Department of Pediatric Hematology and Oncology, Goztepe Medical Park Hospital, Istanbul, Turkey.
Department of Pediatric Hematology and Oncology, Bahcesehir University, Goztepe Medical Park Hospital, Istanbul, Turkey.
Pediatr Transplant. 2024 Feb;28(1):e14688. doi: 10.1111/petr.14688.
Graft-versus-host disease (GvHD) is one of the leading causes of morbidity and mortality in patients undergoing allogeneic HSCT, and effective prevention of GvHD is critical for the success of the HSCT procedure. Calcineurin inhibitors (CNI) have been used for decades as the backbone of GvHD prophylaxis. In this study, the efficacy and safety of Cyclosporine A (CsA) and tacrolimus (TCR) were compared in pediatric HSCT for thalassemia.
This is a retrospective analysis of 129 pediatric patients who underwent HSCT with the diagnosis of thalassemia at Medicalpark Göztepe and Antalya Hospitals between January 2017 and December 2020.
Despite the GvHD prophylaxis, grade II-IV acute GvHD developed in 29 patients. Of these patients, 12 had only gut, 10 had only skin, 6 had combined gut and skin, and one had only liver GvHD. Fifteen of these 29 patients were in the CsA group, and 14 of them were in the TCR group. There was no significant difference between the groups in terms of acute GvHD occurrence, GvHD stage, or involvement sites. In terms of CNI-related toxicity, neurotoxicity in 15 (CsA n = 9, TCR n = 6) and nephrotoxicity in 18 (CsA n = 4, TCR n = 14) patients were observed. While there was no difference between the two groups in terms of neurotoxicity, more nephrotoxicity developed in patients using TCR (p = .013). There was no significant difference between the groups in terms of engraftment syndrome, veno-occlusive disease, CMV reactivation, PRES, or graft rejection.
Regarding GvHD, there was no difference in efficacy between TCR and CsA usage. Patients taking TCR experienced noticeably higher nephrotoxicity in terms of adverse effects. This difference should be considered according to the patient's clinical situation while choosing a CNI.
移植物抗宿主病（GvHD）是接受异基因造血干细胞移植（HSCT）患者发病和死亡的主要原因之一，因此，有效预防 GvHD 对于 HSCT 手术的成功至关重要。钙调磷酸酶抑制剂（CNI）已被用于预防 GvHD 数十年。在这项研究中，比较了环孢素 A（CsA）和他克莫司（TCR）在小儿地中海贫血 HSCT 中的疗效和安全性。
这是一项回顾性分析，纳入了 2017 年 1 月至 2020 年 12 月在 Medicalpark Göztepe 和 Antalya 医院接受 HSCT 治疗的 129 例地中海贫血患儿的临床资料。
尽管进行了 GvHD 预防，但仍有 29 例患者发生 2-4 级急性 GvHD。其中，12 例仅有肠道受累，10 例仅有皮肤受累，6 例同时有肠道和皮肤受累，1 例仅有肝脏 GvHD。这 29 例患者中，15 例在 CsA 组，14 例在 TCR 组。两组在急性 GvHD 发生率、GvHD 分期或受累部位方面无显著差异。在 CNI 相关毒性方面，15 例（CsA 组 n=9，TCR 组 n=6）出现神经毒性，18 例（CsA 组 n=4，TCR 组 n=14）出现肾毒性。两组神经毒性无差异，但 TCR 组肾毒性发生率更高（p=0.013）。两组在植入综合征、静脉闭塞性疾病、CMV 再激活、PRES 或移植物排斥反应方面无显著差异。
在预防 GvHD 方面，TCR 和 CsA 的疗效无差异。但 TCR 组患者的不良反应中肾毒性发生率更高。在选择 CNI 时，应根据患者的临床情况考虑这一差异。
