Skip to main content
medichelpline
Back to Clinical Feed
Try:
Front ImmunolInfectious Disease

Evaluating the impact of leniolisib treatment on symptoms and health-related quality of life in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome

07 May 20264 min read0 viewsJournal Feed

GIST (Key Takeaways)

  • PurposeActivated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) is an ultra-rare inborn error of immunity, characterised by immune deficiency and dysregulation. In a randomised controlled trial (RCT; NCT02435173) and open-label extension (OLE; NCT02859727), leniolisib, a selective PI3Kδ inhibitor, was efficacious and well-tolerated in individuals with APDS. These trials suggested some improvements with leniolisib in health-related quality of life (HRQoL) using generic instruments, with other anecdotal evidence also describing improvements in specific domains.
  • Here, all available qualitative data relating to treatment experience were systematically assessed to evaluate any perceived impacts of leniolisib on patients’ lives. Methods. Changes in APDS-related symptoms and patient HRQoL were assessed using unsolicited qualitative data captured from 36 leniolisib-treated individuals: RCT and OLE clinician-recorded, open-text patient narratives (n=31); case reports (n=4); standalone qualitative study conducted with APDS patient/caregiver interviews/narratives (n=1).Results.
  • Improvements in APDS-related symptoms and HRQoL were reported following treatment with leniolisib: 86.1% (31/36) of individuals mentioned improvements in ≥1 symptom/HRQoL impact. Of these, 87.1% (27/31) explicitly attributed ≥1 improvement to leniolisib. One-third (12/36) of individuals explicitly attributed improvements in fatigue/energy to leniolisib.

Clinical Editorial

Summary

Frontiers in Immunology published a clinical update in Infectious Disease on 07 May 2026.

The item focuses on Evaluating the impact of leniolisib treatment on symptoms and health-related quality of life in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome.

Review the original article for the full source wording and details.

Source Reference

Read the full original publication from the source journal or publisher link below.