Janssen&Janssen schreef op 9 oktober 2023 01:33:
Nog meer goed nieuws, maar dan van de andere kant van de wereld
We present the case of a 26-year-old with severe APDS1 who is the first patient in Australasia to be treated with Leniolisib, a small molecule inhibitor of the p110d catalytic subunit of PI3K.
Results: Our patient demonstrated significant clinical improvement with reduced infections, improvement of lung function and control of her lymphoproliferation. Laboratory data supported correction of B cell dysfunction with improvement of her dysregulated B cell subsets and dysgammaglobulinaema, and of T cell activation exhaustion.
Conclusion: This case demonstrates the efficacy of precision medicine in controlling the clinical features of APDS1 including its effects on lung function and demonstrates the immune mechanisms that may underly the response to therapy.
onlinelibrary.wiley.com/doi/10.1111/i...