Table 4 Summary of initial study results and post hoc analyses
| Death | Need for OLT | Patients with neurological crisis | |
|---|---|---|---|
|
Initial analysis
Early vs late |
Québec cohort
Larochelle 2012 [10] 0/24 vs 2/26 (p = 0.49)a Birmingham study McKiernan 2015 [14] 0/12 vs 2/5 (p = 0.07) |
Québec cohort
Larochelle 2012 [10] 0/24 vs 7/26 (p = 0.01)a Birmingham study McKiernan 2015 [14] 0/12 vs 1/5 (p = 0.29) Bartlett 2014 [17] 0/11 vs 7/20 (p = 0.033) |
Québec cohort
Larochelle 2012 [10] 0/24 vs 5/26 (p = 0.051) Simoncelli 2015 [11] 0/41 vs 5/26 (p = 0.007) |
|
Post hoc 1
Screen detection vs symptomatic detection, all with direct nitisinone initiation |
Québec cohort
Larochelle 2012 [10] 0/24 vs 0/5 Birmingham study McKiernan 2015 [14] 0/12 vs 1/4 (p = 0.25) |
Québec cohort
Larochelle 2012 [10] 0/24 vs 3/5 (p = 0.0027) Birmingham study McKiernan 2015 [14] 0/12 vs 1/4 (p = 0.25) Bartlett 2014 [17] 0/10 vs 5/15 (p = 0.061) |
Québec cohort
Larochelle 2012 [10] 0/24 vs 0/5 Simoncelli 2012 [11] NA |
|
Post hoc 2
Early symptomatic vs late symptomatic,all with direct nitisinone initiation |
Québec cohort
Larochelle 2012 [10] NA Birmingham cohort NA |
Québec cohort
Larochelle 2012 [10] NA Birmingham cohort McKiernan 2015 [14] NA Bartlett 2014 [17] 0/1 vs 5/14 |
Québec cohort
NA |
|
Post hoc 3
Screen detection, direct nitisinone initiation vs screen detection, 1–12 months delayed nitisinone initiation |
Québec cohort
Larochelle 2012 [10] 0/24 vs 0/10 Birmingham cohort NA |
Québec cohort
Larochelle 2012 [10] 0/24 vs 0/10 Birmingham cohort McKiernan 2015 [14] NA Bartlett 2014 [17] NA |
Québec cohort
Larochelle 2012 [10] 0/24 vs 1/10 (p = 0.294) Simoncelli 2015 [11] NA |