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. Author manuscript; available in PMC: 2013 Aug 1.
Published in final edited form as: Phys Med Rehabil Clin N Am. 2012 Aug;23(3):475–493. doi: 10.1016/j.pmr.2012.06.010

Table 3.

Clinical Endpoints used in Duchenne Muscular Dystrophy Prospective Natural History Studies and Clinical Trails (ICF Framework35)

Body Structure / Function

  • Molecular Diagnostics

  • Dystrophin Analysis by Muscle Biopsy (Immunohistochemistry)

  • Health Status / Review of Systems

  • Medications, Clinical Complications

  • Anthropometric measures (standing height, weight, ulnar lenth, tibial length, skinfolds)

  • Vital Signs

  • Body Composition (DEXA)

  • Body Composition (Bioelectrical impedance)

  • Magnetic Resonance Imaging

  • Magnetic Resonance Spectroscopy (Muscle)

  • Ultrasound Imaging (Muscle)

  • Bone Health (DEXA)

  • Passive Range of motion (Goniommetry)

  • Spine Deformity Evaluation

  • Strength: Quantitative Grip strength

  • Strength: Quantitative Tip Pinch and Key Pinch strength

  • Strength: Isometric Strength with Hand-held devices

  • Strength: Isometric Strength with Fixed Devices

  • Strength: Isokinetic strength with fixed devices

  • Strength: Manual Muscle Testing (or MRC%)

  • Pulmonary fuction tests: FVC, FEV1, PEFR, Peak Cough Flow, MIP, MEP

  • Cardiac: ECG

  • Cardiac: Echocardiography

  • Cardiac: Holter Monitoring

  • Cardiac: MR Imaging

  • Cognitive and Neuropsychological Testing
Activities (Clinical Evaluator-Determined Scales)

  • Vignos Lower Extremity Functional Grade

  • Brooke Upper Extremity Functional Grade

  • Northstar Ambulatory Assesssment (NSAA)

  • French Motor Function Measure (MFM)

  • Bayley Scales of Infant Development

  • Hammersmith Functional Motor Scale

  • Modified-Hammersmith Functional Motor Scale (Extended)

  • Gross Notor Function Measure (GMFM)

  • Egen Klassification Scale v. 2 (EK2)
Activities (Functional Tests with Timed Dimension)

  • Time to rise from the floor (supine to stand)

  • Time to climb four steps (beginning and ending standing with arms at sides)

  • Time to walk/run 10 meters or 30 feet (as fast as compatible with safety)

  • Time to stand from a chair (chair height should allow feet to touch floor)

  • Tine to propel a manual wheelchair 10 meters or 30 feet

  • Time to Put on a T-shirt

  • Time to cut out a 3″ × 3″ pre-marked square from a piece of paper with safety scissors (lines do not need to be followed precisely)

  • 6-Minute Walk Test

  • Two-Minute Walk Test

  • 10-minute Walk Test with Energy Expenditure using COSMED K4B2

  • Gait kinematics, kinetics with time-distance parameters

  • Stepwatch Step Activity Monitoring

  • ActiCal

  • 9-Hole Peg test

  • Jebsen Taylor Hand Function Test
Participation Measures

  • Pediatric Evaluation of Disability Inventory (PEDI)

  • Children’s Assessment of Participation and Enjoyment (CAPE)

  • Preferences for Activities of Children (PAC)

  • Assessment of Preschool Children’s Participation (APCP)
Patient-Reported Outcome Measures (PROs)

  • Pediatric Quality of Life Questionnaire PedsQLTM) Generic Core Scale

  • POSNA pediatric musculoskeletal functional health questionnaire / Pediatric Outcomes Data Collection Instrument (PODCI).

  • PedsQL Neuromuscular Module

  • PedsQL Multidime0sional Fatigue Scale

  • NeuroQoL Patient-reported Quality of Life

  • Life Satisfaction Scale (Life Satisfaction Scale for Adolescents)

  • Individualised Neuromuscular QoL (InQoL)

  • Child Behavioral Checklist (ASEBA)

  • Canadian Occupational Performance Measure (COPM)

  • Caregiver Burden Scale

  • WHO Quality of Life – Bref

  • SF-36

  • Pittsburgh Sleep Quality Index