INTRODUCTION:
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy for certain primary immunodeficiencies. Acute graft-versus-host disease (aGvHD) is a serious and potentially life-threatening complication of HSCT that can occur in about half of the patients, with lower gastrointestinal (LGI) tract involvement in about 30%. First-line treatment is generally consisting of high-dose corticosteroids but nearly %30 of patients is unresponsive.1, 2 Steroid refractoriness is associated with poor prognosis and unfortunately, there isn’t consensus about second-line therapies for steroid-refractory (SR) aGvHD.3
Alpha 1 Antitrypsin (AAT) is a novel experimental therapy as a second-line treatment.3 In murine models, it’s shown that AAT can suppress GvHD development and provide an alternative treatment for existing GvHD.4,5 In literature, limited number of studies6-10 are available. There are also recruiting clinical trials both for prevention (NCT03805789) and primary treatment (NCT04167514) of GvHD. However, only one clinical trial (NCT03805789) has participants aged >12 years and there is no published data about its use in children. In this study, we aimed to report our clinical experience with AAT treatment in the pediatric population.