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Table 4 Summary of initial study results and post hoc analyses

From: Evaluation of pre-symptomatic nitisinone treatment on long-term outcomes in Tyrosinemia type 1 patients: a systematic review

  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
  1. aBased on our analysis of data presented in the paper by Larochelle et al. [10] using Fisher exact test
  2. NA not applicable, OLT Orthotopic liver transplantation