Internal medicine
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Multicenter Study Observational Study
Screening for Gaucher Disease Using Dried Blood Spot Tests: A Japanese Multicenter, Cross-sectional Survey.
Objective For patients with Gaucher disease (GD), a rare, inherited lysosomal storage disease, obtaining a definitive diagnosis is currently time-consuming and costly. A simplified screening method to measure the glucocerebrosidase (GBA) activity using dried blood spots (DBS) on filter paper has recently been developed. Using this newly developed screening method, we evaluated real-world GD screening in patients suspected of having GD. ⋯ Of the remaining 55 patients who had 2 DBS assays performed, 11 had a low GBA activity in both assays. Overall, DBS screening identified 12 (1.2%) patients with a low GBA activity, a proportion consistent with prior screening studies. Conclusion These results suggest that the simplified DBS method was less burdensome to patients, was easily utilized by many physicians, and could be a useful first-tier screening assay for GD prior to initiating burdensome genetic testing.
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Objective Persistent hepatitis B virus (HBV) and hepatitis C virus (HCV) infections are major causative factors of hepatic cirrhosis and hepatocellular carcinoma. However, the development of antiviral treatment has enabled their suppression. Therefore, the early detection and treatment of these infections are important. ⋯ The awareness of the need to refer patients to hepatologists increased from 84.7-85.4% before to 93.0% after the lecture. The most frequent reasons for not referring patients previously were "I had no knowledge and/or interest" (28.1% of responses) and "All I did was explain the results orally" (24.2%). Conclusion More widespread education of healthcare personnel is important to increase the number of individuals receiving appropriate treatment from specialist physicians.
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Randomized Controlled Trial
Safety and Efficacy of Zoledronic Acid Treatment with and without Acetaminophen and Eldecalcitol for Osteoporosis.
Objectives We aimed to investigate the safety of zoledronic acid (ZOL) combined with acetaminophen (APAP) regarding both the adverse events and the efficacy of ZOL combined with an eldecalcitol (ELD) in a randomized clinical trial conducted in patients with primary osteoporosis. Methods A total of 109 patients were administered ZOL 5 mg and then were randomly assigned to the following groups (3:2:1): those treated with ZOL, those treated with ZOL combined with APAP and ELD, and those treated with ZOL combined with ELD. For the analyses, the groups were classified into four treatment groups: patients treated with APAP (APAP group) and without APAP (non-APAP group), and those treated with ELD (ELD group) and without ELD (non-ELD group). ⋯ The percent changes in the bone mineral density values from baseline (ΔBMD) in the ELD and non-ELD groups at 12 months were 8.2% and 6.2% for the lumbar spine, 4.2% and 4.0% for the total hip, and 3.9% and 2.2% for the femoral neck, respectively. The ΔBMD of all sites did not differ significantly between the ELD and non-ELD groups. Conclusion In ZOL treatment, the co-administration of APAP should thus be considered as a therapeutic option to reduce the occurrence of symptomatic adverse events stemming from ZOL treatment in Japanese patients with primary osteoporosis, particularly in younger patients.
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Multicenter Study
Impact of Dapagliflozin on the Left Ventricular Diastolic Function in Diabetic Patients with Heart Failure Complicating Cardiovascular Risk Factors.
Objective Our aim was to investigate the impact of the sodium glucose cotransporter type 2 (SGLT2) inhibitor on the left ventricular (LV) diastolic function in type 2 diabetes mellitus (T2DM) patients with chronic heart failure (HF) complicating cardiovascular risk factors. Methods We analyzed data from our previous prospective multicenter study, in which we investigated the effect of dapagliflozin on the LV diastolic function of T2DM patients with stable HF at five institutions in Japan. Patients who had been taking at least 1 antidiabetic drug other than SGLT2 inhibitors started treatment with dapagliflozin. ⋯ A multivariate logistic regression analysis showed that dyslipidemia was the only independent determinant of improvement in the E/e' after the administration of dapagliflozin among cardiovascular risk factors. Furthermore, the relative change in the E/e' from baseline to six months after the administration of dapagliflozin for HF patients with preserved ejection fraction (HFpEF) and dyslipidemia was significantly larger than that for HFpEF patients without dyslipidemia (-15.2% vs. 29.6%, p=0.014), but no such finding was observed in non-HFpEF patients. Conclusion SGLT2 inhibitors may exert a more beneficial effect on the LV diastolic function for T2DM patients with stable HF, especially those with complicating dyslipidemia, than existing treatments.
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Case Reports
Sorafenib Rechallenge and Sorafenib after Lenvatinib Failure in a Patient with Hepatocellular Carcinoma.
A 70-year-old man was diagnosed with multiple lung metastases from hepatocellular carcinoma, and lenvatinib was initiated. Three months later, the response was progressive disease. Sorafenib therapy as a second-line drug was started. ⋯ After the regorafenib failure, sorafenib rechallenge therapy as a fourth-line treatment was initiated. The sorafenib rechallenge, which continued for two months, induced a partial response. Sorafenib after lenvatinib failure and sorafenib rechallenge may be a good option, but further prospective studies are needed.