关键词: craniopharyngioma intracystic treatment pediatric neurooncology pediatric neurosurgery peginterferon alfa-2a

来  源:   DOI:10.3389/fonc.2024.1401761   PDF(Pubmed)

Abstract:
UNASSIGNED: Children with craniopharyngiomas (CPs) typically suffer from a life-long chronic disease. The younger the child, the more vulnerable the maturing brain is to invasive therapies such as surgery or radiotherapy. Therefore, treatment modalities facilitating avoidance or delay of invasive therapies are beneficial for these patients. In the last decade, intracystic injection of interferon alfa-2a or alfa-2b evolved as a treatment of choice based on efficacy and minor toxicity. However, the drug is no longer available internationally. After an extensive pharmacological review, peginterferon alfa-2a was identified as the agent with closest similarity.
UNASSIGNED: A retrospective case series is described, including five patients treated with intracystic peginterferon alfa-2a for cystic CP according to an innovative care protocol. After initial CP cyst aspiration, peginterferon alfa-2a was injected once per week via an Ommaya reservoir for 6 weeks followed by response assessment with MRI.
UNASSIGNED: Patients\' age ranged from 4 to 54 years (four patients <12 years, one adult patient). Intracystic therapy with peginterferon alfa-2a was tolerated well by all five individuals without any major toxicities and resulted in cyst shrinkage in all of the five patients. The importance of a permeability study prior to commencing intracystic therapy became apparent in one patient who suffered from cyst leakage.
UNASSIGNED: Intracystic treatment with peginterferon alfa-2a was found to be a tolerable and efficacious treatment modality in patients with cystic CP. This experience warrants further research with a larger number of patients with measurement of long-term efficacy and safety outcomes.
摘要:
患有颅咽管瘤(CP)的儿童通常患有终身慢性疾病。孩子越年轻,成熟的大脑对侵入性治疗如手术或放射治疗越脆弱。因此,有助于避免或延迟侵入性治疗的治疗方式对这些患者有益.在过去的十年里,干扰素α-2a或α-2b的囊内注射是基于疗效和轻微毒性的选择。然而,这种药物在国际上不再可用。经过广泛的药理学审查,聚乙二醇干扰素α-2a被鉴定为最相似的药物。
描述了回顾性病例系列,包括5例根据创新护理方案接受囊内聚乙二醇干扰素α-2a治疗囊性CP的患者。初次CP囊肿抽吸后,聚乙二醇干扰素α-2a通过Ommaya水库每周注射一次,持续6周,然后用MRI评估反应。
患者的年龄从4到54岁不等(4名患者<12岁,一名成年患者)。聚乙二醇干扰素α-2a的囊内治疗对所有五名患者的耐受性都很好,没有任何主要毒性,并导致所有五名患者的囊肿缩小。在一名患有囊肿漏的患者中,开始进行囊内治疗之前进行渗透性研究的重要性变得显而易见。
发现聚乙二醇干扰素α-2a的囊内治疗是囊性CP患者的一种可耐受且有效的治疗方式。这种经验值得对更多患者进行进一步研究,以测量长期疗效和安全性结果。
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