|
|
Blood, 1 October 2005, Vol. 106, No. 7, pp. 2269-2275.
Prepublished online as a Blood First Edition Paper on June 14, 2005; DOI 10.1182/blood-2004-12-4973.
 Previous Article | Table of Contents | Next Article 
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
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, and
Winfred C. Wang
From St Jude Children's Research Hospital, Memphis, TN; University of Texas Southwestern Medical Center, Dallas, TX; Emory University School of Medicine, Atlanta, GA; and Medical College of Wisconsin, Milwaukee, WI.
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/ 0-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.
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
O. S. Platt
Hydroxyurea for the Treatment of Sickle Cell Anemia
N. Engl. J. Med.,
March 27, 2008;
358(13):
1362 - 1369.
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. M. Higgins, D. T. Eddington, S. N. Bhatia, and L. Mahadevan
Sickle cell vasoocclusion and rescue in a microfluidic device
PNAS,
December 18, 2007;
104(51):
20496 - 20500.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. G. Memtsoudis, R. W. Lekowski JR, P. Rosenberger, Z. Khalpey, D. J. FitzGerald, V. Claypoole, K. D. Courtney, M. J. Landzberg, H. F. Bunn, and P. S. Shekar
Pulmonary thrombectomy in a patient with hemoglobin Nottingham
Perfusion,
July 1, 2007;
22(4):
299 - 301.
[Abstract]
[PDF]
|
|
|
|
|
|
|
E. Coleman and B. Inusa
Sickle Cell Anemia: Targeting the Role of Fetal Hemoglobin in Therapy
Clinical Pediatrics,
June 1, 2007;
46(5):
386 - 391.
[Abstract]
[PDF]
|
|
|
|
|
|
|
M. R. DeBaun and J. J. Field
Limitations of Clinical Trials in Sickle Cell Disease: A Case Study of the Multi-center Study of Hydroxyurea (MSH) Trial and the Stroke Prevention (STOP) Trial
Hematology,
January 1, 2007;
2007(1):
482 - 488.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
P. A. Oneal, N. M. Gantt, J. D. Schwartz, N. V. Bhanu, Y. T. Lee, J. W. Moroney, C. H. Reed, A. N. Schechter, N. L. C. Luban, and J. L. Miller
Fetal hemoglobin silencing in humans
Blood,
September 15, 2006;
108(6):
2081 - 2086.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. D. Villella
Hydroxyurea Therapy for Infants with Sickle Cell Anemia
AAP Grand Rounds,
April 1, 2006;
15(4):
39 - 40.
[Full Text]
[PDF]
|
|
|
| |
