Off-label

Vestibular schwannomas expand slowly in the internal auditory canal, in the cerebellopontine angle, inside the cochlear and the labyrinth. Larger tumors can displace and compress the brainstem. With an annual incidence of 1:100 000 vestibular schwannoma represent 6-7% of all intracranial tumors. In the cerebellopontine angle they are by far the most neoplasm with 90% of all lesions located in this region. Magnetic resonance imaging (MRI), audiometry, and vestibular diagnostics are the mainstays of the clinical workup for patients harboring tumors...

OBJECTIVE: Rodent models of Alcohol Use Disorders (AUD) are used extensively by preclinical researchers to develop new therapeutics for the treatment of AUD. Although these models play an important role in the development of novel, targeted therapeutics, their role in bringing therapeutics to clinical trials is unclear, as off-label use of existing medications not approved for the treatment of AUD is commonly seen in the clinic and clinical trials. METHOD: In the current study, we used the Clinicaltrials...

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OBJECTIVES: There have been few clinical trials (CTs) on antibiotics that inform neonatal and paediatric drug labelling. The rate of unlicensed and off-label prescribing in paediatrics remains high. It is unclear whether the current neonatal and paediatric antibiotic research pipeline is adequate to inform optimal drug dosing. Using the ClinicalTrials.gov registry, this review aims to establish the current global status of antibiotic CTs in children up to 18 years of age. METHODS: Studies were identified using key word searches of the ClinicalTrials...

Introduction. Posaconazole is a triazole antifungal that is used in the treatment of a variety of fungal infections, as well as in the management of mucormycosis (on an off-label basis). Eosinophilia associated with exposure to azole antifungals has been described rarely in the literature. Case presentation. A 31-year-old male on peritoneal dialysis (PD) for end-stage renal disease, secondary to diabetic nephropathy, presented to hospital with abdominal pain after a trip to St Lucia. He was taken to the operating room, where the PD catheter was removed and an abdominal-wall abscess was debrided...

Tardive dyskinesia (TD) is a disorder characterized by involuntary movements, typically of the orofacial muscles and also of the extremities and other muscle groups. The condition is associated with exposure to dopamine receptor blocking agents, including antipsychotics. Because the indications and off-label uses for these agents have expanded over the last 2 decades, a larger number of patients are receiving antipsychotic medications than in the past. While evidence suggests that patients being treated with second-generation antipsychotics have less risk for developing TD than those treated with first-generation antipsychotics, the decreased risk is not as great as was originally expected...

Off-label drug bills are back on track but could run off the rails.

BACKGROUND: Inappropriate sinus tachycardia (IST) is debilitating despite available treatment. Off-label use of ivabradine for IST prompted this systematic analysis of existing data quality and sample size estimates for adequately powered studies. OBJECTIVE: To determine clinical efficacy of ivabradine in IST from pooled prospective studies. METHODS: Analysis included ivabradine studies, for IST subjects without structural heart disease and follow-up of > 2 weeks...

Patients with bicuspid aortic valve (BAV) have traditionally been excluded from large randomized clinical trials involving transcatheter aortic valve replacements (TAVR). Technical enhancements, availability of new generation devices and improved outcomes have led to a marked increase in TAVR volume across the world including off label use in patients with BAV stenosis. Areas covered: In this manuscript, we have reviewed the currently available data regarding safety, efficacy, and outcomes of TAVR in patients with BAV stenosis...

Objective: The aim of this study was to determine the pattern of use of pregabalin and the appropriateness of treatment, so that interventions could be designed to improve various clinical approaches to the use of pregabalin to include unlicensed indications. Design: A descriptive, cross-sectional, multicenter study was performed between April 2014 and January 2015. Setting: Fifty-three primary health care centers covering 1,250,000 inhabitants...

Purpose Cancer drug prices at launch have increased in recent years. It is unclear how individual drug prices change over time after launch and what market determinants influence these changes. We measured the price trajectories of a cohort of cancer drugs after their launch into the US market and assessed the influence of market structure on price changes. Methods We studied the changes in mean monthly costs for a cohort of 24 patented, injectable anticancer drugs that were approved by the US Food and Drug Administration between 1996 and 2012...

OBJECTIVE: Around 30-45% of patients with familial Mediterranean fever (FMF) have been reported to have attacks despite colchicine treatment. Currently, data on the treatment of colchicine-unresponsive or colchicine-intolerant FMF patients are limited; the most promising alternatives seem to be anti-interleukin-1 (anit-IL1) agents. Herein we report our experience with the off-label use of anti-IL1 agents in a large group of FMF patients. METHODS: In all, 21 centers from different geographical regions of Turkey were included in the current study...

