Original Investigation

December 28, 2023

Mesenchymal Stem Cells for Prophylaxis of Chronic Graft-vs-Host Disease After Haploidentical Hematopoietic Stem Cell TransplantAn Open-Label Randomized Clinical Trial

Ruihao Huang, MD¹; Ting Chen, MD¹; Sanbin Wang, MD²; et alJishi Wang, MD³; Yi Su, MD⁴; Jing Liu, MD⁵; Yanqi Zhang, PhD⁶; Xiangyu Ma, PhD⁷; Qin Wen, MD¹; Peiyan Kong, MD¹; Cheng Zhang, MD¹; Lei Gao, MD¹; Jiang F. Zhong, PhD⁸; Li Gao, MD¹; Xi Zhang, MD, PhD^1,9

Author Affiliations Article Information

JAMA Oncol. Published online December 28, 2023. doi:10.1001/jamaoncol.2023.5757

visual abstract icon

Visual

Abstract

Key Points

Question Does infusion of mesenchymal stem cells during the early stage after haploidentical hematopoietic stem cell transplant help prevent chronic graft-vs-host disease?

Findings In this randomized clinical trial of 158 patients with acute leukemia, 4 rounds of prophylactic infusion of umbilical cord mesenchymal stem cells during 45 days to 81 days after haploidentical hematopoietic stem cell transplant significantly decreased the incidence and severity of chronic graft-vs-host disease as well as acute graft-vs-host disease. No effect on leukemia relapse or treatment-related adverse events was observed after repeated infusion of mesenchymal stem cells.

Meaning The results of this randomized clinical trial show that repeated mesenchymal stem cells infusion during the early stage after haploidentical hematopoietic stem cell transplant demonstrated clinically meaningful decline in severe chronic graft-vs-host disease occurrence and improvement in graft-vs-host disease-free and relapse-free survival without increasing the incidence of adverse events; however, further research on the MSCs infusion for haploidentical hematopoietic stem cell transplant complications management should be explored.