Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the ＂ temporary import＂ pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.

Objective:To evaluate the efficacy and safety of nebulized inhalation of recombinant human interferon (IFN) α1b injection in the treatment of respiratory syncytial virus (RSV) associated lower respiratory tract infections (pneumonia and bronchiolitis) in children.Methods:A randomized, double-blind, parallel, placebo-controlled add-on design was used.Children with pneumonia or bronchiolitis aged 2 months to 5 years who tested positive for RSV antigen within 72 hours of onset from 30 clinical trial sites including Beijing Children′s Hospital, Capital Medical University between February 2021 and December 2022 were included in this study and randomly divided into 2 groups at a ratio of 1∶1 based on a stratified-block method.Both groups received basic treatments such as cough control, asthma relieving, expectorant treatment, fever reduction, oxygen therapy, etc.The experimental group received additional nebulized inhalation of IFN α1b injection at a dose of 2.0 μg/(kg·time), twice a day.The control group received nebulized inhalation of placebo twice a day.Clinical efficacy was evaluated based on indicators such as the duration of clinical symptoms and signs, and the Kaplan-Meier method was used to calculate the median and 95% CI of the duration of clinical symptoms and signs.The Log-rank test was used to compared data between groups.Safety was assessed through the incidence of adverse reactions and laboratory tests, and the Chi-square test was used to analyze the difference between groups. Results:There were 123 children in the experimental group and 122 children in the control group.The median durations of all the 5 clinical symptoms and signs [including shortness of breath, wheezing, dyspnea (visible retractions), decreased transcutaneous oxygen saturation, and abnormal mental state] in the experimental group after treatment were slightly shortened than those in the control group [2.7 d(95% CI: 1.9-3.0 d)] vs.[2.9 d(95% CI: 2.6-3.6 d), P=0.027].The improvement in dyspnea (retractions) was especially pronounced in the experimental group, with a relief rate of 50.0% (0, 100%) on the first day of administration[compared with 0 (0, 50.0%) in the control group ( Z=2.002, P=0.025)].The median duration of dyspnea in the experimental group was nearly 1 day shorter than that in the control group [1.0 d(95% CI: 0.7-1.7 d) vs.1.8 d(95% CI: 1.0-2.5 d), P=0.046].There were no significant difference in hospital stay [6.0(5.0, 8.0) d vs.6.5(5.0, 8.0) d, Z=0.675, P=0.500], oxygen therapy duration [32.0(14.0, 96.3) h vs.39.0 (24.0, 83.2) h, Z=0.094, P=0.925], the recovery rate from clinical symptoms during treatment [(105/106, 99.1%) vs.(96/101, 95.0%)], and recurrence rate [(0/106, 0) vs.(2/101, 2.0%)] between the 2 groups (all P>0.05).However, the above-mentioned four indicators in the experimental group showed a trend of clinical benefits.The quantitative virus detection results showed that the RSV viral load in both groups decreased after treatment compared to before treatment.After 2 days of treatment, the decline rate of RSV viral load from the baseline was 0.90 lg copies/(mL·d) in the experimental group and 0.25 lg copies/(mL·d)in the control group, with a statistically significant difference ( P<0.05).Furthermore, there was no statistically significant difference in the incidence of adverse reactions between the 2 groups ( P>0.05).Importantly, no drug-related serious adverse reactions occurred in both groups. Conclusions:The nebulized inhalation therapy of IFN α1b demonstrates efficacy and safety in treating pediatric RSV associated lower respiratory tract infections.It particularly offers outstanding clinical therapeutic value for severe children.

Deflazacort,as a glucocorticoid medication,is conductive to improving motor function and muscle strength,delaying the loss of ambulation,enhancing pulmonary function,reducing the risk of scoliosis,slowing the progression of cardiomyopathy,and increasing survival rates in patients with Duchenne muscular dystrophy(DMD).In February 2017,the U.S.Food and Drug Administration(FDA)approved deflazacort for the treatment of DMD.In May 2024,deflazacort entered Peking Union Medical College Hospital for desig-nated use through the ＂ temporary import＂ pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action,pharmacokinetics,clinical efficacy,and adverse effects,aiming to offer a reference for its rational and safe application in clinical practice.

