Authors' objectives: Remestemcel-L (Prochymal) is intended to be used as second line therapy for the treatment of steroid refractory acute graft versus host disease (GVHD). If licensed, it would offer a novel additional intravenous treatment option for this patient group, who currently have few effective therapies available. Remestemcel-L is a cell therapy product containing human mesenchymal stem cells that are involved in tissue repair through the coordinated release of tissue specific growth factors. Remestemcel-L does not currently have Marketing Authorisation in the EU for any indication. It is estimated that between 20% and 80% of patients undergoing an allogeneic haematopoietic stem cell transplant will develop some form of GVHD. The incidence of acute GVHD varies widely, ranging from 10-80% depending on risk factors such as mismatched donors and older age. Morbidity and mortality rates continue to rise above 70% in steroid refractory patients with acute GVHD. Management of GVHD is largely focused on prevention through immunosuppression of donor T-cells with immunomodulatory agents or through depletion of T-cells before or after transplant, using monoclonal or polyclonal antibodies. Generally, immunosuppression is initiated using methotrexate, followed by ciclosporin or tacrolimus post-transplant. Corticosteroid treatment is recommended in patients with acute GVHD, however this is effective in less than 50%. Remestemcel-L has completed two phase III clinical trials comparing its effect on complete response against treatment with placebo.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/remestemcel-l-prochymal-for-steroid-refractory-acute-graft-versus-host-disease-second-line/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)