Endocrinopathies in paediatric-onset neuromyelitis optica spectrum disorder with aquaporin 4 (AQP4) antibody.

The involvement of the diencephalic regions in neuromyelitis optica spectrum disorder (NMOSD) may lead to endocrinopathies. In this study, we identified the following endocrinopathies in 60% (15/25) of young people with paediatric-onset aquaporin 4-Antibody (AQP4-Ab) NMOSD: morbid obesity ( n = 8), hyperinsulinaemia ( n = 5), hyperandrogenism ( n = 5), amenorrhoea ( n = 5), hyponatraemia ( n = 4), short stature ( n = 3) and central hypothyroidism ( n = 2) irrespective of hypothalamic lesions. Morbid obesity was seen in 88% (7/8) of children of Caribbean origin. As endocrinopathies were prevalent in the majority of paediatric-onset AQP4-Ab NMOSD, endocrine surveillance and in particular early aggressive weight management is required for patients with AQP4-Ab NMOSD.

Sativex in resistant multiple sclerosis spasticity: Discontinuation study in a large population of Italian patients (SA.FE. study).

BACKGROUND: The approval of Sativex for the management of multiple sclerosis (MS) spasticity opened a new opportunity to many patients. In Italy, the healthcare payer can be fully reimbursed by the involved pharma company with the cost of treatment for patients not responding after a 4 week (28 days) trial period (Payment by Results, PbR), and 50% reimbursed with the cost of 6 weeks (42 days) treatment for other patients discontinuing (Cost Sharing, CS). The aim of our study was to describe the Sativex discontinuation profile from a large population of spasticity treated Italian MS patients. METHODS: We collected data of patients from 30 MS centres across the country starting Sativex between January 2014 and February 2015. Data were collected from the mandatory Italian Medicines Agency (AIFA) web-registry. Predictors of treatment discontinuation were assessed using a multivariate Cox proportional regression analysis. RESULTS: During the observation period 631 out of 1597 (39.5%) patients discontinued Sativex. The Kaplan-Meier estimates curve showed that 333 patients (20.8%) discontinued treatment at 4 weeks while 422 patients (26.4%) discontinued at 6 weeks. We found after adjusted modeling that a higher NRS score at T1 (adjHR 2.23, 95% 2.07-2.41, p<0.001) and a lower baseline NRS score (adjHR 0.51 95% CI 0.46-0.56, p<0.001) were predictive of treatment discontinuation. CONCLUSION: These data show that the first 6 weeks are useful in identifying those patients in which Sativex could be effective, thus avoiding the cost of longer term evaluation.

The pharmacokinetics of glatiramer acetate for multiple sclerosis treatment.

INTRODUCTION: Multiple sclerosis (MS) is a T-cell-mediated disease affecting the central nervous system (CNS), characterized by demyelination and axonal degeneration. INF-ß1b was the first drug approved for MS patients in 1993. In 1996, glatiramer acetate (GA), a synthetic copolymer, was approved in the USA for the treatment of relapsing-remitting MS (RRMS) and clinically isolated syndrome (CIS). Although the immunological action of GA has been fully investigated, the exact mechanisms of action of GA are still not completely elucidated. Several in vitro studies on mice and human antigen-presenting cells (APCs) have shown that GA is able to bind to the major histocompatibility complex (MHC), on the surface of APCs, recognizing myelin basic protein (MBP). AREAS COVERED: This review explores the pharmacological characteristics of GA, its mechanism of action and its pharmacokinetics properties. The article also provides information on the efficacy, tolerability and an overview of the most important clinical data on GA. EXPERT OPINION: Despite the development of novel compounds, it is not surprising that GA is, to date, one of the most prescribed drugs for RRMS patients and CIS patients. The proven efficacy and the mild adverse events, makes GA a good therapeutic option in the early stage of the disease. This is particularly useful for patients who suffer flu-like symptoms from other RRMS therapies as an alternative.