Associations Between Short-Term Changes in Disease Activity and Long-Term Clinical Outcomes in Activated Phosphoinositide 3-Kinase Delta Syndrome.
Leniolisib treatment quickly improved immune function in APDS patients, and these early improvements predicted better long-term outcomes including fewer infections.
This PubMed record reports a randomized controlled trial on Associations Between Short-Term Changes in Disease Activity and Long-Term Clinical Outcomes in Activated Phosphoinositide 3-Kinase Delta Syndrome. The authors report that in this clinical trial setting, short-term improvements in immunodeficiency and dysregulation following treatment with leniolisib were associated with longer-term improvements in clinical outcomes, including infection rates and quality of life, for patients with APDS.
What the study was
- Study design
- Randomized controlled trial
- Population
- Human participants or clinical records described in the abstract
- Category
- Genomics/Precision Medicine
- Maturity
- Validated
- Journal
- Journal of immunology research
Why it surfaced
Randomized controlled trial with direct human relevance was scored conservatively from PubMed abstract and citation metadata.
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