Morquio A: Endurance. Endurance testing assesses functional capacity Endurance tests measure the efficiency of performing a task and provide assessments.

Slides:

Advertisements

Similar presentations

Total Hip Replacement and Musculoskeletal disorder research Tosan Okoro NWORTH Seminar May 2012.

Advertisements

Other physiological tests. Factors That Contribute to Physical Performance.

Morquio A: Anesthetic considerations. Morquio A patients are at high risk of anesthesia-related morbidity and mortality due to: –Cervical instability.

Atrial Fibrillation in Patients with Cryptogenic Stroke Gladstone DJ et al. N Engl J Med 2014; 370: Presented by Kris Huston | July 21, 2014.

Majid Mirsadraee Associate professor Islamic Azad University.

Clinical Management Nutr 564: Management Summer 2005.

Overview of the “International Guidelines for the Management and Treatment of Morquio A Syndrome” REFERENCES: None required.

Research Methodology for Rare Diseases Rare Disease Group 14:00 3 rd October 2012 Richard Lilford.

The Health Roundtable Hip Hop A group exercise class for community dwelling adults following a fractured neck of femur (NOF). Tracey Drabsch Orange Innovation.

Summit on Drug Discovery Tuberous Sclerosis Alliance July 7, 2011 Biomarkers in TSC and LAM.

Functional capacity. Total Score: 0-12 It should be calculated by adding the Score in each one of the three parts of the test (Balance, Gait and Chair.

. Exercise testing in survivors of intensive care— is there a role for cardiopulmonary exercise training? Benington S, McWilliams D, Eddleston J, Atkinson.

Medical Management of Claudication: Just Walk it Off!!

ATS Statement: Guidelines for the Six-Minute Walk Test Am J Respir Crit Care Med Vol 166. pp 111–117, 2002.

Development of disability in chronic obstructive pulmonary disease : beyond lung function MarkDEisner, CarlosIribarren, PaulDBlanc, EdwardHYelin, LynnAckerson,

Stepwise approach to assessing cardiac patient risk for noncardiac surgery. (Reproduced, with permission, from Fleisher LA et al. ACC/AHA 2007 Guidelines.

Stepwise approach to assessing cardiac patient risk for noncardiac surgery. (Reproduced, with permission, from Fleisher LA et al. ACC/AHA 2007 Guidelines.

The Minimal Important Difference for St

Algorithms proposed by the Heart Valve Clinic International Database (HAVEC) group for disease staging and management in patients with aortic valve stenosis.

Regulatory perspective

Nat. Rev. Cardiol. doi: /nrcardio

New developments in the preoperative evaluation and perioperative management of coronary artery disease in patients undergoing vascular surgery Stephen.

Genetic sequence analysis of inherited bleeding diseases

Serial imaging while on immunosuppressive therapy (two subjects).

G. Michael Felker et al. JCHF 2014;2:

Percentage of nursing assessments with NEWS documented for patients 21 years and older from March to October Percentage of nursing assessments with.

Kaplan–Meier curves for a, b) progression-free survival (PFS) and c, d) overall survival (OS) in a, c) large-cell neuroendocrine carcinoma patients and.

Survival without tracheostomy

Safety and tolerability of pembrolizumab in patients with relapsed/refractory primary mediastinal large B-cell lymphoma by Pier Luigi Zinzani, Vincent.

Response to Carfilzomib Therapy

Modelled mean (SEM) observed forced vital capacity (FVC) volume change from baseline (mL) over time by dose intensity (>90%, ≤90%), based on actual dose.

ASAS 20/40 response rates, and mean change from baseline in BASDAI through week 156* of treatment. *For patients who discontinued, the end of treatment.

Change from baseline in (A) E-RS total score and (B) E-RS cough and sputum domain score over the study period in ATTAIN and the active-comparator study.

Types of biomarkers/biosignatures to be used for cardiovascular disease. Types of biomarkers/biosignatures to be used for cardiovascular disease. There.

Nat. Rev. Neurol. doi: /nrneurol

Representative diaphragm electromyogram (EMG) tracings at rest (a and b) and during maximum voluntary ventilation (c and d) in a healthy subject (a and.

Baseline Characteristics of the Subjects

Nat. Rev. Gastroenterol. Hepatol. doi: /nrgastro

Serial computed tomography (CT) imaging for monitoring disease progression in patients with idiopathic pulmonary fibrosis. Serial computed tomography (CT)

Electromagnetic navigation image: the icon representing the locatable guide (arrow) can be seen 3 mm away from the centre of a small pulmonary nodule in.

Effects of Pay-for-Performance on the Quality of Primary Care in England Mean Scores for Clinical Quality at the Practice Level for Aspects of Care for.

Catherine M. Pound et al. Hospital Pediatrics 2017;7:

A summary of the pathogenesis, pathophysiology and clinical implications of the pulmonary vascular and cardiac abnormalities in interstitial lung disease.

Simplified Markov diagram of a patient admitted to the ward with no respiratory support. Simplified Markov diagram of a patient admitted to the ward with.

