title: Preliminary Evaluation of Clinician Rated Outcome Measures in Mitochondrial Disease.
abstract: BACKGROUND
  Currently there are no known cures and few effective treatments for mitochondrial disorders. It is also true there is a lack of knowledge about suitable clinician rated outcomes and how these change over time in this patient cohort.
  
  
  OBJECTIVE
  We sought to evaluate the validity and responsiveness to change of clinician rated outcome measures in patients with m.3243A>G-related mitochondrial disease.
  
  
  METHODS
  We assessed the six minute timed walk (6MTW), 10 meter walk / test (10MWT), Timed up and Go (TUG) and the 5 times sit to stand (5XSTS), in 18 patients (12 sedentary controls), at baseline and a subgroup of 10 control-matched patients following a 16-week structured aerobic exercise intervention program.
  
  
  RESULTS
  All outcome measures assessed were valid and able to differentiate between patients and controls. Disease severity, as measured by the Newcastle Mitochondrial Disease Adult Scale, correlated with TUG (r = 0.54, p = 0.020) and 10MWT (r = 0.47, p = 0.050). Receiver Operating Curve analysis revealed 5XSTS to be the most responsive measure (AUC 0.931; 95% CI 0.84- 1.00) with responsiveness to change, post intervention, emulating disease burden variance.
  
  
  CONCLUSIONS
  The 5XSTS can be used to discriminate between mitochondrial patients and sedentary controls with high accuracy. The 10MWT and TUG may serve as suitable and clinically relevant clinician rated measures to track disease progression and assess intervention.
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.|MoveLab, 4th Floor William Leech Building, The Medical School, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.
authors.forename: Jane
authors.initials: J
authors.lastname: Newman
authors.affiliation: Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Brook
authors.initials: B
authors.lastname: Galna
authors.affiliation: MoveLab, 4th Floor William Leech Building, The Medical School, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.
authors.forename: Djordje G
authors.initials: DG
authors.lastname: Jakovljevic
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Matthew G
authors.initials: MG
authors.lastname: Bates
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.
authors.forename: Andrew M
authors.initials: AM
authors.lastname: Schaefer
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Robert
authors.initials: R
authors.lastname: McFarland
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Douglass M
authors.initials: DM
authors.lastname: Turnbull
authors.affiliation: MoveLab, 4th Floor William Leech Building, The Medical School, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.
authors.forename: Michael I
authors.initials: MI
authors.lastname: Trenell
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Robert W
authors.initials: RW
authors.lastname: Taylor
authors.affiliation: Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Lynn
authors.initials: L
authors.lastname: Rochester
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, NE2 4HH, UK.|Newcastle University Institute for Ageing, Institute of Neuroscience, Newcastle upon Tyne, NE4 5PL, UK.
authors.forename: Gráinne S
authors.initials: GS
authors.lastname: Gorman