A preliminary analysis from the ICGG Gaucher registry: Short-term clinical outcomes in Gaucher patients affected by the shortage of imiglucerase

2011 ◽  
Vol 102 (2) ◽  
pp. S45
Author(s):  
Neal Weinreb ◽  
Barry Rosenbloom ◽  
Hans Andersson ◽  
Alexander Cole ◽  
Radhika Tripuraneni ◽  
...  
2021 ◽  
Vol 49 (4) ◽  
pp. 982-993
Author(s):  
Anne-Sofie Agergaard ◽  
Rene B. Svensson ◽  
Nikolaj M. Malmgaard-Clausen ◽  
Christian Couppé ◽  
Mikkel H. Hjortshoej ◽  
...  

Background: Loading interventions have become a predominant treatment strategy for tendinopathy, and positive clinical outcomes and tendon tissue responses may depend on the exercise dose and load magnitude. Purpose/Hypothesis: The purpose was to investigate if the load magnitude influenced the effect of a 12-week loading intervention for patellar tendinopathy in the short term (12 weeks) and long term (52 weeks). We hypothesized that a greater load magnitude of 90% of 1 repetition maximum (RM) would yield a more positive clinical outcome, tendon structure, and tendon function compared with a lower load magnitude of 55% of 1 RM when the total exercise volume was kept equal in both groups. Study Design: Randomized clinical trial; Level of evidence, 1. Methods: A total of 44 adult participants with chronic patellar tendinopathy were included and randomized to undergo moderate slow resistance (MSR group; 55% of 1 RM) or heavy slow resistance (HSR group; 90% of 1 RM). Function and symptoms (Victorian Institute of Sport Assessment–Patella questionnaire [VISA-P]), tendon pain during activity (numeric rating scale [NRS]), and ultrasound findings (tendon vascularization and swelling) were assessed before the intervention, at 6 and 12 weeks during the intervention, and at 52 weeks from baseline. Tendon function (functional tests) and tendon structure (ultrasound and magnetic resonance imaging) were investigated before and after the intervention period. Results: The HSR and MSR interventions both yielded significant clinical improvements in the VISA-P score (mean ± SEM) (HSR: 0 weeks, 58.8 ± 4.3; 12 weeks, 70.5 ± 4.4; 52 weeks, 79.7 ± 4.6) (MSR: 0 weeks, 59.9 ± 2.5; 12 weeks, 72.5 ± 2.9; 52 weeks, 82.6 ± 2.5), NRS score for running, NRS score for squats, NRS score for preferred sport, single-leg decline squat, and patient satisfaction after 12 weeks, and these were maintained after 52 weeks. HSR loading was not superior to MSR loading for any of the measured clinical outcomes. Similarly, there were no differences in functional (strength and jumping ability) or structural (tendon thickness, power Doppler area, and cross-sectional area) improvements between the groups undergoing HSR and MSR loading. Conclusion: There was no superior effect of exercising with a high load magnitude (HSR) compared with a moderate load magnitude (MSR) for the clinical outcome, tendon structure, or tendon function in the treatment of patellar tendinopathy in the short term. Both HSR and MSR showed equally good, continued improvements in outcomes in the long term but did not reach normal values for healthy tendons. Registration: NCT03096067 (ClinicalTrials.gov identifier)


2021 ◽  
Vol 30 ◽  
pp. S112
Author(s):  
O. Al-mukhtar ◽  
S. Vogrin ◽  
S. Noaman ◽  
E. Lampugnani ◽  
D. Dinh ◽  
...  

2021 ◽  
Vol 77 (18) ◽  
pp. 1202
Author(s):  
Tobias Friedrich Ruf ◽  
Rebecca Hahn ◽  
Andres Beiras-Fernandez ◽  
Felix Kreidel ◽  
Michaela Hell ◽  
...  

2021 ◽  
pp. 000486742098347
Author(s):  
John Farhall ◽  
Lisa Brophy ◽  
John Reece ◽  
Holly Tibble ◽  
Long Khanh-Dao Le ◽  
...  