Objective:To evaluate the efficacy and safety of nebulized inhalation of recombinant human interferon (IFN) α1b injection in the treatment of respiratory syncytial virus (RSV) associated lower respiratory tract infections (pneumonia and bronchiolitis) in children.Methods:A randomized, double-blind, parallel, placebo-controlled add-on design was used.Children with pneumonia or bronchiolitis aged 2 months to 5 years who tested positive for RSV antigen within 72 hours of onset from 30 clinical trial sites including Beijing Children′s Hospital, Capital Medical University between February 2021 and December 2022 were included in this study and randomly divided into 2 groups at a ratio of 1∶1 based on a stratified-block method.Both groups received basic treatments such as cough control, asthma relieving, expectorant treatment, fever reduction, oxygen therapy, etc.The experimental group received additional nebulized inhalation of IFN α1b injection at a dose of 2.0 μg/(kg·time), twice a day.The control group received nebulized inhalation of placebo twice a day.Clinical efficacy was evaluated based on indicators such as the duration of clinical symptoms and signs, and the Kaplan-Meier method was used to calculate the median and 95% CI of the duration of clinical symptoms and signs.The Log-rank test was used to compared data between groups.Safety was assessed through the incidence of adverse reactions and laboratory tests, and the Chi-square test was used to analyze the difference between groups. Results:There were 123 children in the experimental group and 122 children in the control group.The median durations of all the 5 clinical symptoms and signs [including shortness of breath, wheezing, dyspnea (visible retractions), decreased transcutaneous oxygen saturation, and abnormal mental state] in the experimental group after treatment were slightly shortened than those in the control group [2.7 d(95% CI: 1.9-3.0 d)] vs.[2.9 d(95% CI: 2.6-3.6 d), P=0.027].The improvement in dyspnea (retractions) was especially pronounced in the experimental group, with a relief rate of 50.0% (0, 100%) on the first day of administration[compared with 0 (0, 50.0%) in the control group ( Z=2.002, P=0.025)].The median duration of dyspnea in the experimental group was nearly 1 day shorter than that in the control group [1.0 d(95% CI: 0.7-1.7 d) vs.1.8 d(95% CI: 1.0-2.5 d), P=0.046].There were no significant difference in hospital stay [6.0(5.0, 8.0) d vs.6.5(5.0, 8.0) d, Z=0.675, P=0.500], oxygen therapy duration [32.0(14.0, 96.3) h vs.39.0 (24.0, 83.2) h, Z=0.094, P=0.925], the recovery rate from clinical symptoms during treatment [(105/106, 99.1%) vs.(96/101, 95.0%)], and recurrence rate [(0/106, 0) vs.(2/101, 2.0%)] between the 2 groups (all P>0.05).However, the above-mentioned four indicators in the experimental group showed a trend of clinical benefits.The quantitative virus detection results showed that the RSV viral load in both groups decreased after treatment compared to before treatment.After 2 days of treatment, the decline rate of RSV viral load from the baseline was 0.90 lg copies/(mL·d) in the experimental group and 0.25 lg copies/(mL·d)in the control group, with a statistically significant difference ( P<0.05).Furthermore, there was no statistically significant difference in the incidence of adverse reactions between the 2 groups ( P>0.05).Importantly, no drug-related serious adverse reactions occurred in both groups. Conclusions:The nebulized inhalation therapy of IFN α1b demonstrates efficacy and safety in treating pediatric RSV associated lower respiratory tract infections.It particularly offers outstanding clinical therapeutic value for severe children.

The gut microbiota plays an important role in regulating the pharmacokinetics and pharmacodynamics of many drugs. FLZ, a novel squamosamide derivative, has been shown to have neuroprotective effects on experimental Parkinson's disease (PD) models. FLZ is under phase Ⅰ clinical trial now, while the underlying mechanisms contributing to the absorption of FLZ are still not fully elucidated. Due to the main metabolite of FLZ was abundant in feces but rare in urine and bile of mice, we focused on the gut microbiota to address how FLZ was metabolized and absorbed.

Parkinson's disease (PD) is the second most common neurodegenerative disease, but none of the current treatments for PD can halt the progress of the disease due to the limited understanding of the pathogenesis. In PD development, the communication between the brain and the gastrointestinal system influenced by gut microbiota is known as microbiota-gut-brain axis. However, the explicit mechanisms of microbiota dysbiosis in PD development have not been well elucidated yet. FLZ, a novel squamosamide derivative, has been proved to be effective in many PD models and is undergoing the phase I clinical trial to treat PD in China. Moreover, our previous pharmacokinetic study revealed that gut microbiota could regulate the absorption of FLZ

Objective:To explore the effect of the discharge preparation plan based on community linkage in the out-of-hospital continuous nursing of chronic obstructive pulmonary disease (COPD) patients.Methods:From March 2019 to March 2020, convenience sampling was used to select 206 COPD patients hospitalized in the Respiratory Department of Henan Provincial People's Hospital. The patients were randomly divided into the observation group and the control group, with 103 cases in each group. The control group received routine management of COPD, and the observation group implemented a community-linked discharge preparation plan on the basis of the control group, and carried out a six-month intervention. The COPD Assessment Test (CAT) score, Patient Assessment of Chronic Illness Care (PACIC) score, body mass index, degree of airflow obstruction, dyspnea and exercise capacity (BODE) index, and the patient readmission rate during the intervention period were compared between the two groups before and after the intervention.Results:After the intervention, the item scores and total scores of the CAT of the observation group were lower than those of the control group. Except for cough and sputum, the differences in the other items and total scores were statistically significant ( P<0.05) . The item scores and total scores of the CAT of the observation group after intervention were lower than those before intervention, and the differences were statistically significant ( P<0.05) . The dimension scores and total score of the PACIC of the observation group after the intervention were higher than those before the intervention, and were higher than those of the control group after the intervention, and the difference was statistically significant ( P<0.05) . The BODE index score of the observation group after intervention was lower than that of the control group, and the difference between the two groups was statistically significant ( P<0.05) . The readmission rate in the observation group within six months was 13.59% (14/103) , which was lower than 31.07% (32/103) in the control group, and the difference between the two groups was statistically significant (χ 2=9.068, P=0.003) . Conclusions:The discharge preparation plan based on community linkage can improve the quality of life and lung function of COPD patients, increase the patient's recognition of chronic disease management, reduce the rate of hospital readmission, and achieve the overall quality improvement in continuous nursing of patients.

