This metric is one of the standard outcome measures used in ALS clinical trials.
(a) The number of patients choosing to experiment with lithium carbonate peaked in the months after publication of a small clinical trial in Italy.
Preliminary negative results from this patient-led study were announced before the first randomized control trial had started recruitment.
The Patients Like Me matching algorithm minimizes the area between the disease progression curves for a target patient and a control.
ALS trials have used a variety of methods to match patients receiving a treatment with appropriate control patients, with trial designs including futility design, multistage adaptive design, lead-in periods, selection design and historical controls.
To investigate whether the major effect of lithium carbonate reported in the original study was corroborated in these 348 patients, we undertook an observational analysis of self-reported outcomes.