Compassionate use of ruxolitinib in acute and chronic graft versus host disease refractory both to corticosteroids and extracorporeal photopheresis

BACKGROUND: Ruxolitinib is a potent inhibitor of JAK1/2 with proven efficacy in myelofibrosis. In recent years, research in graft versus host disease (GVHD) has revealed the role of activation of JAK pathways in alloreactive lymphocytes. Some reports have shown significant responses in refractory GVHD patients. CASES PRESENTATION: In this report we present our experience in 8 patients with acute or chronic GVHD with refractoriness to steroids and extracorporeal photopheresis treated with ruxolitinib. Three patients had acute GVHD (1 pulmonary, 2 cutaneous, 1 multi-systemic) and 5 had chronic GVHD (3 cutaneous); 85% obtained an overall response and 50% a complete response with a tolerable toxicity profile. CONCLUSIONS: In our series, Ruxolitinib was very active as a rescue therapy for patients with acute or chronic GVHD refractory to standard treatment.