Chest
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Randomized Controlled Trial Multicenter Study Comparative Study
Evaluation of the Predictive Value of a Clinical Worsening Definition Using 2-Year Outcomes in Patients With Pulmonary Arterial Hypertension: A REVEAL Registry Analysis.
Time to clinical worsening has been proposed as a primary end point in clinical trials of pulmonary arterial hypertension (PAH); however, neither standardized nor validated definitions of clinical worsening across PAH trials exist. This study aims to evaluate a proposed definition of clinical worsening within a large prospective, observational registry of patients with PAH with respect to its value as a predictor of proximate (within 1 year) risk for subsequent major events (ie, death, transplantation, or atrial septostomy). ⋯ Clinical worsening was highly predictive of subsequent proximate mortality in this analysis from an observational study. These results validate the use of clinical worsening as a meaningful prognostic tool in clinical practice and as a primary end point in clinical trial design.
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Unilateral medical futility policies, which allow health-care providers to limit or withdraw life-sustaining treatment over patient or surrogate objections, are increasingly designed around a procedural approach. Medical or ethics committees follow a prespecified process, the culmination of which is a justified decision about whether ongoing treatment should be withheld or withdrawn. These procedures have three stages. ⋯ We review the available data on procedure-based futility policies, arguing that there is limited information on their potential harms and how these harms are distributed. We consider the ethical implications of policy-making under informational uncertainty, invoking the precautionary principle--in the absence of clear data, if a policy has significant risk of significant harm, the burden of proof that it is not harmful falls on those recommending the policy--as the guiding moral standard for hospitals and professional organizations considering whether to adopt a procedural approach to medical futility. On the basis of this principle, we argue that any new futility guideline must include a significant commitment to collecting prospective data on its application.
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Biomarkers reflective of disease activity in cystic fibrosis (CF) have the potential to improve patient care, particularly during CF pulmonary exacerbations (CFPEs). Although blood-based biomarkers have been studied in CFPE for nearly 3 decades, none have been integrated into routine clinical practice. To facilitate progress in this area, we performed a systematic review evaluating blood-based biomarkers during CFPE. ⋯ Although there are several blood-based biomarkers with evidence for application within the CFPE setting, CRP has been the most widely studied biomarker demonstrating the potential for clinical usefulness. Further validation studies and clinical trials are required to determine whether blood-based biomarkers can be used to ultimately improve health outcomes in the setting of a CFPE.