Umbilical Cord Mesenchymal Stromal Cells for Steroid-Refractory Acute Graft-versus-Host Disease

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Donadel, Camila Derminio ; Pires, Bruno Garcia ; Andre, Nathalia Cristine ; Costa, Thalita Cristina Mello ; Orellana, Maristela Delgado ; Caruso, Samia Rigotto ; Seber, Adriana ; Ginani, Valeria Cortez ; Gomes, Alessandra Araujo ; Novis, Yana ; Barros, George Mauricio Navarro ; Vilella, Neysimelia Costa ; Martinho, Glaucia Helena ; Vieira, Ana Karine ; Kondo, Andrea Tiemi ; Hamerschlak, Nelson ; Filho Schmidt, Jayr ; Xavier, Erick Menezes ; Fernandes, Juliana Folloni ; Rocha, Vanderson ; Covas, Dimas Tadeu ; Calado, Rodrigo Tocantins ; Guerino-Cunha, Renato Luiz ; De Santis, Gil Cunha

• Document type: Journal article
• Source: PHARMACEUTICALS; v. 16, n. 4, p. 12-pg., 2023-04-01.

Abstract

Background: Steroid-refractory acute graft-vs.-host disease (SR-aGVHD) is a complication of allogeneic hematopoietic stem cell transplantation with a dismal prognosis and for which there is no consensus-based second-line therapy. Ruxolitinib is not easily accessible in many countries. A possible therapy is the administration of mesenchymal stromal cells (MSCs). Methods: In this retrospective study, 52 patients with severe SR-aGVHD were treated with MSCs from umbilical cord (UC-MSCs) in nine institutions. Results: The median (range) age was 12.5 (0.3-65) years and the mean +/- SD dose (x10(6)/kg) was 4.73 +/- 1.3 per infusion (median of four infusions). Overall (OR) and complete response (CR) rates on day 28 were 63.5% and 36.6%, respectively. Children (n = 35) had better OR (71.5% vs. 47.1%, p = 0.12), CR (48.6% vs. 11.8%, p = 0.03), overall survival (p = 0.0006), and relapse-free survival (p = 0.0014) than adults (n = 17). Acute adverse events (all of them mild or moderate) were detected in 32.7% of patients, with no significant difference in children and adult groups (p = 1.0). Conclusions: UC-MSCs are a feasible alternative therapy for SR-aGVHD, especially in children. The safety profile is favorable. (AU)

• FAPESP's process: 13/08135-2 - CTC - Center for Cell-Based Therapy
• Grantee: Dimas Tadeu Covas
• Support Opportunities: Research Grants - Research, Innovation and Dissemination Centers - RIDC