Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study. Blood 2005 Oct 01;106(7):2269-75
Date
09/21/2005Pubmed ID
16172253Pubmed Central ID
PMC1895275DOI
10.1182/Blood-2004-12-4973Scopus ID
2-s2.0-27144448031 (requires institutional sign-in at Scopus site) 228 CitationsAbstract
The long-term efficacy and toxicity of hydroxyurea for infants are undefined, and its role in preventing organ dysfunction is unknown. Short-term feasibility of hydroxyurea administration, toxicities, hematologic effects, and effect on spleen function in infants with sickle cell anemia (SCA) were reported (Hydroxyurea Safety and Organ Toxicity [HUSOFT] trial). These infants completing 2 years of hydroxyurea therapy (20 mg/kg/d) were offered study extension with dose escalation to 30 mg/kg/d. Patients were monitored with laboratory tests and biannual imaging studies. Hematologic indices were compared with predicted age-specific values and event rates compared with historic rates. All 21 subjects completing the original trial enrolled in the extension study: median age, 3.4 years old (range, 2.6 to 4.4 years); 12 females; 20 with Hb SS, 1 with Hb S/beta0-thalassemia. Seventeen patients completed 4 years of hydroxyurea, and 11 completed 6 years. After 4 years, hydroxyurea was associated with increased hemoglobin concentration, percentage of fetal hemoglobin (Hb F), and mean corpuscular volume (MCV) and decreased reticulocytes, white blood cells (WBCs), and platelets (P < .01). Patients experienced 7.5 acute chest syndrome (ACS) events per 100 person-years, compared with 24.5 events per 100 person-years among historic controls (P = .001). Treated patients had better spleen function than expected and improved growth rates. Infants with SCA tolerate prolonged hydroxyurea therapy with sustained hematologic benefits, fewer ACS events, improved growth, and possibly preserved organ function.
Author List
Hankins JS, Ware RE, Rogers ZR, Wynn LW, Lane PA, Scott JP, Wang WCMESH terms used to index this publication - Major topics in bold
Anemia, Sickle CellBlood Platelets
Child
Child, Preschool
Clinical Trials as Topic
Dose-Response Relationship, Drug
Erythrocyte Indices
Female
Hematologic Tests
Hemoglobins
Homozygote
Humans
Hydroxyurea
Infant
Leukocytes
Liver
Male
Reticulocytes
Spleen
Time Factors
