Risk Stratification-Based Therapy for Children with E-beta Thalassemia: A 20-Year Follow-Up Study.

IF 1.7 4区医学 Q2 PEDIATRICS

Indian pediatrics Pub Date : 2025-07-01 Epub Date: 2025-05-26 DOI:10.1007/s13312-025-00089-3

Kavitha Ganesan, Suresh Duraisamy, Anupama Nair, Vijayshree Muthukumar, Anurag Nalla Reddy, Minakshi Balwani, Nithya Seshadri, Ramya Uppuluri, Revathi Raj

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引用次数: 0

Abstract

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.

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儿童e - β地中海贫血的风险分层治疗：一项20年随访研究

目的：介绍基于风险分层的e - β地中海贫血儿童随访。方法：采用Mahidol评分法对2002年8月至2022年8月间的e - β型地中海贫血患儿进行轻度、中度和重度分类。中度组患儿的结果：在104例患儿（中位发病年龄3.5岁）中，30例（29%）、46例（44%）和28例（27%）在初次就诊时被分类为轻度、中度和重度疾病。根据nRBC计数将中度组27例和19例患儿重新划分为轻度至中度和中度至重度。56名、19名和29名儿童分别接受了HU、HU输血和每月输血并螯合。在中位8年的随访中，67/93（72%）被分类为“轻度至中度”的儿童在HU上保持良好，26/93（28%）被分类为“中度至重度”的儿童仍然定期输血。结论：利用nrbc和Mahidol评分可以作为个体化治疗的有效工具。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Indian pediatrics 医学-小儿科

CiteScore

3.30

自引率

8.70%

发文量

344

审稿时长

3-8 weeks

期刊介绍： The general objective of Indian Pediatrics is "To promote the science and practice of Pediatrics." An important guiding principle has been the simultaneous need to inform, educate and entertain the target audience. The specific key objectives are: -To publish original, relevant, well researched peer reviewed articles on issues related to child health. -To provide continuing education to support informed clinical decisions and research. -To foster responsible and balanced debate on controversial issues that affect child health, including non-clinical areas such as medical education, ethics, law, environment and economics. -To achieve the highest level of ethical medical journalism and to produce a publication that is timely, credible and enjoyable to read.