Off-label

BACKGROUND: Intracranial aneurysms(IAs) are rare in the pediatric population and are usually considered difficult to treat with traditional microsurgery due to their complex morphology. Endovascular techniques have become the standard option for treating IAs in adults. More recently, flow diverters such as the Pipeline Embolization Device(PED) are being widely adopted for unruptured IAs with safety and efficacy in adults, yet their use in the pediatric population is not well defined. In this paper we present a pediatric patient with a ruptured PCA aneurysm successfully treated with a PED and a review of the literature of the current status of PED use in this subset of patients...

RATIONALE: Atopic dermatitis is a frequent, relapsing, chronic inflammatory skin condition with a deep negative impact on quality of life. Three are the major pathological factors driving its complex pathogenesis: the skin barrier disruption, the altered Th2 cell response and itching. Current management of the disease is often unsatisfactory, unable to induce a complete resolution of signs and symptoms or at least a significant clinical improvement with adequate patient satisfaction. PATIENT CONCERNS: We report the case of a 57-year-old man with severe chronic atopic dermatitis for at least 40 years irresponsive to traditional therapies...

No abstract text is available yet for this article.

The principal finding from this study was the recognition that the α-adrenergic antagonist, phenoxybenzamine, possesses histone deacetylase inhibitory activity. Phenoxybenzamine is approved by the United States Food and Drug Administration for the treatment of hypertensive crises associated with tumors of the adrenal medulla, pheochromocytomas. It has several "off label" indications relative to its capacity to relax vascular smooth muscle and smooth muscle of the urogenital tract. The drug also has a long history of apparent efficacy in ameliorating, and perhaps reversing, the severe symptoms of neuropathic pain syndromes...

Background C3 glomerulopathy (C3G) is a life-threatening kidney disease caused by dysregulation of the alternative pathway of complement (AP) activation. No approved specific therapy is available for C3G, although an anti-C5 mAb has been used off-label in some patients with C3G, with mixed results. Thus, there is an unmet medical need to develop other inhibitors of complement for C3G. Methods We used a murine model of lethal C3G to test the potential efficacy of an Fc fusion protein of complement receptor of the Ig superfamily (CRIg-Fc) in the treatment of C3G...

Refractory pityriasis rubra pilaris (PRP) often is treated off-label with the same biologic therapies that are approved for the treatment of psoriasis, most commonly tumor necrosis factor (TNF) α antagonists and ustekinumab; however, the IL-17A antagonist secukinumab also has shown efficacy in the treatment of PRP. We report 2 new cases of severe refractory PRP that responded rapidly to treatment with secukinumab.

Fentanyl rose to prominence as an alternative analgesic to morphine nearly 50 years ago; today, fentanyl has re-emerged as a dangerous recreational substance. The increased potency and analgesic effect of fentanyl are advantageous in the treatment of pain but are also responsible for the rise in unintentional opioid overdose deaths. In response to this crisis, fentanyl, its analogues, and even precursors are under heightened regulatory scrutiny. Despite this controversial history, derivatization of fentanyl has resulted in numerous synthetic analogues that provide valuable insights into opioid receptor binding and signaling events...

Optimal treatment for advanced cervical cancer after first line chemotherapy remains undefined. Immune checkpoint inhibition with pembrolizumab, a programmed cell death protein 1(PD-1) inhibitor, is under investigation. We analyzed the micro-environmental and molecular genetic profile of tumors from 4 patients with metastatic cervical cancer treated with off-label second-line pembrolizumab in an effort to identify predictive biomarkers. All patients received 2 mg/kg of pembrolizumab, 3-weekly until disease progression...

No abstract text is available yet for this article.

An adolescent with type 1 diabetes and a history of self-harm, which included intentional overdoses and insulin omission, presented with an insulin degludec overdose. She had been commenced on the ultra-long-acting insulin, degludec, with the aim of reducing ketoacidosis episodes in response to intermittent refusal to take insulin. Insulin degludec was administered under supervision as an outpatient. Because it was anticipated that she would attempt a degludec overdose at some stage, the attending clinicians implemented a proactive management plan for this (and related) scenarios...

PURPOSE OF REVIEW: Hypogonadism and the treatment of low testosterone is increasingly a subject of medical inquiry and controversy. The few treatments that are FDA-approved such as testosterone topicals, injections, and pellets create significant demand from patients for treatments with more convenient means of administration, fewer adverse effects, and the ability to maintain male fertility. Off-label drugs are in abundant use for low testosterone, including selective estrogen receptor modulators, gonadotropins, and aromatase inhibitors...

