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1.
Chinese Journal of Experimental Traditional Medical Formulae ; (24): 232-243, 2024.
Artigo em Chinês | WPRIM | ID: wpr-999181

RESUMO

Paridis Rhizoma possesses the functions of clearing heat and detoxifying, alleviating swelling and relieving pain, cooling the liver and calming the convulsion. Saponins are the main active components of Paridis Rhizoma. Studies have shown that total saponins in Paridis Rhizoma have obvious inhibitory effect on solid tumors such as breast cancer, lung cancer, gastric cancer, and liver cancer and non-solid tumors such as leukemia. The saponins may exert the anti-tumor effects by inhibiting the proliferation, migration, and invasion of tumor cells, regulating cell cycle, inducing apoptotic and non-apoptotic death pathways, and regulating metabolism and tumor microenvironment. Furthermore, total saponins in Paridis Rhizoma showed anti-inflammatory, antioxidant, antimicrobial, hemostatic, and uterus-contracting activities. At the same time, they may induce apoptosis of normal cells, inflammation and oxidative stress, and metabolic disorders. In recent years, the reports of liver injury, reproductive injury, gastrointestinal injury, hemolysis, and other adverse reactions caused by total saponins in Paridis Rhizoma have been increasing. Pharmacokinetic studies have shown that there are significant differences in the metabolism of total saponins in Paridis Rhizoma administrated in different ways. Injection has a fast clearance rate, while oral administration may have hepatoenteric circulation. Meanwhile, due to the low solubility and activation of P-glycoprotein (P-gp) molecular pump, the prototype absorption, intestinal permeability, and recovery rate of total saponins in Paridis Rhizoma are poor, which affects the bioavailability. The bioavailability can be improved to some extent by preparing new dosage forms or new drug delivery systems with advanced technology. This paper reviews the pharmacological effect, pharmacokinetics, and adverse reactions of Rhizoma Paridis total saponins by searching the China National Knowledge Infrastructure (CNKI), VIP, and Web of Science with ''Rhizoma Paridis total saponins'' as the keywords, hoping to provide references for the research, development, and clinical application of such components.

2.
Artigo em Inglês | LILACS-Express | LILACS | ID: biblio-1529455

RESUMO

ABSTRACT Tuberculosis stands as one of humanity's oldest afflictions, intrinsically intertwined with social disparities. This formidable disease spares no age group and remains the prevailing cause of infection-induced mortality worldwide, particularly in developing nations. We present a case of a 56-year-old woman with diabetes who was diagnosed with Pulmonary Tuberculosis. After receiving antituberculosis drugs as part of her treatment, she experienced a range of systemic manifestations and suffered from severe ulcerative esophagitis. This adverse reaction led to uncontrollable gastrointestinal intolerance, tragically resulting in her untimely demise. The incident underscores the potential seriousness of adverse reactions that can arise from tuberculosis treatment medications.

3.
Journal of Prevention and Treatment for Stomatological Diseases ; (12): 36-42, 2024.
Artigo em Chinês | WPRIM | ID: wpr-1003442

