Pediatric Drugs最新文献

Background: Anti-interleukin-1 (IL-1) biologic disease-modifying anti-rheumatic drugs are the mainstay for several childhood rheumatic and autoinflammatory diseases. Long-term medication safety is a key consideration for chronic disease management.

Aim: The objective was to synthesise evidence on the long-term safety of anti-IL-1 medications in children and young people with rheumatic diseases, including autoinflammatory diseases.

Methods: The study protocol was registered prospectively (PROSPERO CRD420251000272). Original full text studies of at least ten patients presenting safety data on anti-IL-1 medications in children with rheumatic diseases were eligible for inclusion. Medline, Embase and Web of Science were searched from inception to 27 February 2025. The methodological index for non-randomized studies (MINORS) tool was used to assess risk of bias. All relevant safety outcomes were presented and synthesised. Meta-analysis was not performed owing to study heterogeneity.

Results: A total of 1660 unique records were screened, and 57 unique studies (3690 patients) were included. In total, 31 were retrospective cohort studies, and 10 were prospective interventional trials. Most studies were of moderate (n = 31) or high (n = 25) risk of bias. Rates of adverse events varied significantly between studies. Injection site reactions (particularly with anakinra) and minor infections were common. Infections were the most common type of serious adverse event. Drug reaction with eosinophilia and systemic symptoms (n = 3) and interstitial lung disease (including related conditions) (n = 9) were reported in patients with systemic onset juvenile arthritis only. Deaths (n = 16) and malignancies (n = 7) were uncommon, often occurring long after anti-IL-1 medication discontinuation and were often deemed to be unrelated to the anti-IL-1 medication.

Conclusions: Our results are consistent with the known safety profile of anti-IL-1 medications and show that they are generally safe for use in the context of childhood rheumatic and autoinflammatory diseases. This review of clinical trial and real-world data will help inform clinical decision-making and family counselling when initiating anti-IL-1 medications in children.

Growth hormone (GH) has been used to treat severe GH deficiency for more than 60 years, with other indications dependent upon approval from national medical societies and governmental agencies. Many changes in GH preparations have been made to improve safety, compliance, and efficacy, from the extraction of GH from human pituitary glands to recombinant hormone technology and from the use of vials and syringes to the use of more advanced devices. Adherence to treatment has always been highlighted as a key point in the success of treatment; new depot preparations permitting weekly administration have advantages in this regard. This review summarizes the history of GH use and provides an analysis of the new weekly administration products.

Fremanezumab (fremanezumab-vfrm; AJOVY^®) is a humanized monoclonal antibody developed by Teva Pharmaceuticals to selectively target calcitonin gene-related peptide (a vasodilatory neuropeptide involved in the pathophysiology of migraine). Fremanezumab was first approved in September 2018 in the USA for the prevention of migraine in adults. In August 2025, it was approved in the USA for the preventive treatment of episodic migraine in pediatric patients aged 6-17 years who weigh 45 kg or more. This article summarizes the milestones in the development of fremanezumab leading to this pediatric first approval for migraine.

Background: Pediatric-onset inflammatory bowel diseases (IBD) are associated with an elevated risk of venous thromboembolism (VTE). However, data on the VTE risk in these patients treated with novel biologics, particularly vedolizumab, remain limited.

Aims: To evaluate the association between vedolizumab and the risk of VTE compared with other biologics in patients with pediatric-onset Crohn's disease (CD) or ulcerative colitis (UC).

Methods: We emulated a target trial using electronic health records from the TriNetX research network. The study included patients with CD or UC diagnosed before the age of 18 years who were treated with vedolizumab, ustekinumab, or anti-tumor necrosis factor (TNF) agents. Comparative groups were generated using 1:1 propensity-score matching based on relevant confounders. The primary outcome was the occurrence of any VTE within 12 months of follow-up. Cox proportional hazards models were used to compare the risk of VTE between vedolizumab-treated patients and those treated with anti-TNF agents and ustekinumab.

Results: A total of 1806 matched patient pairs were analyzed across four comparisons: patients treated with vedolizumab versus anti-TNF agents (CD = 407 pairs; UC = 443 pairs), and vedolizumab versus ustekinumab (CD = 563 pairs; UC = 393 pairs), respectively. Patients with CD receiving vedolizumab had a significantly higher risk of VTE compared with those receiving anti-TNF therapy (hazard ratio [HR] 8.63; 95% confidence interval [CI] 1.08-69.10; p = 0.014). A higher VTE risk was also observed in vedolizumab-treated patients with CD compared with those treated with ustekinumab (HR 4.64; 95% CI 1.35-15.97; p = 0.007). In contrast, no significant difference in VTE risk was found between vedolizumab and either comparator group in patients with UC.

Conclusions: Treatment with vedolizumab was associated with a higher incidence of VTE than anti-TNF agents or ustekinumab among individuals with CD. Prospective cohort studies are warranted to validate the safety of biologic therapy for pediatric patients with CD.

The urea cycle, a metabolic pathway comprising six enzymes and two transporters, is necessary for mammalian nitrogen detoxification. A deficiency of any of these components disrupts this process, leading to the accumulation of nitrogen in the form of ammonia, which is especially toxic to the brain. For decades, treatment of urea cycle disorders has consisted of nitrogen scavengers, dietary protein restriction, arginine or citrulline supplementation, calorie support, and liver transplant. In 2011, carglumic acid became available as a substitute for N-acetylglutamate for N-acetylglutamate synthase deficiency. The past 10 years, however, have seen the development of enzyme therapy for arginase deficiency and gene therapy for ornithine transcarbamylase deficiency. This article reviews the current status and availability of treatment options for urea cycle disorders.

Biosimilar use for pediatric rheumatologic conditions, such as juvenile idiopathic arthritis (JIA), is increasing owing to the development and release of multiple biosimilars into the US market. The US biosimilar development process of bio-originator and generic medications differs. Bio-originators typically spend the longest amount of time in research and development and require the largest financial investment, followed by biosimilars and, lastly, generic medications. Data available from European countries, where biosimilars have been made available much earlier than in the USA, support the effectiveness and safety of biosimilars in patients with JIA. However, European rheumatology clinicians surveyed highlight continued concerns regarding biosimilar data and experience. Although there are varying perspectives of major stakeholders on biosimilars in the USA-the patients and caregivers, clinicians, manufacturers, and pharmacy benefit managers (PBMs)-the primary goal of all should be patient benefits. These benefits may include improved medication healthcare access overall and during shortage situations or promoting the innovation of biobetters, but biosimilars may conversely negatively impact provider reimbursement, or manufacturers may drive out competition of competing biosimilar manufacturers. There are risks associated with lack of education for medical staff and patients, such as the nocebo effect, and how to reduce those risks is through assisting patients in understanding their new medication and reasons for the change. Credible biosimilar resources may be used to educate medical staff and patients, including the Food and Drug Administration (FDA), American College of Rheumatology (ACR), and Arthritis Foundation. After a patient has been transitioned to a biosimilar, it is necessary to have appropriate medical follow-up and continuous monitoring for efficacy and safety.