Abstract
BackgroundHepatic fibrosis is a common complication in transfusion-dependent thalassemia patients. Data on the co-transplantation of mesenchymal stem cells (MSCs) with hematopoietic stem cells (HSCs) in beta-thalassemia major patients are scarce. Therefore, we aimed to evaluate the effect of co-transplantation of bone marrow-derived MSC with HSCs on the liver fibrosis alleviation and transplant outcomes in class III beta-thalassemia major.MethodsBetween April 1998 and January 2017, a total of 224 consecutive patients with class III beta-thalassemia major underwent allogeneic HSCT in the Research Institute for Oncology, Hematology and Cell Therapy, Tehran University of Medical Sciences, Tehran, Iran. To assess liver fibrotic changes after transplantation, 47 patients participated in the MSC plus HSC group and 30 patients in the HSC only group at the end of the follow-up period. All patients underwent laboratory tests, especially serum ferritin and liver function testing, hepatic T2* MRI, liver biopsy, and FibroScan before and 2 years after transplantation. Kaplan-Meier curves were derived to determine survival and were compared using the log-rank test. Repeated-measure, mixed-effect linear regression models were used to examine the changes in liver fibrosis over time.ResultsThe 10-year OS rate was 71.84% in the mesenchymal group and 61.89% in the non-mesenchymal group (P value = 0.294), while the 10-year TFS rate was 63.64% in the mesenchymal group and 52.78% in the non-mesenchymal group (P value = 0.285). No significant difference was observed in the 10-year NRM, rejection rate, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD between the two groups. In addition, the results of repeated-measure, mixed-effect linear regression models showed that none of the variables determining hepatic fibrosis had a significant difference between patients receiving MSCs and patients who did not receive MSCs.ConclusionsBased on the results of this study, a single infusion of MSCs at the time of HSCT to patients with class III beta-thalassemia major could not significantly improve the liver fibrosis alleviation and transplantation outcomes, including OS, TFS, TRM, rejection rate, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD.
Highlights
Thalassemia major (TM) is one of the most common inherited genetic hematological diseases worldwide and is associated with significant morbidity and mortality rates [1, 2]
Hepatic fibrosis is a common complication in transfusion-dependent thalassemia patients
Participants who met any of the following criteria were excluded from the study: (1) patients who declined to participate in the study, (2) any severe underlying or pre-existing medical condition other than thalassemia major and its obvious complications, (3) those who lost a regular follow-up visit, (4) patients who did not gain complete hematological recovery after transplantation, (5) patients who had received drugs with hepatic metabolism 6 months before the study, and (6) patients with class I and II thalassemia major
Summary
Thalassemia major (TM) is one of the most common inherited genetic hematological diseases worldwide and is associated with significant morbidity and mortality rates [1, 2]. Among patients with TDTM, the prevalence of liver fibrosis and cirrhosis has been reported to be 40–80% and 10–40%, respectively [12,13,14] It seems that hepatic fibrosis in TM patients depends on the patient’s age, liver iron concentration, and the number of infused packed red blood cell units [15]. Recent evidence has shown that thalassemia patients with moderate to severe liver iron concentrations experience persistently elevated serum iron levels for several years after HSCT [16]. They are at increased risk of developing liver fibrosis and cirrhosis after HSCT [17]. We aimed to evaluate the effect of co-transplantation of bone marrow-derived MSC with HSCs on the liver fibrosis alleviation and transplant outcomes in class III beta-thalassemia major
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
