OBJECTIVE: Hydroxyurea improves hematologic values and decreases vaso-occlusive complications in adults and children with sickle cell anemia (SCA), but has not been tested in infants before the onset of chronic organ dysfunction. We conducted a collaborative pilot trial of hydroxyurea in infants with SCA to assess its (1) feasibility of administration, (2) toxicity, (3) hematologic effects, and (4) effect on spleen function. STUDY DESIGN: Patients with hemoglobin (Hb) SS or Sbeta(0) thalassemia (n = 28, median age 15 months) received hydroxyurea for 2 years at 20 mg/kg/day. Hydroxyurea was temporarily discontinued for predefined toxicity. RESULTS: Seven patients exited the study early: five for noncompliance or refusal to continue, one for mild stroke, and one for fatal splenic sequestration. The predominant toxicity was transient neutropenia, which was usually associated with a viral-like illness. After 2 years of treatment, mean Hb level = 8.8 g/dL and Hb F = 20.3%, both higher than predicted age-specific levels. Radionuclide splenic uptake was absent in 47% of patients at study completion, compared with predicted functional asplenia in 80% of the patients. CONCLUSIONS: Hydroxyurea therapy for infants with SCA is feasible and well tolerated, has hematologic efficacy, and may delay functional asplenia. The potential for hydroxyurea to preserve organ function in SCA should be further evaluated.
OBJECTIVE:Hydroxyurea improves hematologic values and decreases vaso-occlusive complications in adults and children with sickle cell anemia (SCA), but has not been tested in infants before the onset of chronic organ dysfunction. We conducted a collaborative pilot trial of hydroxyurea in infants with SCA to assess its (1) feasibility of administration, (2) toxicity, (3) hematologic effects, and (4) effect on spleen function. STUDY DESIGN:Patients with hemoglobin (Hb) SS or Sbeta(0) thalassemia (n = 28, median age 15 months) received hydroxyurea for 2 years at 20 mg/kg/day. Hydroxyurea was temporarily discontinued for predefined toxicity. RESULTS: Seven patients exited the study early: five for noncompliance or refusal to continue, one for mild stroke, and one for fatal splenic sequestration. The predominant toxicity was transient neutropenia, which was usually associated with a viral-like illness. After 2 years of treatment, mean Hb level = 8.8 g/dL and Hb F = 20.3%, both higher than predicted age-specific levels. Radionuclide splenic uptake was absent in 47% of patients at study completion, compared with predicted functional asplenia in 80% of the patients. CONCLUSIONS:Hydroxyurea therapy for infants with SCA is feasible and well tolerated, has hematologic efficacy, and may delay functional asplenia. The potential for hydroxyurea to preserve organ function in SCA should be further evaluated.
Authors: Sohail Rana; Patricia E Houston; Winfred C Wang; Rathi V Iyer; Jonathan Goldsmith; James F Casella; Caroline K Reed; Zora R Rogers; Myron A Waclawiw; Bruce Thompson Journal: Pediatrics Date: 2014-09 Impact factor: 7.124
Authors: Winfred C Wang; Russell E Ware; Scott T Miller; Rathi V Iyer; James F Casella; Caterina P Minniti; Sohail Rana; Courtney D Thornburg; Zora R Rogers; Ram V Kalpatthi; Julio C Barredo; R Clark Brown; Sharada A Sarnaik; Thomas H Howard; Lynn W Wynn; Abdullah Kutlar; F Daniel Armstrong; Beatrice A Files; Jonathan C Goldsmith; Myron A Waclawiw; Xiangke Huang; Bruce W Thompson Journal: Lancet Date: 2011-05-14 Impact factor: 79.321
Authors: Banu Aygun; Nicole A Mortier; Matthew P Smeltzer; Barry L Shulkin; Jane S Hankins; Russell E Ware Journal: Am J Hematol Date: 2012-12-17 Impact factor: 10.047
Authors: Sophie Lanzkron; John J Strouse; Renee Wilson; Mary Catherine Beach; Carlton Haywood; HaeSong Park; Catherine Witkop; Eric B Bass; Jodi B Segal Journal: Ann Intern Med Date: 2008-05-05 Impact factor: 25.391