Table of Contents
Patients, Families and Friends
Health Care Providers
|Health Care Providers - Care Paths And Protocols - Children & Adolescents|
Higher levels of fetal hemoglobin (Hb F) and lower leukocyte counts are thought to be beneficial in patients with sickle cell disease and can be achieved with daily oral administration of hydroxyurea (HU). A placebo-controlled, double-blind, prospective trial in severely affected adults with Hb SS showed that HU significantly reduced the incidence of vaso-occlusive pain, acute chest syndrome, and blood transfusions. A multi-center phase I/II trial in children >5 years-of-age showed safety and hematologic effects similar to those observed in adults. Clinical benefit in children with Hb SS has been suggested by a number of open-label trials. The drug is FDA-approved for selected adult patients, with the important caution that the drug is not curative and requires close hematologic monitoring for myelotoxicity and the strict use of contraception by both men and women who are sexually active. Use of HU in patients with Hb SC or Sb +- thalassemia is under investigation.
The clinical course of each patient with sickle cell disease should be regularly reviewed by a pediatric hematologist/sickle cell program and the possibility of hydroxyurea treatment and its pros and cons considered. Many patients with severe complications may also be candidates for either a program of chronic transfusions (p. 27) or, if an HLA-matched sibling is available, stem cell transplantation (p.29). HU is generally not considered appropriate for patients with stroke, and it is not useful in the treatment of acute sickle pain. No improvement is expected until the drug has been taken daily for 3-6 months. HU may alter the natural history of the sickle cell disease; for example splenomegaly or splenic sequestration may occur in relatively older patients. HU is a potentially toxic chemotherapeutic agent whose long-term toxicity (including concerns about carcinogenicity and teratogenicity) is unresolved. Thus the drug should be initiated and monitored only by hematologists with expertise in chemotherapy and sickle cell disease and after written documentation of patient education and consent.
Indications (Inclusion criteria)
Toxicity: Toxicity from hydroxyurea is generally defined as any of the following: