The Journal of pediatrics
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The Journal of pediatrics · Dec 2001
Clinical TrialA two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia.
Hydroxyurea improves hematologic values and decreases vaso-occlusive complications in adults and children with sickle cell anemia (SCA), but has not been tested in infants before the onset of chronic organ dysfunction. We conducted a collaborative pilot trial of hydroxyurea in infants with SCA to assess its (1) feasibility of administration, (2) toxicity, (3) hematologic effects, and (4) effect on spleen function. ⋯ Hydroxyurea therapy for infants with SCA is feasible and well tolerated, has hematologic efficacy, and may delay functional asplenia. The potential for hydroxyurea to preserve organ function in SCA should be further evaluated.
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The Journal of pediatrics · Dec 2001
Beneficial effects of intensive therapy of diabetes during adolescence: outcomes after the conclusion of the Diabetes Control and Complications Trial (DCCT).
The Diabetes Control and Complications Trial (DCCT) demonstrated that intensive therapy of type 1 diabetes mellitus reduces the risk of development and progression of microvascular complications. The Epidemiology of Diabetes Interventions and Complications (EDIC) study assessed whether these benefits persisted after the end of DCCT. Results for the adolescent DCCT cohort are reported here. ⋯ These findings provide further support for the DCCT recommendation that most adolescents with type 1 diabetes receive intensive therapy aimed at achieving glycemic control as close to normal as possible to reduce the risk of microvascular complications.
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The Journal of pediatrics · Dec 2001
Randomized Controlled Trial Clinical TrialImpact of chronic transfusion on incidence of pain and acute chest syndrome during the Stroke Prevention Trial (STOP) in sickle-cell anemia.
The Stroke Prevention Trial (STOP) demonstrated that chronic transfusion is highly effective in reducing the risk of stroke in children with sickle-cell disease and an abnormal transcranial Doppler ultrasonography examination result. Our objective was to determine whether chronic transfusion therapy reduces the incidence of pain and acute chest syndrome. ⋯ Compliance with aggressive chronic transfusion reduces the frequency of acute chest syndrome and pain episodes.
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The Journal of pediatrics · Dec 2001
Randomized Controlled Trial Multicenter Study Clinical TrialA two-year randomized, placebo-controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities.
Our objective was to determine whether long-term treatment of young patients with cystic fibrosis (CF) with dornase alfa maintains lung function and reduces respiratory tract exacerbations. ⋯ Treatment of young patients with CF with dornase alfa maintains lung function and reduces the risk of exacerbations over a 96-week period.