The effect of hydroxcarbamide therapy on survival of children with sickle cell disease

• Published in: British journal of haematology Vol. 161; no. 6; pp. 852 - 860
• Main Authors: Lopes de Castro Lobo, Clarisse, Pinto, Jorge F. C., Nascimento, Emilia M., Moura, Patricia G., Cardoso, Gilberto P., Hankins, Jane S.
• Format: Journal Article
• Language: English
• Published: Oxford Blackwell 01.06.2013
• Subjects: Adolescent; Anemia, Sickle Cell - drug therapy; Anemia, Sickle Cell - genetics; Anemia, Sickle Cell - mortality; Anemias. Hemoglobinopathies; Antisickling Agents - administration & dosage; Antisickling Agents - adverse effects; Antisickling Agents - therapeutic use; Biological and medical sciences; Cause of Death; Child; Child, Preschool; Diseases of red blood cells; Female; Genotype; Hematologic and hematopoietic diseases; Hemoglobin, Sickle - genetics; Humans; hydroxycarbamide; Hydroxyurea - administration & dosage; Hydroxyurea - adverse effects; Hydroxyurea - therapeutic use; immunizations; Male; Medical sciences; mortality; paediatric; sickle cell disease; Treatment Outcome; Tumors; Human; Antineoplastic agent; Hemoglobinopathy; Immunization; Sickle cell anemia; Hydroxycarbamide; Hematology; Mortality; Hemopathy; Survival; Genetic disease; paediatric; sickle cell disease; Hemolytic anemia; Cancerology; Treatment; Antimetabolic; immunizations; Child
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.12323

Summary Although evidence is accumulating that hydroxycarbamide decreases mortality among adults with sickle cell disease (SCD), there are no published data regarding the effect of hydroxycarbamide on mortality among children. The Paediatric Hydroxycarbamide Program was established to treat children with SCD aged 3–18 years if they met disease severity criteria. Mortality data and clinical/laboratorial effects of hydroxycarbamide were retrospectively collected for the first 9 years of the Program. Mortality among those who received hydroxycarbamide was compared to that of untreated children. Among 1760 subjects, 267 received hydroxycarbamide at a median dose of 20·8 mg/kg/d (range 10–32) for a median of 2 years (range 0·1–6·5). Survival among hydroxycarbamide‐treated children was significantly greater than that among untreated ones (99·5% vs. 94·5%, P = 0·01), due primarily to fewer deaths from acute chest syndrome and infection. Hydroxycarbamide therapy was significantly associated with increases in haemoglobin concentration, fetal haemoglobin, mean corpuscular volume, and reduction in platelet counts, reticulocytes and neutrophils. Toxicity was minimal and predominantly mild reversible neutropenia. Significantly fewer hospitalizations and emergency room visits, and shorter admissions were observed among hydroxycarbamide‐treated subjects, when compared to the 12‐month period prior to treatment initiation. Hydroxycarbamide therapy reduces disease severity and is probably associated with decreased mortality among children with SCD.