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Stem Cell Transplantation The Use of Extracorporeal Photopheresis for the Treatment of Acute Graft Versus Host Disease Emma Das-Gupta Consultant Haematologist, Centre for Clinical Haematology, Nottingham University Hospitals NHS Trust, Nottingham, UK Abstract Acute graft versus host disease (aGVHD) is a key complication of haematopoietic stem-cell transplantation. The main first-line therapy is steroids, however less than half of patients respond to this treatment, and some patients relapse following an initial response. Extracorporeal photopheresis (ECP) is a therapy that has shown promising efficacy in several immune-mediated disorders, including aGVHD. Used as a second-line treatment, ECP has been associated with good rates of disease resolution, both overall and for individual organ systems involved in this disorder. The responses to ECP have commonly been seen soon after the start of treatment. Patients who respond to ECP have also been shown to have lower rates of transplant-related mortality and greater overall survival than those who do not respond. ECP is well tolerated by patients, including those with a low performance index and children of low body weight. Data on the use of ECP in treating aGVHD in both adults and children are reviewed here. Keywords Acute graft versus host disease (aGVHD), extracorporeal photopheresis, adult, paediatric, complete resolution, transplant-related mortality, survival, tolerability, safety Disclosure: Speaker at an aGVHD meeting in Paris, January 2011, organised by Therakos. Acknowledgements: Writing and editorial assistance was funded by Therakos and provided by Anagram Communications Limited. Received: 7 September 2012 Accepted: 28 September 2012 Citation: European Oncology & Haematology, 2012;8(4):249–53 Correspondence: Emma Das-Gupta, Centre for Clinical Haematology, Nottingham University Hospitals, City Hospital Campus, Hucknall Road, Nottingham, NG5 1PB, UK. E: Support: Educational grant from Therakos to attend ASH, December 2009. Graft versus host disease (GVHD) is a major complication of haematopoietic stem-cell transplantation (HSCT). Historically, GVHD occurring within the first 100 days post HSCT was classified as acute GVHD (aGVHD). More recently, the National Institutes of Health (NIH) consensus criteria for clinical trials proposed that clinical manifestations rather than time after transplantation should be used to distinguish acute from chronic GVHD and introduced the term late acute GVHD, which includes persistent, recurrent, or late-onset aGVHD. 1 Clinical manifestations of aGVHD involve the skin, gastrointestinal tract and liver. 2 The incidence of aGVHD is reported to range from 10 to 80 %, depending on risk factors such as the use of unrelated donors, total body irradiation and transplant conditioning regimen. 3 Acute GVHD is, in turn, a strong risk factor for transplant-related morbidity and mortality (TRM), because of its own pathology and that associated with the immunosuppressive therapies generally used for its treatment. It is also a risk factor for the development of chronic GVHD. Corticosteroids are the main first-line therapy for aGVHD, however these are associated with a response in less than 50 % of patients, 4 and some patients who initially respond subsequently relapse. 5 Response after short-term treatment with prednisolone (five days or 28 days) was significantly associated with a lower TRM than non-response. 6,7 New therapies are therefore being investigated that can © TOUCH MEDICAL MEDIA 2012 quickly and effectively treat aGVHD, and prevent its relapse. One such therapy is extracorporeal photopheresis (ECP). ECP is a therapy that was originally developed over 25 years ago to treat cutaneous T-cell lymphoma. 8 Since that time, its use has been investigated and shown promising efficacy in many other disorders with an auto- or alloimmune aetiology, including GVHD, systemic sclerosis, prevention and treatment of rejection in solid organ transplantation and Crohn’s disease. 9 The treatment has three stages: first leukapheresis, following which the separated white blood cells are photoactivated with 8-methoxypsoralen and exposure to ultraviolet-A light, then return of the activated white blood cells to the patient. ECP has been found to be generally well tolerated, with a good safety profile, in patients with a variety of diseases. 10 This paper reviews studies with ECP in patients with aGVHD. Early Data Much of the early information on the use of ECP in aGVHD was recorded in case studies and series, and small studies (see Table 1). 11–21 The data on 76 patients (age range 5–55 years) from these early studies, all of whom were receiving ECP as second-line treatment for aGVHD, were collated and reviewed in 2002. 11 Overall patient survival was 53 %. ECP was associated with complete resolution (CR) of skin manifestations of aGVHD in 67 % of patients. Rates of CR for liver and gastrointestinal 249