{
  "abstract": "APDS is a rare inborn error of immunity characterized by clinical manifestations, including recurrent infections. This real-world, retrospective analysis evaluated patient-reported infection rates before and after leniolisib initiation. Patients (aged ≥ 12 years) newly initiating leniolisib therapy were eligible for inclusion. Data were retrieved from PANTHERx Rare Pharmacy. Patient-reported infection rates during the baseline period (one year before leniolisib initiation) were compared with infection rates after leniolisib initiation (observation period; from initiation to end of data or death) using generalized estimating equations Poisson regression models with robust standard errors. Patients who had an infection assessment collected via a standardized questionnaire during the baseline and observation periods were included in the analysis (N = 71); subgroups for baseline infection rates (lower and higher than the median) and age (12-17 and ≥ 18 years) were also evaluated. The mean (SD) rate of infections per patient-year (PPY) decreased significantly-by 68%-from 5.8 (5.1) during baseline to 1.9 (2.0) in the observation period (rate ratio [RR], 0.32; 95% CI, 0.24-0.42; P < 0.001); infections PPY decreased by 38% in patients with low baseline infection rates (RR, 0.62; 95% CI, 0.43-0.89; P = 0.010) and 76% in those with high baseline infection rates (RR, 0.24; 95% CI, 0.17-0.34; P < 0.001); infections PPY decreased by 70% in those aged 12 - 17 years (RR, 0.30; 95% CI, 0.20-0.45; P < 0.001) and by 67% in those aged ≥ 18 years (RR, 0.33; 95% CI, 0.23-0.49; P < 0.001). Real-world evidence demonstrated that leniolisib treatment provided a significant reduction in infection rates overall and among subgroups.",
  "abstract_shown": true,
  "affiliations": [
    "Division of Pediatric Allergy and Immunology, Duke University, Durham, NC, US.",
    "PANTHERx Rare Pharmacy, Pittsburgh, PA, US.",
    "Groupe d'analyse SRI, Montréal, QC, Canada.",
    "Division of Allergy, Immunology and Rheumatology, Johns Hopkins University, Baltimore, MD, US.",
    "Consultant for Pharming Healthcare, Inc, Warren, NJ, US.",
    "Pharming Healthcare, Inc, Warren, NJ, US. A.Harrington@pharming.com."
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 9,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Niraj C Patel"
    },
    {
      "affs": [
        1
      ],
      "name": "Jessica Kuivinen"
    },
    {
      "affs": [
        2
      ],
      "name": "Sean D MacKnight"
    },
    {
      "affs": [
        3
      ],
      "name": "M Elizabeth M Younger"
    },
    {
      "affs": [
        2
      ],
      "name": "Guillaume Germain"
    },
    {
      "affs": [
        2
      ],
      "name": "Susannah Ripley"
    },
    {
      "affs": [
        2
      ],
      "name": "François Laliberté"
    },
    {
      "affs": [
        4
      ],
      "name": "Jesse Fishman"
    },
    {
      "affs": [
        5
      ],
      "name": "Amanda Harrington",
      "orcid": "0009-0001-8294-5002"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/bqrkdyr30v2tj.bib",
  "cited_by": 0,
  "doi": "10.1007/s12026-026-09863-2",
  "fulltext_url": "https://europepmc.org/articles/PMC13695588",
  "id": "bqrkdyr30v2tj",
  "issns": [
    "0257-277X",
    "1559-0755"
  ],
  "landing_url": "https://europepmc.org/article/MED/42925385",
  "language": "eng",
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "openalex",
      "2403|Immunodeficiency and Autoimmune Disorders"
    ]
  ],
  "openalex": "W7223448818",
  "pdf_url": "https://europepmc.org/articles/PMC13695588?pdf=render",
  "pmcid": "PMC13695588",
  "pmid": "42925385",
  "published": "2026-10-24",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/bqrkdyr30v2tj.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:20:33Z",
      "native_id": "MED:42925385",
      "source": "europepmc",
      "url": "https://europepmc.org/article/MED/42925385"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "openalex:2403|Immunodeficiency and Autoimmune Disorders",
      "subfield": "immunology"
    }
  ],
  "title": "Effectiveness of leniolisib in reducing infections among patients with activated phosphoinositide 3-kinase δ syndrome (APDS).",
  "title_text": "Effectiveness of leniolisib in reducing infections among patients with activated phosphoinositide 3-kinase δ syndrome (APDS).",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:20:33Z",
  "url": "https://research.draconitic.com/paper/bqrkdyr30v2tj",
  "venue": "Immunologic research",
  "versions": []
}