{"id":"W4409785988","doi":"10.70962/cis2025abstract.6","title":"Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment with PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4–11 Years with Activated PI3Kδ Syndrome (APDS)","year":2025,"lang":"en","type":"article","venue":"Journal of Human Immunity","topic":"Immunodeficiency and Autoimmune Disorders","field":"Immunology and Microbiology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children","funders":"","keywords":"Open label; Medicine; Pediatrics; Internal medicine; Adverse effect","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002847865,0.001649597,0.001962586,0.0004355051,0.0006276482,0.0007403158,0.0006469132,0.001001496,0.002876805],"category_scores_gemma":[0.001531965,0.0005398641,0.001235297,0.0003496445,0.0008021306,0.0009422869,0.000373718,0.002639222,0.0006151244],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007355388,"about_ca_system_score_gemma":0.001680932,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005444358,"about_ca_topic_score_gemma":0.001399375,"domain_scores_codex":[0.9990383,0.0004717041,0.00007198588,0.0001577564,0.0001164596,0.0001438146],"domain_scores_gemma":[0.9987043,0.0002459023,0.0003724511,0.0001071697,0.0001207681,0.0004494672],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.8389863,0.06530833,0.02854004,0.0005562338,0.001579076,0.0003886906,0.0002406101,0.001862126,0.02337585,0.0001332682,0.003052764,0.03597672],"study_design_scores_gemma":[0.347596,0.5530264,0.08844678,0.00004768708,0.0007282117,0.00034195,0.00008608021,0.001423105,0.005500871,0.0001532631,0.002595878,0.00005379267],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9905253,0.001037178,0.0004519444,0.0001549919,0.00003924683,0.003652574,0.002727194,0.00004626511,0.001365408],"genre_scores_gemma":[0.9883687,0.0006728852,0.001280632,0.0004873441,0.0001157529,0.004225533,0.003252857,0.00002097898,0.001575325],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002876805,"threshold_uncertainty_score":0.01506114,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02222860301462419,"score_gpt":0.2734438837276909,"score_spread":0.2512152807130666,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}