Long-term beneficial effects of mirabegron in pediatric patients with therapy-refractory neurogenic lower urinary tract dysfunction.

INTRODUCTION: Neurogenic lower urinary tract dysfunction (NLUTD) in children can cause renal failure and urinary incontinence if not treated sufficiently. Antimuscarinics (AM) and intradetrusor botulinum toxin injections (BoNT-A) with clean intermittent catheterization (CIC) are widely used treatment options for children with NLUTD. However, a considerable number will become refractory to these treatment options. This study aimed to evaluate the efficacy and long-term outcomes of mirabegron in children with NLUTD as an add-on and as a stand-alone treatment. MATERIAL AND METHODS: Patients under 18 years of age with NLUTD who were refractory to AM and/or BoNT-A and were treated with mirabegron 50 mg were retrospectively studied. Mirabegron was either used as monotherapy or in addition to AM and/or BoNT-A. Video-urodynamic studies (VUDSs) were performed before and after treatment with mirabegron. Changes in video-urodynamic parameters, the need for other NLUTD therapy during follow-up, patient-reported side effects, and urinary incontinence were outcomes of interest. RESULTS: A total of 34 patients with NLUTD were included. All patients were on CIC and the median age was 13.1 years (IQR 15.9-10.3). Median follow-up was 31.4 months (IQR 57.4-11.4). Bladder compliance improved by 89.9%, from 14.9 to 28.3 ml/cm H2O (p-value<0.001). Maximum cystometric capacity, end-filling detrusor pressure, volume at first detrusor overactivity, vesicoureteral reflux, and urinary incontinence significantly improved after mirabegron. The add-on therapy group showed more significant improvements in video-urodynamic outcomes compared to the monotherapy group. The median time of requiring other NLUTD therapy was 25.5 months (IQR 39.8-14.8). None of the included patients reported side effects. CONCLUSIONS: Mirabegron is an effective treatment for children with therapy-refractory NLUTD with an average efficacy of 2 years after which additional therapy is required. Despite the retrospective character of this study, our results confirm the beneficial effect of mirabegron in children with therapy-refractory NLUTD, in particular when mirabegron is used as add-on therapy in those with low-compliance bladders.

Does conus morphology have implications for outcome in lumbosacral lipoma?

OBJECTIVE: Lumbosacral lipomas (LSL) of the 'dorsal' type have been associated with more favourable outcomes compared with other conus region lipomas. We hypothesised that integrity of the conus on MRI underpins the improved prognosis in this subgroup of LSL patients. METHODS: The definition of 'dorsal lipomas' included lipomas with attachment to the conus, but where the conus could be delineated on MRI (Morota type 1) as reported by Morota et al. (J Neurosurg Pediatr 19:428-439, 2017). Additional inclusion criteria included asymptomatic status at presentation, age >3 years at follow-up, and neurological and urological evaluation at presentation and at last follow-up. Lipoma extent and conus level were recorded. Outcome measures were the need for untethering surgery and neuro-urological status at last follow-up. Urological outcomes were defined by continence and efficacy of bladder emptying. RESULTS: Twenty-six children were included (median age 8.7 years). Conus level was low (at or below L2) in 92%. Nine required untethering surgery: 5 prophylactic, 4 because of clinical deterioration. Twenty-five children were continent at last follow-up, one had stress incontinence, and none required catheterisation. One had persisting ankle weakness after surgery requiring orthotic support. CONCLUSIONS: In LSL of the conus, visualisation of the conus on MRI is associated with good urological and motor outcomes. The integrity of the conus appears to be a more important prognostic factor than anatomical level. An observational approach to this group of LSL patients does not appear to compromise outcomes. These findings support a selective approach to untethering surgery.

A Patient (46XX) With Congenital Adrenal Hyperplasia and Prostate Cancer: A Case Report.

Congenital adrenal hyperplasia (CAH) can affect sex characteristics. The most common cause of CAH is 21-hydroxylase deficiency, and the cornerstone of treatment is glucocorticoid replacement in adrenocorticotropic hormone-suppressive dosages. A 64-year-old patient (46XX) with CAH resulting from 21-hydroxylase deficiency had been treated with dexamethasone and testosterone since diagnosis at age 12 and was phenotypically male. At age 62, he was diagnosed with prostate carcinoma. The patient received curative treatment with external beam radiotherapy. Genotypically female patients with CAH can develop prostate carcinoma when receiving long-term testosterone replacement therapy.

[A man with a swollen right breast]. / Een man met een gezwollen rechter borst.

A 43-year-old man presented with a rapidly growing right breast. Physical examination and radiological findings demonstrated a subpectoral giant lipoma of the breast. We performed surgical enucleation of the tumour, which weighed 340 g and had a diameter of 20 cm. Histopathological examination confirmed the diagnosis.

Recurrence of ventricular arrhythmias in ischaemic secondary prevention implantable cardioverter defibrillator recipients: long-term follow-up of the Leiden out-of-hospital cardiac arrest study (LOHCAT).

AIMS: To assess the long-term rate of mortality and the recurrence of potentially life-threatening ventricular arrhythmias in secondary prevention implantable cardioverter defibrillator (ICD) patients and to construct a model for baseline risk stratification. METHODS AND RESULTS: Since 1996, all patients with ischaemic heart disease, receiving ICD therapy for secondary prevention of sudden death, were included in the current study. Patients were evaluated at implantation and during long-term follow-up. A total of 456 patients were included in the analysis and followed for 54 +/- 35 months. During follow-up, 100 (22%) patients died and ICD therapy was noted in 216 (47%) patients, of which 138 (30%) for fast, potentially life-threatening ventricular arrhythmia. Multivariate analysis revealed a history of atrial fibrillation or flutter (AF), ventricular tachycardia as presenting arrhythmia, and wide QRS and poor left ventricular ejection fraction as independent predictors of life-threatening ventricular arrhythmias. The strongest predictor was AF with a hazard ratio of 2.1 (95% confidence interval 1.3-3.2). On the basis of the available clinical data, it was not possible to identify a group which exhibited no risk on recurrence of potentially life-threatening ventricular arrhythmias. CONCLUSION: Ischaemic secondary prevention ICD recipients exhibit a high recurrence rate of potentially life-threatening ventricular arrhythmias. Factors that increase risk can be identified but, even with these factors, it was not possible to distinguish a recurrence-free group.