The ulitmate goal of the current study will be the presentation of a specific minimum core set of outcome measures to be used in future clinical and follow-up studies in patients with autoimmune mediated polyneuropathies.
ID
Source
Brief title
Condition
- Autoimmune disorders
- Peripheral neuropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The selected outcome measures will be evaluated on their applicability,
validity, reliability and responsiveness. They are the most important study
parameters.
Secondary outcome
not applicable
Background summary
In the last seven years, efforts have been made by the 'Inflammatory Neuropathy
Cause and Treatment' (INCAT) group to initiate a scientific approach dealing
with the standardisation of outcome measures for studies in patients with
polyneuropathy. These efforts culminated in a workshop, guided by the European
Neuromuscular Centre (ENMC). Based on the recommendations of this workshop,
this study aims to expand the clinimetric knowledge on outcome measures.
The PeriNomS study evaluates a set of often used outcome measures in autoimmune
mediated polyneuropathies. According to the WHO guidelines, the following
levels of outcome are selected: pathology, impairment (loss of body
function/structure), activity limitation and quality of life. The different
outcome measures will be analysed in a cross-sectional and a longitudinal study
group to determine their scientific soundness like being valid, reliable and
responsive to changes over time.
Study objective
The ulitmate goal of the current study will be the presentation of a specific
minimum core set of outcome measures to be used in future clinical and
follow-up studies in patients with autoimmune mediated polyneuropathies.
Study design
This study concerns a multi-centre observational research project with a
cross-sectional and a longitudinal part.
Study burden and risks
For the cross-sectional studies a total of 120 patients will be examined twice
with a time interval of 2-4 weeks. All patients will be asked to complete a set
of questionnaires at home, at their own pace. They will be examined at the
outpatients clinic; routine neurological examination including extra sensory
testing and strength measurements with some instruments. At onset an EMG and a
skin biopsy will be performed (not mandatory for patients).
For the longitudinal part of the study 70 patients, newly diagnosed with GBS or
CIDP will be examined five times during one year. Also 40 patients, newly
diagnosed with MGUSP or MMN will be included and investigated three times
during one year. Above mentioned examination will be performed, including twice
an EMG and skin biopsy (at onset and after one year).
The estimated time for completion of the questionnaires will be about 1,5 hour
eacht time. The visits to the out-patients clinic will take approximately 1
hour, except for the first visit (1,5-2 hours) in both study groups, in the
longitudinal group the last visit will also take about 1,5-2 hours.
The most important burden for patients will be time investment. A skin biopsy
is in many hospitals a routine diagnostic procedure and is a minimal invasive
procedure. There is a small risk of getting an infection and most people get a
scar conform the size of the punch biopsy. EMG evaluation harbours no special
risk, needle examination can be a little painful.
postbus 1738
3000 DR Rotterdam
NL
postbus 1738
3000 DR Rotterdam
NL
Listed location countries
Age
Inclusion criteria
- patients must have clear consciousness
- patients must meet the international criteria for their diagnosis
- written informed consent given by the participant is mandatory before inclusion
- patients with newly diagnosed GBS, CIDP, MGUSP or MMN will be included in the longitudinal part of the study
- patients with CIDP demonstrating a clinical relapse, and not using any medication for their polyneuropathy (e.g. intravenous immunoglobulins, steroids, immunosuppressive agent) for at least 2 months prior to inclusion, can be included in the longitudinal part of the study
- patients with MMN having new (multi)focal nerve lesion, not using any medication for their polyneuropathy (e.g. intravenous immunoglobulins) for at least 2 months prior to inclusion, can also be included in the longitudinal part of the study
- MGUSP: in both parts of the study, patients will only be included in the MGUSP group if they have serologically proven IgM anti-MAG+ antibodies. For the longitudinal study group MGUSP patients must have an indication for therapy (on basis of clear progression of symptoms)
- patients with a clinical diagnosis and a clinical course compatible with CIDP and an IgG MGUSP will be included as CIDP (in both parts of the study)
- for the cross-sectional part of the study, patients with the following diseases will be included: GBS, CIDP, MMN, MGUSP, and AI-SFN in sarcoidosis. Only patients with a clinical stable condition will be included. A clinical stable condition is defined as:
1) An unchanged clinical functionality as declared by the patient to the best of his/her knowledge over 2 months prior to the study and during the study (stable condition at second visit compared to entry)
2) No clear objective changes at neurological examination by the researcher when compared with recorded findings over two month prior to study entry (if available) and during the study (unchanged neurological examination at second visit compared to entry)
- for the cross-sectional study, the selected patients must still have residual symptoms or signs due to their illness that represents a broad range of activity limitations before admission to the current study
- patients with CIDP or MMN receiving (interval) therapy can be included in the cross-sectional part of the study as long as their clinical condition is stable (see definition above)
Exclusion criteria
- age < 18 years
- concomitant disease (e.g. diabetes, renal insufficiency), (prior) treatment with chemotherapy or alcohol abuse that might interfere with general nervous system as well as physical functioning
- patients with an IgM MGUSP without serologically proven IgM anti-MAG+ antibodies will be excluded for both parts of the study
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL14641.078.06 |