scholarly journals Cybernic treatment with wearable cyborg Hybrid Assistive Limb (HAL) improves ambulatory function in patients with slowly progressive rare neuromuscular diseases: a multicentre, randomised, controlled crossover trial for efficacy and safety (NCY-3001)

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Takashi Nakajima ◽  
Yoshiyuki Sankai ◽  
Shinjiro Takata ◽  
Yoko Kobayashi ◽  
Yoshihito Ando ◽  
...  
Keyword(s):  
Crossover Trial ◽  
Muscular Atrophy ◽  
Neuromuscular Diseases ◽  
Walk Test ◽  
Efficacy And Safety ◽  
Muscle Testing ◽  
Ambulatory Ability ◽  
Secondary Endpoints ◽  
Randomised Controlled ◽  
Hybrid Assistive Limb

Abstract Background Rare neuromuscular diseases such as spinal muscular atrophy, spinal bulbar muscular atrophy, muscular dystrophy, Charcot-Marie-Tooth disease, distal myopathy, sporadic inclusion body myositis, congenital myopathy, and amyotrophic lateral sclerosis lead to incurable amyotrophy and consequent loss of ambulation. Thus far, no therapeutic approaches have been successful in recovering the ambulatory ability. Thus, the aim of this trial was to evaluate the efficacy and safety of cybernic treatment with a wearable cyborg Hybrid Assistive Limb (HAL, Lower Limb Type) in improving the ambulatory function in those patients. Results We conducted an open-label, randomised, controlled crossover trial to test HAL at nine hospitals between March 6, 2013 and August 8, 2014. Eligible patients were older than 18 years and had a diagnosis of neuromuscular disease as specified above. They were unable to walk for 10 m independently and had neither respiratory failure nor rapid deterioration in gait. The primary endpoint was the distance passed during a two-minute walk test (2MWT). The secondary endpoints were walking speed, cadence, and step length during the 10-m walk test (10MWT), muscle strength by manual muscle testing (MMT), and a series of functional measures. Adverse events and failures/problems/errors with HAL were also evaluated. Thirty patients were randomly assigned to groups A or B, with each group of 15 receiving both treatments in a crossover design. The efficacy of a 40-min walking program performed nine times was compared between HAL plus a hoist and a hoist only. The final analysis included 13 and 11 patients in groups A and B, respectively. Cybernic treatment with HAL resulted in a 10.066% significantly improved distance in 2MWT (95% confidence interval, 0.667–19.464; p = 0.0369) compared with the hoist only treatment. Among the secondary endpoints, the total scores of MMT and cadence at 10MWT were the only ones that showed significant improvement. The only adverse effects were slight to mild myalgia, back pain, and contact skin troubles, which were easily remedied. Conclusions HAL is a new treatment device for walking exercise, proven to be more effective than the conventional method in patients with incurable neuromuscular diseases. Trial registration: JMACTR, JMA-IIA00156

Efficacy of ethanol ablation for treatment of benign cystic thyroid nodules: the first hospital-based study in Vietnam

MedPharmRes ◽  
2020 ◽  
Vol 4 (1) ◽  
pp. 18-22
Author(s):  
Van Bang Nguyen ◽  
Van Vy Hau Nguyen ◽  
Binh Thang Tran ◽  
Chi Van Le
Keyword(s):  
Thyroid Nodules ◽  
Multiple Logistic Regression Analysis ◽  
Volume Reduction ◽  
Efficacy And Safety ◽  
Inclusion Criteria ◽  
Ethanol Ablation ◽  
Institutional Review ◽  
Thyroid Cyst ◽  
Secondary Endpoints ◽  
Written Informed Consent

Background: In Vietnam, surgery or aspiration is preferred to treat thyroid cysts however each of them still have limitations. Purposes of this study were to evaluate the efficacy and safety of ethanol ablation in treating thyroid cysts and determine factors that predict the outcome of treatment. Methods: This prospective study was approved by the Ethics Committee of the Institutional Review Board of Family hospital and written informed consent for procedures was obtained. From May 2018 to March 2019, 23 patients who underwent treatment for thyroid cysts by EA were enrolled in this study and were followed up for 1 month at Family hospital. The primary endpoint was efficient after one month as the volume reduction ratio was ≥ 50%. Secondary endpoints were improvements in symptoms, cosmetic scores, and safety. Multiple logistic regression analysis was used. Results: In the finding, from May 2018 to March 2019, only 17 patients who matched inclusion criteria were included in the analyst, including 7 purely thyroid cysts, and 10 predominantly cystic nodules. Mean volume decreased significantly from 5.21 ± 3.37 ml to 2.35 ± 2.52 ml in corresponding to 52.87% of volume reduction with p < 0.05. Ethanol ablation (EA) success rate was 52.90% after 1 month. Symptoms and cosmetic scores were improved significantly. The thyroid function was constant. No adverse events occurred. Purely thyroid cyst was a predictive factor contributing to the success of EA. Conclusion: EA seems likely to be a safe and an efficient therapy for patients who had purely or predominantly cystic thyroid nodules.

scholarly journals Randomised, controlled trial of erenumab for the prevention of episodic migraine in patients from Asia, the Middle East, and Latin America: The EMPOwER study

