A randomized clinical trial indicates that levamisole increases the time to relapse in children with steroid-sensitive idiopathic nephrotic syndrome

Levamisole has been considered the least toxic and least expensive steroid-sparing drug for preventing relapses of steroid-sensitive idiopathic nephrotic syndrome (SSINS). However, evidence for this is limited as previous randomized clinical trials were found to have methodological limitations. Therefore, we conducted an international multicenter, placebo-controlled, double-blind, randomized clinical trial to reassess its usefulness in prevention of relapses in children with SSINS. The efficacy and safety of one year of levamisole treatment in children with SSINS and frequent relapses were evaluated. The primary analysis cohort consisted of 99 patients from 6 countries. Between 100 days and 12 months after the start of study medication, the time to relapse (primary endpoint) was significantly increased in the levamisole compared to the placebo group (hazard ratio 0.22 [95% confidence interval 0.11-0.43]). Significantly, after 12 months of treatment, six percent of placebo patients versus 26 percent of levamisole patients were still in remission. During this period, the most frequent serious adverse event (four of 50 patients) possibly related to levamisole was asymptomatic moderate neutropenia, which was reversible spontaneously or after treatment discontinuation. Thus, in children with SSINS and frequent relapses, levamisole prolonged the time to relapse and also prevented recurrence during one year of treatment compared to prednisone alone. However, regular blood controls are necessary for safety issues

Long-term follow-up of thalamic stimulation versus thalamotomy for tremor suppression

Thalamic stimulation and thalamotomy for treatment of tremor due to Parkinson's disease, essential tremor, and multiple sclerosis were compared in a randomized trial. The symptomatic and functional outcome was studied after 5 years of follow-up. Sixty-eight patients were treated (45 Parkinson's disease, 13 essential tremor, 10 multiple sclerosis) by thalamotomy (n = 34) or thalamic stimulation (n = 34). After 5 years, 48 patients were available for follow-up. The primary outcome measure was change in functional status measured by the Frenchay Activities Index (FAI), scores ranging from 0 to 60. Secondary outcome measures were tremor severity, frequency of complications, and patients' assessment of the outcome. The mean difference in FAI scores between thalamic stimulation and thalamotomy was 4.4 (95% CI: 1.1-7.7) after 6 months, 3.3 (95% CI: -0.03-6.6) after 2 years and 4.0 (95% CI: 0.3-7.7) after 5 years in favor of stimulation. Tremor suppression was equally effective after both procedures, and stable in Parkinson patients. In ET and multiple sclerosis, a diminished effect of stimulation was observed in half of the patients. There were six stimulation equipment-related complications, but neurological side effects of surgery were higher after thalamotomy. Subjective outcome-assessment by the patients was more favorable in the stimulation group. (C) 2008 Movement Disorder Societ

Unified Parkinson's disease rating scale motor examination: are ratings of nurses, residents in neurology, and movement disorders specialists interchangeable?

The Unified Parkinson's Disease Rating Scale (UPDRS) is widely used for the clinical evaluation of Parkinson's disease (PD). We assessed the rater variability of the UPDRS Motor examination (UPDRS-ME) of nurse practitioners, residents in neurology, and a movement disorders specialist (MDS) compared to a senior MDS. We assessed the videotaped UPDRS-ME of 50 PD patients. Inter-rater and intra-rater variability were estimated using weighted kappa (kappa(w)) and intraclass correlation coefficients (ICC). Additionally, inter-rater agreement was quantified by calculation of the mean difference between 2 raters and its 95% limits of agreement. Intra-rater agreement was also estimated by calculation of a 95% repeatability limits. The kappa(w) and ICC statistics indicated good to very good inter-rater and intra-rater reliability for the majority of individual UPDRS items and the sum score of the UPDRS-ME in all raters. However, for inter-rater agreement, it appeared that both nurses, residents, and the MDS consistently assigned higher scores than the senior MDS. Mean differences ranged between 1.7 and 5.4 (all differences P < 0.05), with rather wide 95% limits of agreement. The intra-rater 95% repeatability limits were rather wide. We found considerable rater difference for the whole range of UPDRS-ME scores between a senior MDS and nurse practitioners, residents in neurology, and the MDS. This finding suggests that the amount by which raters may disagree should be quantified before starting longitudinal studies of disease progression or clinical trials. Finally, evaluation of rater agreement should always include the assessment of the extent of bias between different rater

