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Hydroxycarbamide treatment reduces transcranial Doppler velocity in the absence of transfusion support in children with sickle cell anaemia, elevated transcranial Doppler velocity, and cerebral vasculopathy: the EXTEND trial.
Rankine-Mullings, Angela; Reid, Marvin; Soares, Deanne; Taylor-Bryan, Carolyn; Wisdom-Phipps, Margaret; Aldred, Karen; Latham, Teresa; Schultz, William H; Knight-Madden, Jennifer; Badaloo, Asha; Lane, Adam; Adams, Robert J; Ware, Russell E.
Afiliação
  • Rankine-Mullings A; Sickle Cell Unit, Caribbean Institute for Health Research, The University of the West Indies, Kingston, Jamaica.
  • Reid M; Tropical Metabolism Research Unit, Caribbean Institute for Health Research Institute, The University of the West Indies, Kingston, Jamaica.
  • Soares D; Radiology Section, Department of Surgery, Radiology, Anaesthesia and Intensive Care, The University of the West Indies, Kingston, Jamaica.
  • Taylor-Bryan C; Tropical Metabolism Research Unit, Caribbean Institute for Health Research Institute, The University of the West Indies, Kingston, Jamaica.
  • Wisdom-Phipps M; Sickle Cell Unit, Caribbean Institute for Health Research, The University of the West Indies, Kingston, Jamaica.
  • Aldred K; Sickle Cell Unit, Caribbean Institute for Health Research, The University of the West Indies, Kingston, Jamaica.
  • Latham T; Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Schultz WH; Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Knight-Madden J; Sickle Cell Unit, Caribbean Institute for Health Research, The University of the West Indies, Kingston, Jamaica.
  • Badaloo A; Tropical Metabolism Research Unit, Caribbean Institute for Health Research Institute, The University of the West Indies, Kingston, Jamaica.
  • Lane A; Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Adams RJ; Department of Paediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
  • Ware RE; Neurology, College of Medicine, Medical University of South Carolina, Charleston, SC, USA.
Br J Haematol ; 195(4): 612-620, 2021 11.
Article em En | MEDLINE | ID: mdl-34291449
EXpanding Treatment for Existing Neurological Disease (EXTEND) investigated whether hydroxycarbamide lowers transcranial Doppler (TCD) velocities in Jamaican children with sickle cell anaemia (SCA) and elevated TCD velocity with or without previous stroke. Forty-three children (age 2-17 years) with baseline maximum time-averaged mean velocity (TAMV) ≥ 170 cm/s were stratified into three risk categories based on treatment status and stroke history: Group 1 (no history of stroke, on hydroxycarbamide, n = 12); and Groups 2 (no stroke, no hydroxycarbamide, n = 21) and 3 (previous stroke, no hydroxycarbamide, n = 10). Open-label hydroxycarbamide at 20 mg/kg/day was commenced, with escalation to maximum tolerated dose (MTD) based on mild marrow suppression (average dose 25·4 ± 4·5 mg/kg/day). TCD was performed every six months with brain magnetic resonance imaging (MRI)/magnetic resonance angiography (MRA) at baseline and after 18-months of hydroxycarbamide. The maximum TAMV decreased significantly compared to baseline (24 ± 30 cm/s, P < 0·0001), with similar declines in all groups. Clinical stroke occurred in five children, one in Group 1, none in Group 2, and four in Group 3, P = 0·0032, comparing group incidence rates. Brain MRI/MRA was stable in children without clinical stroke. EXTEND documents the feasibility and benefits of hydroxycarbamide at MTD to lower TCD velocities and reduce stroke risk in children with SCA and no history of primary stroke in low-resource settings without transfusion management.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Circulação Cerebrovascular / Transtornos Cerebrovasculares / Ultrassonografia Doppler Transcraniana / Hidroxiureia / Anemia Falciforme / Antidrepanocíticos Tipo de estudo: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male País/Região como assunto: Caribe ingles / Jamaica Idioma: En Revista: Br J Haematol Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Jamaica País de publicação: Reino Unido
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Circulação Cerebrovascular / Transtornos Cerebrovasculares / Ultrassonografia Doppler Transcraniana / Hidroxiureia / Anemia Falciforme / Antidrepanocíticos Tipo de estudo: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Male País/Região como assunto: Caribe ingles / Jamaica Idioma: En Revista: Br J Haematol Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Jamaica País de publicação: Reino Unido