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Blood, 1 October 2008, Vol. 112, No. 7, pp. 2667-2674.
Prepublished online as a Blood First Edition Paper on July 11, 2008; DOI 10.1182/blood-2008-03-141481.


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CLINICAL TRIALS AND OBSERVATIONS

A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease

Mary E. D. Flowers1,*, Jane F. Apperley2, Koen van Besien3, Ahmet Elmaagacli4, Andrew Grigg5, Vijay Reddy6, Andrea Bacigalupo7, Hans-Jochem Kolb8, Luis Bouzas9, Mauricette Michallet10, H. Miles Prince11, Robert Knobler12, Dennis Parenti13, Jose Gallo13, and Hildegard T. Greinix14,*

1 Division of Clinical Research, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle; 2 Division of Investigative Science, Royal Hammersmith Hospital, Imperial College of London, London, United Kingdom; 3 Section of Hematology/Oncology, University of Chicago Pritzker School of Medicine, IL; 4 Klinik und Poliklinik fur Knochenmarktransplantation, Universitäts-Klinikum Essen, Essen, Germany; 5 Clinical Haematology and Medical Oncology, Royal Melbourne Hospital, Parkville, Australia; 6 Division of Hematology Oncology, University of Florida, Gainesville; 7 Ospedale S Martino, Genova, Italy; 8 University of Munich, Munich, Germany; 9 National Cancer Institute (CEMO), Rio de Janeiro, Brazil; 10 University of Lyon, Lyon, France; 11 Peter MacCallum Cancer Centre, Melbourne, Australia; 12 Department of Dermatology, Medical University of Vienna, Vienna, Austria; 13 Therakos, Exton, PA; and 14 Department of Internal Medicine I, Bone Marrow Transplant Unit, Medical University of Vienna, Vienna, Austria

Chronic graft-versus-host disease (cGVHD) is a major limitation of successful hematopoietic cell transplantation. The safety and efficacy of extracorporeal photopheresis (ECP) for 12 to 24 weeks together with standard therapy was compared with standard therapy alone in patients with cutaneous manifestations of cGVHD that could not be adequately controlled by corticosteroid treatment. The primary efficacy end point was a blinded quantitative comparison of percent change from baseline in Total Skin Score (TSS) of 10 body regions at week 12. Ninety-five patients were randomized to either ECP and standard therapy (n = 48) or standard therapy alone (n = 47). The median percentage improvement in TSS at week 12 was 14.5% for the ECP arm and 8.5% for the control arm (P = .48). The proportion of patients who had at least a 50% reduction in steroid dose and at least a 25% decrease from baseline in TSS was 8.3% in the ECP arm at week 12 and 0% in the control arm (P = .04). The nonblinded investigator assessment of skin complete or partial responses revealed a significant improvement in favor of ECP (P < .001). ECP was generally well tolerated. These results suggest that ECP may have a steroid-sparing effect in the treatment of cGVHD. Clinical trials registered at www.ClinicalTrials.gov as NCT00054613 [ClinicalTrials.gov] .


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Shedding some LIGHT on chronic GVHD
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