RESUMO

Objective@#To compare the recurrence rates between 755 nm Q-switched alexandrite laser (QSAL) treatment and surgical excision of oral melanotic macules (OMM).@*Methods@#This study was reviewed and approved by the Ethics Committee, and informed consent was obtained from the patients. A retrospective cohort study was designed to collect demographic and clinical characteristics and follow-up data from patients with OMM. Patients who received QSAL or surgical excision in the Department of Oral Medicine, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine from January 2019 to August 2021 were included. The one-year recurrence rate was investigated as the primary outcome. Long-term adverse reaction rates were investigated as safety indicators. Kaplan-Meier analyses were performed to analyze the recurrence-free rates between the groups.@*Results@#A total of 57 patients were enrolled in this study. 16 patients underwent surgical excision, and 41 underwent QSAL. The baseline demographic and clinical characteristics between the groups were not significantly different. No recurrence (0%) of OMM was observed in the surgical excision group, while in the QSAL group, the macule recurred in 12 patients (29.27%). The average duration of recurrence was 6.08 months after treatment. Recurrence was not found to be associated with smoking (P = 1.000), gastrointestinal polyps (P = 1.000), longitudinal melanonychia (P = 0.187), family history (P = 0.552), treatment sessions (P = 0.567) or multiple macule lesions (P = 0.497). Compared with treatment with surgical excision, the odds ratio of recurrence for treatment with QSAL was 4.41, with a 95% confidence interval of 1.27-15.24 (P = 0.020). In the surgical excision group, 3 patients (18.75%) reported depressions and scars on the lesion, while no long-term adverse reactions (0%) were reported in the QSAL group (P = 0.019).@*Conclusion@#Compared with surgical excision, the advantage of QSAL is the low long-term adverse reaction rate, while the disadvantage is the relatively high one-year recurrence rate. It is necessary to communicate the advantages and disadvantages of the two methods with OMM patients to assist in clinical decision-making.

4.
Chinese Journal of Clinical Pharmacology and Therapeutics ; (12): 339-347, 2024.
Artigo em Chinês | WPRIM | ID: wpr-1014545

RESUMO

Immunotherapy has become a global focus in cancer clinical practice and scientific research. In the past two years, PD-1\PD-L1 and CTLA-4 inhibitors, especially Nivolumab, Pembrolizumab, Atezolizumab and Lpilimumab, have been used in non-small cell lung cancer, colon cancer. Promising results have been obtained in malignancies such as melanoma and urinary tract cancer. Traditional Chinese medicine has a long history in China. Modulating immune checkpoints has certain advantages in treating malignant tumors, and it has shown good efficacy in improving its adverse events. This article reviews the role of traditional Chinese medicine in regulating immune checkpoints and improving adverse reactions and its application prospects in immunomodulatory treatment.

5.
China Pharmacy ; (12): 877-880, 2024.
Artigo em Chinês | WPRIM | ID: wpr-1013553

RESUMO

OBJECTIVE To provide reference for the safe use of bevacizumab in cancer patients. METHODS The diagnosis and treatment of a 65-year-old female lung adenocarcinoma patient with diabetic ketoacidosis (DKA) induced by bevacizumab was retrospectively analyzed, and the possible mechanisms and causes were analyzed based on literature review. RESULTS & CONCLUSIONS The diagnosis and treatment process of patients were analyzed, and DKA caused by other drugs and disease factors were excluded. DKA was considered to be caused by the use of bevacizumab according to Naranjo’s ADR evaluation scale; the acidosis of the patient improved rapidly after one hemodialysis treatment. DKA caused by bevacizumab is rare in clinic, clinicians should be aware that bevacizumab may affect pancreatic function and induce DKA, and early detection and treatment should be achieved to improve the prognosis.

6.
Indian J Ophthalmol ; 2023 Jun; 71(6): 2587-2591
Artigo | IMSEAR | ID: sea-225104

RESUMO

Vogt–Koyanagi–Harada (VKH) disease, a bilateral granulomatous panuveitis associated with multisystem involvement, is a T-cell-mediated autoimmune disorder in which cytotoxic T-cell target melanocytes in genetically susceptible individuals. Recently, there has been an increase in literature on the new onset of uveitis and reactivation of previously diagnosed cases of uveitis following Covid-19 vaccinations. It has been postulated that Covid-19 vaccines can lead to an immunomodulatory change resulting in an autoimmune phenomenon in the recipients. VKH following COVID-19 infection was reported in four patients and a total of 46 patients developing VKH or VKH-like disease following COVID-19 vaccinations. There are reports of four patients who had been recovering or recovered from VKH after receiving the first dosage of the vaccine and developed worsening of ocular inflammation after receiving the second dose of the vaccine.

