A Phase II Clinical Trial of an Aromatase Inhibitor for Postmenopausal Women with Lymphangioleiomyomatosis.

Rationale: Lymphangioleiomyomatosis (LAM) is a progressive cystic lung disease that predominantly affects women and can worsen with pregnancy, estrogen treatment, and the menstrual cycle, suggesting an important role for estrogen in disease pathogenesis.

Objectives: To assess the efficacy and safety of the aromatase inhibitor letrozole in the treatment of LAM.

Methods: Seventeen postmenopausal women with LAM were enrolled in this phase II trial and randomized to receive letrozole 2.

Clinical Development Approaches and Statistical Methodologies to Prospectively Assess the Cardiovascular Risk of New Antidiabetic Therapies for Type 2 Diabetes.

In December 2008, the US Food and Drug Administration (FDA) issued a guidance for industry requiring sponsors to demonstrate that a new antidiabetic therapy being developed to treat type 2 diabetes does not increase cardiovascular (CV) risk to an unacceptable extent. CV events reported during phase 2 and phase 3 trials should be prospectively and independently adjudicated. Before submission of a new drug application or biologics license application, sponsors should compare the incidence of major CV events occurring with the investigational agent versus the control group to show that the upper bound of the 2-sided 95% confidence interval (CI) for the estimated risk ratio is less than 1.

Psychometric Comparison of the PROMIS Physical Function CAT With the FAAM and FFI for Measuring Patient-Reported Outcomes.

Background: Selecting optimal patient-reported outcome (PRO) instruments is critical to improving the quality of health care. The purpose of this study was to compare the reliability, responsiveness, and efficiency of three PRO measures: the Foot and Ankle Ability Measure-Activity of Daily Living subscale (FAAM_ADL), the Foot Function Index 5-point verbal rating scale (FFI-5pt), and the PROMIS Physical Function computerized adaptive test (PF CAT).

Methods: Data were aggregated from 10 clinical sites in the AOFAS's National Orthopaedic Foot and Ankle Research (OFAR) Network from 311 patients who underwent elective surgery for a disorder of the foot or ankle.