Objective

Rare tumour management is challenging for clinicians as evidence bases are limited and clinical trials are difficult to conduct. It is even more difficult for patients where self-reliance alone is insufficient to overcome the challenges of navigating care which is often poorly evidence based. In Ireland, a national Gestational Trophoblastic Disease (GTD) service was established as one of 3 initiatives for rare tumours by the National Cancer Control Programme. The service has a national clinical lead, a dedicated supportive nursing service and a clinical biochemistry liaison team. This study sought to assess the impact of a GTD centre using national clinical guidelines and integrating and networking with European and International GTD groups on the clinical management of challenging GTD cases and to consider the application of this model of care to other rare tumour management.

Study Design

In this article, we analyse the impact of a national GTD service on five challenging cases, and review how the service affects patient management in this rare tumour type. These cases were selected from a cohort of patients who were voluntarily registered in the service based on the diagnostic management dilemma they posed.

Results

Case management was impacted by the identification of GTD mimics, the provision of lifesaving treatment of metastatic choriocarcinoma with brain metastasis, networking with international colleagues, the identification of early relapse, the use of genetics to differentiate treatment pathways and prognosis, and supportive supervision of treatment courses of up to 2 years of therapy in a cohort of patients starting or completing families.

Conclusion

The National GTD service could be an exemplar for the management of rare tumours (such as cholangiocarcinoma) in our jurisdiction which could benefit from a similar constellation of supports. Our study demonstrates the importance of a nominated national clinical lead, dedicated nurse navigator support, registration of cases and networking. The impact of our service would be greater if registration was mandatory rather than voluntary. Such a measure would also ensure equity of access for patients to the service, assist in quantifying the need for resourcing and facilitate research to improve outcomes.

中文翻译：

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具有挑战性的妊娠滋养细胞疾病病例和模拟病例：罕见肿瘤治疗的范例

客观的

由于证据基础有限且临床试验难以进行，罕见肿瘤的管理对临床医生来说具有挑战性。对于仅靠自力更生不足以克服通常证据不足的导航护理挑战的患者来说，这更加困难。在爱尔兰，国家妊娠滋养细胞疾病 (GTD) 服务是国家癌症控制计划针对罕见肿瘤的 3 项举措之一。该服务拥有全国临床领导、专门的支持性护理服务和临床生化联络小组。

学习规划

在本文中，我们分析了国家 GTD 服务对五个具有挑战性的病例的影响，并回顾了该服务如何影响这种罕见肿瘤类型的患者管理。这些病例是根据他们提出的诊断管理困境，从自愿注册服务的一组患者中选出的。

结果

病例管理受到 GTD 模拟物的鉴定、转移性绒毛膜癌伴脑转移的挽救生命治疗的提供、与国际同事的联网、早期复发的鉴定、使用遗传学区分治疗途径和预后以及对治疗的支持性监督的影响在一组开始或完成家庭的患者中进行长达 2 年的治疗过程。

结论

国家 GTD 服务可以成为我们管辖范围内罕见肿瘤（如胆管癌）管理的典范，它可以从类似的支持群中受益。我们的研究证明了指定的国家临床负责人、专门的护士导航支持、病例登记和网络的重要性。如果注册是强制性的而不是自愿的，我们服务的影响会更大。此类措施还将确保患者公平获得服务，有助于量化资源需求并促进研究以改善结果。