Journal article
Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.
- Nagy S Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Schädelin S Clinical Trial Unit, University of Basel, Basel, Switzerland.
- Hafner P Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Bonati U Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Scherrer D Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Ebi S Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Schmidt S Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Orsini AL Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- Bieri O Department of Radiology, Division of Radiological Physics, University of Basel Hospital, University of Basel, Basel, Switzerland.
- Fischer D Division of Neuropaediatrics, University of Basel Children's Hospital, University of Basel, Basel, Switzerland.
- 2019-08-31
Published in:
- Muscle & nerve. - 2020
Duchenne muscular dystrophy
clinical outcome measure
longitudinal analysis
motor function measure
quantitative muscle MRI
sample size
Adiposity
Adolescent
Child
Endpoint Determination
Follow-Up Studies
Humans
Longitudinal Studies
Magnetic Resonance Imaging
Male
Movement
Muscle, Skeletal
Muscular Dystrophy, Duchenne
Outcome Assessment, Health Care
Reproducibility of Results
Retrospective Studies
Treatment Outcome
English
INTRODUCTION
The definition of reliable outcome measures is of increasing interest in patients with Duchenne muscular dystrophy (DMD).
METHODS
In this retrospective study, we analyzed the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD. Clinical outcome measures included motor function measure (MFM) and timed function tests, while quantitative MRI data were mean fat fraction (MFF) and T2 relaxation time of thigh muscles. Statistical analysis was based on 3-, 6-, and 12-month follow-up data.
RESULTS
Quantitative MRI using the MFF was the most sensitive and powerful marker of disease progression with a sample size of four at 1-year follow-up, followed by the D1 domain of MFM (standing and transfer function) with a sample size of 12.
DISCUSSION
Our data support the longitudinal design of clinical trials over at least 12 months and the combinational use of clinical and radiological surrogate outcome measures.
The definition of reliable outcome measures is of increasing interest in patients with Duchenne muscular dystrophy (DMD).
METHODS
In this retrospective study, we analyzed the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD. Clinical outcome measures included motor function measure (MFM) and timed function tests, while quantitative MRI data were mean fat fraction (MFF) and T2 relaxation time of thigh muscles. Statistical analysis was based on 3-, 6-, and 12-month follow-up data.
RESULTS
Quantitative MRI using the MFF was the most sensitive and powerful marker of disease progression with a sample size of four at 1-year follow-up, followed by the D1 domain of MFM (standing and transfer function) with a sample size of 12.
DISCUSSION
Our data support the longitudinal design of clinical trials over at least 12 months and the combinational use of clinical and radiological surrogate outcome measures.
- Language
-
- English
- Open access status
- closed
- Identifiers
-
- DOI 10.1002/mus.26690
- PMID 31469921
- Persistent URL
- https://sonar.ch/global/documents/231715
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