Tags

Type your tag names separated by a space and hit enter

Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease.
Blood 2004; 103(6):2039-45Blood

Abstract

Hydroxyurea improves hematologic parameters for children with sickle cell disease (SCD), but its long-term efficacy at maximum tolerated dose (MTD) has not been determined. Between 1995 and 2002, hydroxyurea therapy was initiated for 122 pediatric patients with SCD including 106 with homozygous sickle cell anemia (HbSS), 7 with sickle hemoglobin C (HbSC), 7 with sickle/beta-thalassemia (HbS/ beta-thalassemia [6 HbS/beta0, 1 HbS/beta+]), and 2 with sickle hemoglobin OArab (HbS/OArab). Median age at initiation of therapy was 11.1 years. Hydroxyurea was escalated to MTD, with an average dose of 25.4 +/- 5.4 mg/kg per day; the average duration of hydroxyurea therapy has been 45 +/- 24 months (range, 6-101 months). Hydroxyurea was discontinued for 15 (12%) children with poor compliance. Mild transient neutropenia occurred, but no hepatic or renal toxicity was noted. Hydroxyurea therapy led to significant increases in hemoglobin level, mean corpuscular volume, and fetal hemoglobin (HbF) level, whereas significant decreases occurred in reticulocyte, white blood cell, and platelet counts and serum bilirubin levels. Children with variant SCD genotypes also had hematologic responses to hydroxyurea. HbF induction has been sustained for up to 8 years without adverse effects on growth or increased numbers of acquired DNA mutations. Long-term hydroxyurea therapy at MTD is well tolerated by pediatric patients with SCD and has sustained hematologic efficacy with apparent long-term safety.

Authors+Show Affiliations

Duke Pediatric Sickle Cell Program and Division of Pediatric Hematology/Oncology, Duke University Medical Center, PO Box 2916, Durham, NC 27710, USA. zimme008@mc.duke.eduNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article

Language

eng

PubMed ID

14630791

Citation

Zimmerman, Sherri A., et al. "Sustained Long-term Hematologic Efficacy of Hydroxyurea at Maximum Tolerated Dose in Children With Sickle Cell Disease." Blood, vol. 103, no. 6, 2004, pp. 2039-45.
Zimmerman SA, Schultz WH, Davis JS, et al. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood. 2004;103(6):2039-45.
Zimmerman, S. A., Schultz, W. H., Davis, J. S., Pickens, C. V., Mortier, N. A., Howard, T. A., & Ware, R. E. (2004). Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood, 103(6), pp. 2039-45.
Zimmerman SA, et al. Sustained Long-term Hematologic Efficacy of Hydroxyurea at Maximum Tolerated Dose in Children With Sickle Cell Disease. Blood. 2004 Mar 15;103(6):2039-45. PubMed PMID: 14630791.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. AU - Zimmerman,Sherri A, AU - Schultz,William H, AU - Davis,Jacqueline S, AU - Pickens,Chrisley V, AU - Mortier,Nicole A, AU - Howard,Thad A, AU - Ware,Russell E, Y1 - 2003/11/20/ PY - 2003/11/25/pubmed PY - 2004/4/16/medline PY - 2003/11/25/entrez SP - 2039 EP - 45 JF - Blood JO - Blood VL - 103 IS - 6 N2 - Hydroxyurea improves hematologic parameters for children with sickle cell disease (SCD), but its long-term efficacy at maximum tolerated dose (MTD) has not been determined. Between 1995 and 2002, hydroxyurea therapy was initiated for 122 pediatric patients with SCD including 106 with homozygous sickle cell anemia (HbSS), 7 with sickle hemoglobin C (HbSC), 7 with sickle/beta-thalassemia (HbS/ beta-thalassemia [6 HbS/beta0, 1 HbS/beta+]), and 2 with sickle hemoglobin OArab (HbS/OArab). Median age at initiation of therapy was 11.1 years. Hydroxyurea was escalated to MTD, with an average dose of 25.4 +/- 5.4 mg/kg per day; the average duration of hydroxyurea therapy has been 45 +/- 24 months (range, 6-101 months). Hydroxyurea was discontinued for 15 (12%) children with poor compliance. Mild transient neutropenia occurred, but no hepatic or renal toxicity was noted. Hydroxyurea therapy led to significant increases in hemoglobin level, mean corpuscular volume, and fetal hemoglobin (HbF) level, whereas significant decreases occurred in reticulocyte, white blood cell, and platelet counts and serum bilirubin levels. Children with variant SCD genotypes also had hematologic responses to hydroxyurea. HbF induction has been sustained for up to 8 years without adverse effects on growth or increased numbers of acquired DNA mutations. Long-term hydroxyurea therapy at MTD is well tolerated by pediatric patients with SCD and has sustained hematologic efficacy with apparent long-term safety. SN - 0006-4971 UR - https://www.unboundmedicine.com/medline/citation/14630791/Sustained_long_term_hematologic_efficacy_of_hydroxyurea_at_maximum_tolerated_dose_in_children_with_sickle_cell_disease_ L2 - http://www.bloodjournal.org/cgi/pmidlookup?view=long&pmid=14630791 DB - PRIME DP - Unbound Medicine ER -