基于风险分层的儿童β地中海贫血治疗：一项20年随访研究
Risk Stratification-Based Therapy for Children with E-beta Thalassemia: A 20-Year Follow-Up Study.
作者信息
Ganesan Kavitha, Duraisamy Suresh, Nair Anupama, Muthukumar Vijayshree, Reddy Anurag Nalla, Balwani Minakshi, Seshadri Nithya, Uppuluri Ramya, Raj Revathi
机构信息
Department of Pediatric Hematology, Oncology, Blood, and Marrow Transplantation, Apollo Cancer Hospitals, 320, Padma Complex, Anna Salai, Teynampet, Chennai, Tamil Nadu, 600035, India.
出版信息
Indian Pediatr. 2025 May 26. doi: 10.1007/s13312-025-00089-3.
OBJECTIVE
To present risk stratification-based follow-up of children with E-beta thalassemia.
METHODS
Children with E-beta thalassemia between August 2002 and August 2022 were classified as mild, moderate, or severe disease using Mahidol scoring. Children in the moderate group with < 20 or ≥ 20 nucleated red blood cells (nRBCs) per 100 white blood cells were clubbed with mild or severe phenotype, respectively. Children with mild/moderate severity received hydroxyurea (HU) and those with severe disease received regular transfusions.
RESULTS
Out of 104 children (median age of onset 3.5 years), 30 (29%), 46 (44%), and 28 (27%) were categorized as mild, moderate, and severe disease, at initial presentation. Based on nRBC count, 27 and 19 children in moderate category were recategorized as mild to moderate and moderate to severe. Fifty-six, 19, and 29 children received HU, transfusions with HU, and monthly transfusion and chelation, respectively. At a median follow-up of 8 years, 67/93 (72%) children classified as "mild to moderate" remained well on HU, and 26/93 (28%) children classified as "moderate to severe" remained on regular transfusions.
CONCLUSION
Using nRBCs in addition to Mahidol scoring can serve as a useful tool to individualize therapy.
目的
介绍基于风险分层的E-β地中海贫血患儿随访情况。
方法
采用玛希隆评分法将2002年8月至2022年8月期间的E-β地中海贫血患儿分为轻度、中度或重度疾病。中度组中每100个白细胞中核红细胞（nRBC）<20或≥20的患儿分别归为轻度或重度表型。轻度/中度严重程度的患儿接受羟基脲（HU）治疗，重度疾病患儿接受定期输血。
结果
在104名患儿（发病中位年龄3.5岁）中，初诊时30名（29%）、46名（44%）和28名（27%）被分类为轻度、中度和重度疾病。根据nRBC计数，中度组中的27名和19名患儿分别重新分类为轻度至中度和中度至重度。56名、19名和29名患儿分别接受了HU、HU联合输血以及每月输血和螯合治疗。在中位随访8年时，分类为“轻度至中度”的93名患儿中有67名（72%）接受HU治疗情况良好，分类为“中度至重度”的93名患儿中有26名（28%）仍在接受定期输血。
结论
除玛希隆评分外，使用nRBC可作为个体化治疗的有用工具。
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