Treatment of chronic inflammatory demyelinating polyneuropathy.

Purpose Of Review: Chronic inflammatory demyelinating poly(radiculo)neuropathy (CIDP) is a treatable disorder. There are three proven effective treatments available. Randomized controlled trials have only focused on short-term effects, but most patients need long-term therapy.

Perception of prognostic risk in patients with multiple sclerosis: the relationship with anxiety, depression, and disease-related distress.

Objective: The aim of the study was to investigate the impact of perception of prognostic risk on anxiety, depression, and disease-related distress in patients with multiple sclerosis (MS).

Study Design And Setting: Perceived risk and perceived seriousness of the 2-year, 10-year, and lifetime prognosis of wheelchair dependence, disability status, anxiety, depression, and disease-related distress were assessed in 101 patients. Distress was measured as the intrusion and avoidance of MS-related thoughts and feelings.

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.

Objective: Recent reports warn that the worldwide cell culture capacity is insufficient to fulfill the increasing demand for human protein drugs. Production in milk of transgenic animals is an attractive alternative. Kilogram quantities of product per year can be obtained at relatively low costs, even in small animals such as rabbits.

Functional polymorphisms in LPS receptors CD14 and TLR4 are not associated with disease susceptibility or Campylobacter jejuni infection in Guillain-Barré patients.

Guillain-Barré syndrome (GBS) is an acute immune-mediated polyneuropathy preceded by infections. Campylobacter jejuni is the most frequent pathogen and its lipopolysaccharide (LPS) induces antibodies cross-reactive with gangliosides. In this study we assessed whether known functional polymorphisms in the LPS receptors CD14 and Toll-like receptor 4 (TLR4) are associated with an increased susceptibility for GBS or with C.

Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up.

Pompe's disease is an autosomal recessive myopathy. The characteristic lysosomal storage of glycogen is caused by acid alpha-glucosidase deficiency. Patients with late-onset Pompe's disease present with progressive muscle weakness also affecting pulmonary function.

Chlamydia pneumoniae and the risk for exacerbation in multiple sclerosis patients.

In this prospective study of 73 relapsing remitting multiple sclerosis patients followed up for a mean of 1.7 years, the relation was tested between serologically defined Chlamydia pneumoniae (CP) infection periods and exacerbation rate. Episodes of serologically defined CP infections were observed in a subgroup, and these episodes were associated with increased risk for exacerbation.

Comparison between impairment and disability scales in immune-mediated polyneuropathies.

The ability of a scale to detect clinical relevant changes over time, i.e., its "responsiveness," may help clinicians to choose among valid and reliable measures.

Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy.

Pompe's disease (glycogen storage disease type II) is an autosomal recessive myopathy caused by lysosomal alpha-glucosidase deficiency. Enzyme replacement therapy (ERT) is currently under development for this disease. We evaluated the morphological changes in muscle tissue of four children with infantile Pompe's disease who received recombinant human alpha-glucosidase from rabbit milk for 72 weeks.

Anti-GM1 IgG antibodies induce leukocyte effector functions via Fcgamma receptors.

Guillain-Barré syndrome (GBS) is an immune-mediated neuropathy, in which leukocytes and humoral components of the immune system proposedly initiate localized inflammation. An important pathogenic role for anti-GM1 ganglioside antibodies has been suggested. Therefore, we evaluated anti-GM1 IgG antibody-induced leukocyte effector functions such as degranulation and phagocytosis using serum of 24 GBS patients.

Screening for anti-ganglioside antibodies in hypocretin-deficient human narcolepsy.

Narcolepsy is a sleep disorder caused by defective hypocretin (orexin) neurotransmission. It is thought to result from an autoimmune destruction of hypocretin producing neurons. Recently, low hypocretin levels were found in patients with Guillain-Barré syndrome, a post-infectious immune-mediated disorder in which a variety of circulating antibodies against neuronal gangliosides are found.

Treatment of immune neuropathies.

Purpose Of The Review: Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating poly(radiculo)neuropathy (CIDP) and multifocal motor neuropathy (MMN) are potentially treatable disorders. The use of appropriate assessment scales to evaluate the effects of treatment is essential. Recent therapeutic trials and the question of whether patients with mild disease or other variants of these disorders need to be treated are discussed.

Comparison of weakness progression in inclusion body myositis during treatment with methotrexate or placebo.

We investigated whether 5 to 20mg per week oral methotrexate could slow down disease progression in 44 patients with inclusion body myositis in a randomized double-blind placebo-controlled study over 48 weeks. Mean change of quantitative muscle strength testing sum scores was the primary study outcome measure. Quantitative muscle strength testing sum scores declined in both treatment groups, -0.

Psychometric evaluation of a new handicap scale in immune-mediated polyneuropathies.

A new handicap measure, the Rotterdam nine-item handicap scale, was developed and its validity, reliability, and responsiveness evaluated in patients with immune-mediated polyneuropathies. We evaluated 113 stable patients, of whom 83 had Guillain--Barré syndrome (GBS), 22 had chronic inflammatory demyelinating polyneuropathy (CIDP), and 8 had a gammopathy-related polyneuropathy. We also studied 20 patients with recently diagnosed GBS (n = 7) or CIDP (n = 13) and changing clinical conditions (longitudinal group).