title: Feasibility and efficacy of chronic transfusion for stroke prevention in children with sickle cell disease.
abstract: OBJECTIVE
  In children with sickle cell disease (SCD), chronic transfusion to maintain haemoglobin S (HbS) below 30% markedly decreases both the risk of a first stroke when transcranial Doppler (TCD) ultrasonography shows abnormal cerebral blood flow velocities and the risk of recurrent stroke. Maintaining HbS below 30% may be difficult, especially in countries where blood donors and recipients belong to different ethnic groups and where the availability of closely matched blood products is limited. We assessed the feasibility and efficacy of chronic transfusion with an HbS target of 30% in children with SCD living in the Paris area.
  
  
  METHODS
  We retrospectively studied 29 children aged 6.8 +/- 3.0 yr (3-15 yr) at inclusion who received chronic transfusion either because of abnormal TCD findings (primary prevention group, PPG, n = 17) or because of a previous cerebrovascular event (secondary prevention group, SPG, n = 12 including nine with a history of stroke and three of transient ischaemic attacks).
  
  
  RESULTS
  Mean follow-up was 3.5 +/- 3.0 yr (0.5-12 yr). No cases of stroke occurred in the PPG. In the SPG, one patient with a history of stroke and severe cerebrovascular disease had a recurrence after 11 yr of chronic transfusion, when the HbS level was 20%. The stroke recurrence rate (SPG group) was 1.6/100 patient-years. Mean HbS levels before and after transfusion were 30 +/- 10% and 20.6 +/- 7%, respectively. Two patients acquired red-cell alloantibodies. Of the 29 patients, 22 required iron chelation.
  
  
  CONCLUSIONS
  Regular transfusion maintaining HbS below 30% is feasible and safe in children with SCD in France and protects from overt stroke.
authors.affiliation: Service de Pédiatrie Générale, Hôpital Necker-Enfants Malades, AP-HP, Université Paris Descartes, 75006Paris, France.
authors.forename: Elsa
authors.initials: E
authors.lastname: Mirre
authors.forename: Valentine
authors.initials: V
authors.lastname: Brousse
authors.forename: Laureline
authors.initials: L
authors.lastname: Berteloot
authors.forename: Karen
authors.initials: K
authors.lastname: Lambot-Juhan
authors.forename: Suzanne
authors.initials: S
authors.lastname: Verlhac
authors.forename: Claire
authors.initials: C
authors.lastname: Boulat
authors.forename: Marie-Dominique
authors.initials: MD
authors.lastname: Dumont
authors.forename: Gérard
authors.initials: G
authors.lastname: Lenoir
authors.forename: Mariane
authors.initials: M
authors.lastname: de Montalembert
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000150:complications / Q000628:therapy*; D001803:Blood Transfusion; D002560:Cerebrovascular Circulation; D015913:Chelation Therapy; D002648:Child; D002675:Child, Preschool; D004912:Erythrocytes / Q000276:immunology; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006451:Hemoglobin, Sickle / Q000032:analysis; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy / Q000209:etiology; D007518:Isoantibodies / Q000096:biosynthesis; D008279:Magnetic Resonance Imaging; D008297:Male; D010297:Paris; D012189:Retrospective Studies; D055502:Secondary Prevention; D020521:Stroke / Q000000981:diagnostic imaging / Q000209:etiology / Q000517:prevention & control*; D017585:Ultrasonography, Doppler, Transcranial
chemical_list: D006451:Hemoglobin, Sickle; D007502:Iron Chelating Agents; D007518:Isoantibodies