BACKGROUND: Epidemiological data on pulmonary hypertension (PH) are scarce from developing countries including India. METHODS: We established a multi-center registry of PH, the PRO-KERALA registry, in Kerala, India. Fifty hospitals enrolled consecutive adult (>18 years) patients for one year. Echocardiographic criteria (right ventricular systolic pressure - RVSP > 50 mmHg) or invasively obtained mean pulmonary artery pressure > 25 mmHg was the criteria for entry...

Naltrexone, a non-selective antagonist of opioid receptors, is mainly used as rehabilitation therapy for discharged opiate addicts to eliminate addiction in order to maintain a normal life and prevent or reduce relapse. In recent years, there have been some novel and significant findings on the off-label usage of naltrexone. Within a specific dosage window, LDN can act as an immunomodulator in multiple autoimmune diseases and malignant tumors as well as alleviate the symptoms of some mental disorders. The results of increasing studies indicate that LDN exerts its immunoregulatory activity by binding to opioid receptors in or on immune cells and tumor cells...

Ketamine, a channel blocking NMDA receptor antagonist, is used off-label for its psychedelic effects, which may arise from a combination of several inter-related actions. Firstly, reductions of the contribution of NMDA receptors to afferent information from external and internal sensory inputs may distort sensations and their processing in higher brain centres. Secondly, reductions of NMDA receptor-mediated excitation of GABAergic interneurons can result in glutamatergic overactivity. Thirdly, limbic cortical disinhibition may indirectly enhance dopaminergic and serotonergic activity...

WHAT IS KNOWN AND OBJECTIVE: Prothrombin complex concentrate (PCC) is a plasma-derived concentrate used to replenish clotting factors. There are limited recommendations for treating coagulopathy induced by direct oral anticoagulants (DOAC). Data are limited regarding both total dose and repeated dosing with this population. CASE SUMMARY: We describe a case of an adult patient anticoagulated with apixaban who received two 35 unit/kg doses of PCC resulting in suspected pulmonary embolism...

OBJECTIVES: Although off-label use of recombinant activated factor VII against refractory bleeding is incorporated in current guideline recommendations, safety concerns persist predominantly with respect to thromboembolic complications. We analyzed the safety and efficacy of recombinant activated factor VII at a very low dose in cardiosurgical patients with refractory bleeding. METHODS: This prospective study includes 1180 cardiosurgical patients at risk of bleeding...

Intracardiac thrombus most commonly develops in the left atrial appendage (LAA) and left ventricle (LV) in the setting of atrial fibrillation (AF) and post-myocardial fibrillation (MI), respectively. Current guidelines recommend that patients with post-MI LV or LAA thrombus should be treated with vitamin K antagonist (VKA). However, the use of VKA may be limited by bleeding complications, interactions with various food and drugs, and a narrow therapeutic window requiring frequent monitoring. Thus, non-VKA oral anticoagulants (NOACs) have been attempted as an off-label use for the treatment of intracardiac thrombosis in light of their favorable pharmacologic profile...

Valproate and formulations thereof are approved for the management of seizure disorders, bipolar disorder, and migraine. Valproate is also used for many off-label indications. Unfortunately, data from observational studies indicate that valproate is perhaps the most teratogenic drug in the neuropsychiatric pharmacopeia. It is significantly more teratogenic than many other antiepileptic drugs (AEDs). In observational studies, gestational exposure to valproate is also associated with higher risks of cognitive, language, and psychomotor delay during early childhood and possibly with an increased risk of autism, as well...

Ranitidine is an H2 antihistamine used as an off-label therapy for recalcitrant verruca vulgaris. We describe a man who developed a sleep disturbance after initiating therapy with ranitidine and review similar adverse effects associated with other drugs in this class. The patient was a 40-year-old man with an eight-year history of a wart on his right plantar foot that was recalcitrant to several topical therapies. Adjunctive treatment with ranitidine 150 mg twice daily was initiated. He developed sleep disturbance with bizarre dreams and gastrointestinal symptoms...

OBJECTIVE: When is off-label use permitted by law in the statutory health insurance? Which approach is recommended for medical professionals to avoid recourse? RESULTS: The statutory health insurance in Germany is characterized among others by the precepts of efficiency and quality. Thus medicinal products may only be prescribed after an examination of quality, safety and efficacy. For using medicinal products off label, i. e. without applicable authorization or outside the authorized indications, these requirements are slightly reduced...