{"id":"W4413745962","doi":"10.1182/bloodadvances.2025016702","title":"Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel","year":2025,"lang":"en","type":"article","venue":"Blood Advances","topic":"Hemoglobinopathies and Related Disorders","field":"Medicine","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia; University of Toronto; BC Children's Hospital; Hospital for Sick Children","funders":"National Institutes of Health; Swedish Orphan Biovitrum; bluebird bio; Jazz Pharmaceuticals; Alexion Pharmaceuticals; Vertex Pharmaceuticals; Chiesi USA; Silence Therapeutics; Sanofi; Amgen; Pfizer; National Center for Advancing Translational Sciences; Agios Pharmaceuticals; Bristol-Myers Squibb","keywords":"Thalassemia; Medicine; Quality of life (healthcare); Blood transfusion; Beta thalassemia; Intensive care medicine; Pediatrics; Internal medicine; Nursing","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":[0.0003356508,0.0001770073,0.0005869329,0.0002314517,0.00002825457,0.000004099659,0.00007932144,0.0001368677,0.00004768313],"category_scores_gemma":[0.00007791058,0.0001338293,0.00005686826,0.0005456783,0.00006460639,0.00009519015,0.00002694565,0.0002719458,0.000001970576],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00007384688,"about_ca_system_score_gemma":0.0002978493,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001132343,"about_ca_topic_score_gemma":0.0009861329,"domain_scores_codex":[0.9979305,0.0001043729,0.001003841,0.0003363094,0.0003124336,0.0003125378],"domain_scores_gemma":[0.9993536,0.00004521269,0.0002000329,0.0002330456,0.00006526696,0.000102804],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.001325276,0.001910089,0.9852708,0.0007353894,0.00008580371,0.00001112247,0.0003310909,0.0001196888,0.0004062954,0.00003067376,0.000005393773,0.009768393],"study_design_scores_gemma":[0.0154539,0.000574653,0.9815475,0.001009684,0.00003567865,4.979341e-7,0.0003724793,0.00001569377,0.0006187678,0.0001072847,0.0001315841,0.0001322215],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9895092,0.008007785,0.00002043581,0.0005558247,0.0001346874,0.0009166233,0.00001799484,0.00002445019,0.0008130157],"genre_scores_gemma":[0.9980854,0.0009975254,0.0002093041,0.0003654244,0.000004413904,0.00005140244,0.00002148397,0.00001323806,0.0002517914],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01412862,"threshold_uncertainty_score":0.5457402,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.005443854165594448,"score_gpt":0.2535369022453521,"score_spread":0.2480930480797576,"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."}}