Outcomes of Infant Medulloblastoma Treated With Standard-Dose Chemotherapy—A Single-Institution Experience From a Low-Middle-Income Country

IF 2.3 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer Pub Date : 2025-09-22 DOI:10.1002/pbc.32056

Gnanamani Senguttuvan, Leenu Lizbeth Joseph, Rikki Rorima John, Geeta Chacko, Ranjani Jayachandran, Krishna Prabhu, Ranjit K. Moorthy, Baylis Vivek Joseph, Rajesh Balakrishnan, Patricia Sebastin, Leni G. Mathew

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Abstract

Background

Infant medulloblastoma (iMBL) is predominantly desmoplastic nodular (DN) or medulloblastoma with extensive nodularity (MBEN) histology, sonic hedgehog (SHH) driven. Intensified chemotherapy (intraventricular chemotherapy or high-dose chemotherapy with autologous stem cell rescue) is recommended to compensate for the omission of radiation. This study aims to analyze the outcomes of iMBL treated without treatment intensification.

Methods

This retrospective study was performed on 28 patients with iMBL (children with medulloblastoma <3 years at diagnosis or 3–4 years with DN/MBEN histology) treated between January 2004 and April 2024 in a tertiary care institute in southern India.

Results

The median age at diagnosis and duration of symptoms were 24.5 months and 1 month, respectively. Vomiting (82%) and ataxia (71.4%) were the common presentations. Ventriculoperitoneal shunt or endoscopic third ventriculostomy was done in 57% of patients. Twenty-four patients (∼86%) underwent gross/near-total resection. The most common surgical complication was cerebellar mutism (n = 6, 21%). Half of the cohort had nonmetastatic disease. Sixteen patients had DN/MBEN histology. Only 68% (n = 19) opted to continue postoperative treatment. Chemotherapy protocols used were baby SFOP, HIT SKK, and modified Packer. One fourth (n = 7) received radiation (DN/MBEN = 3; other histology = 4) as a part of their initial treatment. There was no treatment-related mortality. The 5-year event-free survival (EFS) of patients who received postoperative chemotherapy ± RT was 70% (those with DN/MBEN were 82.5% and non-DN/MBEN were 35.7%). The 5-year EFS of patients with DN/MBEN histology who received only chemotherapy (n = 9) was 88.9%.

Conclusions

Managing medulloblastoma in infants is challenging in LMIC, as a significant proportion of families opt against postoperative treatment. Although traditionally considered high risk, good survival can be achieved with non-intensified therapy without an increase in treatment-related mortality, especially in patients with DN/MBEN histology.

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标准剂量化疗治疗婴儿髓母细胞瘤的结果-来自中低收入国家的单机构经验

背景：婴儿髓母细胞瘤（iMBL）主要是纤维增生性结节（DN）或广泛结节性髓母细胞瘤（MBEN）组织学，由音hedgehog基因（SHH）驱动。建议强化化疗（脑室化疗或自体干细胞拯救的大剂量化疗）来弥补放疗的遗漏。本研究旨在分析未经强化治疗的iMBL的治疗结果。方法：回顾性研究了28例iMBL（髓母细胞瘤患儿）。结果：诊断时的中位年龄为24.5个月，症状持续时间为1个月。呕吐（82%）和共济失调（71.4%）是常见的表现。57%的患者行脑室腹腔分流术或内镜下第三脑室造瘘术。24例患者（约86%）接受了大体/近全切除。最常见的手术并发症是小脑性缄默症（n = 6, 21%）。该队列中有一半患有非转移性疾病。16例患者有DN/MBEN组织学。只有68% （n = 19）的患者选择继续术后治疗。使用的化疗方案为婴儿SFOP、HIT SKK和改良Packer。四分之一（n = 7）接受放射治疗（DN/MBEN = 3；其他组织学= 4）作为初始治疗的一部分。没有与治疗相关的死亡率。术后化疗±放疗患者的5年无事件生存率（EFS）为70% （DN/MBEN患者为82.5%，非DN/MBEN患者为35.7%）。仅接受化疗的DN/MBEN组织学患者（n = 9）的5年EFS为88.9%。结论：在LMIC中，婴儿髓母细胞瘤的治疗是具有挑战性的，因为相当大比例的家庭选择不进行术后治疗。虽然传统上被认为是高风险，但通过非强化治疗可以获得良好的生存率，而不会增加与治疗相关的死亡率，特别是对于具有DN/MBEN组织学的患者。

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

Pediatric Blood & Cancer 医学-小儿科

CiteScore

4.90

自引率

9.40%

发文量

546

审稿时长

1.5 months

期刊介绍： Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.