{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0005362076,0.0003028753,0.001132642,0.0006217752,0.0002798834,0.00002566167,0.0005073656,0.0001348003,0.00003263878],"category_scores_gemma":[0.00004854133,0.000215032,0.00006807561,0.0004562541,0.0002739705,0.0003537681,0.0002107774,0.0005303689,7.548617e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001948234,"about_ca_system_score_gemma":0.0002762102,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008910677,"about_ca_topic_score_gemma":0.0001729654,"domain_scores_codex":[0.9976557,0.0006290847,0.001100408,0.0002091154,0.0001118178,0.0002938442],"domain_scores_gemma":[0.998118,0.0002156543,0.001059614,0.0003916474,0.0002007152,0.00001441184],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.01636854,0.1437506,0.6070564,0.000217839,0.002342778,0.00008916728,0.01803873,0.00003133649,0.2051893,0.00002918112,0.00003011435,0.006856015],"study_design_scores_gemma":[0.06538616,0.04717486,0.8776101,0.0002725617,0.0004288665,0.00003189689,0.004972883,2.09638e-7,0.003829806,0.00001691695,0.00004496961,0.0002307093],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9971818,0.001656986,0.000003224398,0.00001788082,0.0001407341,0.0008110451,0.00002472962,0.000009038968,0.0001545981],"genre_scores_gemma":[0.9996094,0.0001135996,0.00002942532,0.00002009311,0.000001327698,0.00001078869,0.00003808023,0.0000192938,0.0001580118],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2705538,"threshold_uncertainty_score":0.876875,"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."}}