scholarly journals Corrigendum: Off-Label Use of Ataluren in Four Non-ambulatory Patients With Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

2019 ◽  
Vol 7 ◽  
Author(s):  
Daniel Ebrahimi-Fakhari ◽  
Ulrich Dillmann ◽  
Marina Flotats-Bastardas ◽  
Martin Poryo ◽  
Hashim Abdul-Khaliq ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Pulmonary Function ◽  
Off Label Use ◽  
Off Label ◽  
Ambulatory Patients ◽  
Label Use

scholarly journals Perioperative Evaluation of Respiratory Muscle Strength after Scoliosis Correction in Patients with Duchenne Muscular Dystrophy

2017 ◽  
Vol 11 (5) ◽  
pp. 787-792 ◽  
Author(s):  
Wataru Saito ◽  
Kosuke Mizuno ◽  
Gen Inoue ◽  
Takayuki Imura ◽  
Toshiyuki Nakazawa ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Pulmonary Function ◽  
Respiratory Function ◽  
Respiratory Muscle ◽  
Vital Capacity ◽  
Respiratory Muscle Strength ◽  
Scoliosis Correction ◽  
The Mean

<sec><title>Study Design</title><p>Retrospective cohort study.</p></sec><sec><title>Purpose</title><p>To investigate the effect of spinal correction on respiratory muscle strength in patients with Duchenne muscular dystrophy (DMD).</p></sec><sec><title>Overview of Literature</title><p>Several studies have reported that scoliosis correction in patients with DMD does not improve pulmonary function. In these studies, pulmonary function was evaluated using the traditional spirometric values of percent vital capacity (%VC) and percent forced vital capacity (%FVC). However, traditional spirometry may not be suitable for patients with DMD because the results can be influenced by patient fatigue or level of understanding. Therefore, we evaluated respiratory function focusing on respiratory muscle strength using maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP), and sniff nasal inspiratory pressure (SNIP), in addition to %VC and %FVC.</p></sec><sec><title>Methods</title><p>We retrospectively reviewed 16 patients with DMD who underwent spinal correction surgery between 2006 and 2011 at Kitasato University Hospital. All patients were males, and the mean age was 13.5 years. Respiratory muscle strength was evaluated using MIP, MEP, and SNIP. Measurements were obtained preoperatively and at 1 and 6 months postoperatively, and %VC and %FVC were obtained preoperatively and within 6 months postoperatively.</p></sec><sec><title>Results</title><p>The mean preoperative and postoperative %VC values were 54.0% and 51.7%, whereas the mean %FVC values were 53.9% and 53.2%, respectively. The mean MIP, MEP, and SNIP values obtained preoperatively and at 1 and 6 months postoperatively were as follows: MIP, 40.5, 42.7 and 47.2 cm H<sub>2</sub>O; MEP, 26.0, 28.0, and 29.0 cm H<sub>2</sub>O; and SNIP, 33.4, 33.0, and 33.0 cm H<sub>2</sub>O; respectively. The mean MIP and MEP values significantly improved postoperatively. There were no significant differences in SNIP, %VC, or %FVC preand postoperatively.</p></sec><sec><title>Conclusions</title><p>By focusing on respiratory muscle strength, our results suggest that scoliosis correction in patients with DMD might have a favorable effect on respiratory function.</p></sec>

scholarly journals Relationship between muscle strength and motor function in Duchenne muscular dystrophy

2016 ◽  
Vol 74 (7) ◽  
pp. 530-535 ◽  
Author(s):  
Milene F. Nunes ◽  
Michele E. Hukuda ◽  
Francis M. Favero ◽  
Acary B. Oliveira ◽  
Mariana C. Voos ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Motor Function ◽  
Medical Research Council ◽  
Correlation Coefficients ◽  
Strongly Correlated ◽  
Ambulatory Patients ◽  
The Relationship ◽  
Function Measure

ABSTRACT Measuring muscle strength and motor function is part of Duchenne muscular dystrophy (DMD) assessment. However, the relationship between these variables is controversial. Objective To investigate the relationship between muscle strength and motor function and between these variables and age. Method Muscle strength was measured by Medical Research Council (MRC) scale and motor function, by Motor Function Measure (MFM), in 40 non-ambulatory patients. Spearman tests investigated the relationships between muscle strength, motor function and age. Results Total MRC and MFM scores were strongly related to each other (r = 0.94; p < 0.001), but not to age (r = -0.19, r = -0.31, respectively; p > 0.05). Strong and moderate relationships between partial muscle strength and motor function scores were found. Higher correlation coefficients were found between total scores and Dimensions 2 (axial/ proximal control) and 3 (distal control) of MFM. Conclusion Muscle strength and motor function are strongly correlated and seem to decrease proportionally in DMD.

Indikationsfremde Anwendung von Medikamenten und Medizinprodukten in der Notfallmedizin

2019 ◽  
Vol 14 (04) ◽  
pp. 361-371
Author(s):  
Karl Peter Ittner ◽  
Joachim Koppenberg ◽  
Ute Walter
Keyword(s):  
Off Label Use ◽  
Off Label ◽  
Label Use

ZusammenfassungWenn zugelassene Arzneimittel außerhalb der in der entsprechenden Fachinformation dargelegten Beschreibungen angewendet werden, dann spricht man von einer nicht zulassungskonformen Anwendung oder von einem Off-Label-Use. Wie in fast allen medizinischen Fachgebieten gibt es auch im Rettungsdienst sogenannte Off-Label-Use-Pharmakotherapien. Sofern evidenzbasierte Informationen zu einer nicht zulassungskonformen Anwendung vorliegen, und insbesondere im konkreten Notfall keine zulassungskonforme Möglichkeit besteht, dann ist diese gerechtfertigt. Verwendet ein Notarzt aber ein Medizinprodukt außerhalb der Zulassung, dann stellt er ein neues Produkt her und haftet persönlich bei einem Patientenschaden.

Botulinumtoxin in der Urologie

2010 ◽  
Vol 29 (09) ◽  
pp. 551-555
Author(s):  
W. N. Vance ◽  
J. Wissel
Keyword(s):  
Off Label Use ◽  
Botulinumtoxin A ◽  
Off Label ◽  
Label Use

ZusammenfassungAlle Indikationen zur Anwendung von Botulinumtoxin A (BoNT A) in der Urologie befinden sich im Status des sogenannten off label use, entsprechend sind Kernfragen wie z. B. die Kostenübernahme nicht geklärt. Erst 20 Jahre nach der ersten Anwendung in der Urologie werden Zulassungsstudien durchgeführt. Andererseits sind insbesondere die Behandlungsmöglichkeiten im Bereich der neurogenen Harnblase so etabliert, dass sie bereits in die urologischen Leitlinien Einzug gefunden haben. Hinsichtlich der Dosierung von BoNT A und der optimalen Anwendungstechnik bestehen weder allgemein anerkannte Handlungsanweisungen noch offizielle Empfehlungen. Dies gilt auch im Bereich der Anästhesie zur Injektion von BoNT A im urologischen Gebiet. Nicht wenige Patienten schrecken davor zurück, sich regelmäßig, zum Teil jährlich, einer Allgemeinanästhesie zu unterziehen. Ein besonderer Hoffnungsschimmer stellt für multimorbide Patienten mit hohem OP-Risiko die sich entwickelnde Behandlung der Prostatahyperplasie mittels BoNT A dar, insbesondere da in allen Studien die Lokalanästhesie angewandt wurde und nur wenige Nebenwirkungen auftraten.

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