Key outcomes in treatment of activated phosphoinositide 3-kinase delta syndrome: An e-Delphi panel study and responder threshold application
This study provides expert consensus on outcomes important in assessing APDS treatment effectiveness and improvement thresholds in 6 treatment outcomes indicative of a clinically meaningful benefit. These outcomes may help optimize APDS treatment in the clinic.
Authors
- João Bana e Costa (ORCID: https://orcid.org/0009-0009-3595-1571)
- Ana Sofia Santos (ORCID: https://orcid.org/0000-0001-8824-228X)
- Chad Gwaltney (ORCID: https://orcid.org/0000-0001-8130-2024)
- Jason Bradt
- Julia Upton (ORCID: https://orcid.org/0000-0001-5320-4232)
- Kelli W. Williams (ORCID: https://orcid.org/0000-0003-2318-6023)
- Andrew J. Cant
- Amanda Harrington
Institutions
- University of Lisbon (PT)
- Medical University of South Carolina (US)
- University of Toronto (CA)
- Universidade Politécnica de Lisboa (PT)
- Hospital for Sick Children (CA)
- Westerly Hospital (US)
- Lisbon School of Design (PT)
- Michigan Surgical Hospital (US)
- Newcastle University (GB)
Publication Details
- Journal
- PLoS ONE
- Published
- 2025-10-15
- DOI
- https://doi.org/10.1371/journal.pone.0333341
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00