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

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.MethodsChanges 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).ResultsImprovements 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.