Objective:To explore the clinical features of dropped head syndrome(DHS) related to mitogen-activated extracellular signal regulated kinase inhibitors.Methods:Case reports of dropped head syndrome caused by MEK inhibitors were collected by searching PubMed and Embase databases as of December 20, 2020. The following information of patients including gender, age, primary diseases, use of MEK inhibitors, occurrence time and clinical symptoms of DHS, and treatments and outcomes were extracted and analyzed descriptively.Results:A total of 7 patients were entered; 4 from the United States, 2 from France, and 1 from Germany. There were 4 males and 3 females, aged from 56 to 76 years. The primary diseases were metastatic melanoma in 6 patients and Erdheim-Chester disease in 1 patient. Of them, 3 patients were treated with selumetinib, 2 were with cobimetinib, and 1 was with binimetinib and trametinib respectively. Time from the first application of MEK inhibitors to the onset of DHS was from 0.5 to 20 months with a median time of 1 (1, 2) month. Major symptoms of DHS were neck pain, neck extensor weakness, and limited head lifting, which might be accompanied by neck stiffness in some patients. The pain on the neck could spread to the shoulders and occipital region, and a few patients might only have interscapular pain. At the time of diagnosis of DHS, the serum concentration of creatine kinase (CK) was elevated (150~1 011 U/L). After the diagnosis of DHS, 5 patients stopped taking MEK inhibitors and DHS symptoms were relieved or disappeared; 2 patients were treated with glucocorticoids for 1 to 4 weeks, but DHS symptoms were not relieved, then they stopped taking MEK inhibitors and DHS symptoms were improved. In total, the symptoms of DHS in the 7 patients were relieved after 14-30 days of MEK inhibitors withdrawal, with serum CK returning to within normal range. Three patients rechallenged MEK inhibitors with reduced doses; 1 patient had no recurrence of DHS and the remaining 2 patients had mild recurrences of DHS, which could resolve spontaneously or stay a stable condition.Conclusions:DHS related to MEK inhibitors usually occurs within 1 month of medication, accompanied by an increase in CK level. The symptoms can be relieved or disappear after stopping medication in time and CK level will return to normal.

Objective:To explore the clinical features of dropped head syndrome(DHS) related to mitogen-activated extracellular signal regulated kinase inhibitors.Methods:Case reports of dropped head syndrome caused by MEK inhibitors were collected by searching PubMed and Embase databases as of December 20, 2020. The following information of patients including gender, age, primary diseases, use of MEK inhibitors, occurrence time and clinical symptoms of DHS, and treatments and outcomes were extracted and analyzed descriptively.Results:A total of 7 patients were entered; 4 from the United States, 2 from France, and 1 from Germany. There were 4 males and 3 females, aged from 56 to 76 years. The primary diseases were metastatic melanoma in 6 patients and Erdheim-Chester disease in 1 patient. Of them, 3 patients were treated with selumetinib, 2 were with cobimetinib, and 1 was with binimetinib and trametinib respectively. Time from the first application of MEK inhibitors to the onset of DHS was from 0.5 to 20 months with a median time of 1 (1, 2) month. Major symptoms of DHS were neck pain, neck extensor weakness, and limited head lifting, which might be accompanied by neck stiffness in some patients. The pain on the neck could spread to the shoulders and occipital region, and a few patients might only have interscapular pain. At the time of diagnosis of DHS, the serum concentration of creatine kinase (CK) was elevated (150~1 011 U/L). After the diagnosis of DHS, 5 patients stopped taking MEK inhibitors and DHS symptoms were relieved or disappeared; 2 patients were treated with glucocorticoids for 1 to 4 weeks, but DHS symptoms were not relieved, then they stopped taking MEK inhibitors and DHS symptoms were improved. In total, the symptoms of DHS in the 7 patients were relieved after 14-30 days of MEK inhibitors withdrawal, with serum CK returning to within normal range. Three patients rechallenged MEK inhibitors with reduced doses; 1 patient had no recurrence of DHS and the remaining 2 patients had mild recurrences of DHS, which could resolve spontaneously or stay a stable condition.Conclusions:DHS related to MEK inhibitors usually occurs within 1 month of medication, accompanied by an increase in CK level. The symptoms can be relieved or disappear after stopping medication in time and CK level will return to normal.