Long-term hydroxyurea therapy for infants with sickle cell anemia

The HUSOFT extension study

Jane S. Hankins, Russell E. Ware, Zora R. Rogers, Lynn W. Wynn, Peter A. Lane, J. Paul Scott, Winfred C. Wang

Research output: Contribution to journalArticle

184 Citations (Scopus)

Abstract

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/β0-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.

Original languageEnglish (US)
Pages (from-to)2269-2275
Number of pages7
JournalBlood
Volume106
Issue number7
DOIs
StatePublished - Oct 2005

Fingerprint

Hydroxyurea
Sickle Cell Anemia
Acute Chest Syndrome
Toxicity
Fetal Hemoglobin
Therapeutics
Spleen
Erythrocyte Indices
Thalassemia
Reticulocytes
Growth
Platelets
Hemoglobins
Leukocytes
Blood
Blood Platelets
Cells
Safety
Imaging techniques

ASJC Scopus subject areas

  • Hematology

Cite this

Hankins, J. S., Ware, R. E., Rogers, Z. R., Wynn, L. W., Lane, P. A., Scott, J. P., & Wang, W. C. (2005). Long-term hydroxyurea therapy for infants with sickle cell anemia: The HUSOFT extension study. Blood, 106(7), 2269-2275. https://doi.org/10.1182/blood-2004-12-4973

Long-term hydroxyurea therapy for infants with sickle cell anemia : The HUSOFT extension study. / Hankins, Jane S.; Ware, Russell E.; Rogers, Zora R.; Wynn, Lynn W.; Lane, Peter A.; Scott, J. Paul; Wang, Winfred C.

In: Blood, Vol. 106, No. 7, 10.2005, p. 2269-2275.

Research output: Contribution to journalArticle

Hankins, JS, Ware, RE, Rogers, ZR, Wynn, LW, Lane, PA, Scott, JP & Wang, WC 2005, 'Long-term hydroxyurea therapy for infants with sickle cell anemia: The HUSOFT extension study', Blood, vol. 106, no. 7, pp. 2269-2275. https://doi.org/10.1182/blood-2004-12-4973
Hankins, Jane S. ; Ware, Russell E. ; Rogers, Zora R. ; Wynn, Lynn W. ; Lane, Peter A. ; Scott, J. Paul ; Wang, Winfred C. / Long-term hydroxyurea therapy for infants with sickle cell anemia : The HUSOFT extension study. In: Blood. 2005 ; Vol. 106, No. 7. pp. 2269-2275.
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