Attention-deficit hyperactivity disorder (ADHD) is a neurobehavioral disorder with an onset of symptoms before 7 years of age, often starting as early as the preschool years. The off-label use of methylphenidate (MPH) for 3- to 6-year-old preschool children with ADHD is being more common, although MPH is not licensed for use in children younger than age 6 years, in most countries. Despite the limited data, the review of the literature suggests that MPH meets evidence based criteria as beneficial and safe for carefully diagnosed ADHD preschool children. Carefully monitored administration of evidence-based psychotropic medication in preschool children with ADHD may be indicated if psychosocial treatments are ineffective.
Child ; Child, Preschool ; Humans ; Methylphenidate ; Off-Label Use ; Phenazines

OBJECTIVETo investigate the off-label use of oral glucocorticoids in outpatients.

METHODSThe information of outpatient glucocorticoids prescriptions from January 1st to June 30th in 2012 were collected from the information system in our hospital, then the software of Excel was employed to statistically analyze the data including the amount of drugs used in different departments,as well as the age, sex, and diagnosis of the patients. The diagnoses were compared with those included in the labels approved by China Food and Drug Administration and US Food and Drug Administration and domestic and foreign guidelines.

RESULTSIt was found that 16.53% of the cases were off-label use,and dexamethasone had the highest proportion (60.50%) of off-label use. Most of the off-label use had evidence support, such as multiple myeloma and myasthenia gravis, while some cases did not, such as epilepsy and sudden deafness.

CONCLUSIONThe management of off-label use should be further strengthened to promote the safe and rational use of glucocorticoids.

Anticonvulsants ; administration & dosage ; therapeutic use ; Drug Prescriptions ; Humans ; Off-Label Use

Children should be treated with medicines that have been tested for efficacy and safety in the given age groups. In reality the majority of drugs being used in children lack such information, inevitably leading to the "off-label use." Over decades there have been concerns regarding risk of adverse effects with such off-label uses. However, in most countries rational application of off-label uses have not been prohibited, since it may be the only available and perhaps the best possible treatment option. In contrast, in an effort to minimize such use and per se 'protect' children from possible harm associated with it, Korea has launched "Drug Utilization Review" system, which included age group-specific contraindications, based on the labeling information approved by Ministry of Food and Drug Safety. The problem lies in the definition and scope of 'contraindication.' Lack of evidence for safety and concern for risk of serious adverse drug reactions are the basis for contraindication. This leads to branding of off-label uses as contraindications in certain age groups. This policy is restricting the already scarce treatment options and suffocating the initiative for developing better pharmacotherapy for children. We should learn from other countries that have travailed more to provide better medicines to children. Clinical trials of new drugs as well as existing drugs in pediatric populations are strongly encouraged. Simple prohibition of off-label use is not the answer, but rather systematized management of off-label use as well as conduct of clinical trials in pediatric age groups must be encouraged and supported.
Child* ; Drug Therapy* ; Drug-Related Side Effects and Adverse Reactions ; Humans ; Korea ; Off-Label Use*

The introduction of intravenous nutritional therapy for cosmetic and health purposes in Korea has been controversial. Because it brings ethical problems of off-label prescribing on the basis of low level of medical evidences for claims of fatigue reduction, detoxification, anti-inflammation effect, skin whitening, antioxidant and anti-aging effects. Despite the lack of clinical studies, the verification of the efficacy and safety of intravenous nutritional therapy and the need to provide objective information to patients are prerequisite for the correct intravenous use. In addition, efforts should be made to prevent the abuse for commercial purposes.
Fatigue ; Fursultiamin ; Glutathione ; Glycyrrhizic Acid ; Humans ; Korea ; Off-Label Use ; Skin ; Thioctic Acid

Over the last two decades, the systemic treatment of cancer has evolved from cytotoxic chemotherapy to targeted therapy and now immunotherapy. Next-generation sequencing (NGS) is entering clinical applications for cancer treatment through the help of more powerful computational analyses. The increasing number of targeted therapies approved by regulatory authorities (RAs) with or without biomarkers necessitates the screening of multiple biomarkers using NGS, which is now approved and reimbursed by Korean RAs for some types of malignancies. However, the clinical utility of NGS remains to be established as a prerequisite for its routine incorporation into clinical practice. Currently, the best scenario of NGS use in clinics is to enroll patients into clinical trials based on the detection of biomarkers, but this is only possible in the hospitals conducting the specific trial. The other scenario is the off-label use of a targeted drug, but this requires social consensus for future implementation. The clinical applications of NGS are expanding in terms of its platforms, from targeted sequencing to whole exome and RNA sequencing, and in terms of systemic therapy, from targeted therapy to immunotherapy. Research into tumor mutational burden and neoantigens is shedding new light on the clinical use of NGS in immunotherapy.
Biomarkers ; Consensus ; Drug Therapy ; Exome ; Humans ; Immunotherapy ; Mass Screening ; Off-Label Use ; Sequence Analysis, RNA

