Chaturvedi Aprajita, Sadashiva Nishanth, Kalahasti Sathyarao, Konar Subhas, Krishna Uday, Ar Prabhuraj, Shukla Dhaval, Beniwal Manish, Pruthi Nupur, Arimappamagan Arivazhagan, Saini Jitender, Rao Shilpa, Santosh Vani
Department of Neurosurgery, National Institute of Mental Health and Neurosciences (NIMHANS), Bangalore, 560029, India.
Consultant Radiation Oncology, Apollo Proton Cancer Center, Chennai, India.
Childs Nerv Syst. 2024 Dec 27;41(1):71. doi: 10.1007/s00381-024-06730-z.
Diffuse intrinsic pontine glioma (DIPG) in children comprises 80% of brainstem gliomas. In 2021, 5th edition of WHO CNS tumor classification defined H3K27M altered diffuse midline gliomas (DMGs) which replaced this entity. Lesion location precludes resection and the only current option available is radiotherapy. Patient age, duration of symptoms, histone subtype mutation etc. may helpl in prognostication but the disease remains incurable with a median overall survival of 9-12 months.
This is a prospective observational study from a tertiary health care center in a low to middle-income country. We included patients with DIPG (radiological and/or histopathological H3K27M altered) from June 2018 to April 2023. Clinical, radiological, histology, and molecular features were reviewed and prognostic factors for 3 months, 6 months, and overall survival was analysed for all patients.
We included 92 pediatric patients. The median age of our study population was 8.5 years. Median LPS was 80. Cranial nerve palsy was the most common presenting complaint. Hydrocephalus requiring CSF diversion was present in 38 patients (41.3%). Lesion biopsy was performed in 36 patients (39.1%) and exophytic component decompression was done in 11 patients (11.9%). Seven patients were lost to follow-up. Adjuvant therapy was received by 51 patients (51/85, 60%). Radiotherapy was the only significant prognostic indicator of 3 months, 6 months, and overall survival (HR: 0.39). The presence of necrosis on histopathology was also an indicator of poor prognosis (HR: 2.38). There were 7 long-term survivors in our study but we did not find any significant survival prognostic indicator amongst this group.
Conventional adjuvant therapy has not proven of much benefit. With the advancement in molecular understanding of the entity, there is an upsurge in the development of targeted therapy but with no promising results so far. In this study, we have attempted to explore the prognostic factors and unique challenges we face in a resource-limited setting against this disease.
儿童弥漫性脑桥内在型胶质瘤（DIPG）占脑干胶质瘤的80%。2021年，世界卫生组织中枢神经系统肿瘤分类第5版定义了H3K27M改变的弥漫性中线胶质瘤（DMG），取代了这一实体。病变位置妨碍手术切除，目前唯一可用的选择是放疗。患者年龄、症状持续时间、组蛋白亚型突变等可能有助于预后评估，但该疾病仍然无法治愈，中位总生存期为9至12个月。
这是一项来自低收入至中等收入国家三级医疗保健中心的前瞻性观察性研究。我们纳入了2018年6月至2023年4月期间患有DIPG（放射学和/或组织病理学H3K27M改变）的患者。回顾了所有患者的临床、放射学、组织学和分子特征，并分析了3个月、6个月和总生存期的预后因素。
我们纳入了92名儿科患者。研究人群的中位年龄为8.5岁。中位淋巴细胞百分比为80。颅神经麻痹是最常见的主诉。38名患者（41.3%）存在需要脑脊液分流的脑积水。36名患者（39.1%）进行了病变活检，11名患者（11.9%）进行了外生性成分减压。7名患者失访。51名患者（51/85，60%）接受了辅助治疗。放疗是3个月、6个月和总生存期的唯一显著预后指标（风险比：0.39）。组织病理学上出现坏死也是预后不良的指标（风险比：2.38）。我们的研究中有7名长期存活者，但在该组中未发现任何显著的生存预后指标。
传统辅助治疗尚未证明有多大益处。随着对该实体分子理解的进展，靶向治疗的开发激增，但迄今为止没有取得有希望的结果。在本研究中，我们试图探索在资源有限的情况下针对这种疾病所面临的预后因素和独特挑战。
