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J Clin Neurosci. 2018 Nov 22. pii: S0967-5868(18)31742-9. doi: 10.1016/j.jocn.2018.11.034. [Epub ahead of print]

Establishment and 12-month progress of the New Zealand Motor Neurone Disease Registry.

Author information

1
Department of Neurology, Auckland City Hospital, Private Bag 92024, Auckland 1142, New Zealand. Electronic address: mndregistry@adhb.govt.nz.
2
Department of Neurology, Auckland City Hospital, Private Bag 92024, Auckland 1142, New Zealand.
3
Motor Neurone Disease New Zealand, PO Box 24-036, Royal Oak, Auckland 1345, New Zealand.
4
Department of Pharmacology and Clinical Pharmacology, Faculty of Medical and Health Sciences, University of Auckland, Private Bag 92019, Auckland 1023, New Zealand.
5
Department of Neurology, Christchurch Hospital, Private Bag 4710, Christchurch 8140, New Zealand.
6
Palliative Care Service, Wellington Hospital, Private Bag 7902, Wellington 6242, New Zealand.
7
Department of Neurology, Auckland City Hospital, Private Bag 92024, Auckland 1142, New Zealand; Centre for Brain Research, Neurogenetics Research Clinic, Faculty of Medical and Health Sciences, University of Auckland, Private Bag 92019, Auckland 1023, New Zealand.

Abstract

There are only limited treatments currently available for Motor Neurone Disease, each with modest benefits. However, there is a large amount of research and drug discovery currently underway worldwide. The New Zealand Motor Neurone Disease Registry was established in 2017 to facilitate participation in research and clinical trials, and to aid researchers in planning and recruitment. The NZ MND Registry is an opt in patient registry which collects demographic, contact and clinical data for those who choose to enrol. We report anonymised aggregated data from the first year's enrolment. 12th July 2018, there were 142 participants enrolled in the NZ MND Registry. Participant sex distribution reflects the demographics reported worldwide, but ethnicity is divergent from what is seen in New Zealand overall, with an over-representation of people who identify as New Zealand European. 85.5% of participants are diagnosed with sporadic MND and 6.1% with familial MND. The remainder were participants who have not been diagnosed but have a family history, or positive genetic test for a MND-causing mutation. Levels of disability are reported using ALSFRS-R scores, and show that the majority of participants are within the higher range of the scale. The registry has facilitated entry of patients into three studies to date. The establishment of the NZ MND Registry illustrates a swift launch of a rare disease patient registry. The role of patient registries is an ever changing one, but with clear utility at every point of along the pathway to drug discovery.

KEYWORDS:

ALS; Epidemiology; Motor neuron disease; Patient registry

PMID:
30473407
DOI:
10.1016/j.jocn.2018.11.034

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