{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T21:00:14Z","timestamp":1791320414492,"version":"4.3.1"},"reference-count":22,"publisher":"American Society of Hematology","issue":"11","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2001,6,1]]},"abstract":"<jats:p>The short-term beneficial effect of hydroxyurea (HU) in sickle cell disease (SCD) has been proven by randomized studies in children and adults. The Belgian registry of HU-treated SCD patients was created to evaluate its long-term efficacy and toxicity. The median follow-up of the 93 patients registered is 3.5 years; clinical and laboratory data have been obtained for 82 patients at 1 year, 61 at 2 years, 44 at 3 years, 33 at 4 years, and 22 after 5 years. On HU, the number of hospitalizations and days hospitalized dropped significantly. Analysis of the 22 patients with a minimum of 5 years of follow-up confirm a significant difference in the number of hospitalizations (P\u2009=\u2009.0002) and days in the hospital (P\u2009&amp;lt;\u2009.01), throughout the treatment when compared to prior to HU therapy. The probabilities of not experiencing any event or any vaso-occlusive crisis requiring hospitalization during the 5 years of treatment were, respectively, 47% and 55%. On HU, the rate per 100 patient-years of severe events was estimated to be 3.5% for acute chest syndrome, 1.2% for aplastic crisis, 0.4% for splenic sequestration; it was 0% for the 9 patients with a history of stroke or transient ischemic attack followed for an average of 4 years. No important adverse effect occurred. Long-term chronic treatment with HU for patients with SCD appears feasible, effective, and devoid of any major toxicity; in patients with a history of stroke, HU may be a valid alternative to chronic transfusion support.<\/jats:p>","DOI":"10.1182\/blood.v97.11.3628","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T08:46:47Z","timestamp":1027759607000},"page":"3628-3632","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":170,"title":["Five years of experience with hydroxyurea in children and young adults with sickle cell disease"],"prefix":"10.1182","volume":"97","author":[{"given":"Alina","family":"Ferster","sequence":"first","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parvine","family":"Tahriri","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christiane","family":"Vermylen","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Genevie\u0300ve","family":"Sturbois","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francis","family":"Corazza","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Fondu","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Devalck","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie F.","family":"Dresse","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Walter","family":"Feremans","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen","family":"Hunninck","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michele","family":"Toppet","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Philippet","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Van Geet","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Sariban","sequence":"additional","affiliation":[{"name":"From the Hemato-Oncology Unit, Ho\u0302pital Universitaire des Enfants Reine Fabiola; the Department of Pediatric Hematology, Cliniques Universitaires St Luc; and the Department of Hematology, Ho\u0302pital Erasme, Brussels, Belgium; Service de Pe\u0301diatrie, Clinique de l'Espe\u0301rance, Montegne\u0301e, Belgium; Service de Pe\u0301diatrie, Ho\u0302pital de la Citadelle, Lie\u0300ge, Belgium; Kliniek voor Kinderziekten, Universitair Ziekenhuis, Ghent, Belgium; and UZ Gasthuisberg, Leuven, Belgium."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"key":"2020021217431815800_B1","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJM199107043250103","article-title":"Pain in sickle cell disease.","volume":"325","author":"Platt","year":"1991","journal-title":"N Engl J Med."},{"key":"2020021217431815800_B2","first-page":"360","article-title":"The variable expression of sickle cell disease is genetically determined.","volume":"27","author":"Powars","year":"1990","journal-title":"Semin Hematol."},{"key":"2020021217431815800_B3","doi-asserted-by":"crossref","first-page":"1639","DOI":"10.1056\/NEJM199406093302303","article-title":"Mortality in sickle cell disease: life expectancy and risk factors for early death.","volume":"330","author":"Platt","year":"1994","journal-title":"N Engl J Med."},{"key":"2020021217431815800_B4","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1097\/00005792-198801000-00005","article-title":"Sickle cell chronic lung disease: prior morbidity and the risk of pulmonary failure.","volume":"67","author":"Powars","year":"1988","journal-title":"Medicine."},{"key":"2020021217431815800_B5","first-page":"202","article-title":"Sickle cell anemia: beta-S-gene-cluster haplotypes as prognostic indicators of vital organ failure.","volume":"28","author":"Powars","year":"1991","journal-title":"Semin Hematol."},{"key":"2020021217431815800_B6","doi-asserted-by":"crossref","first-page":"1317","DOI":"10.1056\/NEJM199505183322001","article-title":"Effect of hydroxyurea on the frequency of painful crisis in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia.","volume":"332","author":"Charache","year":"1995","journal-title":"N Engl J Med."},{"key":"2020021217431815800_B7","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1097\/00005792-199611000-00002","article-title":"Hydroxyurea and sickle cell anemia: clinical utility of a myelosuppressive \u201cswitching\u201d agent. The multicenter study of hydroxyurea in sickle cell anemia.","volume":"75","author":"Charache","year":"1996","journal-title":"Medicine."},{"key":"2020021217431815800_B8","doi-asserted-by":"crossref","first-page":"1960","DOI":"10.1182\/blood.V88.6.1960.bloodjournal8861960","article-title":"Hydroxyurea for treatment of severe sickle cell anemia : a pediatric clinical trial.","volume":"88","author":"Ferster","year":"1996","journal-title":"Blood."},{"key":"2020021217431815800_B9","first-page":"30","article-title":"Use of hydroxyurea in children with sickle cell disease: what comes next?","volume":"34","author":"Ohene-Frempong","year":"1997","journal-title":"Semin Hematol."},{"key":"2020021217431815800_B10","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1016\/S0140-6736(88)92239-8","article-title":"Bone marrow transplantation in 5 children with sickle cell anemia.","volume":"1","author":"Vermylen","year":"1988","journal-title":"Lancet."},{"key":"2020021217431815800_B11","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1111\/j.1365-2141.1992.tb06407.x","article-title":"Bone marrow transplantation for severe sickle cell anemia.","volume":"80","author":"Ferster","year":"1992","journal-title":"Br J Haematol."},{"key":"2020021217431815800_B12","first-page":"18","article-title":"Bone marrow transplantation for sickle cell disease: the European experience.","volume":"16","author":"Vermylen","year":"1994","journal-title":"Am J Pediatr Hematol Oncol."},{"key":"2020021217431815800_B13","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/sj.bmt.1701291","article-title":"Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium.","volume":"22","author":"Vemylen","year":"1998","journal-title":"Bone Marrow Transplant."},{"key":"2020021217431815800_B14","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1097\/00043426-199707000-00009","article-title":"Three year follow-up of hydroxyurea treatment in children with sickle cell disease. The French Study Group on Sickle Cell Disease.","volume":"19","author":"De Montalembert","year":"1997","journal-title":"Am J Pediatr Heamatol Oncol."},{"key":"2020021217431815800_B15","first-page":"1550","article-title":"Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I\/II trial.","volume":"94","author":"Kinney","year":"1999","journal-title":"Blood."},{"key":"2020021217431815800_B16","first-page":"288","article-title":"and the Cooperative Study of Sickle Cell Disease. 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