Off-label

Topical budesonide irrigations are frequently prescribed after endoscopic sinus surgery (ESS) to manage mucosal inflammation. However, this off-label indication may conflict with health insurance formularies. We sought to quantify the relative frequency of postoperative budesonide prescriptions to determine if this could be considered common practice after ESS. We extracted and analyzed postoperative prescription data for patients undergoing ESS from 2016 to 2022 within our health care system. Overall, among 8157 ESS patients, 15...

BACKGROUND: First-line treatment for symptomatic intracranial atherosclerotic disease (ICAD) is medical management; however, interventional approaches are increasingly considered for refractory disease. The Resolute Onyx in TIA management (ROTIA) study is a post-market evaluation of the Resolute Onyx (R-Onyx) drug-eluting stent in the treatment of recurrent transient ischemic attacks (TIAs) due to refractory ICAD (off-label use). METHODS: This is a single-center, retrospective case series of consecutive patients who underwent angioplasty and stenting with R-Onyx for treatment of recurrent TIAs due to refractory ICAD from October 2019 to November 2022...

BACKGROUND: Dalbavancin has been used off-label to treat invasive bacterial infections in vulnerable populations like people who use drugs (PWUD) because of its broad gram-positive coverage and unique pharmacological properties. This retrospective, multisite study examined clinical outcomes at 90 days in PWUD versus non-PWUD after secondary treatment with dalbavancin for bacteremia, endocarditis, osteomyelitis, septic arthritis, and epidural abscesses. METHODS: Patients at 3 teaching hospitals who received dalbavancin for an invasive infection between March 2016 and May 2022 were included...

INTRODUCTION: Gastrointestinal bleeding (GI) is a prevalent condition among pediatric patients, with a reported incidence of 6.4%, often severe enough to require admission to the pediatric intensive care unit (PICU). There are multiple therapies utilized in the management of GI bleeding in pediatrics, among which continuous intravenous (IV) infusion of omeprazole is used off-label without standard pediatric dosing recommendations. Reviewing the current literature reveals a lack of studies assessing the efficacy, safety, and appropriate dosing regimen of continuous omeprazole infusion in children with GI bleeding...

Some stakeholders called on medical boards to sanction physicians for spreading misinformation during COVID-19 and discussing off-label medications. In 2022, California passed AB 2098, which classifies physician dissemination of misinformation as unprofessional conduct subject to discipline by the state medical board. This article describes the purpose and function of state medical boards, the law relating to off-label prescribing, and why using medical boards to discipline physicians for discussing controversial opinions exceeds the traditional role of state medical boards...

Pyoderma gangrenosum (PG) is a neutrophilic dermatosis that is challenging to diagnose and treat. Clinicians frequently use fast-acting corticosteroids, which are subsequently combined with slower-acting immunosuppressants to progressively taper the corticosteroid dosage. Current research is focused on the use of monoclonal antibodies (mAbs) directed against target molecules involved in the pathogenesis of PG. However, available data on their efficacy are based on sporadic case reports and clinical experiences, so the authors aimed to evaluate the efficacy of risankizumab, an anti-interleukin-23 mAb, in the management of two complex PG cases...

Targeting the mammalian target of rapamycin (mTOR) pathway represents a potentially novel approach to treat basal cell carcinoma (BCC), but activation of this pathway has not been well described in human BCCs. The purpose of this study was to assess whether mTOR pathway activation occurs in BCCs (both sporadic and syndromic) and report a case of a patient with Gorlin syndrome (GS) whose clinically suspicious BCCs responded to mTOR inhibition through topical sirolimus treatment. After Stanford Institutional Review Board Approval, archived BCCs from patients with GS (n = 25), sporadic BCCs (n = 35), and control tissues were subjected to immunohistochemical analysis for the activation of mTOR pathway, and immunohistochemical staining intensity was evaluated by a dermatopathologist...

Medicines management in children and young people presents specific challenges because children differ from adults in their response to medicines. The way in which medicines work inside the human body, or pharmacokinetics, varies according to age and stage of development. Accurate drug calculations for a child rely on the careful consideration of a series of factors, such as weight and height, pharmacokinetics and drug characteristics. This article focuses on three fundamental aspects: pharmacokinetics, drug calculations, and unlicensed and off-label drug use...

BACKGROUND: Treatment of invasive gram-positive infections in complex patient populations is challenging. Dalbavancin, approved for skin and soft tissue infections, offers advantages in this setting due to its long half-life and infrequent dosing. However, less is known about the outcomes of off-label dalbavancin for deeper infections. OBJECTIVES: The objective of this study is to examine the feasibility and outcomes of patients with complex gram-positive infections treated with dalbavancin as an alternative to standard outpatient parenteral antimicrobial therapy (OPAT)...

BACKGROUND: Ferumoxytol (Ferahame, AMAG Pharmaceuticals, Waltham, MA) is increasingly used off-label as an MR contrast agent due to its relaxivity and safety profiles. However, its potent T2∗ relaxivity limits achievable T1-weighted positive contrast and leads to artifacts in standard MRI protocols. Optimization of protocols for ferumoxytol deployment is necessary to realize its potential. METHODS: We present first-in-human clinical results of the Quantitative Ultrashort Time-to-Echo Contrast Enhanced (QUTE-CE) MRA technique using the superparamagnetic iron oxide nanoparticle agent ferumoxytol for vascular imaging of the head/brain in 15 subjects at 3...

