title: Prognostic significance of early vaso-occlusive complications in children with sickle cell anemia.
abstract: Sickle cell anemia (SS) is highly phenotypically variable, and early predictors of outcome could guide clinical care. To determine whether early vaso-occlusive complications predicted subsequent adverse outcomes in the Dallas Newborn Cohort, we studied all members with SS or sickle-beta0-thalassemia who presented in their first year of life and had 5 years or more of follow-up. We defined 3 potential early predictors: hospitalizations in the first 3 years of life for (1) painful events other than dactylitis, (2) dactylitis, and (3) acute chest syndrome (ACS). We studied the associations of these predictors with the following late adverse outcomes (occurring after the third birthday): death, first overt stroke, use of disease-modifying therapy, and hospitalizations for pain events and ACS. None of the early events predicted death or stroke. Early pain and ACS both predicted a modest, temporary increase in the number of later painful episodes, but early ACS strongly increased the odds of more frequent ACS throughout childhood. Dactylitis had limited utility as a predictor. Although we still lack a useful prognostic framework for young children with SS, those who experience early ACS might be candidates for higher risk interventions to mitigate or cure their disease.
authors.affiliation: Division of Hematology-Oncology, Department of Pediatrics, University of Texas Southwestern Medical Center, 5323 Harry Hines Blvd, Dallas, TX 75390, USA. charles.quinn@utsouthwestern.edu
authors.forename: Charles T
authors.initials: CT
authors.lastname: Quinn
authors.forename: Elizabeth P
authors.initials: EP
authors.lastname: Shull
authors.forename: Naveed
authors.initials: N
authors.lastname: Ahmad
authors.forename: Nancy J
authors.initials: NJ
authors.lastname: Lee
authors.forename: Zora R
authors.initials: ZR
authors.lastname: Rogers
authors.forename: George R
authors.initials: GR
authors.lastname: Buchanan
mesh_terms: D000208:Acute Disease; D000755:Anemia, Sickle Cell / Q000150:complications* / Q000188:drug therapy / Q000235:genetics / Q000401:mortality / Q000601:surgery; D001157:Arterial Occlusive Diseases / Q000453:epidemiology / Q000209:etiology*; D001803:Blood Transfusion; D015331:Cohort Studies; D003131:Combined Modality Therapy; D016208:Databases, Factual; D005260:Female; D005385:Fingers / Q000473:pathology; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006579:Heterozygote; D006720:Homozygote; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010146:Pain / Q000453:epidemiology / Q000209:etiology; D011379:Prognosis; D012189:Retrospective Studies; D012805:Sickle Cell Trait / Q000150:complications* / Q000188:drug therapy / Q000235:genetics / Q000401:mortality / Q000601:surgery; D020521:Stroke / Q000453:epidemiology / Q000209:etiology; D013781:Texas / Q000453:epidemiology; D017086:beta-Thalassemia / Q000150:complications* / Q000235:genetics / Q000401:mortality / Q000601:surgery
chemical_list: D006918:Hydroxyurea