Granulostim (filgrastim biosimilar) / UCB - LARVOL DELTA

[VIRTUAL] EARLY DETECTION AND STEM CELL TRANSPLANTATION OF A RARE IMMUNODEFICIENCY (ASPHO 2021) - "Flow cytometry was remarkable for decreased CD3+ T cells (305 cells/microL), CD19+ B cells (398 cells/microL), and naïve CD45RA+ T cells (18%, normal 64 -95%), and severe neutropenia (0.3x109cells/L) refractory to filgrastim...At age 3 months, the patient underwent 5/6-matched UCB SCT with alemtuzumab, fludarabine, and melphalan conditioning. SCT was complicated by sinusoidal obstructive syndrome, treated with defibrotide, and grade 2 acute graft-versus-host disease of her skin, steroid-responsive... Phenotypic variability in patients with PMG3 deficiency can include isolated neutropenia and immunodeficiency. TREC screening allows prompt identification of newborns with rare immunodeficiencies associated with T cell lymphopenia. Reduced-intensity UCB SCT can achieve an excellent outcome in PGM3 deficiency, with resolution of neutropenia and improvement in immune status."

IO biomarker • Aplastic Anemia • Dermatology • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Neutropenia • Primary Immunodeficiency • Transplantation • CD19