Off-label

Pancreatic acinar cell carcinoma (PACC) is a rare cancer. When the tumor is metastatic, few therapeutic options are available. Precision medicine using next-generation sequencing is defined by the administration of drugs based on the tumor genetic mutations. The usage of precision medicine for finding new therapeutic options for rare cancers is an emerging field. We have reported here the case of a patient bearing a multitreated metastatic PACC. This patient underwent somatic and constitutional exome analyses...

Livedoid vasculopathy (LV) is a thrombotic skin disease characterized by episodic painful ulcerations of the distal aspects of the legs. Its healing process typically leaves small porcelain-white scars called atrophie blanche as a result of the occlusion of cutaneous microcirculation. The main goals of the treatment are pain management and the prevention of ulceration and of progressive scarring in the malleolar area. The therapeutic management is still a challenge, however, and most treatments were based on anecdotal off-label protocols...

BACKGROUND: To describe frequency, preventability and seriousness of adverse drug reactions (ADRs) in children as cause of emergency department (ED) admission and to evaluate the association between specific factors and the reporting of ADRs. METHODS: A retrospective analysis based on reports of suspected ADRs collected between January 1st, 2012 and December 31st, 2016 in the ED of Meyer Children's Hospital (Italy). Demographics, clinical status, suspected drugs, ADR description, and its degree of seriousness were collected...

BACKGROUND: Topical minoxidil and oral finasteride are the only drugs approved for the treatment of androgenetic alopecia (AGA) in Spain. However, the management of this condition is highly variable because numerous treatments are used off-label. The main aim of this study was to describe the prescribing habits of dermatologists in Spain for male AGA (MAGA) and female AGA (FAGA). MATERIAL AND METHODS: Descriptive cross-sectional study using online questionnaires completed by dermatologists working in Spain...

With specialized care, patients with myasthenia gravis can have very good outcomes. The mainstays of treatment are acetylcholinesterase inhibitors, and immunosuppressive and immunomodulatory therapies. There is good evidence thymectomy is beneficial in thymomatous and nonthymomatous disease. Nearly all of the drugs used for MG are considered "off-label." The 2 exceptions are acetylcholinesterase inhibitors and complement inhibition with eculizumab, which was recently approved by the US Food and Drug Administration for myasthenia gravis...

Chronic spontaneous urticaria (CSU) is a common skin disease characterised by intermittent weals (hives), angioedema or both lasting for at least 6 weeks.1-3 Second-generation antihistamines are widely used to manage symptoms but are not completely effective in many patients at licensed doses.4 Some guidelines recommend off-label use of high-dose antihistamines as the next therapeutic step.2,3 Here, we review the evidence supporting this recommendation.

Biologic disease modifying antirheumatic drugs (bDMARDs) can be highly efficient in the treatment of various non-infectious uveitis entities. Currently, the TNF-α-inhibitor Adalimumab is the only in-label therapeutic option, whereas, all other bDMARDs need to be given as an off-label therapy. bDMARDs are indicated in diseases refractory to conventional synthetic DMARD therapy and/or systemic steroids, or in patients in whom treatment with those is not possible due to side effects. Therapeutic mechanisms currently employed are cytokine-specific (interferons, inhibition of TNF-α or of interleukin [IL]-1-, IL-6- or IL-17-signalling), inhibit T cell costimulation (CTLA-4 fusion protein), or act via depletion of B cells (anti-CD20)...

Veno-venous extracorporeal membrane oxygenation can be performed either by two cannulae or by a single dual-lumen cannula. The dual-lumen cannulation configuration offers multiple advantages: it avoids the femoral site which may be at greater risk of infection, it improves patient mobility, eases prone positioning and greatly reduces recirculation. The Avalon was the first commercially available dual-lumen cannula for adults. It has gained much popularity, but, for more than a year now, the adult vascular access kit with the 30Fr dilator has been discontinued in the United Kingdom...

Osteoporosis is a common skeletal disorder characterized by low bone mass, which leads to reduced bone strength and an increased risk of fractures. Anabolic agents have been shown to improve bone mass and decrease fracture risk in osteoporosis patients by directly stimulating osteoblasts to produce new bone. Currently, two anabolic agents are available in the USA: recombinantly produced teriparatide (TPTD), which is the fully active (1-34) amino active sequence of human parathyroid hormone (PTH), and abaloparatide (APTD), a synthetic analog of parathyroid hormone-related peptide (PTHrP)...

Interstitial lung disease in children (chILD) comprises a range of different rare diseases. There is limited evidence for the treatment of chILD and no randomised clinical trials of treatment have been undertaken. Most treatments are therefore prescribed off-label based on expert opinion. The off-label nature of prescription of drugs for chILD highlights the importance of a solid understanding of the side effects to facilitate risk-benefit assessment. The European Respiratory Society chILD guidelines recommend the use of systemic glucocorticosteroids, hydroxychloroquine and azithromycin...

