Mesenchymal Stromal Cells for Treating Steroid-Resistant Acute and Chronic Graft Versus Host Disease: A Multicenter Compassionate Use Experience.
María Del Mar Macías-SánchezCynthia Morata-TarifaNatividad CuendeAna Cardesa-GilMaría Ángeles Cuesta-CasasMaría Jesús Pascual-CasconAntonia PascualCarmen Martín-CalvoManuel JuradoJosé Antonio Perez-SimónIldefonso EspigadoSebastián Garzón LópezGloria Carmona SánchezRosario Mata-Alcázar-CaballeroRosario Sanchez-PernautePublished in: Stem cells translational medicine (2022)
Graft versus host disease (GVHD) is a severe complication after allogenic hematopoietic cell transplantation (HSCT). Several clinical trials have reported the use of mesenchymal stromal cells (MSCs) for the treatment of GVHD. In March 2008, the Andalusian Health Care System launched a compassionate use program to treat steroid-resistant GVHD with MSC. Clinical-grade MSC were obtained under GMP conditions. MSC therapy was administered intravenously in four separate doses of 1 × 106 cells/kg. Sixty-two patients, 45 males (7 children) and 17 females (2 children), received the treatment. Patients had a median age of 39 years (range: 7-66) at the time of the allogenic HSCT. The overall response was achieved in 58.7% of patients with acute (a)GVHD. Two years' survival for aGVHD responders was 51.85%. The overall response for patients with chronic (c)GVHD was 65.50% and the 2-year survival rate for responders was 70%. Age at the time of HSCT was the only predictor found to be inversely correlated with survival in aGVHD. Regarding safety, four adverse events were reported, all recovered without sequelae. Thus, analysis of this compassionate use experience shows MSC to be an effective and safe therapeutic option for treating refractory GVHD, resulting in a significant proportion of patients responding to the therapy.
Keyphrases
- end stage renal disease
- newly diagnosed
- ejection fraction
- clinical trial
- chronic kidney disease
- allogeneic hematopoietic stem cell transplantation
- young adults
- prognostic factors
- randomized controlled trial
- stem cells
- peritoneal dialysis
- bone marrow
- liver failure
- staphylococcus aureus
- oxidative stress
- hepatitis b virus
- acute lymphoblastic leukemia
- patient reported
- free survival
- extracorporeal membrane oxygenation
- pseudomonas aeruginosa
- intensive care unit
- cross sectional
- biofilm formation
- umbilical cord
- pi k akt
- combination therapy
- phase ii
- open label