RELIABILITY AND VALIDITY OF DISABILITY MEASURES IN PERIPHERAL NEUROPATHIES
Molenaar D.S.M.(1), Vermeulen M.(1), de Haan R.(2) (1)Department of Neurology, (2)Department of Clinical Epidemiology and Biostatistics, Academic Medical Center, PO Box 22700, 1100 DE Amsterdam, The Netherlands
The aim of this study was to evaluate disability measures that were used in patients with peripheral neuropathies. Only six measures we found in the literature focused on disability: functional abilities, Guillain-Barré syndrome (GBS) leg index, Hauser ambulation index, modified Rankin scale, Rivermead mobility index, and timed 10 meter walk test. We studied agreement of disability measures between observers, homogeneity, clinical and convergent validity in patients with various neuromuscular disorders. The functional abilities and the Rankin scale showed substantial interobserver agreement (weighted kappa (Kw) = 0.76 and 0.73, respectively), whereas almost perfect agreement was found in the Hauser ambulation index (Kw = 0.95), GBS leg index (Kw = 0.84), and Rivermead mobility index (ICC = 0.82). Homogeneity of the Rivermead mobility index was high (Cronbach's alpha = 0.91). Receiver Operator Characteristics curves were constructed and areas under the curves (AUCs) calculated to investigate the association between the scores on the disability measures and dichotomised scores (mild to moderate disability versus severe disability) of the physical dimension of the extensively validated Sickness Impact Profile (SIP). Points estimates of the AUCs (0.79 - .91) reveal that the instruments were able to discriminate patients with a mild to moderate level of disability versus a severe level of disability. The highest correlations between the selected disability measures and the physical dimension of SIP were observed in the Hauser ambulation index (Spearman correlation coefficient (rs = 0.76) and the Rivermead mobility index (Pearson's r = -0.75).
PURE MOTOR CIDP. A DISTINCT ENTITY?
Sabatelli M., Mignogna T., Lippi G., De Armas L., Madia F., Mereu M.L., Tonali P. Istituto di Neurologia. Università Cattolica. Rome. Italy.
The spectrum of clinical, pathological and electrophysiological features of CIDP is very heterogeneous. The onset may be acute or slowly progressive and clinical course monophasic or relapsing-remitting. Motor-sensory, ataxic sensory and pure motor variants have been described. The importance of the classification of CIDP is not an academic one since each subtype might respond to specific treatments. We describe clinical features and long term follow-up of 4 patients affected by pure motor CIDP. 4 out of our 30 CIDP patients showed no clinical, electrophysiological and (in two patients) sural nerve biopsy features of sensory fibers involvement. All four patients disclosed similar clinical and electrophysiological features: the age of onset was before 30 years, clinical course was relapsing-remitting and electrophyisiological examination showed features of pure motor demyelinating neuropathy with normal distal CMAP amplitude and clear evidence of conduction blocks. None of them showed significant response to corticosteroids, while high dose intravenous immunoglobulins were very effective in all patients. Our findings suggest that pure motor CIDP represent a distinct entity. Supported by Telethon Italy and U.IL.D.M. Rome.
TREATMENT OF MULTIFOCAL MOTOR NEUROPATHY WITH INTERFERON BETA-1A
van den Berg-Vos R.M., van den Berg L.H., Franssen H., Wokke J.H.J., van Doom. P.A.(1), Martina I.S.J.(1). University Hospital of Utrecht, (1)University Hospital of Rotterdam, the Netherlands
BACKGROUND: Intravenous immunoglobulins (IVIg) treatment has shown to be effective as maintenance treatment in multifocal motor neuropathy (MMN), but is expensive and frequent infusions may be aggravating for patients. Interferon beta-1a is an effective treatment in multiple sclerosis, a multifocal demyelinating disease of the central nervous system. OBJECTIVE: To study whether interferon beta-1a (Rebif(R) ) is a comparable or better treatment in MMN than IvIg. METHODS: Ten patients were included in this open study of 6 months duration and were treated with Rebif(R), 6 MIU 3 times subcutaneously a week. Muscle strength, dexterity, ambulation and disability were evaluated every three weeks during treatment. Electrophysiological examination was done before and after treatment. RESULTS: Side-effects were moderate and consisted of flu-like symptoms and skin reactions on injection sites, gradually improving after several weeks of treatment. In 7 patients there was no effect of treatment. Six of these patients had had IVIg as maintenance treatment and deteriorated such that IvIg had to be restarted during Rebif(R) treatment. Three patients with shorter disease durations showed some improvement. CONCLUSIONS: Treatment of MMN with Rebif(R) is not as effective as IVIg, although a subgroup of patients with shorter disease durations may show some improvement.
INTRAVENOUS IMMUNOGLOBULIN (IVIg) IN THE TREATMENT OF MULTIFOCAL MOTOR NEUROPATHY : A RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED STUDY OF 19 PATIENTS
Léger J.M., Chassande B., Musset L., Keime F., Le Forestier N., Bagot d'Arc M., Bouche P., Baumann N. Hôpital de la Sâlpetrière Paris, France.
Patients fulfilling the eligibility criteria for MMNCB were
enrolled and stratified according to whether they had been previously
treated with IVIg (group 2 : 9 patients) or not (group 1 : 10
patients). They were randomized to receive either IVIg (Endobulin,
Baxter) or placebo at dose of 500 mg/kg/day for 5 consecutive
days, every month for 3 months. At month 4, patients found to
be non-responders were switched to the other treatment, while
responders kept the same treatment, both for the following 3 months.
Clinical assessment was conducted on MRC score in 28 muscles and
self-evaluation scale (5 daily activities scored from 0 to 5).
Electrophysiological studies and anti-GM1 antibody titers were
performed every month for 7 months. In group 1, 9/10 patients
completed the study of whom 5 received firstly placebo and 4 IVIg.
Four patients responded to IVIg : 2 during the first and 2 during
the second course, while 2 other patients responded to placebo.,
The last 3 patients were non-responders. In group 2, the 4 patients
treated firstly with placebo switched at month 4, while the 5
patients treated firstly with IVIg kept their treatment. In summary,
7 of the 9 patients receiving IVIg as the first treatment were
responders, versus 2 out of the 9 patients receiving placebo (p=0.03).
In addition, there was a significant improvement of the only self-evaluation
scale in the IVIg group at month 4. No significant changes in
anti-GM1 titers were observed in the responders, but the 5 patients
with anti-GM1 titers > 1/3200 were always responders to IVIg
in the 2 groups.
Supported by: Baxter and BIOMED Contract (European Community)
N°BMH4-CT96-0324
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