The role of calcium accumulation in the pathogenesis of Duchenne muscular dystrophy (DMD) has already been discussed. Several trials with different calcium-blocking drugs have revealed no clinical benefit. In addition, the present study includes histological investigations and computer tomography to verify therapeutic effects. In a randomized placebo-controlled double-blind study, 13 DMD patients aged from 3-10 years (mean, 7 years) were treated with 5 mg/kg diltiazem daily for 1 year. Compared with before therapy, the number of calcium-positive muscular fibres was remarkably reduced in the treated DMD patients, but not in the placebo group. The evaluation of all other biochemical and clinical parameters revealed no significant effects of the diltiazem therapy. The muscular X-ray density measured by computer tomography decreased under treatment. After the evaluation of the double-blind study, the code was broken. Therapy, however, was continued in the treated group and started in the placebo group. After 3 years of diltiazem therapy the clinical status of all 26 patients of the study and 20 additional DMD patients who were treated with diltiazem was compared with 46 untreated DMD patients of the same age and stage in our department. No obvious clinical benefit of diltiazem therapy could be observed.

Nine severely ill patients with a confirmed diagnosis of systemic juvenile rheumatoid arthritis were treated with recombinant y-interferon (y-IFN) in addition to the therapy they were previously receiving for their disease. Improvements in clinical symptoms were noted in 7 of the patients, and median laboratory values also showed a marked improvement after y-IFN treatment. A relapse occurred in 1 patient. The results of this study should stimulate further research on the use of y-IFN in systemic juvenile rheumatoid arthritis, particularly in determining the appropriate effective dosage.Over 800 adults with rheumatoid arthritis (RA) have been treated with y-interferon (yIFN) in Germany since 1983 (Brzoska J: unpublished data). Severe side effects have not been observed. In a recent placebo-controlled double-blind trial, 58% of the RA patients responded to y-IFN (I). However, despite its apparent efficacy, its mode of action is not yet clearly understood (2). The present pilot study of y-IFN was Submitted for publication April 5, 1988; accepted in revised form December 13, 1988. performed with patients with systemic juvenile rheumatoid arthritis (JRA), since this disease often has a high morbidity and mortality rate ( 3 ) despite the current methods of treatment, which include azathioprine, gold, D-penicillamine, and cytotoxic drugs.Patients and methods. Nine severely ill patients with a confirmed diagnosis of systemic JRA according to the criteria of the American Rheumatism Association (4) were selected for study. They had not responded satisfactorily to nonsteroidal antiinflammatory drugs (NSAIDs) and immunosuppressive therapy, and therefore had been given corticosteroids for long-term treatment (Table 1). In addition to their current regimen, the patients were treated with recombinant y-IFN (Biogen, Cambridge, MA, through Bioferon, Laupheim, FRG). The initial dosage during the first 3 weeks was 0.5 pglkg body weight, daily, administered subcutaneously; the same dosage was given for 3 further weeks 3 times a week. Then, depending upon each patient's response to therapy, the dosage was individually reduced by lowering the frequency of injections to either twice or once a week. Clinical parameters, joint function, and laboratory data (hemoglobin level, leukocyte and thrombocyte count, erythrocyte sedimentation rate [ESR], levels of C-reactive protein [CRP] , serum glutamic oxaloacetic transaminase, serum glutamic pyruvic transaminase, and lactate dehydrogenase, and urinalysis) were assessed before initiating y-IFN treatment and once monthly during therapy.Results. Diverse improvements were observed in 7 of 9 patients who had a history of systemic JRA progressing over several years. These patients' general condition improved during the first weeks of treatment. In 2 boys, intermittent attacks of fever

Group A streptococcal (GAS) disease shows increasing incidence worldwide. We characterised children admitted with GAS infection to European hospitals and studied risk factors for severity and disability. This is a prospective, multicentre, cohort study (embedded in EUCLIDS and the Swiss Pediatric Sepsis Study) including 320 children, aged 1 month to 18 years, admitted with GAS infection to 41 hospitals in 6 European countries from 2012 to 2016. Demographic, clinical, microbiological and outcome data were collected. A total of 195 (61%) patients had sepsis. Two hundred thirty-six (74%) patients had GAS detected from a normally sterile site. The most common infection sites were the lower respiratory tract (LRTI) (22%), skin and soft tissue (SSTI) (23%) and bone and joint (19%). Compared to patients not admitted to PICU, patients admitted to PICU more commonly had LRTI (39 vs 8%), infection without a focus (22 vs 8%) and intracranial infection (9 vs 3%); less commonly had SSTI and bone and joint infections ( p < 0.001); and were younger (median 40 (IQR 21–83) vs 56 (IQR 36–85) months, p = 0.01). Six PICU patients (2%) died. Sequelae at discharge from hospital were largely limited to patients admitted to PICU (29 vs 3%, p < 0.001; 12% overall) and included neurodisability, amputation, skin grafts, hearing loss and need for surgery. More patients were recruited in winter and spring ( p < 0.001). Conclusion : In an era of observed marked reduction in vaccine-preventable infections, GAS infection requiring hospital admission is still associated with significant severe disease in younger children, and short- and long-term morbidity. Further advances are required in the prevention and early recognition of GAS disease. What is Known: • Despite temporal and geographical variability, there is an increase of incidence of infection with group A streptococci. However, data on the epidemiology of group A streptococcal infections in European children is limited. What is New: • In a large, prospective cohort of children with community-acquired bacterial infection requiring hospitalisation in Europe, GAS was the most frequent pathogen, with 12% disability at discharge, and 2% mortality in patients with GAS infection. • In children with GAS sepsis, IVIG was used in only 4.6% of patients and clindamycin in 29% of patients. Supplementary Information The online version contains supplementary material available at 10.1007/s00431-022-04718-y.

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