SciCombinator

Discover the most talked about and latest scientific content & concepts.

ML Ganz, S Stern, A Ward, L Nalysnyk, M Selzer, A Hamed and N Weinreb
Abstract
The Disease Severity Scoring System (DS3) is a validated measure for evaluating Gaucher disease type 1 (GD1) severity. We developed a new framework, consisting of health states, transition probabilities between those states, and preferences for those states (utilities) based on the DS3 to predict long-term outcomes of patients starting treatment. We defined nine mutually exclusive (alive) health states based on three DS3 categories: mild (0 ≤ DS3 ≤ 3.5) without symptoms of bone disease; mild with bone pain, mild with severe skeletal complications (SSC) defined as lytic lesions, avascular necrosis, or fracture; moderate (3.5 < DS3 ≤ 6.5) without SSC; moderate with SSC; marked (6.5 < DS3 ≤ 9.5) without SSC; marked with SSC; severe (9.5 < DS3 ≤ 19) without SSC; and severe with SSC. Health-state transition probabilities and utilities were estimated from a longitudinal sample of patients with GD1 who started enzyme replacement therapy (the DS3 Score Study). Age dependent GD1-specific mortality was derived from published data. We used a Markov state-transition model to illustrate how to estimate time spent in each health state.
Tweets*
13
Facebook likes*
0
Reddit*
0
News coverage*
1
Blogs*
0
SC clicks
0
Concepts
Medicine, Skeletal system, Statistics, Therapy, Osteoporosis, Avascular necrosis, Gaucher's disease, Epidemiology
MeSH headings
-
comments powered by Disqus

* Data courtesy of Altmetric.com