Zaucha-Prażmo Agnieszka, Sadurska Elżbieta, Pieczonka Anna, Goździk Jolanta, Dębski Robert, Drabko Katarzyna, Zawitkowska Joanna, Lejman Monika, Wachowiak Jacek, Styczyński Jan, Kowalczyk Jerzy R
Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, University Children's Hospital, Lublin, Poland.
Department of Pediatric Cardiology, Medical University of Lublin, University Children's Hospital, Lublin, Poland.
Ann Transplant. 2019 Jun 25;24:374-382. doi: 10.12659/AOT.915330.
BACKGROUND The objective of this study was the analysis of transplant outcomes and survival in children treated with allogeneic hematopoietic cell transplantation (alloHCT) for non-malignant disorders, with a focus on risk factor analysis of transplant-related mortality (TRM). MATERIAL AND METHODS The treatment outcome was analyzed retrospectively in 10 consecutive years in 4 pediatric transplant centers in Poland. To compare the outcomes, patient data were analyzed according to the diagnosis, age at transplant, donor type, stem cell source, conditioning regimens, transplanted CD34+ cells dose, and pediatric TRM score. RESULTS From 183 analyzed patients, 27 (14.8%) died, all of them due to transplant-related complications. TRM occurred more frequently in matched unrelated donor (MUD) transplant recipients vs. matched sibling donor (MSD) transplant recipients (p=0.02); in peripheral blood (PB) recipients vs. bone marrow (BM) recipients (p=0.004); and in patients receiving >5×10⁶/kg CD34+ cells (p<0.0001). OS differed significantly according to underlying disease comparing to other diagnoses. Lower survival was found in patients transplanted from MUD (p=0.02). OS was higher in patients receiving BM (p=0.001) and in those receiving ≤5×10⁶/kg CD34+ cells (p<0.001). Multivariate analysis showed lower probability of TRM in BM recipients (p=0.04). The probability of TRM was higher in SCID patients (p=0.02) and in patients receiving >5×10⁶/kg CD34+ cells (p=0.0001). CONCLUSIONS Underlying disease, stem cell source, and CD34+ dose higher than 5×10⁶/kg were the most important risk factors for TRM, and they all affected OS.
背景 本研究的目的是分析接受异基因造血细胞移植（alloHCT）治疗非恶性疾病的儿童的移植结局和生存率，重点是移植相关死亡率（TRM）的危险因素分析。
材料与方法 对波兰4个儿科移植中心连续10年的治疗结局进行回顾性分析。为比较结局，根据诊断、移植时年龄、供体类型、干细胞来源、预处理方案、移植的CD34+细胞剂量和儿科TRM评分对患者数据进行分析。
结果 在183例分析患者中，27例（14.8%）死亡，均死于移植相关并发症。TRM在匹配无关供体（MUD）移植受者中比匹配同胞供体（MSD）移植受者更频繁发生（p = 0.02）；在外周血（PB）受者中比骨髓（BM）受者更频繁发生（p = 0.004）；以及在接受>5×10⁶/kg CD34+细胞的患者中更频繁发生（p < 0.0001）。与其他诊断相比，总体生存率（OS）根据基础疾病有显著差异。从MUD移植的患者生存率较低（p = 0.02）。接受BM的患者OS较高（p = 0.001），接受≤5×10⁶/kg CD34+细胞的患者OS也较高（p < 0.001）。多因素分析显示BM受者TRM的概率较低（p = 0.04）。TRM的概率在重症联合免疫缺陷（SCID）患者中较高（p = 0.02），在接受>5×10⁶/kg CD34+细胞的患者中较高（p = 0.0001）。
结论 基础疾病、干细胞来源和高于5×10⁶/kg的CD34+剂量是TRM最重要的危险因素，它们均影响OS。
