Ruxolitinib as Salvage Therapy for Chronic Graft-versus-Host Disease

Autor: Michael Hernandez-Henderson, Dongyun Yang, Ryotaro Nakamura, Pablo Parker, Monzr M. Al Malki, Haris Ali, Ricardo Spielberger, Jonathan Cotliar, Stephen J. Forman, Jasmine Zain, Erin Kopp, Sally Mokhtari, Karen Huelsman, Badri Modi, Sanjeet Dadwal, Amandeep Salhotra, Jeremy Klein
Rok vydání: 2018
Předmět:
Zdroj: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 25(2)
ISSN: 1523-6536
Popis: Chronic graft-versus-host disease (cGVHD) continues to be a major complication after allogeneic hematopoietic cell transplantation, significantly affecting patients' quality of life. A regimen of systemic corticosteroids is considered first-line therapy but is often associated with inadequate responses and multiple side effects. In patients with refractory disease, an evidenced-based consensus is lacking as to the single best approach to managing symptoms. Ruxolitinib, a selective JAK1/2 inhibitor, has recently gained favor as a second-line approach in patients with steroid-refractory cGVHD. In this retrospective study, we evaluated the outcomes of 46 patients who received ruxolitinib for cGVHD between March 2016 and December 2017 at our institution, and evaluated ruxolitinib's impact at 6 and 12 months, based on the National Institutes of Health Severity Scale, including organ-specific responses, and mean prednisone dose. Furthermore, we present the first reported probability of ruxolitinib's treatment failure-free survival (FFS) in patients with cGVHD. After 12 months of ruxolitinib therapy, complete response, partial response, and stable disease was observed in 13% (n = 6), 30.4% (n = 14), and 10.9% (n = 5) of patients, respectively. The 1-year probability of FFS was 54.2% (95% confidence interval, .388 to .673), and ruxolitinib use was associated with a reduction in prednisone dose. In conclusion, our data, which represent the largest cohort of patients with cGVHD reported to date, support the use of ruxolitinib for cGVHD refractory to steroids and currently available salvage therapies, discontinued due to lack of response and high cost.
Databáze: OpenAIRE