7.
Med. U.P.B ; 42(1): 96-99, ene.-jun. 2023.
Artigo em Espanhol | LILACS, COLNAL | ID: biblio-1416211

RESUMO

La pancreatitis en pediatría se consideraba anteriormente una enfermedad poco fre­cuente; en la actualidad se reportan 13.2 casos por 100 000 niños/año. La causa más importante de pancreatitis en la población pediátrica, después de la etiología biliar, son los medicamentos (13% de los casos). Uno de los principales medicamentos como causa de pancreatitis en pediatría es el ácido valproico (AV); el cual puede inducir una pancreatitis aguda. Aquí se presentará el primer caso de pancreatitis por AV en población pediátrica reportado en Colombia. Se trata de un paciente de cuatro años, con trastorno en el neurodesarrollo por un síndrome de TORCH, quien tomaba AV a largo plazo por un trastorno de la conducta. Ingresó a una institución de alta complejidad donde se diagnostica pancreatitis aguda con signos de necrosis en tejido pancreático secundario a uso de AV. Se suspendió el medicamento con resolución de su cuadro clínico y alta médica hacia el día 15


Pediatric pancreatitis was previously considered a rare disease. Currently, 13.2 cases are reported per 100,000 children/year. The most important cause of pancreatitis in the pediatric population, after biliary etiology, are medications (13% of cases). One of the main medications as a cause of pediatric pancreatitis is valproic acid (VA), which can lead to acute pancreatitis. Here we will present the first case of VA pancreatitis in the pediatric population reported in Colombia. This is a four-year-old patient, with a neurodevelopmental disorder due to TORCH syndrome, who was taking VA long-term for a conduct disorder. He was admitted to a highly complex institution where acute pancreatitis was diagnosed with signs of necrosis in pancreatic tissue secondary to the use of VA. The medication was discontinued with resolution of his set of symptoms and medical discharge around day 15.


A pancreatite pediátrica era anteriormente considerada uma doença rara; atualmente, 13,2 casos por 100 000 crianças/ano são relatados. A causa mais importante de pancreatite na população pediátrica, depois da etiologia biliar, são os medicamentos (13% dos casos). Uma das principais medicações como causa de pancrea-tite em pediatria é o ácido valpróico (VA); que podem induzir pancreatite aguda. Aqui apresentaremos o primeiro caso de pancreatite AV na população pediátrica relatado na Colômbia. Trata-se de uma paciente de quatro anos de idade, com transtorno do neuro-desenvolvimento devido à síndrome TORCH, que fazia uso de AV de longa duração para um transtorno de conduta. Ele foi internado em uma instituição de alta complexidade onde foi diagnosticado pancreatite aguda com sinais de necrose no tecido pancreático secundário ao uso de AV. A medicação foi suspensa com resolução do quadro clínico e alta médica por volta do 15º dia


Assuntos
Humanos , Recém-Nascido , Lactente , Pré-Escolar , Criança , Adolescente , Pancreatite , Pediatria , Preparações Farmacêuticas , Ácido Valproico
8.
Chinese Journal of Hematology ; (12): 490-494, 2023.
Artigo em Chinês | WPRIM | ID: wpr-984649

