Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
Department of Pediatric Stem Cell Transplantation, Children's Medical Center, Tehran University of Medical Sciences, Tehran, Iran.
Int J Hematol. 2021 Jan;113(1):145-157. doi: 10.1007/s12185-020-02998-4. Epub 2020 Oct 8.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) potentially renders thalassemia patients disease-free with presumably cessation of associated complications. This study analyzes the liver fibrosis status and the determinants of its progression in ex-thalassemic patients. The liver fibrosis status of 108 pediatric transfusion-dependent β-thalassemia major patients was evaluated before and one year after allo-HSCT using transient elastography (TE). All patients achieved normal hematopoiesis. In univariate analyses, not in all, but in patients developing significant post-HSCT iron overload or hepatic graft-versus-host disease (GvHD), as well as recipients of bone marrow stem cells (BMSC), significant TE increment occurred. In multivariable analyses, through a model with large effect size (Adj.R = 26%, F = 13.53, P < 0.001), post-HSCT serum ferritin and hepatic GvHD were ascertained as independent determinants of significant TE increase, and the effect of stem cell graft source approached the level of significance. Excluding the patients with intermediate/high Lucarelli risk classes, the TE increase was significantly greater only in BMSC recipients (P = 0.033). Although the risk impact of allograft source on liver fibrosis progression requires further evaluation; hepatic status of ex-thalassemic patients can be preserved after HSCT, if hepatic GvHD is controlled and adequate post-transplantation iron depletion is ensured.
同种异体造血干细胞移植 (allo-HSCT) 有可能使地中海贫血患者无病生存,并且可能停止相关并发症。本研究分析了 ex-thalassemic 患者的肝纤维化状况及其进展的决定因素。使用瞬态弹性成像 (TE) 评估了 108 例依赖输血的β-地中海贫血重型儿科患者 allo-HSCT 前后的肝纤维化状况。所有患者均实现了正常的造血功能。在单因素分析中,并非所有患者,而是在发生显著 post-HSCT 铁过载或肝移植物抗宿主病 (GvHD) 的患者中,以及接受骨髓干细胞 (BMSC) 的患者中,TE 值显著增加。在多变量分析中,通过一个具有较大效应量的模型 (Adj.R = 26%,F = 13.53,P < 0.001),post-HSCT 血清铁蛋白和肝 GvHD 被确定为 TE 显著增加的独立决定因素,而干细胞移植物来源的影响接近显著性水平。排除 Lucarelli 风险等级中/高的患者,只有 BMSC 受者的 TE 增加显著更大 (P = 0.033)。尽管同种异体移植物来源对肝纤维化进展的风险影响需要进一步评估;但如果控制肝 GvHD 并确保移植后充分去铁,HSCT 后 ex-thalassemic 患者的肝脏状况可以得到保留。