Current APDS management is mainly empirical. Treatment burden includes immunomodulatory therapies, prophylactic antimicrobials, immunoglobulin replacement therapy (IRT), and procedures such as splenectomies, repeated otosinopulmonary surgeries, and hematopoietic stem cell transplantation. Most strategies do not target the disease pathogenesis, hyperactive PI3Kd signaling.5-7
Leniolisib, a novel, orally bioavailable small molecule inhibitor, was engineered to selectively target PI3Kd signaling.24 We previously reported leniolisib use in 6 patients with APDS in a 12-week dose-finding clinical trial. Leniolisib was well tolerated, reduced PI3Kd pathway hyperactivity, partially reconstituted lymphocyte subsets, and decreased lymphoproliferation.25
Here we report outcomes from a phase 3 trial (NCT02435173), a 12-week, randomized, triple-blinded, placebo-controlled, fixed-dose study of 31 patients.
Kortom....alle studies hebben goede resultaten laten zien...kwestie van tijd mbt goedkeuring in Europa, Japan en UK