Kohli-Kumar Mudra, Marandi Hossain, Keller Margaret A, Guertin Kathy, Hvizdala Eva
Pediatrics, University of South Florida, 17th Davis Boulevard 2nd floor, Tampa, FL 33606-3475, USA.
J Pediatr Hematol Oncol. 2002 Dec;24(9):777-8. doi: 10.1097/00043426-200212000-00021.
This report describes the sustained response of an Iranian girl with homozygous beta(0) thalassemia (IVS-II-1G-->A) to hydroxyurea (HU) and recombinant erythropoietin (rEPO). Since the start of this regimen 7 years ago, she has been transfusion-independent and her hemoglobin is maintained between 9.5-11.0 gm/dL. She is maintaining consistent growth around the 10th percentile for age and enjoys a good quality of life. She has not had any therapy-related adverse effects. This experience suggests that therapy with HU and rEPO may be useful long-term in some patients with beta thalassemia.
本报告描述了一名患有纯合子β(0)地中海贫血（IVS-II-1G→A）的伊朗女孩对羟基脲（HU）和重组促红细胞生成素（rEPO）的持续反应。自7年前开始这一治疗方案以来，她已不再依赖输血，血红蛋白维持在9.5 - 11.0克/分升之间。她的生长发育一直保持在同龄人第10百分位左右，生活质量良好。她没有出现任何与治疗相关的不良反应。这一经验表明，HU和rEPO联合治疗可能对一些地中海贫血患者具有长期疗效。