PURPOSE: Current off-label uses of acetazolamide in hospitalized patients are reviewed. SUMMARY: Acetazolamide is a carbonic anhydrase inhibitor typically used for indications including epilepsy, glaucoma, edema, and altitude sickness but it may be prescribed in hospitalized patients for off-label indications. It inhibits carbonic anhydrase, which leads to reduced hydrogen ion secretion in the proximal renal tubule, resulting in increased bicarbonate and cation excretion and causing urinary alkalization and diuresis...

Adaptation and evolution of transcatheter aortic valve replacement (TAVR) technologies has led to approval of TAVR for consideration in patients at intermediate risk for surgical aortic valve intervention. As TAVR becomes more mainstream, attention is shifting toward percutaneous mitral valve (MV) repair and transcatheter MV replacement (TMVR) techniques. Transcatheter heart valves (both purpose-built and off-label-use TAVR valves) are being implanted during TMVR procedures to treat clinically significant MV disease (native disease, degenerated bioprosthetic valves, and dysfunctional surgical MV annuloplasty repairs) when the risk of open heart MV surgery is prohibitive...

BACKGROUND: Anti-angiogenesis Tyrosine kinase inhibitors (TKIs) have been proved to show promising effects on prolonging progression-free survival (PFS) for advanced sarcoma after failure of standard multimodal Therapy. Methylsulfonic apatinib is one of those TKIs which specifically inhibits VEGFR-2. This paper summarizes the experience of three Peking University affiliated hospitals in off-label use of apatinib in the treatment of extensively pre-treated sarcoma. METHODS: We retrospectively analysed files of patients with advanced sarcoma not amenable to curative treatment, who were receiving an apatinib-containing regimen between June 1, 2015 and December 1, 2016...

OBJECTIVES: Little is known about off-label use and manipulations to achieve the prescribed dose of antiepileptic drugs (AEDs) in outpatient prescriptions. This study aimed to evaluate this practice in a tertiary center for child epilepsy. METHODS: We reviewed off-label use and manipulations of AEDs delivered to the outpatient's epilepsy clinic. Multivariate logistic regressions were used to determine the factors associated with off-label and manipulated uses. RESULTS: Five hundred eleven consultations generated 897 AED deliveries (1...

INTRODUCTION: Evidence for drug use in newborn is sparse, which may cause large differences in drug prescriptions. We aimed to investigate the differences between neonatal intensive care units (NICUs) in the Netherlands in currently prescribed drugs. MATERIALS AND METHODS: This multicenter study included neonates admitted during 12 months to four different NICUs. Drugs were classified in accordance with the Anatomical Therapeutic Chemical (ATC) classification system and assessed for on/off-label status in relation to neonatal age...

Cluster headache is a severe headache disorder with considerable impact on quality of life. The pathophysiology of the disease remains poorly understood. With few specific targets for treatment, current guidelines mainly include off-label treatment with medication. However, new targets for possible treatment options are emerging. Calcitonin gene-related peptide (CGRP)-targeted medication could become the first (cluster) headache-specific treatment option. Other exciting new treatment options include invasive and non-invasive neuromodulation techniques...

This article is the first reported case describing the off-label use of enteral immediate-release guanfacine, a long-acting α-2 adrenergic agonist most commonly used in the treatment of attention-deficit hyperactivity disorder, for sedation in a patient with severe anxiety and agitation limiting mechanical ventilation weaning several days after cardiac surgery. In this case, after several days of unsuccessful attempts to control his agitation and anxiety with conventional therapies, guanfacine therapy was initiated, and the patient was rapidly weaned from all other sedatives and mechanical ventilation shortly thereafter...

A 26-year-old woman presented to our clinic with years of headaches and mental clouding. She had a nonfocal neurological examination including benign ophthalmological examination. Head computed tomography and brain magnetic resonance imaging (MRI) demonstrated asymmetry of the lateral ventricles due to a lesion arising from the foramen of Monro. At the top of our differential diagnosis were choroid plexus tumor and colloid cyst. We offered the patient treatment via an endoscopic approach. Here, we demonstrate the feasibility of using tumor-localizing dyes such as indocyanine green (ICG) in conjunction with endoscopes equipped with lasers to localize and treat intraventricular lesions...

INTRODUCTION: To investigate the presentation, aetiology, management and outcomes of type IIIb endoleak after endovascular aneurysm repair (EVAR). EVIDENCE ACQUISITION: Electronic bibliographic databases were searched to identify published reports of type IIIb endoleak after EVAR, according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) standards. EVIDENCE SYNTHESIS: In total 33 articles were identified reporting on a total of 50 patients spanning 19 years of EVAR (1998-2017)...

Most antiepileptic drugs (AEDs) receive regulatory approval for children years after the drug is available in adults, encouraging off-label use of the drug in children and hindering attempts to obtain quality pediatric data in controlled trials. Extrapolating adult efficacy data to pediatrics can reduce the time between approval in adults and that in children. To extrapolate efficacy from adults to children, several assumptions must be supported, such as (1) a similar disease progression and response to interventions in adults and children, and (2) similar exposure response in adults and children...