Ruxolitinib for treating chronic graft versus host disease after allogeneic stem cell transplant in paediatric patients

Ruxolitinib is in clinical development for the treatment of chronic graft versus host disease (cGvHD), after allogeneic stem cell transplant (AlloSCT) in children. AlloSCT is a type of haematopoietic cell transplant (HCT), in which stem cells from a healthy donor are transplanted to the patient to replace stem cells that were destroyed during chemotherapy treatment. Chronic graft versus host disease (cGvHD), is a frequent complication of AlloSCT, where the donor’s T-cells (white blood cells) fight the patient’s organs and tissue, reducing their ability to carry out their normal function. This occurs 100 days after AlloSCT. The most frequently affected areas are the skin, mouth, lungs, and eyes. Treatment for cGvHD can be very complex and requires a combination of therapies. Corticosteroids remain the first-line treatment for chronic GvHD.