RESUMO

Objective: To investigate the efficacy and safety of Bruton tyrosine kinase inhibitors (BTKi) ibrutinib or zanubrutinib monotherapy in newly diagnosed patients with Waldenström macroglobulinemia (WM) . Methods: The efficacy and adverse effects of 58 patients with newly diagnosed WM receiving BTKi monotherapy in Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine were analyzed retrospectively from January 2018 to August 2022. Results: The response of 55 patients may be examined. Forty patients received ibrutinib monotherapy for a median of 15 months, with an overall response rate (ORR) of 85%, a main remission rate (MRR) of 70%, and a very good partial remission (VGPR) rate of 10%. Fifteen patients received zanubrutinib monotherapy for a median of 13 months, with an ORR of 93%, an MRR of 73%, and a VGPR rate of 0%. For various reasons, 10 patients were converted from ibrutinib to zanubrutinib. Ibrutinib treatment lasted an average of 7.5 months before conversion. The median duration of zanubrutinib therapy after conversion was 3.5 months. The ORRs before and after conversion were 90% and 100%, MRRs were 80% and 80%, and VGPR rates were 10% and 50%, respectively. After a median of 16 months, the 24-month progression-free survival (PFS) rate of patients who received both BTKi was 86%. PFS did not differ statistically across individuals with low, medium, and high-risk ISS scores (P=0.998). All of the patients survived. The most common side effects of BTKi were neutropenia and thrombocytopenia, which occurred in 12% and 10% of all patients, respectively. Ibrutinib accounts for 5% of atrial fibrillation, and zanubrutinib has a 7% risk of bleeding. Conclusions: In treating WM, ibrutinib or zanubrutinib provides good efficacy and tolerable adverse effects.


Assuntos
Humanos , China , Estudos Retrospectivos , Resultado do Tratamento , Inibidores de Tirosina Quinases/uso terapêutico , Macroglobulinemia de Waldenstrom/tratamento farmacológico
9.
Acta Pharmaceutica Sinica ; (12): 2551-2559, 2023.
Artigo em Chinês | WPRIM | ID: wpr-999023

RESUMO

Single cell RNA sequencing (scRNA-seq) is an advanced technology to study the transcriptome information at the single cell level. The application of this technology can attribute to analyze the heterogeneous map of cells in the process of disease development, and precisely identify the specific cell subsets that are responsive to pharmacological therapy. Currently, scRNA-seq technology has been widely applied in the field of drug research, including studies on therapeutic targets, drug-induced adverse reactions, drug resistance and vaccine. This work reviews the application of scRNA-seq technology in drug discovery, which offers a scientific basis for personalized and accurate medication therapy.

10.
China Pharmacy ; (12): 2766-2769, 2023.
Artigo em Chinês | WPRIM | ID: wpr-998563

RESUMO

OBJECTIVE To analyze the effects of tigecycline on coagulation function in patients with severe renal insufficiency, and to provide a reference for safe clinical drug use. METHODS Retrospective analysis was performed for the clinical data of patients with severe renal dysfunction complicated with infection receiving tigecycline admitted to nephrology department of our hospital from January 2021 to October 2022. The levels of prothrombin time (PT), activated partial thromboplastin time (APTT), international normalized ratio (INR), platelet (PLT) and fibrinogen (FIB) were compared 3 days before medication, with 1-5, 6-10, 11-15 and 16-20 days after medication, 5 days after withdrawal and/or after symptomatic treatment. RESULTS Finally, 14 patients were included, and 9 patients developed coagulopathy, with an incidence of 64.29%. Compared with 3 days after medication, the levels of FIB at 6-10 and 11-15 days after medication, and PLT at 1-5 , 6-10 and 11- 15 days after medication were decreased significantly, while the levels of PT at 1-5 and 6-10 days after medication, APTT at 1-5, 6-10 and 11-15 days after medication were significantly prolonged, and INR increased significantly at 1-5 and 6-10 days after medication (P<0.05). Compared with 3 days before medication, there were no statistically significant changes in FIB, PT, INR, APTT and PLT at 16-20 days after medication and 5 days after withdrawal and/or symptomatic treatment(P>0.05). CONCLUSIONS Patients with severe renal insufficiency should be cautious with tigecycline, which can lead to prolonged PT and APTT, increased INR, and decreased PLT and FIB. If medication time is over 14 days, dynamic monitoring of coagulation function indicators is recommended to reduce the risk of adverse reactions.

