JAMA

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IMPORTANCE: Individual cohort studies concur that the amyloidogenic V142I variant of the transthyretin (TTR) gene, present in 3% to 4% of US Black individuals, increases heart failure (HF) and mortality risk. Precisely defining carrier risk across relevant clinical outcomes and estimating population burden of disease are important given established and emerging targeted treatments. OBJECTIVES: To better define the natural history of disease in carriers across mid to late life, assess variant modifiers, and estimate cardiovascular burden to the US population...

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IMPORTANCE: Many medical journals, including JAMA, restrict the use of causal language to the reporting of randomized clinical trials. Although well-conducted randomized clinical trials remain the preferred approach for answering causal questions, methods for observational studies have advanced such that causal interpretations of the results of well-conducted observational studies may be possible when strong assumptions hold. Furthermore, observational studies may be the only practical source of information for answering some questions about the causal effects of medical or policy interventions, can support the study of interventions in populations and settings that reflect practice, and can help identify interventions for further experimental investigation...

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IMPORTANCE: In newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL), disease progression due to acquired resistance to first- or second-generation BCR::ABL1 tyrosine kinase inhibitors is common. Ponatinib inhibits BCR::ABL1 and all single-mutation variants, including T315I. OBJECTIVE: To compare frontline ponatinib vs imatinib in adults with newly diagnosed Ph+ ALL. DESIGN, SETTING, AND PARTICIPANTS: Global registrational, phase 3, open-label trial in adults aged 18 years or older with newly diagnosed Ph+ ALL...