Trial reportFrontiers in immunology2026
Evaluating the impact of leniolisib treatment on symptoms and health-related quality of life in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome.
Trial report in Frontiers in immunology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor Blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 (Leniolisib) in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/ p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)
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Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: Activated 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. This was associated with reported HRQoL improvements explicitly attributed to leniolisib: physical activity (33.3% [12/36]), work/school performance/attendance (13.9% [5/36]) and travel ability (8.3% [3/36]). Moreover, 36.1% (13/36) of participants explicitly attributed improvements in their overall wellbeing to leniolisib. Conclusion: As data were unsolicited and baseline symptoms/HRQoL were not available, results may not represent all changes experienced, and is not possible to determine the amount of change in symptoms/HRQoL associated with leniolisib. Nevertheless, these analyses indicate that participants with APDS experienced improvements in symptoms and overall wellbeing following treatment with leniolisib.
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Registered trials
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