11.
International Eye Science ; (12): 1983-1987, 2023.
Artigo em Chinês | WPRIM | ID: wpr-998476

RESUMO

Thyroid-associated ophthalmopathy(TAO)is an autoimmune disease associated with thyroid dysfunction that can significantly impact quality of life, result in visual impairment and facial disfigurement. Traditional treatments are often unsatisfactory. Studies have shown that teprotumumab, a human monoclonal antibody that can inhibit insulin-like growth factor 1 receptor(IGF-1R), has become an emerging targeted drug for TAO. Although the drug has proven to be effective and relatively safe in the treatment of TAO, adverse reactions are worthy of attention of ophthalmologists with the continuous promotion of clinical application, including hearing impairment, hyperglycemia, diarrhea, muscle spasms, infusion reactions, cognitive decline, thyroid suppression, alopecia, nausea and fatigue. Teprotumumab was generally well tolerated, with most adverse events being mild or moderate in severity. This paper aims to review the adverse reactions and precautions of teprotumumab in the treatment of TAO.

12.
Chinese Journal of Schistosomiasis Control ; (6): 398-406, 2023.
Artigo em Chinês | WPRIM | ID: wpr-997254

RESUMO

Echinococcosis is a zoonotic parasitic disease caused by infection with Echinococcus species. As the drug of first choice for treatment of echinococcosis, albendazole suffers from problems of large doses and remarkable adverse reactions in clinical therapy. Development of novel drugs against echinococcosis is of urgent need. Recently, great advances have been achieved in the research on traditional Chinese medicine for treatment of echinococcosis. This review summarizes the progress of researches on traditional Chinese medicine for treatment of echinococcosis, aiming to provide insights into development of anti-echinococcosis drugs.

13.
Chinese Journal of Experimental Traditional Medical Formulae ; (24): 254-264, 2023.
Artigo em Chinês | WPRIM | ID: wpr-996832

RESUMO

Paridis Rhizoma, a traditional valuable Chinese herbal medicine, has the functions of clearing heat and removing toxin, relieving edema and pain, cooling liver and calming convulsion, which can be used to treat various diseases such as mumps, abscess, burn, bleeding, and tumor. It has been used in folk medicine for a long time and is the main raw material of various Chinese patent medicines such as Gongxuening Capsules and Yunnan Baiyao. Polyphyllin Ⅰ, an isospirostanol saponin and one of the main active components in Paridis Rhizoma, is distributed in the rhizome, pericarp, and leaves of Paris polyphylla. With high polarity, polyphyllin Ⅰ is mainly extracted by n-butanol extraction and macroporous adsorption resin chromatography, separated by silica gel column chromatography and preparative high performance liquid chromatography, and purified with the combination of methods. With anti-tumor, anti-inflammatory, antibacterial, and anti-virus effects, it is generally employed to treat liver cancer, lung cancer, gastric cancer and other cancers as well as arthritis, influenza, sore toxin, and bacterial infection. However, polyphyllin Ⅰ may cause stomach irritation, hemolysis, liver damage, kidney damage, heart damage, and other adverse reactions. Pharmacokinetic studies show that it has problems such as low bioavailability and poor intestinal absorption and permeability, which affect the clinical application of polyphyllin Ⅰ. This paper summarizes the research on the plant sources, extraction and separation methods, pharmacological effects, adverse reactions, and pharmacokinetics of polyphyllin Ⅰ in recent years, which is expected to provide a reference for the rational clinical application and other in-depth research work of polyphyllin Ⅰ.

