Achieving valid patient-reported outcomes measurement: a lesson from fatigue in multiple sclerosis.

Background: The increasing influence of patient-reported outcome (PRO) measurement instruments indicates their scrutiny has never been more crucial. Above all, PRO instruments should be valid: shown to assess what they purport to assess.

Objectives: To evaluate a widely used fatigue PRO instrument, highlight key issues in understanding PRO instrument validity, demonstrate limitations of those approaches and justify notable changes in the validation process.

Randomized controlled trial of atorvastatin in clinically isolated syndrome: the STAyCIS study.

Objective: To test efficacy and safety of atorvastatin in subjects with clinically isolated syndrome (CIS).

Methods: Subjects with CIS were enrolled in a phase II, double-blind, placebo-controlled, 14-center randomized trial testing 80 mg atorvastatin on clinical and brain MRI activity. Brain MRIs were performed quarterly.

Cost-effectiveness of disease-modifying therapy for multiple sclerosis: a population-based study.

Objective: To evaluate the cost-effectiveness of disease-modifying therapies (DMTs) in the United States compared to basic supportive therapy without DMT for patients with relapsing multiple sclerosis (MS).

Methods: Using data from a longitudinal MS survey, we generated 10-year disease progression paths for an MS cohort. We used first-order annual Markov models to estimate transitional probabilities.

Multicenter randomized clinical trial of donepezil for memory impairment in multiple sclerosis.

Objectives: The goal of this study was to determine if memory would be improved by donepezil as compared to placebo in a multicenter, double-blind, randomized clinical trial (RCT).

Methods: Donepezil 10 mg daily was compared to placebo to treat memory impairment. Eligibility criteria included the following: age 18-59 years, clinically definite multiple sclerosis (MS), and performance ≤ ½ SD below published norms on the Rey Auditory Verbal Learning Test (RAVLT).

Broadening the spectrum of controls for skin biopsy in painful neuropathies.

Epidermal nerve fiber density (ENFD) is useful in the evaluation of small-fiber neuropathies (SFSNs). A recent evidence-based review highlighted the need to broaden the spectrum of ENFD controls to include lower limb pain states other than polyneuropathy. We studied epidermal innervation in multiple sclerosis (MS) and distal lower limb burning pain (DLLBP).