Effectiveness and safety of PCSK9 inhibitor therapy in patients with familial hypercholesterolemia within a therapeutic program in Poland: Preliminary multicenter data.

BACKGROUND: In Poland, treatment with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors has become available free of charge in a therapeutic program. Assessed herein, is the efficacy and safety of alirocumab and evolocumab in patients with heterozygous familial hypercholesterolemia (FH). METHODS: Data of 55 adult FH patients who participated in the program were analyzed upon meeting the criteria established by the Ministry of Health (low density lipoprotein cholesterol [LDL-C] above 160 mg/dL on max. tolerated statin dose and ezetimib). The efficacy of PCSK9 inhibitors in reducing LDL-C with drug administration every 2 weeks was assessed after 3 months and 1 year of therapy. A safety profile evaluation was performed at each visit. 48 patients completed the 3-month and 21 for the 1-year observation periods (34 patients treated with alirokumab and 14 with evolocumab). RESULTS: The mean concentration of direct-measured LDL-C decreased from the initial level of 215.1 ± 74.5 mg/dL to 75.3 ± 64.1 mg/dL, i.e., by 65 ± 14% following 3 months of treatment. This effect was stable in 1-year observation (77.7 ± 72.8 mg/dL). Adverse effects were flu-like symptoms (13.0%), injection site reactions (11.1%), fatigue (5.6%) and musculoskeletal symptoms (5.6%). Seven patients failed to complete the 3-month treatment period due to side effects or non-compliance, and 1 patient failed to complete the 1-year treatment due to myalgia. CONCLUSIONS: This study confirmed high effectiveness of PCSK9 inhibitors in reducing LDL-C levels in patients with FH. Due to restrictive inclusion criteria with LDL-C threshold level > 160 mg/dL (> 4.1 mmol/L) required for participation in the therapeutic program, a relatively small number of FH patients were eligible for treatment.

Clinical side effects of continuous positive airway pressure in patients with obstructive sleep apnoea.

CPAP is considered the gold standard treatment in OSA and is highly efficacious in controlling OSA symptoms. However, treatment effectiveness is limited because of many factors including low adherence due to side effects. This review highlights the range of side effects associated with CPAP therapy in patients with OSA. This information is important for the initiation of patients onto CPAP as well as their continued care while on treatment, given the increase in non-medically supervised CPAP care models in use globally.

A Progress on ADME and the Toxicity of Fructus Psoralea / 世界科学技术-中医药现代化

Fructus psoralea is a tonic traditional Chinese herb commonly used in clinic.The chemical constituents of Fructus psoralea are complicated,mainly containing coumarins,flavonoids and monoterpene phenols with various pharmacological effects.Since the increasing number of reports on the toxicity of Fructus psoralea in clinic,the side effects including toxicity on the liver and kidney,as well as skin allergies have gradually attracted attention.The toxicity of Fructus psoralea is produced from ADME (absorption,distribution,metabolism and excretion) in vivo.In this paper,we collected and clarified the studies of ADME of Fructus psoralea in vivo,and summarized recent adverse clinical events and research over its toxicity.We propose to make a thorough study on the toxic material basis of Fructus psoralea and the toxicological mechanism of its extraction,fractions and compounds.The review provided a possible reference and the direction of research for the safe clinical use of Fructus psoralea.