Castleberry Chesney, Ziniel Sonja, Almond Christopher, Auerbach Scott, Hollander Seth A, Lal Ashwin K, Fenton Matthew, Pahl Elfriede, Rossano Joseph W, Everitt Melanie D, Daly Kevin P
Washington University in St. Louis, St. Louis, MO, USA.
Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Pediatr Transplant. 2017 Aug;21(5). doi: 10.1111/petr.13013. Epub 2017 Jul 3.
Clinical practice variations are a barrier to the study of pediatric heart transplants and coordination of multicenter RCTs in this patient population. We surveyed centers to describe practice patterns, understand areas of variation, and willingness to modify protocol. Pediatric heart transplant centers were identified, and one survey was completed per center. Simple descriptive statistics were used. The response rate was 77% (40 responses from 52 contacted centers, 37 with complete responses). Median center volume of respondents was eight transplants/year (IQR 3-19). Most centers reported tacrolimus (36/38, 95%) and mycophenolate mofetil (36/38, 95%) as maintenance immunosuppression. Other immunosuppression agents reported were cyclosporine (7/38, 18%), everolimus or sirolimus (3/38, 8%), and azathioprine (2/38, 5%). Overall, respondents answered similarly for questions regarding clinical practices including induction therapy, maintenance immunosuppression, and rejection treatment threshold (>85% agreement for all). Additionally, willingness to change clinical practices was over 70% for all practices surveyed (35 total respondents), and 97% of centers (36/37) were willing to participate in a RCT of maintenance immunosuppression. In conclusion, we found many similar clinical practice protocols. Most centers are willing to collaborate on a common protocol in order to participate in a RCT and support a trial investigating maintenance immunosuppression.
临床实践差异是小儿心脏移植研究以及该患者群体多中心随机对照试验协调工作的障碍。我们对各中心进行了调查，以描述实践模式、了解差异领域以及修改方案的意愿。确定了小儿心脏移植中心，并每个中心完成一份调查问卷。使用了简单的描述性统计方法。回复率为77%（52个联系中心中有40个回复，37个回复完整）。回复者所在中心的移植中位数为每年8例（四分位间距3 - 19）。大多数中心报告使用他克莫司（36/38，95%）和霉酚酸酯（36/38，95%）作为维持免疫抑制药物。报告的其他免疫抑制药物有环孢素（7/38，18%）、依维莫司或西罗莫司（3/38，8%）以及硫唑嘌呤（2/38，5%）。总体而言，对于包括诱导治疗、维持免疫抑制和排斥反应治疗阈值等临床实践问题，回复者的回答相似（所有问题的一致性均超过85%）。此外，对于所有调查的实践，改变临床实践的意愿超过70%（共35名回复者），97%的中心（36/37）愿意参与维持免疫抑制的随机对照试验。总之，我们发现了许多相似的临床实践方案。大多数中心愿意就共同方案进行合作，以参与随机对照试验并支持一项关于维持免疫抑制的试验研究。
