In a phase II clinical trial, Martinez et al (2009) examined the safety and effectiveness of alemtuzumab in treating steroid-refractory acute GVHD (aGVHD) grade II or higher after stem cell transplantation. A total of 10 adult patients (6 with aGVHD grade III and 4 with aGVHD grade IV) were included in the study. Nine patients had gastrointestinal tract involvement, 7 had skin involvement, and 5 had liver involvement. Five patients responded to treatment, 2 with CR and 3 with partial response. Eight infectious events (4 of grade 3 to 4) and 7 CMV re-activations were observed. Six patients had grade 3 to 4 cytopenia. All 10 patients died (7 resulting from aGVHD progression, 2 from severe infection, and 1 from to leukemia relapse), at a median of 40 days (range of 4 to 88 days) after alemtuzumab treatment. Overall, these findings suggested that steroid-refractory aGVHD may be improved by treatment with alemtuzumab, but that this treatment does not overcome the dismal prognosis of patients with severe aGVHD, demonstrating the need for alternative therapies to treat this complication.
Ruxolitinib therapy to treat corticosteroid-refractory acute and chronic graft-versus-host disease (GVHD) in transplant recipients can achieve response rates >80%, according to a multi-center study of 95 patients treated with this JAK1/2 inhibitor. In this retrospective study, 54 patients had grades III or IV acute GVHD and 41 patients had moderate or severe chronic GVHD. In each group, the median number of previous therapies to treat GVHD was 3. The overall response rate was % in acute GVHD patients, including 25 complete responses (%). In chronic GVHD patients, the overall response rate was %. In patients responding to ruxolitinib, the rate of GVHD-relapse was % and % for acute GVHD and chronic GVHD, respectively.