{"id":"W4410544331","doi":"10.1111/bjh.20058","title":"Long‐term efficacy and safety of mitapivat in non‐transfusion‐dependent α‐ or β‐thalassaemia: An open‐label phase 2 study","year":2025,"lang":"en","type":"article","venue":"British Journal of Haematology","topic":"Hemoglobinopathies and Related Disorders","field":"Medicine","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto","funders":"Agios Pharmaceuticals","keywords":"Medicine; Haemolysis; Adverse effect; Pyruvate kinase; Pyruvate kinase deficiency; Internal medicine; Anemia; Erythropoiesis; Ineffective erythropoiesis; Gastroenterology; Immunology; Metabolism; Glycolysis","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":[0.001444259,0.000899013,0.001740615,0.0002650703,0.0003314306,0.0009590641,0.0005787668,0.000976783,0.003576503],"category_scores_gemma":[0.001173288,0.000356271,0.001089312,0.0002453788,0.0005342135,0.0008791944,0.0002120076,0.002337383,0.0007340235],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003597196,"about_ca_system_score_gemma":0.0005122445,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002073674,"about_ca_topic_score_gemma":0.0004879776,"domain_scores_codex":[0.9996505,0.0001478852,0.00002552203,0.00006254565,0.00005116169,0.00006249319],"domain_scores_gemma":[0.9992461,0.0001708523,0.000214684,0.00007122692,0.00007246356,0.0002246981],"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":[0.6593693,0.06707695,0.00760416,0.002245041,0.002910568,0.0006606713,0.0004615797,0.002314754,0.07256975,0.0002550897,0.005636752,0.1788953],"study_design_scores_gemma":[0.3548708,0.5997216,0.02930594,0.000140538,0.001151492,0.0009904426,0.0001008901,0.001881499,0.006443135,0.0003027199,0.005024606,0.00006636741],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9920696,0.003865971,0.0005718583,0.0002528901,0.00009792686,0.0009587968,0.0008546025,0.0001110478,0.001217299],"genre_scores_gemma":[0.9909654,0.002438186,0.0009557237,0.0007524848,0.0002027837,0.001159578,0.00156028,0.0000291488,0.001936421],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003576503,"threshold_uncertainty_score":0.01196462,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01959694867621731,"score_gpt":0.3406461953865784,"score_spread":0.3210492467103611,"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."}}