A safety and feasibility study with platelet lysate expanded bone marrow mesenchymal stromal cells for the treatment of acute graft-versus-host disease in Brazil

摘要

Allogeneic hematopoietic stem cell transplant (AlloSCT) is the treatment of choice for many malignant and non-malignant hematological disorders [1]. However, this treatment is frequently complicated by acute graft-versus-host disease (aGVHD), which can be associated with high morbidity and mortality [2]. Steroids are still the first-line treatment for established aGVHD, with a response rate of 30-50%, and there is no established and effective therapy for severe steroid-refractory aGVHD. The outcome for patients is poor and overall survival low, with as few as 16% of patients alive at 2 years [3]. In the case of failure after corticosteroid treatment, different therapeutic options have been introduced as second- or third-line strategies [4]. In this scenario, infusion of ex vivo expanded mesenchymal stromal cells (MSCs) has emerged as an additional tool for the treatment of GVHD. Owing to their proprieties the infusion of MSCs had shown promising results in studies of the treatment of aGVHD [5-7]. Here we report the results of a safety and feasibility pilot study including eight patients with steroid-resistant aGVHD treated with Good Manufacturing Practice (GMP) clinical grade, platelet lysate (PL) expanded MSCs.