%) had recurrent stroke after switching to hydroxyurea; seven were previously reported and three new strokes occurred during extended follow-up. The overall secondary stroke event rate was 4.6 per 100 patient-years. Children on hydroxyurea received serial phlebotomy and had lower mean serum ferritin values than children on transfusions (591 ng/mL vs. 3410 ng/mL, P = 0.02). In this cohort, long-term hydroxyurea treatment reduced but did not eliminate the risk of stroke recurrence and, uniquely, allowed phlebotomy to reduce iron overload. Long-term assessments of this therapy should evaluate risk factors for secondary stroke and assessments of hemosiderosis, neurocognitive outcome, and health-related quality of life.
authors: Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
authors: Anthea
authors: A
authors: Greenway
authors: Russell E
authors: RE
authors: Ware
authors: Courtney D
authors: CD
authors: Thornburg
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000150:complications / Q000628:therapy*; D000986:Antisickling Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D015331:Cohort Studies; D003131:Combined Modality Therapy; D005500:Follow-Up Studies; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000627:therapeutic use*; D019190:Iron Overload / Q000209:etiology / Q000628:therapy*; D008297:Male; D008499:Medical Records; D018962:Phlebotomy / Q000379:methods*; D012189:Retrospective Studies; D012306:Risk; D055502:Secondary Prevention; D020521:Stroke / Q000517:prevention & control*; D013997:Time Factors; D065227:Transfusion Reaction; D055815:Young Adult
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea