Prospective Study of Second Line Therapy for Acute GVHD(IRAS version1)
Research type
Research Study
Full title
Prospective Outcomes of Second-line Therapy in Acute Graft-versus-Host Study including ECP (POSTAGE)
IRAS ID
149631
Contact name
Emma Das-Gupta
Contact email
Clinicaltrials.gov Identifier
Research summary
Acute graft versus host disease (aGVHD) is a complication of haematopoietic stem cell transplantation (HSCT) that can be associated with significant morbidity and mortality. It is graded using the Modified Glucksberg/ Keystone criteria from 0 (no aGVHD) to IV (severe aGVHD). The transplant related mortality (TRM) for grades 0-IV aGVHD is 28%, 27%, 43%, 68% and 92% respectively. First line therapy of aGVHD is with systemic corticosteroids. However only around 50% of patients with aGVHD will have a complete response (CR) to therapy with steroids. The remainder are classed as having steroid-refractory (SR) aGVHD (unresponsive to steroids) or steroid-dependent (SD) aGVHD (responds to steroids but recurs on steroid withdrawal). These patients are hard to manage and there is no consensus on what second line therapies to give. The BCSH (British Committee for Standards in Haematology) and BSBMT (British Society for Blood and Marrow Transplantation) recommend 5 second line therapies: extracorporeal photopheresis (ECP), anti-tumour necrosis factor (TNF)a antibodies, mammalian target of rapamycin (mTOR) inhibitors, mycophenolate mofetyl (MMF), interleukin 2 (IL2) receptor antibodies. Due to the paucity of published high quality clinical outcome data the BCSH/BSBMT group were not able to make recommendations relating to the efficacy of individual second line therapies and it was accepted that prospective comparative data would be required in the future to inform this section of the guideline. This study aims to contribute to this unmet need. It will also gather the necessary information to act as an audit of compliance with the current BCSH/BSBMT guideline. The study will prospectively collect outcome data on patients receiving second line therapy for aGVHD. The aim will be to compare the effectiveness of the different treatments in order to guide future treatment recommendations and to identify the need for a randomised prospective clinical trial.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
14/EM/1035
Date of REC Opinion
8 Jul 2014
REC opinion
Favourable Opinion