Department of Nuclear Medicine, University of Pretoria, Pretoria, South Africa.
Semin Nucl Med. 2020 Sep;50(5):399-404. doi: 10.1053/j.semnuclmed.2020.04.001. Epub 2020 May 24.
The development of peptide receptor radionuclide therapy (PRRT) in disseminated neuroendocrine tumors (NETs) has been a long and protracted process. The idea was born within nuclear medicine academia but its translation to clinical practice has been marked by misunderstanding of the rigors of the processes used in drug registration. There were several false starts and some of the required basic science did not occur until after first in man studies. The standard process of preclinical, phase 1, 2 and 3 clinical trials were sometimes blurred and the required data including the assurances that patients were studied on protocol was missing from subsequent publications. Despite this there was a growing conviction and increasing evidence that the use of PRRT had a positive benefit in both survival and symptom relief in about 80% of treated patients. After a decade and a half of false starts and incomplete data a formal randomized controlled trial was conducted comparing PRRT with high dose somatostatin which clearly proved that PRRT was both safe, effective and the treatment of choice in hormone refractory NETs.
肽受体放射性核素治疗（PRRT）在弥散性神经内分泌肿瘤（NETs）中的发展是一个漫长而曲折的过程。这个想法源于核医学学术界，但将其转化为临床实践却因对药物注册过程中严谨性的误解而受阻。在此过程中出现了一些失误，一些必要的基础科学研究直到首次人体研究之后才得以进行。临床前、1 期、2 期和 3 期临床试验的标准流程有时会混淆，后续出版物中也缺少必要的数据，包括对患者按方案进行研究的保证。尽管如此，人们越来越相信，PRRT 的使用在大约 80%的治疗患者的生存和症状缓解方面具有积极的益处。经过十五年的反复尝试和不完整的数据收集，终于进行了一项 PRRT 与高剂量生长抑素的正式随机对照试验，该试验清楚地证明 PRRT 既安全、有效，又是激素难治性 NETs 的首选治疗方法。
