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.