14.
Chinese Journal of Internal Medicine ; (12): 84-87, 2023.
Artigo em Chinês | WPRIM | ID: wpr-994392

RESUMO

The study aimed to analyze the efficacy and safety of rituximab in the treatment of 23 cases of lupus nephritis and explore the prospect of half-dose rituximab in lupus nephritis treatment. Twenty-three patients with lupus nephritis hospitalized in the Department of Rheumatology and Immunology at the First Medical Center of the PLA General Hospital from May 2013 to December 2021 were selected. Eighteen patients received rituximab 375 mg/m 2 on the first and 14th days, 5 patients received 500 mg of rituximab on the first and 14th days, and rituximab was used as needed 6 months later. Methylprednisolone (80-120 mg) was given together with rituximab. Afterward, 1 mg/kg prednisone was used for 4 weeks, which was progressively tapered to maintenance doses or discontinued. B lymphocyte level, renal function, 24-h urine protein level, and systemic lupus erythematosus (SLE) disease activity index 2000 (SLEDAI2K) score before and after treatment were recorded. The efficacy and adverse reactions were analyzed. The results showed that 11 patients suffered from renal insufficiency [creatinine (162.7±58.6) μmol/L ] at baseline, while the creatinine level of 9 patients returned to normal 12 months after the treatment [ (66.3±10.1)μmol/L ]. Normal renal function of the other 12 patients was maintained during treatment. After 12 months, the 24-h urine protein level decreased from 4.00 (2.00,6.80) g in the baseline period to 0.10 (0.08,0.40) g. SLEDAI2K score decreased from 22 (18,26) in the baseline period to 3 (0,6) 12 months after the treatment. The B lymphocyte level reached 0.00 (0.00,0.01)% at 3 months. Of 23 patients, 13 patients achieved complete remission, and 7 patients achieved partial remission after 6 months of rituximab treatment. Five patients experienced adverse reactions related to rituximab, including 1 case of transfusion reaction, 1 case of perioral herpes with pulmonary infection, and 3 cases of decreased IgG levels. Therefore, rituximab regimen used in this study can be an effective treatment strategy for lupus nephritis.

15.
Chinese Journal of Radiation Oncology ; (6): 235-240, 2023.
Artigo em Chinês | WPRIM | ID: wpr-993180

RESUMO

Objective:To identify dose-volume parameters to predict the incidence of acute intestinal toxicity in cervical cancer patients after postoperative adjuvant radiotherapy.Methods:Clinical data of 93 cervical cancer patients who underwent postoperative adjuvant intensity-modulated radiotherapy (IMRT) were retrospectively evaluated. The dose-volume parameters comprised the absolute volume of the bowel receiving 5-45 Gy (5 Gy interval) radiation dose and the total volume of the bowel. The acute radiation-induced intestinal toxicity was evaluated by Radiation Therapy Oncology Group (RTOG) criteria. The association between the irradiated bowel volume and acute intestinal toxicity was analyzed.Results:A total of 26 (28%) patients experienced grade ≥2 acute intestinal toxicity. A strong relationship between grade ≥2 acute intestinal toxicity and the irradiated small bowel volume was observed at the total volume of small bowel, small bowel V 5 Gy, V 10 Gy and V 15 Gy (all P<0.05). Small bowel V 10 Gy ( HR=1.028, 95% CI, 0.993-1.062, P=0.029) and small bowel ?V 15 Gy( HR=0.991, 95% CI, 0.969-1.013, P=0.034)were the independent risk factors for evident acute intestinal toxicity. Conclusion:Dose-volume parameters of the small bowel can be used as predictors for the occurrence of grade ≥2 acute intestinal toxicity in cervical cancer patients undergoing postoperative adjuvant radiotherapy.