Objective: In Victoria, Prevention and Recovery Care Services have been established to provide a partial alternative to inpatient admissions through short-term residential mental health care in the community. This study set out to determine whether Prevention and Recovery Care Services are achieving their objectives in relation to reducing service use and costs, fostering least restrictive care and leading to positive clinical outcomes. Methods: We matched 621 consumers whose index admission in 2014 was to a Prevention and Recovery Care (‘PARCS consumers’) with 621 similar consumers whose index admission in the same year was to an acute inpatient unit and who had no Prevention and Recovery Care stays for the study period (‘inpatient-only consumers’). We used routinely collected data to compare them on a range of outcomes. Results: Prevention and Recovery Care Services consumers made less subsequent use of acute inpatient services and, on balance, incurred costs that were similar to or lower than inpatient-only consumers. They were also less likely to spend time on an involuntary treatment order following their index admission. Prevention and Recovery Care Services consumers also experienced positive clinical outcomes over the course of their index admission, but the magnitude of this improvement was not as great as for inpatient-only consumers. This type of clinical improvement is important for Prevention and Recovery Care Services, but they may place greater emphasis on personal recovery as an outcome. Conclusion: Prevention and Recovery Care Services can provide an alternative, less restrictive care option for eligible consumers who might otherwise be admitted to an acute inpatient unit and do so at no greater cost.


Pancreatology ◽  
2013 ◽  
Vol 13 (3) ◽  
pp. S68
Author(s):  
Wei Huang ◽  
Jia Guo ◽  
Xiao Yang ◽  
Tao Jin ◽  
Kiran Altaf ◽  
...  

2021 ◽  
pp. 175857322110671
Author(s):  
Edoardo Franceschetti ◽  
Edoardo Giovannetti de Sanctis ◽  
Pietro Gregori ◽  
Michele Paciotti ◽  
Alessio Palumbo ◽  
...  

Background Two surgical techniques were compared : Standard BIO-RSA, performed with a glenoid eccentric reaming along with a cylindric bone graft augmentation vs. the Angled BIO-RSA, performed with a glenoid concentric reaming and a defect correction with an angled bone graft. Methods Patients undergoing RSA from January 2016 to April 2019, with one of the two techniques being performed, were retrospectively reviewed. Glenoids were classified according to Favard. Clinical (Constant-Murley, VAS and ROM) and radiographic (superior tilt correction) data were collected pre-operatively and at 12 months post-operatively. Results 141 shoulders were included. Angled BIO-RSA group showed statistically significant better outcomes in terms of forward flexion (149.9° Vs 139.3°) and abduction (136.4° Vs 126.7°). The use of an Angled BIO-RSA showed a statistically significant better superior tilt correction (1.252° Vs 4.09°). Angled BIO-RSA, leads to a better inclination correction and a mean postoperative tilt value inferior to 5° in E1 and E3 differently from standard BIO-RSA. Discussion Both techniques were able to correct glenoid superior inclination with excellent postoperative short-term results. However, angled BIO-RSA technique appears to be more effective in ensuring a correct inclination of the prosthetic glenoid component with better clinical outcomes.


Author(s):  
William D. Gerull ◽  
Zhizhou Yang ◽  
Daniel Kreisel ◽  
Ruben Nava ◽  
Bryan F. Meyers ◽  
...  
Keyword(s):  

2021 ◽  
Author(s):  
Lei Yang ◽  
Ke Gao ◽  
Xin-Ye Yao ◽  
Yong-lan Tang ◽  
Wan-Ying Yang ◽  
...  

Abstract Background: Liver cirrhosis is a confirmed risk factor for worse clinical outcomes of stroke, however the contribution of liver fibrosis to cardioembolic stroke (CES) and its short-term outcomes are poorly understood. This study aimed to investigate whether liver fibrosis is associated with more severe stroke, worse short-term clinical outcomes of acute CES, due to nonvalvular atrial fibrillation (NVAF), as well as the impact of sex on the association. Methods: Using data of 522 patients with NVAF admitted within 48 hours after acute symptom of CES onset. We calculated Fibrosis-4 score (FIB-4) and defined liver fibrosis as: likely advanced fibrosis (FIB-4>3.25), indeterminate (FIB-4, 1.45-3.25), unlikely advanced fibrosis (FIB-4<1.45). We invested the impact of liver fibrosis degree on stroke severity on admission, major disability at discharge and all cause death at 90 days stratified by sex. Results: Among 522 acute CES patients with NVAF, the mean FIB-4 on admission reflected intermediate fibrosis, whereas liver enzymes were largely normal. After adjusting for possible confounders, multivariate analyses revealed that likely advanced liver fibrosis was associated with severe stroke (OR=2.21, 95% CI: 1.04-3.54), major disability at discharge (OR=4.59, 95% CI: 1.88-11.18), and 90-days mortality (HR=1.25, 95% CI: 1.10-1.56). Further grouped by sex, these associations were stronger in males but not significant in females.Conclusions: In patients with largely normal liver enzyme, likely advanced liver fibrosis is associated with severe stroke, major disability and all cause death after acute CES due to NVAF; the association unfolded more obvious in males, but not for females.


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