{"id":"W4387335440","doi":"10.1016/j.jaci.2023.09.032","title":"Interim analysis: Open-label extension study of leniolisib for patients with APDS","year":2023,"lang":"en","type":"article","venue":"Journal of Allergy and Clinical Immunology","topic":"Immunodeficiency and Autoimmune Disorders","field":"Immunology and Microbiology","cited_by":40,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Children's Hospital of Pittsburgh; National Institute of Allergy and Infectious Diseases; Irving Medical Center, Columbia University; Walter Reed National Military Medical Center; Novartis Pharma; Leonard M. Miller School of Medicine; Qatar University; Medical Center, University of Pittsburgh; King Faisal Specialist Hospital and Research Centre; University of Pittsburgh; Leidos; University of Alabama; Perelman School of Medicine, University of Pennsylvania; Vanderbilt University Medical Center; University of Arkansas for Medical Sciences; University of Alabama at Birmingham; AstraZeneca; University of Cincinnati; Cincinnati Children's Hospital Medical Center; Hamad Medical Corporation; Roy J. and Lucille A. Carver College of Medicine, University of Iowa; College of Medicine, University of Cincinnati; Texas Children's Hospital; Division of Intramural Research, National Institute of Allergy and Infectious Diseases; CSL Behring; Pfizer; University of Miami; Alberta Health Services; NHLBI Division of Intramural Research; University of Pennsylvania; Vanderbilt University; National Institutes of Health; U.S. Department of Health and Human Services","keywords":"Medicine; Internal medicine; Adverse effect; Interim analysis; Clinical endpoint; Placebo; Immunology; Immune dysregulation; Immune system; Gastroenterology; Pediatrics; Clinical trial; Pathology","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001130654,0.0001923251,0.001064399,0.0003884702,0.000196514,0.00001823289,0.0006344582,0.0002937771,0.00005979727],"category_scores_gemma":[0.0003652509,0.0001337348,0.0001957036,0.000550803,0.0004201304,0.000187537,0.0003120884,0.0004673965,0.00001132386],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001043526,"about_ca_system_score_gemma":0.00007612358,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005224254,"about_ca_topic_score_gemma":0.00003311982,"domain_scores_codex":[0.9974768,0.0004336888,0.001423933,0.0002907529,0.00006135919,0.0003134546],"domain_scores_gemma":[0.9974501,0.0006623899,0.001019035,0.0003392883,0.0005163756,0.00001277072],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"observational","study_design_scores_codex":[0.1041543,0.09315494,0.2350718,0.00008843668,0.2073776,0.00008157866,0.009527898,0.0006155604,0.03235627,0.002291672,0.003353875,0.3119261],"study_design_scores_gemma":[0.03501152,0.03291553,0.9158928,0.00008590983,0.00149761,0.00001945906,0.005423244,0.00004165223,0.000183376,0.0002965949,0.008337458,0.0002948196],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9934493,0.004915073,0.0001719118,0.0001323529,0.00090871,0.0003509436,0.000007852546,0.00001407976,0.00004976541],"genre_scores_gemma":[0.9984268,0.001058174,0.00004535865,0.00007424924,0.000005977085,0.00001016808,0.00004496219,0.0000150448,0.0003192856],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.6808211,"threshold_uncertainty_score":0.5453548,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04781694176036012,"score_gpt":0.3561429483666915,"score_spread":0.3083260066063314,"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."}}