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Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group.
Blood 1999; 94(5):1550-4Blood

Abstract

Previous studies have determined the short-term toxicity profile, laboratory changes, and clinical efficacy associated with hydroxyurea (HU) therapy in adults with sickle cell anemia. The safety and efficacy of this agent in pediatric patients with sickle cell anemia has not been determined. Children with sickle cell anemia, age 5 to 15 years, were eligible for this multicenter Phase I/II trial. HU was started at 15 mg/kg/d and escalated to 30 mg/kg/d unless the patient experienced laboratory toxicity. Patients were monitored by 2-week visits to assess compliance, toxicity, clinical adverse events, growth parameters, and laboratory efficacy associated with HU treatment. Eighty-four children were enrolled between December 1994 and March 1996. Sixty-eight children reached maximum tolerated dose (MTD) and 52 were treated at MTD for 1 year. Significant hematologic changes included increases in hemoglobin concentration, mean corpuscular volume, mean corpuscular hemoglobin, and fetal hemoglobin parameters, and decreases in white blood cell, neutrophil, platelet, and reticulocyte counts. Laboratory toxicities typically were mild, transient, and were reversible upon temporary discontinuation of HU. No life-threatening clinical adverse events occurred and no child experienced growth failure. This Phase I/II trial shows that HU therapy is safe for children with sickle cell anemia when treatment was directed by a pediatric hematologist. HU in children induces similar laboratory changes as in adults. Phase III trials to determine if HU can prevent chronic organ damage in children with sickle cell anemia are warranted.

Authors+Show Affiliations

Duke Pediatric Sickle Cell Program, Duke Children's Hospital, Durham, NC 27710, USA. kinne001@mc.duke.eduNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Clinical Trial, Phase I
Clinical Trial, Phase II
Journal Article
Multicenter Study
Research Support, U.S. Gov't, P.H.S.

Language

eng

PubMed ID

10477679

Citation

Kinney, T R., et al. "Safety of Hydroxyurea in Children With Sickle Cell Anemia: Results of the HUG-KIDS Study, a Phase I/II Trial. Pediatric Hydroxyurea Group." Blood, vol. 94, no. 5, 1999, pp. 1550-4.
Kinney TR, Helms RW, O'Branski EE, et al. Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Blood. 1999;94(5):1550-4.
Kinney, T. R., Helms, R. W., O'Branski, E. E., Ohene-Frempong, K., Wang, W., Daeschner, C., ... Ware, R. E. (1999). Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Blood, 94(5), pp. 1550-4.
Kinney TR, et al. Safety of Hydroxyurea in Children With Sickle Cell Anemia: Results of the HUG-KIDS Study, a Phase I/II Trial. Pediatric Hydroxyurea Group. Blood. 1999 Sep 1;94(5):1550-4. PubMed PMID: 10477679.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. AU - Kinney,T R, AU - Helms,R W, AU - O'Branski,E E, AU - Ohene-Frempong,K, AU - Wang,W, AU - Daeschner,C, AU - Vichinsky,E, AU - Redding-Lallinger,R, AU - Gee,B, AU - Platt,O S, AU - Ware,R E, PY - 1999/9/9/pubmed PY - 1999/9/9/medline PY - 1999/9/9/entrez SP - 1550 EP - 4 JF - Blood JO - Blood VL - 94 IS - 5 N2 - Previous studies have determined the short-term toxicity profile, laboratory changes, and clinical efficacy associated with hydroxyurea (HU) therapy in adults with sickle cell anemia. The safety and efficacy of this agent in pediatric patients with sickle cell anemia has not been determined. Children with sickle cell anemia, age 5 to 15 years, were eligible for this multicenter Phase I/II trial. HU was started at 15 mg/kg/d and escalated to 30 mg/kg/d unless the patient experienced laboratory toxicity. Patients were monitored by 2-week visits to assess compliance, toxicity, clinical adverse events, growth parameters, and laboratory efficacy associated with HU treatment. Eighty-four children were enrolled between December 1994 and March 1996. Sixty-eight children reached maximum tolerated dose (MTD) and 52 were treated at MTD for 1 year. Significant hematologic changes included increases in hemoglobin concentration, mean corpuscular volume, mean corpuscular hemoglobin, and fetal hemoglobin parameters, and decreases in white blood cell, neutrophil, platelet, and reticulocyte counts. Laboratory toxicities typically were mild, transient, and were reversible upon temporary discontinuation of HU. No life-threatening clinical adverse events occurred and no child experienced growth failure. This Phase I/II trial shows that HU therapy is safe for children with sickle cell anemia when treatment was directed by a pediatric hematologist. HU in children induces similar laboratory changes as in adults. Phase III trials to determine if HU can prevent chronic organ damage in children with sickle cell anemia are warranted. SN - 0006-4971 UR - https://www.unboundmedicine.com/medline/citation/10477679/Safety_of_hydroxyurea_in_children_with_sickle_cell_anemia:_results_of_the_HUG_KIDS_study_a_phase_I/II_trial__Pediatric_Hydroxyurea_Group_ L2 - http://www.bloodjournal.org/cgi/pmidlookup?view=long&pmid=10477679 DB - PRIME DP - Unbound Medicine ER -