Steroid Refractory Chronic Graft-Versus-Host Disease: Cost-Effectiveness Analysis

• Published in: Biology of blood and marrow transplantation 2018-09, Vol.24 (9), p.1920-1927
• Main Authors: Yalniz, Fevzi F., Murad, Mohammad H., Lee, Stephanie J., Pavletic, Steven Z., Khera, Nandita, Shah, Nilay D., Hashmi, Shahrukh K.
• Format: Article
• Language: English
• Subjects: Allogeneic; Chronic Disease; Cost; Cost-Benefit Analysis - trends; Female; Graft vs Host Disease - economics; GVHD; Health Care Costs; Humans; Male; Survival; Transplant
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2018.03.008

•cGVHD remains a serious complication of cell transplantation.•Available options on SR-cGVHD vary in both cost and effectiveness.•Attention to economic issues when treating SR-cGVHD can help guide how treatments should be sequenced. Given the increasing incidence of chronic graft-versus-host disease (cGVHD) and its rapidly escalating costs due to many lines of drug treatments, we aimed to perform a meta-analysis to assess the comparative effectiveness of various treatment options. Using these results, we then conducted a cost-effectiveness analysis for the frequently utilized agents in steroid-refractory cGVHD. We searched for studies examining tacrolimus, sirolimus, rituximab, ruxolitinib, hydroxychloroquine, imatinib, bortezomib, ibrutinib, extracorporeal photopheresis, pomalidomide, and methotrexate. Studies with a median follow-up period shorter than 6 months and enrolling fewer than 5 patients were excluded. Meta-analysis for overall and organ system–specific GVHD response (overall response [ORR], complete response [CR], and partial response [PR]) was conducted for each intervention. Cost per CR and cost per CR + PR were calculated as the quotient of the 6-month direct treatment cost by CR and CR + PR. Forty-one studies involving 1047 patients were included. CR rates ranged from 7% to 30% with rituximab and methotrexate, respectively, and ORR ranged from 30% to 85% with tacrolimus and ruxolitinib, respectively. Cost per CR ranged from US$1,187,657 with ruxolitinib to US$680 with methotrexate. Cost per ORR ranged from US$453 for methotrexate to US$242,236 for ibrutinib. The most cost-effective strategy was methotrexate for all of the organ systems. Pomalidomide was found to be the least cost-effective treatment for eye, gastrointestinal, fascia/joint, skin, and oral GVHD, and imatinib was found to be the least cost-effective treatment for liver and extracorporeal photopheresis for lung GVHD. We observed huge cost-effectiveness differences among available agents. Attention to economic issues when treating cGVHD is important to recommend how treatments should be sequenced, knowing that many patients will cycle through available agents.