Abstract

Background

Craniopharyngiomas account for approximately 1.2–4% of all CNS tumors. They are typically treated with a combination of surgical resection and focal radiotherapy. Unfortunately, treatment can lead to permanent deleterious effects on behavior, learning, and endocrine function.

Methods

The Pediatric Brain Tumor Consortium performed a multicenter phase 2 study in children and young adults with unresectable or recurrent craniopharyngioma (PBTC-039). Between December 2013 and November 2017, nineteen patients (median age at enrollment, 13.1 y; range, 2–25 y) were enrolled in one of 2 strata: patients previously treated with surgery alone (stratum 1) or who received radiation (stratum 2).

Results

Eighteen eligible patients (8 male, 10 female) were treated with weekly subcutaneous pegylated interferon alpha-2b for up to 18 courses (108 wk). Therapy was well tolerated with no grade 4 or 5 toxicities. 2 of the 7 eligible patients (28.6%) in stratum 1 had a partial response, but only one response was sustained for more than 3 months. None of the 11 stratum 2 patients had an objective radiographic response, although median progression-free survival was 19.5 months.

Conclusions

Pegylated interferon alpha-2b treatment, in lieu of or following radiotherapy, was well tolerated in children and young adults with recurrent craniopharyngiomas. Although objective responses were limited, progression-free survival results are encouraging, warranting further studies.

中文翻译：

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聚乙二醇干扰素α-2b用于不可切除或复发性颅咽管瘤患者的II期研究：小儿脑肿瘤联合会的报告。

摘要

背景

颅咽管瘤约占所有CNS肿瘤的1.2–4％。通常将其与手术切除和局部放疗相结合进行治疗。不幸的是，治疗可能对行为，学习和内分泌功能造成永久性有害影响。

方法

小儿脑肿瘤联合会在患有不可切除或复发性颅咽管瘤的儿童和年轻人中进行了一项多中心2期研究（PBTC-039）。在2013年12月至2017年11月之间，共有19例患者（入组中位年龄13.1岁；范围2-25岁）纳入以下两个层级之一：先前接受手术治疗的患者（第1层）或接受放射治疗的患者（第2层） ）。

结果

每周用皮下聚乙二醇化干扰素α-2b治疗18例合格患者（男8例，女10例），疗程最多18个疗程（108周）。该疗法耐受良好，无4级或5级毒性。在第1层的7名合格患者中，有2名（28.6％）有部分缓解，但只有一种缓解持续了3个月以上。尽管中位无进展生存期为19.5个月，但11层2例患者均未发生客观的影像学反应。

结论

在复发性颅咽管瘤的儿童和年轻人中，聚乙二醇化干扰素α-2b治疗代替放疗或在放疗后具有良好的耐受性。尽管客观反应有限，但无进展生存期的结果令人鼓舞，值得进一步研究。