title: Long-term treatment follow-up of children with sickle cell disease monitored with abnormal transcranial Doppler velocities.
abstract: Stroke risk in sickle cell anemia (SCA), predicted by high transcranial Doppler (TCD) velocities, is prevented by transfusions. We present the long-term follow-up of SCA children from the Créteil newborn cohort (1992-2012) detected at risk by TCD and placed on chronic transfusions. Patients with normalized velocities and no stenosis were treated with hydroxyurea, known to decrease anemia and hemolytic rate. Trimestrial Doppler was performed and transfusions restarted immediately in the case of reversion to abnormal velocities. Patients with a genoidentical donor underwent transplant. Abnormal time-averaged maximum mean velocities (TAMMV) ≥200 cm/s were detected in 92 SCA children at a mean age of 3.7 years (range, 1.3-8.3 years). No stroke occurred posttransfusion after a mean follow-up of 6.1 years. Normalization of velocities (TAMMV < 170 cm/s) was observed in 83.5% of patients. Stenosis, present in 27.5% of patients, was associated with the risk of non-normalization (P< .001). Switch from transfusions to hydroxyurea was prescribed for 45 patients, with a mean follow-up of 3.4 years. Reversion, predicted by baseline reticulocyte count ≥400 × 10(9)/L (P< .001), occurred in 28.9% (13/45) patients at the mean age of 7.1 years (range, 4.3-9.5 years). Transplant, performed in 24 patients, allowed transfusions to be safely stopped in all patients and velocities to be normalized in 4 patients who still had abnormal velocities on transfusions. This long-term cohort study shows that transfusions can be stopped not only in transplanted patients but also in a subset of patients switched to hydroxyurea, provided trimestrial Doppler follow-up and immediate restart of transfusions in the case of reversion.
authors: Referral Center for Sickle Cell Disease, Department of Pediatrics, and.
authors: Françoise
authors: F
authors: Bernaudin
authors: Referral Center for Sickle Cell Disease, Medical Imaging Department, Centre Hospitalier Intercommunal de Créteil, Université Paris-Est Créteil Val de Marne, Créteil, France;
authors: Suzanne
authors: S
authors: Verlhac
authors: Referral Center for Sickle Cell Disease, Department of Pediatrics, and.
authors: Cécile
authors: C
authors: Arnaud
authors: Referral Center for Sickle Cell Disease, Department of Pediatrics, and.
authors: Annie
authors: A
authors: Kamdem
authors: Department of Pediatrics, and.
authors: Isabelle
authors: I
authors: Hau
authors: Referral Center for Sickle Cell Disease, Department of Pediatrics, and.
authors: Emmanuella
authors: E
authors: Leveillé
authors: Medical Imaging Department, Centre Hospitalier Intercommunal de Créteil, Université Paris-Est Créteil Val de Marne, Créteil, France;
authors: Manuela
authors: M
authors: Vasile
authors: Medical Imaging Department, Centre Hospitalier Intercommunal de Créteil, Université Paris-Est Créteil Val de Marne, Créteil, France;
authors: Florence
authors: F
authors: Kasbi
authors: Department of Pediatrics, and.
authors: Fouad
authors: F
authors: Madhi
authors: Department of Pediatrics, and.
authors: Christine
authors: C
authors: Fourmaux
authors: Department of Pediatrics, and.
authors: Sandra
authors: S
authors: Biscardi
authors: Department of Hematology, Saint-Louis Hospital and.
authors: Eliane
authors: E
authors: Gluckman
authors: Department of Hematology, Saint-Louis Hospital and.
authors: Gérard
authors: G
authors: Socié
authors: Department of Pediatric Hematology, Robert Debré Hospital, Paris Diderot University, Paris, France; and.
authors: Jean-Hugues
authors: JH
authors: Dalle
authors: Department of Pediatrics, and INSERM Unité 955, Université Paris-Est Créteil Val de Marne, Créteil, France.
authors: Ralph
authors: R
authors: Epaud
authors: Referral Center for Sickle Cell Disease, Department of Pediatrics, and INSERM Unité 955, Université Paris-Est Créteil Val de Marne, Créteil, France.
authors: Corinne
authors: C
authors: Pondarré
mesh_terms: D000755:Anemia, Sickle Cell / Q000000981:diagnostic imaging / Q000503:physiopathology / Q000628:therapy; D001783:Blood Flow Velocity; D001803:Blood Transfusion; D002560:Cerebrovascular Circulation; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage*; D007223:Infant; D008297:Male; D020521:Stroke / Q000000981:diagnostic imaging / Q000503:physiopathology / Q000517:prevention & control; D017585:Ultrasonography, Doppler, Transcranial
chemical_list: D006918:Hydroxyurea