16.
Chinese Critical Care Medicine ; (12): 88-92, 2023.
Artigo em Chinês | WPRIM | ID: wpr-991984

RESUMO

Objective:To investigate the effects of continuous renal replacement therapy (CRRT) on plasma concentration, clinical efficacy and safety of colistin sulfate.Methods:Clinical data of patients received with colistin sulfate were retrospectively analyzed from our group's previous clinical registration study, which was a prospective, multicenter observation study on the efficacy and pharmacokinetic characteristics of colistin sulfate in patients with severe infection in intensive care unit (ICU). According to whether patients received blood purification treatment, they were divided into CRRT group and non-CRRT group. Baseline data (gender, age, whether complicated with diabetes, chronic nervous system disease, etc), general data (infection of pathogens and sites, steady-state trough concentration, steady-state peak concentration, clinical efficacy, 28-day all-cause mortality, etc) and adverse event (renal injury, nervous system, skin pigmentation, etc) were collected from the two groups.Results:A total of 90 patients were enrolled, including 22 patients in the CRRT group and 68 patients in the non-CRRT group. ① There was no significant difference in gender, age, basic diseases, liver function, infection of pathogens and sites, colistin sulfate dose between the two groups. Compared with the non-CRRT group, the acute physiology and chronic health evaluation Ⅱ (APACHE Ⅱ) and sequential organ failure assessment (SOFA) were higher in the CRRT group [APACHE Ⅱ: 21.77±8.26 vs. 18.01±6.34, P < 0.05; SOFA: 8.5 (7.8, 11.0) vs. 6.0 (4.0, 9.0), P < 0.01], serum creatinine level was higher [μmol/L: 162.0 (119.5, 210.5) vs. 72.0 (52.0, 117.0), P < 0.01]. ② Plasma concentration: there was no significant difference in steady-state trough concentration between CRRT group and non-CRRT group (mg/L: 0.58±0.30 vs. 0.64±0.25, P = 0.328), nor was there significant difference in steady-state peak concentration (mg/L: 1.02±0.37 vs. 1.18±0.45, P = 0.133). ③ Clinical efficacy: there was no significant difference in clinical response rate between CRRT group and non-CRRT group [68.2% (15/22) vs. 80.9% (55/68), P = 0.213]. ④ Safety: acute kidney injury occurred in 2 patients (2.9%) in the non-CRRT group. No obvious neurological symptoms and skin pigmentation were found in the two groups. Conclusions:CRRT had little effect on the elimination of colistin sulfate. Routine blood concentration monitoring (TDM) is warranted in patients received with CRRT.

17.
Chinese Journal of Applied Clinical Pediatrics ; (24): 465-468, 2023.
Artigo em Chinês | WPRIM | ID: wpr-990062

RESUMO

Spinal muscular atrophy (SMA) was a severe inherited neuromuscular disease caused by the deficiency of the survival motor neuron (SMN) protein encoded by SMN1 gene with pathogenic variants.According to the age of onset and maximal achievement of motor function, SMA was classified into 4 subtypes.The most common and severe subtype was SMA type 1.During the natural course, most of patients with SMA type 1 die of respiratory failure within 2 years of age if not treated.Patients with other subtypes have progressive muscle weakness and atrophy at varying degrees.Before the emergence of disease-modifying therapy, a multidisciplinary management, including physical therapy, respiratory and nutritional support, has been the only approach to delay the disease progression and enhance the survival rate of SMA.However, motor milestone progress is unable to achieve by SMA patients, because the lack of SMN protein has not been solved.In the past 5 years, 3 drugs have been approved for the treatment of SMA.Clinical trials and a growing number of real-world study data have shown that medications of disease-modifying agents contribute to effectively improve motor function in SMA patients with different subtypes, and promote the motor milestone progress in some patients, which significantly reduce the mortality.However, treatment response of disease-modifying agents to SMA varies with the subtypes of SMA, individualized conditions and courses of disease.Therefore, it is particularly important to choose an optimal treatment to ensure the quality of life of patients.This review focuses on recent advances and emerging challenges in the treatment of SMA.

18.
International Journal of Pediatrics ; (6): 272-276, 2023.
Artigo em Chinês | WPRIM | ID: wpr-989080

RESUMO

Ketogenic diet(KD)is a formulation diet with a high proportion of fat, low proportion of carbohydrates, appropriate protein and other nutrients, which has been used for centuries in the treatment of refractory epilepsy.In recent years, KD has been shown to be effective in the treatment of other diseases, such as amyotrophic lateral sclerosis, traumatic brain injury, diabetes, obesity, etc.Although KD has a positive effect on the treatment of a variety of diseases, the short-term and long-term adverse reactions caused by the imbalance of its nutritional structure should not be ignored.This article reviews the adverse reactions of KD in the treatment of children with refractory epilepsy and the corresponding prevention and treatment measures, to guide safe and efficient implementation of KD therapy in the clinic.