BACKGROUND: This study aims to evaluate the outcomes of using calcaneal plate in fixation of comminuted posterior wall (PW) acetabular fractures especially that have cranial (dome) or posterior extension (posterior column edge), and to evaluate its safety. To our knowledge, this is the first study that utilizes this off label implant technique in fixation of such fracture. METHODS: Twenty-two patients enrolled in the study with a minimum follow up of one year. After reducing the PW fragments sequentially, calcaneal plate was applied, fixing its distal part at ischial tuberosity upper ends using 3 screws in a triangular fashion, while its proximal part and radial wings were firmly fixed along the acetabular rim together with the classic longitudinal anchorage...

BACKGROUND: Domperidone is a commonly prescribed galactagogue used off-label for lactation insufficiency. Prescriber unfamiliarity or safety concerns can lead to therapeutic delay and potential early breastfeeding discontinuation. To facilitate access, the study site pharmacy department developed a Structured Administration and Supply Arrangement (SASA) for International Board-Certified Lactation Consultants (IBCLC) to screen and initiate domperidone using a checklist. MATERIAL: To validate a domperidone screening tool via analysis of its use and compliance, together with a staff satisfaction survey...

BACKGROUND: Granuloma formation is an uncommon and persistent skin inflammatory condition caused by the injection of dermal fillers. The exact cause of this reaction is not well understood, but it may be associated with irritating components or abnormal immune function. Treating granulomas can be difficult. However, recent research has shown that Janus kinase (JAK) inhibitors hold promise as a potential therapy for refractory granulomatous diseases. OBJECTIVES: The aim was to evaluate the efficacy and safety of tofacitinib as a treatment for granulomas secondary to filler injection and the possible mechanisms were discussed and summarized...

OBJECTIVE: To analyze the characteristics of the new medicines approved in the pediatric population in the last 3 years, both those with studies only in the pediatric population and those that extend their indication in this population group, as well as the current situation in relation to their marketing and financing. METHODS: Descriptive observational study of all drugs that include an indication in the pediatric population in Spain (by extension of the indications of drugs already authorized or because they are new drugs that already include an indication in this population group), from January 2019 to March 2022...

PURPOSE: Systematic repurposing of approved medicines for another indication may accelerate drug development in oncology. We present a strategy combining biomarker testing with drug repurposing to identify new treatments for patients with advanced cancer. METHODS: Tumours were sequenced with the Illumina TruSight Oncology 500 (TSO-500) platform or the FoundationOne CDx panel. Mutations were screened by two medical oncologists and pathogenic mutations were categorised referencing literature...

The discovery of antivascular endothelial growth factor medications has resulted in a substantial change in diabetic retinopathy treatment. The most common cause of diabetic retinopathy blindness is Diabetic Macular Edema. The pathophysiology of Diabetic Macular Edema is thought to include the well-known pro-angiogenic and pro-permeability factor vascular endothelial growth factor. Over the past decade, drugs that impede the functions of vascular endothelial growth factors have established themselves as a standard-of-care treatment for a range of ocular ailments and improved patients' clinical results with diabetic retinopathy and Diabetic Macular Edema, and their frequency has grown exponentially with the introduction of these agents Pegaptanib, Ranibizumab, and Aflibercept which are approved for ophthalmic indications, while Bevacizumab is used off-label...

BACKGROUND: Immune checkpoint inhibitors (ICI) have improved outcomes in a variety of adult cancers and are prescribed with increasing frequency across oncology. However, patterns of off-label use of ICI in pediatrics remain unclear. METHODS: This is a single-institution, retrospective cohort study evaluating off-label ICI use in pediatric and young adult patients with cancer treated at our institution from 2014 to 2022. Response was based on clinician assessment derived from clinical records...

Background: Oral and inhalation-based cannabidiol (CBD) administration has been clinically evaluated for various therapeutic indications, alongside widespread off-label use. However, the long-term exposure kinetics and varied bioavailability have not been fully characterized. Methods: Human CBD plasma concentration-time profiles from six studies evaluating the oral administration of Epidiolex® and three studies evaluating inhalation-based delivery were obtained. A four-compartment pharmacokinetic (PK) model with Weibull-based oral absorption kinetics was employed to describe the long-term PKs of CBD...

The classification of medical devices by the Food and Drug Administration (FDA) involves rigorous scrutiny from specialized panels that designate devices as Class I, II, or III depending on their levels of relative risk to patient health. Posterior rigid pedicle screw systems were first classified by the FDA in 1984 and have since revolutionized the treatment of many spine pathologies. Despite this early classification by the FDA, posterior cervical pedicle and lateral mass screws were not reclassified from unclassified to Class III and then to Class II until 2019, nearly 35 years after their initial classification...

This study employed evidence mapping to systematically sort out the clinical studies about the treatment of premature ventricular contractions with Chinese patent medicines and to reveal the distribution of evidence in this field. The articles about the treatment of premature ventricular contractions with Chinese patent medicines were searched against PubMed, Cochrane Library, Web of Science, CNKI, Wanfang, and VIP with the time interval from January 2016 to December 2022. Evidence was analyzed and presented by charts and graphs combined with text...