Nusinersen treatment in patients with spinal muscular atrophy: A safety analysis of laboratory parameters / 西安交通大学学报(医学版)

【Objective】 To explore the changes of laboratory parameters and safety of nusinersen in the treatment of spinal muscular atrophy (SMA). 【Methods】 Retrospective analysis was made on the six SMA patients treated with nusinersen in the Department of Neurology at The First Affiliated Hospital of Xi’an Jiaotong University from December 2021 to December 2022. We summarized the patients’ clinical data, including genetic diagnosis results, disease classification, and clinical manifestations. Intrathecal injection of 5 mL/12 mg of nusinersen administered on the 1st, 14th, 28th, and 63rd days, followed by maintenance treatment every 4 months. After each administration, we compared and evaluated the patients’ cerebrospinal fluid, blood routine, liver function, kidney function, and coagulation function with baseline values. We regularly followed up the patients and recorded adverse reactions after administration to evaluate medication safety. 【Results】 A total of six patients were diagnosed with SMA, including four cases of SMA3 type and two cases of SMA4 type. The deletion of exon 7 of the survival motor neuron gene 1 (SMN1) in patients led to changes in motor neuron, most of which were caused by limb weakness; in severe cases, the patients were unable to stand. The baseline abnormal test indicators of patients included the increase of cerebrospinal fluid lactate dehydrogenase (CSF-LDH), the increase of creatine kinase (CK), and the decrease of creatinine (Cr). After four times of treatment, the patients’ CSF-WBC, CSF-Glu, CSF-Pro, CSF-LDH, WBC, PLT, RBC, ALT, AST, GGT, BUN, uric acid (UA), INR, aPTT, and D-dimer had no significant difference from the baseline (P>0 05). The patients had no other significant adverse reactions except headaches, dizziness, and back pain after puncture. 【Conclusion】 Nusinersen has good safety on SMA patients.

The impact of development of population-based study in the biomedical field on laws and regulations: a cross-strait experience on biobank development.

Together with the completion of the Human Genome Project, biomedical research has marched into the "Post-Genomic Era." In order to take advantage of this extracted gene related information extensively and precisely so as to realize man's biological phenomena as well as the mechanism of pathogenesis, consequentially, a large scale sample collection of different geological areas and/or ethnic groups becomes necessary for the future population based genetic research of a country and, in turn, the construction of population-based genetic database (Biobank). In recent years, both mainland China and Taiwan have not only made great progress in information and computation technologies, but have also gradually taken a close look into the quality of medicine delivery. Thus, it becomes unavoidable for both sides to create each one's population-based genetic databases (Biobank). Theoretically speaking, the Biobank development shall benefit the study on the correlation between genes and disease and also the solution for disease treatment as well. At the same time, medical diagnostic technology has also been significantly improved. It is believable that the population-based genetic database might be utilized to promote medical quality and to reduce the cost of public health delivery. Further; in the near future, it might become the "raw materials "for medical research application. However when taking promotion of public welfare as the premises for a Biobank development, the severe and multi challenge occurred against the traditional legal rules in terms of the privacy protection, public trust development, the compliance of informed consent principle, the implementation of benefit-sharing doctrine and the possible discrimination concern about the population/participants selection and some other ELSI issues. In this paper, the major legal issues encountered by the Biobank development will first be reviewed accompanied by the background information concerning the Biobank development scenario crossing the Taiwan Strait. Also, mainly following the realm of comparative policy or legal approaches, the paper learning from the fruits of this comparative study, tries to propose some recommendations for future legislative consideration by both mainland China and Taiwan. It's been this author's wish that, when establishing a large scale population based Biobank, the promotion of public trust shall be placed as the primary goal together with the emphasis on supporting publicity and transparency on the administrative practices, so as to encourage the public participation in observing the principle of altruism and, in turn, benefit the future biomedicine development.

High-performance liquid chromatography-based determination of plasma and renal tissue cisplatin levels after subcutaneous cisplatin implantation in mice / 南方医科大学学报

<p><b>OBJECTIVE</b>To develop a method for determining plasma and renal tissue concentrations of cisplatin (DDP) after subcutaneous DDP implantation in mice.</p><p><b>METHODS</b>DDP was extracted from the plasma and tissue of mice receiving subcutaneous DDP implantation and reacted with sodium diethyldithiocarbamate (DDTC). The product Pt (DDTC)(2) extracted by diethyl ether was determined using high-performance liquid chromatography (HPLC) with the mobile phase of water and methanol at the ratio of 25:75 and the flow rate of 1.0 ml/min. The derivatives of DDP and nickel chloride were detected at the wavelength of 254 nm.</p><p><b>RESULTS</b>The linear range of DDP was 0.1-10 microg/ml (r=0.9998 for plasma and 0.9993 for kidney). The intra-day and inter-day RSD was below 10%, and the minimum concentration detectable was 50 ng.</p><p><b>CONCLUSION</b>The method is accurate and effective for determining plasma and tissue DDP levels after subcutaneous DDP administration and can be used in pharmacokinetic study of DDP.</p>

Protecting effect of brevifolin and 8,9-single-epoxy brevifolin of Phyllanthus simplex on rat liver injury / 中国中药杂志

<p><b>OBJECTIVE</b>To investigate the effect of protecting liver of brevifolin and 8,9-single-epoxy brevifolin of Phyllanthus simplex.</p><p><b>METHOD</b>Rats were administered with CCl4 (ip) or alcohol (ig) to establish acute or chronic liver injured model, respectively. ALT, AST and TBIL in serum were measured using colorimetric analysis to evaluate liver function. MDA content or SOD activity in serum and liver tissue was measured by thiobarbituric acid chromatometry and xanthine oxidase methods, respectively. The hemorheological parameters were observed.</p><p><b>RESULT</b>Brevifolin and 8,9-single-epoxy brevifolin reduced the increase of ALT induced by CCl4, but they did not influence the increase of AST. And it could inhibit the pathologic increase of serum TBIL induced by alcohol. They could ameliorate the MDA increase or SOD decrease in serum and liver tissue in rats with liver injury, and decrease abnormal changed hemorheological parameters.</p><p><b>CONCLUSION</b>Brevifolin and 8,9-single-epoxy brevifolin show protective effective against acute and chronic liver injuries, and the mechanism is relevant to antagonizing the lipid peroxidation of free radical and improving the blood circulation.</p>

Preparation of Paracetamol multiloculated implant and its drug release profile in vitro / 西安交通大学学报(医学版)

Objective To prepare Paracetamol (APP) multiloculated implant loaded with poly-lactide-co-glycolide acid (PLGA) and to study the drug release profile in vitro. Methods APP multiloculated implant was fabricated by micro-electro-mechanical system (MEMS), and high-performance liquid chromato graphy (HPLC) measurement was used to investigate in vitro drug release profile. HPLC analysis was carried out by employing C18 column and a mixture of methanol-water (15∶85) as mobile phase. The detection wavelength was 215nm and flow rate was 0.8mL/min. Results With different multiloculated shape, the rate of the drug release in vitro was varied significantly. Moreover, the releasing of APP multiloculated implant with ecto-tetragonum ento-hexagon in vitro conformed to Higuchi equation. Conclusion The technology of the preparations is feasible, and the structural and morphological characteristics of the multiloculated implant have a significant impact on the release speed of the drug delivery system.