Treatment of severe acute graft-versus-host disease refractory to multiple lines of therapy: Real-world data from 317 adults treated with the multiple-donor bone marrow-derived mesenchymal stromal cell preparation tomostrocel.

Authors

M Verbeek, H Bonig, F Ayuk, G Bug, M D'Aveni-Piney, D Heidenreich, P Herhaus, U Holtick, I Kos, W Krüger, M T Lupo-Stanghellini, T Mika, C Neuerburg, E Osswald, V Panagiota, C Reicherts, C Schmid, M Srour, F Stölzel, M Tribanek, F Wortmann, G Wulf, A Zens, R Zeiser

Published in

Transplantation and cellular therapy. Sep 12, 2026. Epub Sep 12, 2026.

Abstract

Patients with severe, multi-refractory acute graft-versus-host disease (aGvHD) after hematopoietic stem cell transplantation (HSCT) have a dismal prognosis, but mesenchymal stromal cells (MSC) have emerged as a promising treatment option. A unique bone marrow-derived off-the-shelf MSC product generated from multiple donors, i.e. human allogeneic mesenchymal stromal cells DRK-BaWü-He-FFM (tomostrocel, formerly referred to as MSC-FFM), is currently under development. Tomostrocel is also distributed under Hospital Exemption in Germany and Named Patient Use elsewhere in Europe, with data of treated patients captured in a dedicated surveillance program.
The purpose of this retrospective analysis of prospectively collected surveillance data is to describe outcomes of adult patients with multi-refractory aGvHD treated with tomostrocel in clinical practice.
We retrospectively assessed real-world data of 317 adults with refractory aGvHD treated with tomostrocel in multiple transplant centers across Europe between 2017 and 2025. This uncontrolled observational study includes pseudonymized data actively collected for all patients receiving tomostrocel outside of clinical trials. Evaluated outcomes include response rates at different time points, overall survival and adverse reactions. Patients were followed-up for 24 months from first administration of tomostrocel.
Median follow-up since the last HSCT was 26.9 months (range 2.4 to 63.2 months). aGvHD was mostly severe (4, 37, and 55% grade II, III, and IV, respectively) and a median of three (reported range 0-10) prior therapeutic lines had been exhausted, including ruxolitinib in 75% of patients. The predominant site of aGvHD was the lower gastrointestinal (GI) tract (30%/52% with stage 3/4). The overall response rate in this heavily pre-treated cohort at Day 28 was 45% (95% confidence interval [CI] 40-51%). Probability of overall survival (OS) at 6, 12, and 24 months was 44% (95% CI 38-49%), 33% (95% CI 27-38%), and 28% (95% CI 23-34%), respectively (median 4.3 months). Several subgroup analyses suggested signs of efficacy compared to expected outcomes according to the literature. The safety profile of tomostrocel was favorable, consistent with previous reports.
This real-world data analysis showed several efficacy signals and a reassuring safety profile for tomostrocel in multi-refractory aGvHD. Response on Day 28 was predictive for OS, regardless of prior exposure to ruxolitinib. Despite the inherent limitations associated with real-world data collection, these findings suggest that tomostrocel represents a promising treatment option for heavily pretreated and severely ill SR-aGvHD patients. Results of the ongoing randomized controlled trials are eagerly awaited.

PMID:
42731730
Bibliographic data and abstract were imported from PubMed on 13 Sep 2026.

Read full publication at:
Please sign in to see all details.

Stats

1-terrible, 9-excellent. How would you rate this publication? Sign in in to submit your rating.

Post a comment

You need to be signed in to post comments. You can sign in here.

Comments

There are no comments yet.