Combined JAK- and TNFα-targeting in severe steroid-refractory graft-versus-host disease in children

Steroid-refractory graft-versus-host disease (SR-GvHD) after hematopoietic stem cell transplantation (HSCT) remains a major cause of morbidity and mortality [ 1 , 2 ]. The REACH trials demonstrated a better overall response and a better failure free survival in patients treated with the JAK inhibitor ruxolitinib for acute and chronic SR-GvHD compared to standard of care [ 3 , 4 , 5 ]. Of the 16 pediatric patients with higher grades acute GvHD (aGvHD) III–IV however, only 68.8% showed a response on day 28 while almost half of patients failed to maintain a durable response (ORR day 56, 56.3%) [ 5 ]. Other treatment options or combinations are needed for these patients.