Trial design. Study design schematic of the PF-ILD trial followed by a separate open-label trial (optional): part A (visits 1 through 9 over 52 weeks)

Study institution’s PEWS

Algorithm for the assessment of fitness to fly in chronic obstructive pulmonary disease patients. Algorithm for the assessment of fitness to fly in chronic.

Figure 2. Patient stratification by the reported duration of symptoms and disease severity at the time of assessment Patient stratification by the reported.

The 6MWT results showing improvement from baseline at 1, 6 and 12 months in patients receiving an implant (mean±SE of mean). The 6MWT results showing improvement.

Illustration of a trial design to help evaluate the clinical accuracy of a test of ischaemia. Illustration of a trial design to help evaluate the clinical.

Plasma levels of GLP-1 are increased in patients with critical illness and correlate with disease severity. Plasma levels of GLP-1 are increased in patients.

Distribution of patients in the first- or second-year follow-up according to the number of acute exacerbations of chronic obstructive pulmonary disease.

P aeruginosa-free survival curves analyzed by center for screened and control CF patients using information either from all respiratory secretion cultures.

P aeruginosa-free survival curves analyzed by center and group using information either from all respiratory secretion cultures (A) or from specimens taken.

The telesystem. The telesystem. Patients received regular automated calls from a computer-linked telesystem. Patients could also directly contact the telesystem.

Forest plot from meta-analysis carried out on four studies including high-dose N-acetylcysteine (NAC) treatment a) assessing the relative risk of chronic.

Maximal rates of fat oxidation in men and women across different submaximal exercise intensities in the untrained state and after 12 weeks of SIT. *Indicates.

T2-weighted MR imaging appearance of a healthy 60-year-old woman (A), a 66-year-old woman with idiopathic Parkinson disease (B), and a 16-year-old female.

Progression in the patient’s pulmonary function tests from 2010 to a) Forced vital capacity (FVC); b) total lung capacity (TLC); c) diffusing capacity.

Examples for true-positive, false-positive, and false-negative cord signs in the evaluation of sinus thrombosis. Examples for true-positive, false-positive,

Antitumor activity. Antitumor activity. A, maximum change in target lesion size from baseline assessed according to RECIST 1.0 (n = 26). Six patients had.

Integrating evidence quality appraisal with an assessment of the anticipated balance between benefits and harms leads to designation of a policy as a strong.

Scatterplots showing baseline correlation between forced expiratory volume in 1 s (FEV1) % pred and a) COPD Assessment Test (CAT), b) Clinical COPD Questionnaire.

Mean change from baseline in percentage predicted forced vital capacity (FVC) in the a) phase III CAPACITY [27] and b) ASCEND [14] studies. #: n=174; ¶:

Study protocol. Study protocol. (A) Patients were randomly allocated to placebo or amitriptyline treatment for four weeks. At the end of the month there.

A) Dyspnoea response at rest, iso-time, and peak exercise in 20 patients with fibrotic interstitial lung disease during constant work-rate cycle exercise.

Mean BMI z scores in 6- to 9-year-old white girls with and without breast development, based on the PROS study data reported by Kaplowitz et al.13 The.

SMD (95% CI) for outcomes at 12 weeks

Comparison of total lung capacity (TLC), slow vital capacity (SVC) and carbon monoxide transfer factor (Tlco) in patients with stable interstitial lung.

Presentation transcript:

Morquio A: Endurance

Endurance testing assesses functional capacity Endurance tests measure the efficiency of performing a task and provide assessments of: Functional status of the cardiac, respiratory and musculoskeletal systems Overall disease progression Quality of life Response to treatments McDonald et al, J Pediatr Rehabil Med, 2010; Hendriksz et al, J Inherit Metab Dis, 2012

Image courtesy of Elizabeth Wright Recommended endurance tests for assessing submaximal functional capacity in Morquio A patients include: 6 minute walk test (6MWT) measures how far a person can walk on a hard, flat surface in 6 minutes a standardized test (American Thoracic Society, 2002) used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) 3 minute stair climb (3MSCT) measures how many steps a person can ascend in 3 minutes, using rails and resting as needed used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) Harmatz et al, Mol Genet Metab, 2013; Harmatz et al, Pediatrics, 2005; McDonald et al, J Pediatr Rehabil Med, 2010; Hendriksz et al, J Inherit Metab Dis, 2012 Submaximal intensity tests are sensitive indicators of endurance for MPS patients

Endurance is impaired in Morquio A Mean 6MWT = ±152.2 m (n = 316 subjects) Mean 3MSCT = 30.0 ±24.0 stairs (n= 274 subjects) Harmatz et al, Mol Genet Metab, 2013 MorCAP baseline data Mean 6MWTMean 3MSCT n=248n=68n=316 n=215n=59n=274

Assessment At diagnosis Annually As clinically indicated Evaluation by physiotherapistXXX 6 MWT (if physically and developmentally able)XXX 3 MSCT (if physically and developmentally able)XXX Regular assessments are recommended for optimal patient quality of life Hendriksz et al, J Inherit Metab Dis, 2012