Jakafi for GVHD – pro
Patients with acute graft-versus-host disease (aGVHD) that is resistant to steroid therapy have few treatment options. Ruxolitinib, a selective inhibitor of Janus kinase (JAK) 1/2, suppresses a variety of pro-inflammatory cytokines, several of which might contribute to the severity of aGVHD.
To my knowledge only one report of its use in 6 patients had been published by Spoerl et al. Ruxolitinib (5 mg twice daily, increasing to 10 mg twice daily after 3 days) promoted complete resolution of intestinal GVHD in two patients and normalized serum bilirubin levels in one patient with liver GVHD. It also decreased the involved skin area from 50% to <25% and enabled a reduction in steroid dose in all patients with cutaneous GVHD. In all patients studied, ruxolitinib decreased levels of IL-6 and soluble IL-2 receptor. Treatment-related adverse effects such as thrombocytopenia and anemia were absent.
Although this report encompasses only six patients, it is an important guide to future research, because this drug was effective. Second, ruxolitinib represents a novel therapeutic approach and proved effective in patients who had failed with a number of other agents. Third, hematologic toxicity was not observed, an important consideration in post-transplant patients. Farther research is necessary before it can routinely b eused.
Spoerl S et al. Activity of therapeutic JAK 1/2 blockade in graft-versus-host disease. Blood 2014 Jun 12; 123:3832.
Garnett, Jane F. Apperley, and Jiří Pavlůc, Treatment and management of graft-versus-host disease: improving response and survival. Ther Adv Hematol. 2013 Dec; 4(6): 366–378.Catherine