Del Baldo Giada, Vennarini Sabina, Cacchione Antonella, Amelio Dante, De Ioris Maria Antonietta, Fabozzi Francesco, Colafati Giovanna Stefania, Mastronuzzi Angela, Carai Andrea
Department of Onco-Hematology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, Scientific Institute for Reasearch, Hospitalization and Healthcare (IRCCS), 00165 Rome, Italy.
Pediatric Radiotherapy Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, 20133 Milan, Italy.
Diagnostics (Basel). 2022 Nov 9;12(11):2745. doi: 10.3390/diagnostics12112745.
Craniopharyngioma (CP) is a rare brain tumor involving the sellar region. The best management is still debated. Gross total resection (GTR) is considered the best option to improve recurrence-free survival, but considerable long-term sequelae with a significant impact on quality of life have been reported. Subtotal resection followed by radiotherapy achieves similar disease control compared to GTR with less complications.
We retrospectively reviewed 10 pediatric patients affected by CP treated with partial resection and subsequent proton therapy (PBT). We reviewed visual, endocrinological, and neuropsychological data at baseline, after surgery, and after radiation for all patients.
At the time of diagnosis, visual impairment was detected in 70% of patients and endocrinological abnormalities in 50%. All patients were subject to one or more surgical procedures. Surgery had no impact on visual status; however, it caused a worsening of endocrine function in half of patients. After surgery, all patients underwent PBT, achieving a partial response in 7 out of 10 patients (70%), while stable disease was observed in the other three patients (30%) at a median follow-up of 78 months from the end of PBT. Both visual and endocrine deficits were stable after PBT, with neurocognitive performance scores unchanged from baseline.
A conservative surgical approach followed by PBT represents a safe and effective strategy to manage CP and limit long-term sequelae.
颅咽管瘤（CP）是一种累及鞍区的罕见脑肿瘤。其最佳治疗方案仍存在争议。根治性全切除（GTR）被认为是提高无复发生存率的最佳选择，但据报道会出现相当多的长期后遗症，对生活质量有重大影响。与GTR相比，次全切除后放疗能达到相似的疾病控制效果，且并发症更少。
我们回顾性分析了10例接受部分切除及后续质子治疗（PBT）的小儿颅咽管瘤患者。我们回顾了所有患者在基线、术后及放疗后的视力、内分泌和神经心理学数据。
诊断时，70%的患者存在视力障碍，50%存在内分泌异常。所有患者均接受了一次或多次手术。手术对视力状况无影响；然而，有一半患者的内分泌功能恶化。术后，所有患者均接受了PBT，10例患者中有7例（70%）获得部分缓解，另外3例患者（30%）病情稳定，自PBT结束后的中位随访时间为78个月。PBT后视力和内分泌缺陷均稳定，神经认知表现评分与基线无变化。
采用保守手术方法并结合PBT是治疗颅咽管瘤和限制长期后遗症的一种安全有效的策略。
