Article
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety
Leniolisib, a PI3Kδ inhibitor, improves the pathophysiology of APDS. This publication demonstrates the safety and efficacy of leniolisib in patients with APDS ≥12 years of age receiving up to 7 years of treatment, illustrating a positive tolerability profile and sustained disease burden reduction.
Research Letter
Hematopoietic cell transplantation using treosulfan-based conditioning for IL-10 receptor deficiency
Lakkaraja and Schmitt et al. observe that IL-10 receptor deficiency is a life-threatening inborn error of immunity associated with early-onset inflammatory bowel disease and found that employing hematopoietic cell transplantation (HCT) using treosulfan-based conditioning results in excellent survival with full donor engraftment. Importantly, anakinra effectively serves as a bridge to HCT.
