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ORIGINAL RESEARCH article

Front. Pediatr.
Sec. Pediatric Hematology and Hematological Malignancies
Volume 12 - 2024 | doi: 10.3389/fped.2024.1375493

Mesenchymal Stromal Cells in the treatment of Pediatric Hematopoietic Cell Transplantation-related complications (graft versus host disease, hemorrhagic cystitis, graft failure and poor graft function): a single center experience Provisionally Accepted

  • 1Department of Paediatric Oncology and Haematology, Vall d'Hebron University Hospital, Spain
  • 2Vall d'Hebron University Hospital, Spain
  • 3Banc de Sang i Teixits, Spain
  • 4Department of Clinical Pharmacology, Vall d'Hebron University Hospital, Spain
  • 5Department of Medicine, Faculty of Medicine, Autonomous University of Barcelona, Spain
  • 6Vall d'Hebron Research Institute (VHIR), Spain

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Objectives: to describe mesenchymal stromal cells (MSCs) in the treatment of hematopoietic stem cell transplantation (HSCT) complications and to assess its safety and efficacy.Methods Single-center retrospective study (2016-2023). Patients under 20 years who received MSCs for the treatment of HSCT-related complications were included.Results: Thirty patients (53.7% boys), median age at transplant 11 years (range 2-19) were included. MSCs indications were: graft-versus-host disease (GVHD) in 18 patients (60%), of them 13 had acute GVHD (43.3%) and 5 chronic GVHD (16.6%); Grade 3-4 hemorrhagic cystitis (HC) in 4 (13.3%); poor graft function (PGF) in 6 (20%), 5 of them receiving MSCs with a CD34 stem cell-boost coinfusion; graft failure (GF) in 2 (6.7%), to enhance engraftment after a subsequent HSCT. Infusion-related-adverse-events were not reported. Overall response (OR) was 83%; 44% patients (11/25) showed complete response (CR). OR for GVHD, HC, PGF and GF was 83.3%, 100%, 66.7% and 100% respectively. Response rate was 40% (95%CI: 20-55) and 79% (95%CI:57-89) at 15 and 30 days. With a median follow-up of 21 months (IQR11-52.5), overall survival (OS) was 86% (95%CI:74-100) and 79% (95%CI:65-95) at 6 and 12 months post-MSCs infusion.In our study, the most frequent indication of MSCs was refractory aGVHD (43.3%). Response rates were high (OR 83%) and safety profile was good.

Keywords: Mesenchymal Stromal Cells, Hematopoietic Stem Cell Transplantation, graft versus host disease, Graft failure, poor graft function, hemorrhagic cystitis, Children

Received: 23 Jan 2024; Accepted: 28 Mar 2024.

Copyright: © 2024 Pérez-Torres Lobato, BENITEZ-CARABANTE, Alonso, Torrents, Castillo Flores, Uria Oficialdegui, Panesso, Alonso-Martínez, Oliveras, Renedo-Miró, Vives and Diaz De Heredia. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Dr. Cristina Diaz De Heredia, Department of Paediatric Oncology and Haematology, Vall d'Hebron University Hospital, Barcelona, 08035, Catalonia, Spain