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. 2023 Mar 30;16(4):512.
doi: 10.3390/ph16040512.

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

Camila Derminio Donadel  1 , Bruno Garcia Pires  2 , Nathália Cristine André  1 , Thalita Cristina Mello Costa  2 , Maristela Delgado Orellana  1 , Sâmia Rigotto Caruso  1 , Adriana Seber  3   4 , Valéria Cortez Ginani  3   4 , Alessandra Araújo Gomes  5 , Yana Novis  5 , George Maurício Navarro Barros  6 , Neysimélia Costa Vilella  6 , Gláucia Helena Martinho  7 , Ana Karine Vieira  7 , Andrea Tiemi Kondo  8 , Nelson Hamerschlak  8 , Jayr Schmidt Filho  9 , Erick Menezes Xavier  10 , Juliana Folloni Fernandes  8   11 , Vanderson Rocha  10 , Dimas Tadeu Covas  1   2 , Rodrigo Tocantins Calado  1   2 , Renato Luiz Guerino-Cunha  2 , Gil Cunha De Santis  1   2
Affiliations

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

Camila Derminio Donadel et al. Pharmaceuticals (Basel). .

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 (×106/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.

Keywords: acute graft-versus-host disease; hematopoietic stem cell transplantation; mesenchymal stromal cell.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Flowchart showing the reasons for the exclusion of 6 patients, which resulted in a total of 52 patients included in the analyses.
Figure 2
Figure 2
Overall (A) and relapse-free survival (B) in children and adults (after MSC infusion).
Figure 3
Figure 3
Overall survival according to response (complete and partial) at day 28 after MSCs.
See this image and copyright information in PMC

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