BACKGROUND

Immunosuppression strategies have changed over time in pediatric heart transplantation. Thus, comorbidity profiles may have evolved. Clinical Trials in Organ Transplantation in Children-04 is a multicenter, prospective, cohort study assessing the impact of pre-transplant sensitization on outcomes after pediatric heart transplantation. This sub-study reports 1-year outcomes among recipients without pre-transplant donor-specific antibodies (DSAs).

METHODS

We recruited consecutive candidates (<21 years) at 8 centers. Sensitization status was determined by a core laboratory. Immunosuppression was standardized as follows: Thymoglobulin induction with tacrolimus and/or mycophenolate mofetil maintenance. Steroids were not used beyond 1 week. Rejection surveillance was by serial biopsy.

RESULTS

There were 240 transplants. Subjects for this sub-study (n = 186) were non-sensitized (n = 108) or had no DSAs (n = 78). Median age was 6 years, 48.4% were male, and 38.2% had congenital heart disease. Patient survival was 94.5% (95% confidence interval, 90.1–97.0%). Freedom from any type of rejection was 67.5%. Risk factors for rejection were older age at transplant and presence of non-DSAs pre-transplant. Freedom from infection requiring hospitalization/intravenous anti-microbials was 75.4%. Freedom from rehospitalization was 40.3%. New-onset diabetes mellitus and post-transplant lymphoproliferative disorder (PTLD) occurred in 1.6% and 1.1% of subjects, respectively. There was no decline in renal function over the first year. Corticosteroids were used in 14.5% at 1 year.

CONCLUSIONS

Pediatric heart transplantation recipients without DSAs at transplant and managed with a steroid avoidance regimen have excellent short-term survival and a low risk of first-year diabetes mellitus and PTLD. Rehospitalization remains common. These contemporary observations allow for improved caregiver and/or patient counseling and provide the necessary outcomes data to help design future randomized controlled trials.

中文翻译：

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低风险儿科心脏移植受者的早期结果和避免类固醇：一项多中心队列研究（儿童器官移植临床试验 - CTOTC-04）。

背景

儿科心脏移植中的免疫抑制策略随着时间的推移发生了变化。因此，合并症特征可能已经演变。儿童器官移植临床试验 - 04 是一项多中心、前瞻性队列研究，评估移植前致敏对小儿心脏移植后结果的影响。这项子研究报告了没有移植前供体特异性抗体 (DSA) 的受者的 1 年结果。

方法

我们在 8 个中心招募了连续候选人（<21 岁）。致敏状态由核心实验室确定。免疫抑制标准化如下：用他克莫司和/或吗替麦考酚酯维持诱导胸腺球蛋白。超过 1 周未使用类固醇。通过连续活检进行排斥反应监测。

结果

共有240例移植手术。本子研究的受试者 ( n  = 186) 未致敏 ( n  = 108) 或没有 DSA ( n  = 78)。中位年龄为6岁，48.4%为男性，38.2%患有先天性心脏病。患者存活率为 94.5%（95% 置信区间，90.1–97.0%）。未遭受任何类型拒绝的比例为 67.5%。排斥反应的危险因素是移植时年龄较大和移植前存在非 DSA。无需住院/静脉注射抗微生物药物的感染率为 75.4%。免于再住院的率为 40.3%。新发糖尿病和移植后淋巴组织增生性疾病 (PTLD) 的发生率分别为 1.6% 和 1.1%。第一年肾功能没有下降。1 年时使用皮质类固醇的比例为 14.5%。

结论

移植时未接受 DSA 并采用避免类固醇疗法的儿童心脏移植受者具有良好的短期生存率，并且第一年糖尿病和 PTLD 的风险较低。再住院仍然很常见。这些当代观察结果可以改善护理人员和/或患者的咨询，并提供必要的结果数据来帮助设计未来的随机对照试验。