A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease.
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.
Flowers
Mary E D
ME
Division of Clinical Research, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, USA. mflowers@fhcrc.org
Apperley
Jane F
JF
van Besien
Koen
K
Elmaagacli
Ahmet
A
Grigg
Andrew
A
Reddy
Vijay
V
Bacigalupo
Andrea
A
Kolb
Hans-Jochem
HJ
Bouzas
Luis
L
Michallet
Mauricette
M
Prince
H Miles
HM
Knobler
Robert
R
Parenti
Dennis
D
Gallo
Jose
J
Greinix
Hildegard T
HT
D000293:Adolescent; D000328:Adult; D000368:Aged; D002908:Chronic Disease; D003710:Demography; D005260:Female; D005712:Galvanic Skin Response; D006086:Graft vs Host Disease / Q000401:mortality / Q000503:physiopathology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008875:Middle Aged; D009026:Mortality; D017893:Photopheresis / Q000009:adverse effects / Q000379:methods*; D011446:Prospective Studies; D011788:Quality of Life; D013256:Steroids / Q000627:therapeutic use; D013997:Time Factors; D014481:United States / Q000453:epidemiology
D013256:Steroids