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Table 3 Model Results (Projected Lifetime Outcomes) in Gaucher Disease Type 1

From: A new framework for evaluating the health impacts of treatment for Gaucher disease type 1

  Starting Health State    
Intact Spleen No Spleen
Mild Moderate Marked with SSC Mild Moderate Marked with SSC
Discounted QALYs 15.73 15.69 14.92 15.70 15.65 14.89
Undiscounted QALYs 31.16 31.20 29.73 31.13 31.16 29.71
% of Remaining Lifetime Spent in:
 Mild 69.72 70.61 33.86 68.94 69.57 19.55
 Mild with BP 12.86 12.26 16.13 13.15 12.23 13.37
 Mild with SSC 0.73 0.69 1.17 0.75 0.70 1.07
 Moderate 15.69 15.58 42.20 16.14 16.50 52.97
 Moderate with SSC 0.53 0.46 2.76 0.55 0.54 5.40
 Marked 0.24 0.21 1.36 0.25 0.24 3.01
 Marked with SSC 0.20 0.17 2.38 0.21 0.20 3.02
 Severe 0.01 0.00 0.03 0.01 0.01 1.28
 Severe with SSC 0.02 0.02 0.12 0.02 0.02 0.33
  1. Results for cohorts starting treatment at age 32 (patients in all cohorts have 42.29 remaining life years). QALYs discounted 3.5% per annum