Co‐transplantation of bone marrow‐derived mesenchymal stem cells with hematopoietic stem cells does not improve transplantation outcome in class III beta‐thalassemia major: A prospective cohort study with long‐term follow‐up

Abstract

Bone marrow transplantation is the only curative treatment for beta-thalassemia major. Data on the co-transplantation of MSCs with HSCs in beta-thalassemia major patients are scarce. We aimed to investigate the outcomes of thalassemia major patients who underwent bone marrow-derived MSC co-transplantation with HSCs compared with those who only received HSCs. This prospective randomized study included patients with class III thalassemia major undergoing HSCT divided randomly into two groups: Thirty-three patients underwent co-transplantation of bone marrow-derived MSCs with HSCs, and 26 patients only received HSCs. Five-year OS, TFS, TRM, graft rejection rate, and GVHD were estimated. The 5-year OS was 66.54% (95% CI, 47.8% to 79.9%) in patients who underwent co-transplantation of MSCs with HSCs vs 76.92% (95% CI, 55.7% to 88.9%) in patients who only received HSCs (P = .54). No significant difference was observed in the 5-year TFS between the two groups (59.1% vs 69.2%; P = .49). The 5-year cumulative incidence of TRM was not statistically significant among patients who underwent co-transplantation of MSCs with HSCs (27.27%) vs those who only received HSCs (19.23%; P = .61). There was no statistically significant difference in graft rejection, acute GvHD, and chronic GvHD between the two groups. Based on our findings, the co-transplantation of MSCs and HSCs to class III thalassemia major patients does not alter their transplantation outcomes including OS, TFS, rejection rate, transplant-related mortality, and GvHD. K E Y W O R D S beta-thalassemia, hematopoietic stem cells, mesenchymal stem cells, outcome How to cite this article: Rostami T, Maleki N, Kasaeian A, et al. Co-transplantation of bone marrow-derived mesenchymal stem cells with hematopoietic stem cells does not improve transplantation outcome in class III beta-thalassemia major: A prospective cohort study with long-term follow-up. Pediatr

The effect of bone marrow-derived mesenchymal stem cell co-transplantation with hematopoietic stem cells on liver fibrosis alleviation and survival in patients with class III β-thalassemia major

“…To date, no studies have investigated the use of bone marrow-derived MSCs for the improvement of liver fibrosis in thalassemia major. As reported in our previous study on a subgroup of thalassemia major patients, the co-transplantation of MSCs and HSCs to class III thalassemia major patients does not alter their transplantation outcomes [47]. In the present study, the cotransplantation of bone marrow-derived MSCs and HSCs in patients with LRC class III beta-thalassemia major could not significantly improve the liver fibrosis alleviation and transplantation outcomes, including OS, TFS, TRM, reject incidence, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD.…”

The Effect of Bone Marrow-Derived Mesenchymal Stem Cells Co-transplantation with Hematopoietic Stem Cells on Liver Fibrosis Alleviation and Survival in Patients with Class III β-thalassemia Major

“…As reported in our previous study on a subgroup of thalassemia major patients, the co-transplantation of MSCs and HSCs to class III thalassemia major patients does not alter their transplantation outcomes. 47 In the present study, the co-transplantation of bone marrow-derived MSCs and HSCs in patients with LRC class III beta-thalassemia major could not signi cantly improve the liver brosis alleviation and transplantation outcomes, including OS, TFS, TRM, reject incidence, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD.…”

Presencia del antígeno CD34 como marcador.

“…Pero dado el caso que se necesite, es posible estimular la movilización de células madres de la médula ósea hacia la circulación periférica. Para que se pueda dar dicha movilización, los donadores deben pasar por un procedimiento 4 a 5 días previos a la extracción, en el cual se les aplicará una inyección diaria de factor estimulante de colonia de granulocitos (G-CSF) (1,3,9).…”

“…El trasplante de médula ósea (TMO) es un procedimiento terapéutico que consiste en reemplazar la médula ósea con células afectadas del paciente por médula ósea con células sanas totipotenciales; este tejido puede ser obtenido de manera alogénica o autóloga por el paciente (1,2). Los TMO suelen darse como una alternativa terapéutica en pacientes que están cursando enfermedades que afectan las células de la médula ósea como son la anemia drepanocítica, anemia aplásica o en casos de cáncer como son los linfomas, leucemias, mieloma múltiple y mielodisplasia (3,4).…”

“…Al ser una intervención delicada, se deben tomar todas las medidas de seguridad y prevención necesarias para evitar complicaciones al momento del trasplante, por lo que es indispensable saber si el trasplante es exitoso o no, para poder continuar con el tratamiento (3). Por esto, en este caso se busca medir los niveles de células madre con antígeno CD34 en sangre periférica para evaluar cómo se está dando el curso del trasplante, debido a que este antígeno se encuentra en la superficie de células sanguíneas, por lo cual al ser detectado en sangre estaría indicando la formación de células totipotenciales, que cuentan con los elementos necesarios para producción de médula ósea (4,5).…”