Background: No oral systemic treatments are approved for pediatric patients with psoriasis.Objective: To evaluate the pharmacokinetics and safety of apremilast, an oral phosphodiesterase 4 inhibitor, in pediatric patients with psoriasis.Methods: This phase 2, multicenter, open-label study enrolled pediatric patients with moderate to severe plaque psoriasis. Patients received apremilast twice daily without titration for 2 weeks (group 1 [age, 12-17 years; weight, $35 kg]: apremilast 20 or 30 mg; group 2 [age, 6-11 years; weight, $15 kg]: apremilast 20 mg), followed by a 48-week extension. Primary endpoints were pharmacokinetics and safety. Other endpoints were taste/acceptability and change from baseline in score on the Psoriasis Area and Severity Index.Results: A total of 42 enrolled patients (21 adolescents [age, 12-17 years] and 21 children [age, 6-11 years]) received apremilast. Pharmacokinetics modeling and noncompartmental analyses showed that weight-based dosing with apremilast 20 mg twice daily in children or apremilast 20 or 30 mg twice daily in adolescents provides exposure (area under the concentration-time curve from time 0 to 12 hours after the dose) that is comparable to that achieved with apremilast 30 mg twice daily in adults. The safety profile was generally similar to that in adults. Most study participants liked the taste of the tablet. Improvements from baseline in mean Psoriasis Area and Severity Index score were 68% for adolescents (overall) and 79% for children. Limitations:No children weighing less than 20 kg were enrolled.Conclusions: This first-time-in-children phase 2 study supports weight-based apremilast dosing for future phase 3 studies of pediatric plaque psoriasis. ( J Am Acad Dermatol 2020;82:389-97.)
Purpose of review Hypertrophic cardiomyopathy (HCM) is one of the leading causes of sudden cardiac death (SCD) in younger people and athletes. It is crucial to identify the risk factors for SCD in individuals with HCM. This review, based on recent systematic literature studies, will focus on the risk factors for SCD in patients with HCM. Recent findings An increasing number of studies have further explored the risk factors for SCD in patients with HCM, and new risk markers have emerged accordingly. In addition, more accurate SCD risk estimation and stratification methods have been proposed and continuously improved. Summary The identification of independent risk factors for HCM-related SCD would likely contribute to risk stratification. However, it is difficult to predict SCD with absolute certainty, as the annual incidence of SCD in adult patients with HCM is approximately 1%. The review discusses the established risk factors, such as a family history of SCD, unexplained syncope and some new risk factors. Taken together, the findings of this review demonstrate that there is a need for further research on individual risk factors and that SCD risk stratification in HCM patients remains a clinical challenge.
Backgroud/aim: The aim of this study was to assess the effect of a combination use of methimazole (MMI) and selenium (Se) in the treatment of Graves’ disease (GD). Materials and methods: A total of 103 newly-diagnosed hyperthyroidism patients were randomized to MMI and MMI+Se combination group. After treatment for six months, the levels of triiodothyronine (FT3), free thyroxine (FT4), thyrotrophin receptor antibody (TRAb), thyroid peroxidase antibody (TPOAb), and thyroglobulin antibody (TGAb) were observed. Besides, an in vitro culture model of thyroid cells was established and the protein expression and mRNA levels of TRAb, TPOAb and TGAb were determined by western blot and RT-PCR. Results: A significant decrease in the levels of FT3, FT4, TRAb, TPOAb and TGAb were observed in both groups along with a marked increase in TSH levels. Furthermore, the in vitro experiments showed that the protein expression and mRNA levels of TRAb, TPOAb and TGAb decreased significantly. Also, compared to the MMI group, there was a greater improvement of these indices in the MMI+Se group. Conclusion: We suggest that the combined use of MMI and Se could improve the thyroid activity in patients which may provide effective therapy for the treatment of GD in clinical settings. Key words: Graves’ disease; methimazole; selenium; TRAb; TPOAb; TGAb
BACKGROUND Primary cardiac tumors are uncommon, of which cardiac myxoma accounts for 50%-80%. Left ventricular myxoma has been rarely reported, accounting for only 3%-4% of all cardiac myxomas. Multiple left ventricular myxomas are, relatively, even rarer. CASE SUMMARY In this report, we present a case of multiple left ventricular myxomas combined with severe rheumatic valve lesions. Symptomatically, the patient presented with fatigue, shortness of breath, and palpitation after activities. The patient underwent complete surgical resection of multiple left ventricular myxomas combined with mechanical replacement of the mitral and aortic valves, tricuspid valvuloplasty. The patient recovered well after the operation, with no obvious related complications. CONCLUSION Multiple left ventricular myxomas may coexist with severe rheumatic valve disease. Operation is an effective treatment.
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