7. First CD3/CD45RA depleted haploidentical transplant for severe combined immunodeficiency disease with Rotavirus gastroenteritis in Vietnam

Tóm tắt

Severe combined immunodeficiency includes inherited diseases with impairment of T cells development, often associated with profound defects of B and/or NK cells differentiation. Rotavirus infection is responsible for severe acute gastroenteritis and over 400,000 deaths a year in children aged < 5 years, especially in low and middle-income countries. We report a nine-month-old girl presented with failure to thrive left axillary lymphadenitis post-BCG-vaccination, persistent, nonvaccine-associated rotavirus gastroenteritis. An immune evaluation revealed absent T lymphocytes, B lymphocytes, normal NK cells with genetic testing confirmed RAG2 severe combined immunodeficiency. Rotavirus gastroenteritis persisted despite the administration of oral immunoglobulin pre-transplantation. The patient was treated by a haploidentical transplant -from mother as a donor using CD3/CD45RA+ depletion kit, CliniMACs system (Miltenyi). The first transplant was performed without conditioning. One month post-transplant, rotavirus was cleared. The second transplant was performed three months later, with myeloablative conditioning (Bu-Flu-rATG). Neutrophils engrafted on day +10 post-transplant, platelet engrafted on day +45. Whole blood chimerism on day +28 post 2nd transplant was 100%. Serum IgG level has been normalized without IVIG infusion three months post 2nd transplant.