Extracorporeal photopheresis with TC-V in Japanese patients with steroid-resistant chronic graft-versus-host disease
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There are few established therapies for chronic graft-versus-host disease (cGVHD) refractory to first-line treatment with steroids. We evaluated the efficacy and safety of extracorporeal photopheresis (ECP) with a third-generation TC-V device in Japanese patients with cGVHD. Fifteen patients with steroid-resistant or -intolerant cGVHD after allogeneic hematopoietic stem cell transplantation participated in this multicenter open-label study. Extracorporeal photopheresis was conducted on days 1–3, week 1; days 1–2, weeks 2–12; and days 1–2, weeks 16, 20, and 24. The composite primary endpoint consisted of evaluation of response and changes in steroid dose 24 weeks after ECP initiation. Secondary endpoints included response over time, concomitant drug dose, quality of life, and safety. Twelve patients completed scheduled ECP therapy; eight (66.7%) showed a response at week 24. In all 15 patients, the mean (± standard deviation) steroid dose decreased 0.115 ± 0.230 mg/kg/day from screening to week 24. Five serious, potentially treatment-related adverse events (heart failure, thrombosis in the device, pneumonia, edema, and wheezing) occurred; none were fatal. This study confirmed that ECP using the TC-V device was effective, with an acceptable toxicity profile. Further studies in larger cohorts are clearly warranted to determine its optimal use in Japanese patients with cGVHD.
KeywordsChronic graft-versus-host disease Extracorporeal photopheresis (ECP) Hematopoietic stem cell transplantation Steroid resistant
The authors thank the doctors, nurses, and medical engineers at each site for their dedicated care and support of the patients.
This study was sponsored by Therakos Inc., a Mallinckrodt Pharmaceuticals company. Vorpal Technologies KK, an independent contract research organization, was solely responsible for the administration of the clinical trial and the verification of the data points. A representative of Mallinckrodt Pharmaceuticals was a member of the steering committee but did not participate in the design of the study. Thomas Chin, of Mallinckrodt Pharmaceuticals, was responsible for the statistical analysis; however, he neither participated in the verification of the data from the investigational sites (performed by Vorpal Technologies KK) nor in the data interpretation or the writing of this manuscript. We thank Vorpal Technologies KK for their role in the conduct of the study. We also thank Gail Flores, PhD, and Brian Bass, both of Bass Global, Inc., for medical writing and editorial assistance with this manuscript, which was financed by Mallinckrodt Pharmaceuticals. Finally, we thank all of the patients who participated in this study.
Compliance with ethical standards
Conflict of interest
The authors declare that they have no conflict of interest.
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