No abstract

MS in juvenile patients under the age of 16 occurred in 31 (5%) of our whole MS population of 620 patients in the time from 1975-1991. It does not differ clinically from the disease as observed in 72 patients with later onset MS in respect to symptoms at onset, course, progression rate, rate of relapses and abnormalities in CSF and MRI. However, fever, headache, nausea and vomiting with pleocytosis in CSF during the first episode and development of oligoclonal bands with passage of time may be characteristic in some juvenile patients. The presence of oligoclonal bands and MRI results are of high diagnostic value in this special group of patients.

IntroductionEvidence from a number of open-label, uncontrolled studies has suggested that rituximab may benefit patients with autoimmune diseases who are refractory to standard-of-care. The objective of this study was to evaluate the safety and clinical outcomes of rituximab in several standard-of-care-refractory autoimmune diseases (within rheumatology, nephrology, dermatology and neurology) other than rheumatoid arthritis or non-Hodgkin's lymphoma in a real-life clinical setting.MethodsPatients who received rituximab having shown an inadequate response to standard-of-care had their safety and clinical outcomes data retrospectively analysed as part of the German Registry of Autoimmune Diseases. The main outcome measures were safety and clinical response, as judged at the discretion of the investigators.ResultsA total of 370 patients (299 patient-years) with various autoimmune diseases (23.0% with systemic lupus erythematosus, 15.7% antineutrophil cytoplasmic antibody-associated granulomatous vasculitides, 15.1% multiple sclerosis and 10.0% pemphigus) from 42 centres received a mean dose of 2,440 mg of rituximab over a median (range) of 194 (180 to 1,407) days. The overall rate of serious infections was 5.3 per 100 patient-years during rituximab therapy. Opportunistic infections were infrequent across the whole study population, and mostly occurred in patients with systemic lupus erythematosus. There were 11 deaths (3.0% of patients) after rituximab treatment (mean 11.6 months after first infusion, range 0.8 to 31.3 months), with most of the deaths caused by infections. Overall (n = 293), 13.3% of patients showed no response, 45.1% showed a partial response and 41.6% showed a complete response. Responses were also reflected by reduced use of glucocorticoids and various immunosuppressives during rituximab therapy and follow-up compared with before rituximab. Rituximab generally had a positive effect on patient well-being (physician's visual analogue scale; mean improvement from baseline of 12.1 mm).ConclusionsData from this registry indicate that rituximab is a commonly employed, well-tolerated therapy with potential beneficial effects in standard of care-refractory autoimmune diseases, and support the results from other open-label, uncontrolled studies.

A cross-sectional cost-of-care study was performed to assess the economic burden of multiple sclerosis (MS) in France, Germany and the UK. Patients were stratified into 3 groups according to the Expanded Disability Severity Scale (EDSS): stages I, II and III, corresponding to mild (EDSS 1.0 to 3.5), moderate (EDSS 4.0 to 6.0) and severe (EDSS 6.5 to 8.0) MS, respectively. 90 patients with MS and 30 non-MS control patients were recruited in each country. Control patients were matched to the patients with MS on the basis of age and gender. Demographic, clinical and economic data during the 3-month period prior to entry were collected in patient interviews. Total costs included actual expenditures, such as direct medical and non-medical costs, as well as indirect costs. From the societal perspective, the total cost of MS for 3 months was estimated at 1,928 US dollars, 3,941 US dollars and 5,678 US dollars in France, 2,772 US dollars, 2,056 dollars and 5701dollars in Germany, and 5,125 US dollars, 6,751 US dollars and 14, 622 US dollars in the UK, for stage I, II and III patients, respectively. The major medical cost driver in the UK was outpatient consultations, whereas hospitalisations were the major component in Germany and France. The major cost in the UK arose from the dependence of patients with MS on caregivers, which caused high non-medical, societal costs compared with France and Germany. From both the societal and health insurance perspectives in each country, costs for control patients were lower than those for stage I MS patients. MS represents a major financial burden on the individual, the family, health services and society, and these costs increase with MS progression.

Whereas treatment for spasticity and depression is common in our cohort, sexual dysfunction, dysphagia, cognitive dysfunction, and fatigue are treated to a far lesser extent. The need for psychological support, physical, and occupational therapy has to be recognized as neuropsychological symptoms have a great impact on retirement at an early stage. Overall symptomatic treatment rates for the most common symptoms have increased over the last years ( p < 0.001).