Precision Medicine in Pediatric Autoimmunity: Leniolisib Treatment of Childhood‐Onset Lupus Nephritis Due to Activated Phosphoinositide 3‐Kinase δ Syndrome
This investigation advances rheumatology by demonstrating that APDS1-associated LN displays a specific tissue immune signature and responds to targeted inhibition of the causative molecular pathway. Our findings provide mechanistic insights into genetic drivers of autoimmunity and support pathway-specific therapeutic approaches for refractory autoimmune manifestations in primary immunodeficiencies.
Authors
- Tusharkanti Ghosh (ORCID: https://orcid.org/0000-0002-7537-6374)
- Elena W.Y. Hsieh (ORCID: https://orcid.org/0000-0003-3969-6597)
- Heng Du (ORCID: https://orcid.org/0000-0001-9067-0910)
- Kimberly Rae Jordan (ORCID: https://orcid.org/0000-0001-8380-7157)
- Jason P. Weinman (ORCID: https://orcid.org/0000-0001-6540-3757)
- Nicholas Willard (ORCID: https://orcid.org/0000-0003-4912-201X)
- Angela Minic (ORCID: https://orcid.org/0000-0001-5395-0032)
- Mansooreh Ahmadian (ORCID: https://orcid.org/0000-0002-5020-3979)
- Clara Lin (ORCID: https://orcid.org/0000-0002-5840-875X)
- Fuyong Xing (ORCID: https://orcid.org/0000-0003-0982-8675)
- Christian Rickert (ORCID: https://orcid.org/0000-0001-7396-4335)
- Jonathan T. Lim (ORCID: https://orcid.org/0009-0007-9139-8841)
- Vijaya Knight (ORCID: https://orcid.org/0000-0002-7514-0097)
- Jennifer C. Cooper
- Debashis Ghosh (ORCID: https://orcid.org/0000-0001-6618-1316)
- Sara A. Johnson (ORCID: https://orcid.org/0009-0009-4696-6955)
- Melisha Hanna
Institutions
- Colorado School of Public Health (US)
- Children's Hospital Colorado (US)
- University of Colorado Anschutz Medical Campus (US)
- University of Colorado Denver (US)
Publication Details
- Journal
- Arthritis & Rheumatology
- Published
- 2025-05-19
- DOI
- https://doi.org/10.1002/art.43254
- Citations
- 1
- Primary Topic
- Systemic Lupus Erythematosus Research
- Type
- article
- Field-Weighted Citation Impact
- 1.67