Quality of life in patients with vascular malformations of the lower extremity

With the exception of capillary malformations (port-wine stains), the adverse psychosocial effects of vascular malformations have not received much attention in the medical literature. We, therefore, studied health related quality of life of patients with vascular malformations located primarily on the lower extremity, who presented to us over a 10-year period. Patient's self-assessment of quality of life was measured by the Multiple Outcomes Study (MOS) Short Form Health Survey Questionnaire (SF-36(TM)). Several possible predictors were also examined. Eighty-one patients (33 mate, 48 female), aged 14-61 years, completed the SF-36. Seventy-one patients (88%) had a low flow lesion. Twenty-three patients (28%) had hypertrophy of the tower extremity, while 11 patients (14%) had hypotrophy. Sixty-nine patients (85%) had the vascular malformation located only on the tower extremity. Six (7%) also had their upper extremity (hand) involved, and 20 patients (24.7%) had > 10% TBSA affected. Eight patients (10%) needed special shoes. Fifty-one patients (63%) had a previously performed MRI, of which 62.7% (32/51) had muscle involvement. Fifty-one patients (63%) had been operated on. Of the 34 patients wearing elastic compression stockings, 25 patients (74%) indicated that they were satisfied with the stockings. Compared to the general population sample, the SF-36 questionnaire indicated that our vascular malformation patients reported impaired vitality and higher levels of pain, white no differences were seen regarding the other dimensions of quality of life. Demographic, clinical and therapy characteristics could explain quality of life only to a limited extent. Explained variability of the SF-36 dimensions ranged from 0% for mental health to 34.5% for rote functioning physical. To our surprise, this study suggests that patients with vascular malformations located primarily on the lower extremity do not have a greatly decreased quality of life when compared to the general Dutch population. (C) 2004 The British Association of Plastic Surgeons. Published by Elsevier Ltd. All rights reserve

Effect of Hydrocortisone Therapy Initiated 7 to 14 Days After Birth on Mortality or Bronchopulmonary Dysplasia Among Very Preterm Infants Receiving Mechanical Ventilation A Randomized Clinical Trial

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Effect of Systemic Hydrocortisone Initiated 7 to 14 Days after Birth in Ventilated Preterm Infants on Mortality and Neurodevelopment at 2 Years' Corrected Age: Follow-up of a Randomized Clinical Trial

This follow-up to the STOP-BPD randomized clinical trial, which investigated the efficacy and safety of systemic hydrocortisone treatment initiated 7 to 14 days after birth in ventilator-dependent, very preterm infants in the Netherlands and Belgium at 36 weeks’ postmenstrual age, examines the prespecified composite of death and neurodevelopmental impairment among these infants at 2 years’ corrected age

A randomized clinical trial indicates that levamisole increases the time to relapse in children with steroid-sensitive idiopathic nephrotic syndrome

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Systemic Hydrocortisone To Prevent Bronchopulmonary Dysplasia in preterm infants (the SToP-BPD study): statistical analysis plan

Abstract Background Bronchopulmonary dysplasia (BPD) is the most common complication of preterm birth with short-term and long-term adverse consequences. Although the glucocorticoid dexamethasone has been proven to be beneficial for the prevention of BPD, there are concerns about an increased risk of adverse neurodevelopmental outcome. Hydrocortisone has been suggested as an alternative therapy. The aim of the Systemic Hydrocortisone To Prevent Bronchopulmonary Dysplasia in preterm infants (SToP-BPD) trial is to assess the efficacy and safety of postnatal hydrocortisone administration for the reduction of death or BPD in ventilator-dependent preterm infants. Methods/design The SToP-BPD study is a multicentre, double-blind, placebo-controlled hydrocortisone trial in preterm infants at risk for BPD. After parental informed consent is obtained, ventilator-dependent infants are randomly allocated to hydrocortisone or placebo treatment during a 22-day period. The primary outcome measure is the composite outcome of death or BPD at 36 weeks postmenstrual age. Secondary outcomes are short-term effects on pulmonary condition and long-term neurodevelopmental sequelae assessed at 2 years corrected age. Complications of treatment, other serious adverse events and suspected unexpected serious adverse reactions are reported as safety outcomes. This pre-specified statistical analysis plan was written and submitted without knowledge of the unblinded data. Trial registration Netherlands Trial Register, NTR2768. Registered on 17 February 2011. EudraCT, 2010-023777-19. Registered on 2 November 2010

Gut-directed hypnotherapy in children with irritable bowel syndrome or functional abdominal pain (syndrome): a randomized controlled trial on self exercises at home using CD versus individual therapy by qualified therapists

Irritable bowel syndrome (IBS) and functional abdominal pain (syndrome) (FAP(S)) are common pediatric disorders, characterized by chronic or recurrent abdominal pain. Treatment is challenging, especially in children with persisting symptoms. Gut-directed hypnotherapy (HT) performed by a therapist has been shown to be effective in these children, but is still unavailable to many children due to costs, a lack of qualified child-hypnotherapists and because it requires a significant investment of time by child and parent(s). Home-based hypnotherapy by means of exercises on CD has been shown effective as well, and has potential benefits, such as lower costs and less time investment. The aim of this randomized controlled trial (RCT) is to compare cost-effectiveness of individual HT performed by a qualified therapist with HT by means of CD recorded self-exercises at home in children with IBS or FAP(S). 260 children, aged 8-18 years with IBS or FAP(S) according to Rome III criteria are included in this currently conducted RCT with a follow-up period of one year. Children are randomized to either 6 sessions of individual HT given by a qualified therapist over a 3-month period or HT through self-exercises at home with CD for 3 months.The primary outcome is the proportion of patients in which treatment is successful at the end of treatment and after one year follow-up. Treatment success is defined as at least 50% reduction in both abdominal pain frequency and intensity scores. Secondary outcomes include adequate relief, cost-effectiveness and effects of both therapies on depression and anxiety scores, somatization scores, QoL, pain beliefs and coping strategies. If the effectiveness of home-based HT with CD is comparable to, or only slightly lower, than HT by a therapist, this treatment may become an attractive form of therapy in children with IBS or FAP(S), because of its low costs and direct availability. Dutch Trial Register number NTR2725 (date of registration: 1 February 2011