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As a journalist, you can create a free Muck Rack account to customize your profile, list your contact preferences, and upload a portfolio of your best work.Articles
Development and validation of the predictive aplastic score system (PASS): a simplified tool to diagnose acquired aplastic anemia in adults
Abstract Acquired aplastic anemia (AA) can present similarly to inherited bone marrow failure syndromes (IBMFS) but treatment differs. AA diagnosis relies on excluding IBMFS; however, genetic testing is not always available, may delay care or be inconclusive. We developed the Predictive Aplastic Score System (PASS), a clinical tool using readily available data to distinguish AA from IBMFS in adults. The training cohort included 212 adults (162 AA, 50 IBMFS).
Controversial issues in clinical investigation of rare disease drug development
For rare disease drug development, a single-stage hypothesis testing procedure is often considered for clinical investigation regarding safety and efficacy of a test treatment study. Under the hypothesis testing framework, “p-value less than 0.05 with at least 80% power” is often used as the gold standard in the regulatory review/approval process.
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