The authors compared survival and multiple comorbidities in children diagnosed with craniopharyngioma who underwent gross-total resection (GTR) versus subtotal resection (STR) with radiation therapy (RT), either intensity-modulated radiation therapy (IMRT) or proton beam therapy (PBT). The authors hypothesized that there are differences between multimodal treatment methods with respect to morbidity and progression-free survival (PFS).

The medical records of children diagnosed with craniopharyngioma and treated surgically between February 1997 and December 2018 at Texas Children’s Hospital were reviewed. Surgical treatment was stratified as GTR or STR + RT. RT was further stratified as PBT or IMRT; PBT was stratified as STR + PBT versus cyst decompression (CD) + PBT. The authors used Kaplan-Meier analysis to compare PFS and overall survival, and chi-square analysis to compare rates for hypopituitarism, vision loss, and hypothalamic obesity (HyOb).

Sixty-three children were included in the analysis; 49% were female. The mean age was 8.16 years (95% CI 7.08–9.27). Twelve of 14 children in the IMRT cohort underwent CD. The 5-year PFS rates were as follows: 73% for GTR (n = 31), 54% for IMRT (n = 14), 100% for STR + PBT (n = 7), and 77% for CD + PBT (n = 11; p = 0.202). The overall survival rates were similar in all groups. Rates of hypopituitarism (96% GTR vs 75% IMRT vs 100% STR + PBT, 50% CD + PBT; p = 0.023) and diabetes insipidus (DI) (90% GTR vs 61% IMRT vs 85% STR + PBT, 20% CD + PBT; p = 0.004) were significantly higher in the GTR group. There was no significant difference in the HyOb or vision loss at the end of study follow-up among the different groups. Within the PBT group, 2 patients presented a progressive vasculopathy with subsequent strokes. One patient experienced a PBT-induced tumor.

GTR and CD + PBT presented similar rates of 5-year PFS. Hypopituitarism and DI rates were higher with GTR, but the rate of HyOb was similar among different treatment modalities. PBT may reduce the burden of hypopituitarism and DI, although radiation carries a risk of potential serious complications, including progressive vasculopathy and secondary malignancy. Further prospective study comparing neurocognitive outcomes is necessary.

作者比较了接受大体全切除 (GTR) 与次全切除 (STR) 和放射治疗 (RT)（调强放射治疗 (IMRT) 或质子束治疗 (PBT)）的颅咽管瘤儿童的生存率和多种合并症. 作者假设多模式治疗方法在发病率和无进展生存期 (PFS) 方面存在差异。

回顾了 1997 年 2 月至 2018 年 12 月在德克萨斯儿童医院诊断为颅咽管瘤并接受手术治疗的儿童的医疗记录。手术治疗分层为 GTR 或 STR + RT。RT 进一步分层为 PBT 或 IMRT；PBT 分层为 STR + PBT 与囊肿减压 (CD) + PBT。作者使用 Kaplan-Meier 分析来比较 PFS 和总生存率，并使用卡方分析来比较垂体功能减退、视力丧失和下丘脑肥胖 (HyOb) 的发生率。

63 名儿童被纳入分析；49% 是女性。平均年龄为 8.16 岁（95% CI 7.08-9.27）。IMRT 队列中的 14 名儿童中有 12 名接受了 CD。5 年 PFS 率如下：GTR 为 73%（n = 31），IMRT 为 54%（n = 14），STR + PBT 为 100%（n = 7），CD + PBT 为 77%（ n = 11；p = 0.202）。所有组的总生存率相似。垂体功能减退率（96% GTR vs 75% IMRT vs 100% STR + PBT，50% CD + PBT；p = 0.023）和尿崩症（DI）（90% GTR vs 61% IMRT vs 85% STR + PBT，20 % CD + PBT；p = 0.004）在 GTR 组中显着更高。在研究随访结束时，不同组之间的 HyOb 或视力丧失没有显着差异。在 PBT 组中，2 名患者出现进行性血管病变并随后发生中风。

GTR 和 CD + PBT 的 5 年 PFS 率相似。GTR 的垂体功能减退和 DI 发生率较高，但不同治疗方式的 HyOb 发生率相似。PBT 可以减轻垂体功能减退和 DI 的负担，尽管放疗有潜在严重并发症的风险，包括进行性血管病变和继发性恶性肿瘤。需要进一步比较神经认知结果的前瞻性研究。