Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, Maryland.
Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, Maryland.
J Pediatr Adolesc Gynecol. 2023 Dec;36(6):566-568. doi: 10.1016/j.jpag.2023.08.005. Epub 2023 Aug 25.
Now that ovarian tissue cryopreservation (OTC) has become standard of care for patients receiving gonadotoxic therapies, discussion has turned toward offering OTC to pediatric patients with Turner syndrome outside of research. Although patients with Turner syndrome have unmet fertility needs and the authors support efforts for fertility preservation in these individuals, safety and efficacy data about OTC in this population are limited. Building on longstanding debates around offering experimental therapies as research or outside of research (as "innovative therapy"), we considered the suitability of offering OTC for patients with Turner syndrome as innovative therapy. On the basis of pathophysiology and preliminary research data, we argue that there is significant uncertainty about whether the risk-benefit profile of OTC for patients with Turner syndrome is favorable. This reduces the weight of arguments in favor of offering it as innovative therapy. Furthermore, as Turner syndrome is rare, widespread availability of OTC could make it difficult to develop generalizable knowledge. The benefits of innovative therapy for acquiring experience from use in humans and avoiding research-related procedures are of limited importance too, as OTC is already an established procedure, and current studies involve limited procedures that restrict access. OTC should therefore only be offered to patients with Turner syndrome in research settings until additional data suggest that the risk-benefit profile is likely favorable.
如今，卵巢组织冷冻保存（OTC）已成为接受性腺毒性治疗患者的标准治疗方法，因此人们开始讨论是否应在研究之外向特纳综合征的儿科患者提供 OTC。尽管特纳综合征患者有未满足的生育需求，且作者支持在这些个体中进行生育力保存的努力，但关于该人群中 OTC 的安全性和有效性数据有限。在围绕着将实验性疗法作为研究或研究之外的“创新性疗法”提供的长期争论的基础上，我们考虑了将 OTC 作为创新性疗法提供给特纳综合征患者的适宜性。基于发病机制和初步研究数据，我们认为，关于 OTC 对特纳综合征患者的风险效益比是否有利，存在很大的不确定性。这降低了支持将其作为创新性疗法提供的论点的权重。此外，由于特纳综合征较为罕见，如果广泛提供 OTC，可能难以获得可推广的知识。创新性疗法在获取人类使用经验和避免与研究相关程序方面的益处也很有限，因为 OTC 已经是一种既定的程序，而且当前的研究涉及限制准入的有限程序。因此，只有在有更多数据表明风险效益比可能有利的情况下，才应在研究环境中向特纳综合征患者提供 OTC。