19.
Chinese Journal of Blood Transfusion ; (12): 75-79, 2023.
Artigo em Chinês | WPRIM | ID: wpr-1004893

RESUMO

【Objective】 To observe the regulation of autonomic nerves in blood donors during blood donation by heart rate variability analysis and explore the possible mechanism of donation related vasovagal reaction. 【Methods】 Electrocardiogram (ECG) of 90 blood donors was monitored by Fontaine Ⅰlead during the whole process of blood donation, and the 5-min heart rate variation before, during and after blood donation was analyzed. 【Results】 During the whole process of blood donation, the sympathetic HRV index (LF nu) and the sympathetic and vagal balance ability index (LF/HF) increased, whereas the vagal nerve index (pNN50, RMSSD, HFnu) and heart rate variability index (SDNN, Total power) decreased. For baseline heart rate variability of different blood donors (first-time vs. repeated, male vs. female, 18-24 years old vs. ≥25 years old, <400 mL vs. 400 mL) before blood donation, the pNN50, RMSSD and Total power of 18-24 years old blood donors were higher, but other indicators showed no significant difference. There were differences in HRV indexes of different types of blood donors during blood donation compared with before blood donation. The decrease of pNN50 and HFnu and the increase of LF/HF were larger in experienced blood donors than in first-time blood donors. The decrease of RMSSD was larger in male blood donors than in female blood donors; the change of LF/HF was larger in blood donors aged≥25 years than in blood donors aged 18-24 years; other indicators had no significant difference. 【Conclusion】 Blood donation leads to reflex readjustment of the cardiac autonomic tone: the sympathetic nerve is excited while the vagal nerve is suppressed. The cardiac autonomic nerve function of first-time blood donors, female donors and low-age (18-24 years old) donors to blood donation stress is not fully regulated. Donation related vasovagal reaction may be related to the autonomic nerve regulation function of blood donors.

20.
Chinese Journal of Blood Transfusion ; (12): 889-891, 2023.
Artigo em Chinês | WPRIM | ID: wpr-1004715

RESUMO

【Objective】 To explore the relationship between the storage time of leukodepleted red blood cells and transfusion adverse reactions by analyzing the occurrence of transfusion adverse reactions of patients after leukodepleted red blood cells transfusion from four hospitals. 【Methods】 By using the electronic medical record management system, the collection and transfusion dates of leukodepleted red blood cells from four hospitals in Enshi Prefecture from 2018 to 2022, as well as the information on transfusion adverse reactions, were retrieved. 【Results】 From 2018 to 2022, a total of 697 61 bags of leukodepleted red blood cells were transfused in four hospitals, resulting in 166 cases of transfusion adverse reactions, among which 93 were allergic reactions, 63 were non hemolytic febrile reactions, and 10 were others, with a total incidence rate of transfusion adverse reactions at 0.24%. The average storage time of leukodepleted red blood cells with and without transfusion adverse reactions was (20.25±6.31) and (19.88±5.50) days, respectively. With a storage time of 7 days as the threshold, the incidence of transfusion adverse reactions was the lowest for a storage time of 15~21 days. The incidence of transfusion adverse reactions of leukodepleted red blood cells in two groups (with storage days ≤21 days and >21 days) was not statistically significant(P>0.05). 【Conclusion】 Allergic reactions were the main type of transfusion adverse reaction caused by leukodepleted red blood cells, and the incidence of transfusion adverse reactions decreased and then increased with the prolongation of the storage time of leukodepleted red blood cells. There was no significant difference in the incidence of transfusion adverse reactions with leukodepleted red blood cells stored for ≤ 21 days and >21 days.

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