Cephalalgia ◽  
2021 ◽  
pp. 033310242110241
Author(s):  
Shuu-Jiun Wang ◽  
Artemio A Roxas ◽  
Bibiana Saravia ◽  
Byung-Kun Kim ◽  
Debashish Chowdhury ◽  
...  
Keyword(s):  
Latin America ◽  
Middle East ◽  
Primary Endpoint ◽  
Episodic Migraine ◽  
Acute Migraine ◽  
Efficacy And Safety ◽  
Medication Treatment ◽  
Significant Difference ◽  
Specific Medication ◽  
Secondary Endpoints

Objective EMPOwER, a double-blind, randomised, phase 3 study, evaluated the efficacy and safety of erenumab in adults with episodic migraine from Asia, the Middle East, and Latin America. Methods Randomised patients (N = 900) received monthly subcutaneous injections of placebo, erenumab 70 mg, or 140 mg (3:3:2) for 3 months. Primary endpoint was change from baseline in monthly migraine days at Month 3. Other endpoints included achievement of ≥50%, ≥75%, and 100% reduction in monthly migraine days, change in monthly acute migraine-specific medication treatment days, patient-reported outcomes, and safety assessment. Results At baseline, mean (standard deviation) age was 37.5 (9.9) years, 81.9% were women, and monthly migraine days was 8.2 (2.8). At Month 3, change from baseline in monthly migraine days (primary endpoint) was −3.1, −4.2, and −4.8 days for placebo, erenumab 70 mg, and erenumab 140 mg, respectively, with a statistically significant difference for erenumab versus placebo (P = 0.002 [70 mg], P < 0.001 [140 mg]). Both erenumab doses were also significantly superior to placebo on all secondary endpoints, including the proportion of patients achieving ≥50% reduction from baseline in monthly migraine days, change from baseline in monthly acute migraine-specific medication treatment days and change from baseline in the Headache Impact Test-6™ scores. The safety profile of erenumab was comparable with placebo; no new safety signals were observed. Conclusions This study of erenumab in patients with episodic migraine from Asia, the Middle East, and Latin America met all primary and secondary endpoints. A consistent numerical benefit was observed with erenumab 140 mg versus erenumab 70 mg across all efficacy endpoints. These findings extend evidence of erenumab’s efficacy and safety to patients under-represented in previous trials. ClinicalTrials.gov identifier: NCT03333109

scholarly journals Comparative efficacy and safety of different regimens of ranibizumab for neovascular age-related macular degeneration: a network meta-analysis of randomised controlled trials

BMJ Open ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. e040906
Author(s):  
Xinyu Zhao ◽  
Lihui Meng ◽  
Youxin Chen
Keyword(s):  
Adverse Events ◽  
Macular Degeneration ◽  
Randomised Controlled Trials ◽  
Meta Analysis ◽  
Age Related Macular Degeneration ◽  
Controlled Trials ◽  
Cochrane Central Register ◽  
Efficacy And Safety ◽  
Age Related ◽  
Randomised Controlled

ObjectiveTo give a comprehensive efficacy and safety ranking of different therapeutic regimens of ranibizumab for neovascular age-related macular degeneration (nAMD).DesignA systematic review and network meta-analysis.MethodsThe PubMed, Embase, Cochrane Central Register of Controlled Trials, and other clinical trial registries were searched up to 1 October 2019 to identify related randomised controlled trials (RCT) of different regimens of ranibizumab for nAMD. The primary efficacy outcome was the changes of best-corrected visual acuity (BCVA) at 1 year, the primary safety outcome was the incidence of severe ocular adverse events. Secondary outcomes such as changes of central retinal thickness (CRT) were evaluated. We estimated the standardised mean difference (SMD), ORs, 95% CIs, the surface under the cumulative ranking curves and the mean ranks for each outcome using network meta-analyses with random effects by Stata 14.0.ResultsWe identified 26 RCTs involving 10 821 patients with nAMD randomly assigned to 21 different therapeutic regimens of ranibizumab or sham treatment. Ranibizumab 0.5 mg (treat and extend, T&E) is most effective in terms of changes of BCVA (letters, SMD=21.41, 95% CI 19.86 to 22.95) and three or more lines of BCVA improvement (OR=2.83, 95% CI 1.27 to 4.38). However, it could not significantly reduce retreatment times compared with monthly injection (SMD=−0.94, 95% CI −2.26 to 0.39). Ranibizumab 0.5 mg (3+pro re nata)+non-steroidal anti-inflammatory drugs (NSAIDs) is most effective in reducing CRT and port delivery system of ranibizumab (100 mg/mL) could reduce the number of retreatment most significantly. All regimes have no more risk of severe ocular complications (including vitreous haemorrhage, rhegmatogenous retinal detachment, endophthalmitis, retinal tear and retinal pigment epithelium tear) or cardiocerebral vascular complications.ConclusionsRanibizumab 0.5 mg (T&E) is most effective in improving the visual outcome. The administration of topical NSAIDs could achieve additional efficacy in CRT reduction and visual improvement. Both interventions had acceptable risks of adverse events.

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