PURPOSE: To evaluate the safety and feasibility of a new femtosecond laser-assisted method for posterior capsule opacification (PCO) prevention. SETTING: University Eye Hospital Bochum, Bochum, Germany. DESIGN: Prospective randomized intraindividual case series. METHODS: Eyes scheduled for bilateral cataract surgery between April 2015 and January 2016 were enrolled in the study. In 1 eye, routine manual cataract surgery with intraocular lens (IOL) implantation was performed with a primary posterior laser capsulotomy as the last step in the procedure...

Clonidine hydrochloride is administered to opioid-addicted mothers' neonates to reduce neonatal abstinence syndrome. It is prescribed off-label to neonates at 0.5-1 µg/kg/6 hours, alone or in combination. The commercially injectable form of clonidine - Catapressan® 0.15 mg/mL - is being orally given after an appropriate dilution in water. However, this practice is not suitable for a perfectly safe and accurate administration. The objectives were to design a 10 µg/mL oral solution of clonidine hydrochloride in Inorpha® and to study the stability of this solution by a validated stability indicating liquid chromatography (LC) method...

INTRODUCTION: The neonatal population remains one of the populations in which appropriate dosing regimens are still lacking, resulting in a large off-label or unlicensed use. Clinical research in these small infants remains a challenge, which sparks the need for modeling and simulation as an additional tool for neonatal drug research. METHODS: The use of physiologically based pharmacokinetic modeling in preterm and term neonates is investigated. RESULTS: Throughout the last decade, the use of this modeling technique in this vulnerable population has received increased attention, but still many knowledge gaps exist...

Proper diagnosis of dementia with Lewy bodies (DLB) in clinical practice remains suboptimal as many cases are misdiagnosed, usually as Alzheimer disease (AD) or Parkinson's disease (PD) and, in rare cases, psychosis. Therefore, it is important for patients with dementia to be thoroughly evaluated by a specialist who is familiar with current diagnostic tests and treatment options. New diagnostic criteria from the Dementia with Lewy Bodies Consortium have been developed to increase diagnostic sensitivity for DLB (Diagnosis and management of dementia with Lewy bodies: fourth consensus report of the DLB Consortium; McKeith et al...

STUDY DESIGN: Retrospective database review. OBJECTIVES: After the Food and Drug Administration approved bone morphogenetic protein-2 (BMP) in 2002, BMP was used off-label in the cervical spine to increase bone growth and bony fusion. Since then, concerns have been raised regarding complication rates and safety. This study was conducted to examine the use of BMP in anterior cervical discectomy and fusion (ACDF) in the Medicare population and to determine risk of complications and associated costs within 90 days of surgery...

Pansclerotic morphoea (PM) is a subtype of juvenile localised scleroderma characterised by severe course with generalised full-thickness skin involvement and possible growth and functional impairment. PM treatment comprises a combination of immunosuppressive agents such as corticosteroids, methotrexate, mycophenolate mofetil, PUVA and antithymocyte globulin and biological agents used in off-label. A possible role of IL-6 in the regulation of firoblast differentiation and stimulation of collagen synthesis has been suggested and in patients with systemic sclerosis (SSc) the treatment with tocilizumab (TCZ) was associated to improvement of skin thickness and joint motion...

Contrast-enhanced ultrasound (CEUS) is a radiation-free, safe, and in specific clinical settings, highly sensitive imaging modality. Over the recent decades, there is cumulating experience and a large volume of published safety and efficacy data on pediatric CEUS applications. Many of these applications have been directly translated from adults, while others are unique to the pediatric population. The most frequently reported intravenous abdominal applications of CEUS in children are the characterization of focal liver lesions, monitoring of solid abdominal tumor response to treatment, and the evaluation of intra-abdominal parenchymal injuries in selected cases of blunt abdominal trauma...

Implants used to correct pathological varus-valgus deformities (VVD) and leg length discrepancies (LLD) may not be optimized for the specific treatment, as suggested by their off-label use. Detailed analysis of this issue has been limited by the poorly understood mechanical behavior of the growing physis and ignorance of the loads acting on the implants. The aim of this study was to predict and compare the loading conditions of a growth modulation implant in VVD and LLD treatments. Idealized finite element (FE) models of the juvenile distal femur treated with the eight-Plate implant were developed for VVD and LLD...