1.Literature Review of Using Bisphosphonates in the Treatment of Calciphylaxis
Chunsu LIANG ; Shaohong WANG ; Yizhou ZHANG ; Xin LIU ; Ziran NIU ; Yili SHI ; Bo ZHANG
JOURNAL OF RARE DISEASES 2024;3(2):214-220
Objective To analyze the effectiveness and safety of bisphosphonates in the treatment of pa-tients with calcification defense.Methods PubMed,Embase databases,CNKI and Wanfang were searched to collect the case reports and clinical studies of bisphosphonates for calcification defense.Then,the relevant infor-mation of patients was extracted for statistical analysis.Results A total of 18 case reports were selected involving 20 patients.Thirteen patients(65.0%)were treated with pamidronate,four(20.0%)were treated with etidr-onate,two(10.0%)were treated with alendronate,and one(5.0%)was treated with zoledronic acid.Thirteen patients(65.0%)recovered completely,the recovery time of whom ranged from half month to nine months.The tolerance of bisphosphonates in most patients(90.0%)was good,while one patient who did not tolerate pamidr-onate recovered after the frequency of administration was adjusted and one patient with high dosage of etidronate returned to normal after the discontinuation of the usage.Conclusions Bisphosphonates,an inhibitor of bone resorption,is effective and safe in the treatment of patients with calcification defense.
2.Separation and simultaneous quantitation of PGF2 and its epimer 8--PGF2 using modifier-assisted differential mobility spectrometry tandem mass spectrometry.
Chunsu LIANG ; Hui SUN ; Xiangjun MENG ; Lei YIN ; J Paul FAWCETT ; Huaidong YU ; Ting LIU ; Jingkai GU
Acta Pharmaceutica Sinica B 2018;8(2):228-234
Because many therapeutic agents are contaminated by epimeric impurities or form epimers as a result of metabolism, analytical tools capable of determining epimers are increasingly in demand. This article is a proof-of-principle report of a novel DMS-MS/MS method to separate and simultaneously quantify epimers, taking PGF2 and its 8-epimer, 8--PGF2, as an example. Good accuracy and precision were achieved in the range of 10-500 ng/mL with a run time of only 1.5 min. Isopropanol as organic modifier facilitated a good combination of sensitivity and separation. The method is the first example of the quantitation of epimers without chromatographic separation.
3.Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Tingting XU ; Wei ZUO ; Xin LIU ; Qianqian FAN ; Yang GAO ; Chunsu LIANG ; Bo ZHANG
JOURNAL OF RARE DISEASES 2022;1(2):199-205
In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystrophin gene. Currently, the four ASO drugs approved internationally for DMD are all targeted at dystrophin, including eteplirsen, golodirsen, viltolarsen and casimersen. They all belong to phosphorodiamidate morpholino oligomers (PMO) antisense oligonucleotide drugs, so that their pharmacokinetic characteristics are similar. The drugs quickly spread to other tissues after intravenous administration. Because of the electrical neutrality of the PMO, they have a low binding rate to plasma proteins and are quickly metabolized by the kidney and excreted in the urine as archetypes. In addition, the likelihood of drug-drug interactions of ASO is low. Existing clinical studies have shown that they have certain clinical benefits and good tolerability, bringing new options for DMD treatment. This paper mainly discusses the pharmacological effects, pharmacokinetic characteristics, efficacy, and safety of ASO drugs for the treatment of DMD, hoping to provide scientific reference for the rational and safe clinical use of such drugs.
4.Palpitations, Shortness of Breath, Weakness in Limbs, Edema, and Dyspnea: A Rare Inflammatory Myopathy with Positive Aniti-mitochondrial Antibodies and Cardiac Involvement
Chunsu LIANG ; Xuchang ZHANG ; Ning ZHANG ; Lin KANG ; Xiaohong LIU ; Jiaqi YU ; Yingxian LIU ; Lin QIAO ; Yanli YANG ; Xiaoyi ZHAO ; Ruijie ZHAO ; Na NIU ; Xuelian YAN
Medical Journal of Peking Union Medical College Hospital 2025;16(1):248-255
This article presents a case study of a patient who visited the Geriatric Department of Peking Union Medical College Hospital due to "palpitations, shortness of breath for more than 2 years, limb weakness for 6 months, edema, and nocturnal dyspnea for 2 months". The patient exhibited decreased muscle strength in the limbs and involvement of swallowing and respiratory muscles, alongside complications of heart failure and various arrhythmias which were predominantly atrial. Laboratory tests revealed the presence of multiple autoantibodies and notably anti-mitochondrial antibodies. Following a comprehensive multidisciplinary evaluation, the patient was diagnosed with anti-mitochondrial antibody-associated inflammatory myopathy. Treatment involved a combination of glucocorticoids and immunosuppressants, along with resistance exercises for muscle strength and rehabilitation training for lung function, resulting in significant improvement of clinical symptoms. The case underscores the importance of collaborative multidisciplinary approaches in diagnosing and treating rare diseases in elderly patients, where careful consideration of clinical manifestations and subtle abnormal clinical data can lead to effective interventions.