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Identification and outcomes of clinical phenotypes in amyotrophic lateral sclerosis/motor neuron disease: Australian National Motor Neuron Disease observational cohort

Talman, Paul, Duong, Thi, Vucic, Steve, Mathers, Susan, Venkatesh, Svetha, Henderson, Robert, Rowe, Dominic, Schultz, David, Edis, Robert, Needham, Merrilee, Macdonnell, Richard, McCombe, Pamela, Birks, Carol and Kiernan, Matthew 2016, Identification and outcomes of clinical phenotypes in amyotrophic lateral sclerosis/motor neuron disease: Australian National Motor Neuron Disease observational cohort, BMJ open, vol. 6, no. 9, Article number : e012054, pp. 1-8, doi: 10.1136/bmjopen-2016-012054.

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Title Identification and outcomes of clinical phenotypes in amyotrophic lateral sclerosis/motor neuron disease: Australian National Motor Neuron Disease observational cohort
Author(s) Talman, Paul
Duong, Thi
Vucic, Steve
Mathers, Susan
Venkatesh, Svetha
Henderson, Robert
Rowe, Dominic
Schultz, David
Edis, Robert
Needham, Merrilee
Macdonnell, Richard
McCombe, Pamela
Birks, Carol
Kiernan, Matthew
Journal name BMJ open
Volume number 6
Issue number 9
Season Article number : e012054
Start page 1
End page 8
Total pages 8
Publisher BMJ Publishing Group
Place of publication London, Eng.
Publication date 2016-09-30
ISSN 2044-6055
Keyword(s) EPIDEMIOLOGY
Summary OBJECTIVE: To capture the clinical patterns, timing of key milestones and survival of patients presenting with amyotrophic lateral sclerosis/motor neuron disease (ALS/MND) within Australia.

METHODS: Data were prospectively collected and were timed to normal clinical assessments. An initial registration clinical report form (CRF) and subsequent ongoing assessment CRFs were submitted with a completion CRF at the time of death.

DESIGN: Prospective observational cohort study.

PARTICIPANTS: 1834 patients with a diagnosis of ALS/MND were registered and followed in ALS/MND clinics between 2005 and 2015.

RESULTS: 5 major clinical phenotypes were determined and included ALS bulbar onset, ALS cervical onset and ALS lumbar onset, flail arm and leg and primary lateral sclerosis (PLS). Of the 1834 registered patients, 1677 (90%) could be allocated a clinical phenotype. ALS bulbar onset had a significantly lower length of survival when compared with all other clinical phenotypes (p<0.004). There were delays in the median time to diagnosis of up to 12 months for the ALS phenotypes, 18 months for the flail limb phenotypes and 19 months for PLS. Riluzole treatment was started in 78-85% of cases. The median delays in initiating riluzole therapy, from symptom onset, varied from 10 to 12 months in the ALS phenotypes and 15-18 months in the flail limb phenotypes. Percutaneous endoscopic gastrostomy was implemented in 8-36% of ALS phenotypes and 2-9% of the flail phenotypes. Non-invasive ventilation was started in 16-22% of ALS phenotypes and 21-29% of flail phenotypes.

CONCLUSIONS: The establishment of a cohort registry for ALS/MND is able to determine clinical phenotypes, survival and monitor time to key milestones in disease progression. It is intended to expand the cohort to a more population-based registry using opt-out methodology and facilitate data linkage to other national registries.
Language eng
DOI 10.1136/bmjopen-2016-012054
Field of Research 080109 Pattern Recognition and Data Mining
Socio Economic Objective 970108 Expanding Knowledge in the Information and Computing Sciences
HERDC Research category C1 Refereed article in a scholarly journal
ERA Research output type C Journal article
Copyright notice ©2016, The Authors
Free to Read? Yes
Use Rights Creative Commons Attribution non-commercial licence
Persistent URL http://hdl.handle.net/10536/DRO/DU:30087599

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Every reasonable effort has been made to ensure that permission has been obtained for items included in DRO. If you believe that your rights have been infringed by this repository, please contact drosupport@deakin.edu.au.