Alpha-1 antitrypsin for treating high risk, acute graft versus host disease in people aged ≥ 12 years

Alpha-1 antitrypsin is currently in development for the treatment of high risk, acute graft versus host disease (aGvHD) in people aged ≥ 12 years following an allogeneic haematopoietic stem cell transplant (HSCT) for any indication. A stem cell transplant replaces damaged blood cells with healthy ones from another person (a donor). GvHD is a frequent complication of allogenic stem cell transplantation and involves a reaction between the donor cells and the recipient's native tissues, leading to injury of the recipient's tissues. GvHD occurs in acute and chronic form. aGVHD usually manifests within 100 days following HSCT whereas chronic GVHD generally manifests later (>100 days). Systemic corticosteroids are used as a first-line treatment for aGvHD, however, roughly half of patients become refractory to this treatment, meaning that the disease progresses or there is no response after treatment. As the number of patients undergoing allogenic HSCT increases, developing safe and effective treatments for aGvHD is increasingly important, especially for those whose disease becomes refractory to systemic steroid therapy.