Objective: To understand the current situation regarding pediatric off-label use of drugs recommendations in Chinese clinical practice guidelines and to make recommendations for standardized reporting format regarding off-label use of drugs for children. Methods: This cross-sectional study was carried out by systematically searching the databases for Chinese guideline consensus articles published in journals between 2018 and 2020 and extracting recommendations regarding off-label use of drugs from those articles. The essential characteristics of the included guidelines, the ranking of off-label drug types, the order of drug information, the type of off-label drug use, and the percentage of citation studies on which the recommendations were based were analyzed. Results: Among 108 studies that included Chinese off-label guidelines and consensus, 364 recommendations on pediatric off-label use of drugs were included. The Chinese Medical Association published the most, 48 out of the 108 studies (44.4%), and of those 14 studies (13.0%) were on infectious and parasitic diseases. Of the 364 recommendations on off-label use of drugs, the most commonly addressed drugs were 16 recommendations (4.4%) for cyclosporine A, 11 recommendations (3.0%) for methotrexate , and 11 recommendations (3.0%) for fentanyl. The most commonly addressed drug categories were as follows: 68 recommendations (18.6%) were immune system drugs, 66 recommendations (18.1%) were anti-infectives, and 56 recommendations (15.4%) were oncology drugs. The most commonly addressed drug information accounts were as follows: 364 recommendations (100.0%) were indications, 204 recommendations (56.0%) were dosages, and 198 recommendations (54.4%) were the route of administration. Based on the instructions approved by the Chinese Food and Drug Administration, the main forms of the off-label drug were as follows: 175 recommendations (48.1%) were unapproved indications, 127 recommendations (34.9%) were unapproved populations, and 72 recommendations (19.8%) were unapproved ages. Only 129 recommendations (35.4%) were cited, mainly including clinical guidelines (48 studies, 23.4%), reviews (22 studies, 10.7%), and pediatric randomized controlled trials (22 studies, 10.7%). Conclusions: Off-label use of drugs is commonly recommended in pediatric guidelines and consensus documents written by Chinese authors. However, the reporting of the recommendations varies widely, and the quality of the supporting evidence is poor.
Child ; China ; Consensus ; Cross-Sectional Studies ; Humans ; Off-Label Use ; Pharmaceutical Preparations

BACKGROUND AND OBJECTIVES: While the off-label use of implantable medical devices for treating congenital heart disease is not uncommon, the present conditions and outcomes of their off-label use have rarely been described. Therefore, this study evaluated the prevalence and results of using implantable devices off-label to treat congenital heart disease at a single institute. SUBJECTS AND METHODS: This was a retrospective study based on the medical records of catheter-based interventions for congenital heart disease performed from July 1, 1995 to June 1, 2015. The inclusion criterion was the off-label use of an implantable device. Patient demographic data, procedural success, and follow-up status regarding late complications were investigated, and the results of the off-label use of each device were compared. RESULTS: Off-label use occurred in 144 of 1730 interventions with device implantation, accounting for 8.3% of the interventions. The median patient age and mean body weight were 51.0 months and 16.3 kg, respectively. Immediate and late failures were found in 9 cases, and 3 cases of mortality were not directly related to the devices used. The overall success rate was 93.8%. There were no long-term complications of the off-label use of occlusion devices. No procedural failures resulted from stent implantation, but one case of stent malposition and two cases of stent fracture were identified after procedure completion. CONCLUSION: In general, the off-label use of implantable devices for treating congenital heart disease is safe and effective.
Body Weight ; Follow-Up Studies ; Heart Defects, Congenital* ; Humans ; Medical Records ; Mortality ; Off-Label Use* ; Prevalence ; Retrospective Studies ; Stents

A 75-year-old patient with severe comorbidities was treated with an Endurant® (Medtronic, USA) II endograft due to a ruptured abdominal aortic aneurysm (AAA). After four years of unremarkable follow-up, bilateral limb separation was detected. The patient underwent endovascular bridging without any complication. Although rarely detected in newer grafts, late bilateral type IIIa endoleaks can present and should be promptly repaired. Complex or ruptured AAAs treated with off-label use of endografts should be under closer surveillance using imaging tools for potential endoleaks or aneurysm sac growth.
Aged ; Aneurysm ; Aortic Aneurysm, Abdominal ; Comorbidity ; Endoleak* ; Endovascular Procedures ; Extremities* ; Follow-Up Studies ; Humans ; Off-Label Use ; Retreatment ; Transplants

Breast cancer is the most common cancer in the world, and 5-year survival rate of metastatic breast cancer is about 20%. The treatment of metastatic breast cancer is mainly chemotherapy, endocrine therapy and targeted therapy. However, after multiline treatment, patients with MBC especially the triple negative breast cancer face the problem of drug resistance. Tumor angiogenesis theory suggests that blocking angiogenesis can inhibit tumor growth and migration. Based on this, angiogenesis treatment strategy is proposed. Antiangiogenic drugs mainly include biological macromolecular drugs targeting vascular endothelial growth factor (VEGF) or vascular endothelial growth factor receptor (VEGFR) and small molecule VEGFR inhibitors. Angiogenesis is known to play a key role in the growth and metastasis of breast cancer. Therefore, anti-angiogenetic therapy has potential in metastatic breast cancer patients. Since the approval of tumor drug indications by NPMA in China is often later than the release of the latest research data, the National Health Commission issued "the guiding principles for the clinical application of new antitumor drugs" in 2020. The principle pointed out that under special circumstances such as the absence of better treatment, medical institutions should manage the usage of drugs that are not clearly defined in the instructions but have evidence-based data. Based on the latest research progress in breast cancer, the consensus writing expert group collated published reports, international academic conferences, conducted analysis, discussion and summary, collected data on the use of small molecule anti-vascular targeting drugs for advanced breast cancer, and formulated "expert consensus on the application of small molecule anti-angiogenic drugs in the treatment of advanced breast cancer" . For clinicians' reference only.
Angiogenesis Inhibitors/therapeutic use* ; Breast Neoplasms/pathology* ; Consensus ; Female ; Humans ; Neovascularization, Pathologic/pathology* ; Off-Label Use ; Vascular Endothelial Growth Factor A/metabolism*