The Relationship Between Patient Reported Dysphagia Symptom Severity And Swallowing Related Quality Of Life In Patients With Neurological Disorders

Objective: The aim of this study was to determine the relationship between patient reported dysphagia symptom severity and swallowing related quality of life (SRQOL) in patients with neurological disorders. Methods: Patients with a diagnosis of neurological disorders who aged between 25 to 60 years were included. The dysphagia symptom severity was assessed with the Turkish Eating Assessment Tool (T-EAT-10). SRQOL was assessed by the Turkish version of the Swallow Quality of Life Questionnaire (T-SWAL-QOL). Results: Eighty-four patients with neurological disorder were included. The mean T-EAT-10 score was 19.59 +/- 11.16 (min = 0, max = 40), and the mean total score of T-SWAL-QOL was 50.63 +/- 23.52 (min = 0, max = 90). A negative, good, statistically significant correlation between T-EAT-10 and total T-SWAL-QOL was found (p < 0.05). Conclusion: Higher patient reported dysphagia symptom severity is associated with lower scores of SRQOL. Therefore, appropriate management strategies which also focus on patient reported symptoms may be necessary to minimize negative SRQOL issues of patients with neurogenic dysphagia.Wo

Head And Neck Cancer: Changes In Artrokinematic Parameters Of Neck And Swallowing Function After Radiotherapy

The study aimed to show the changes on artrokinematic parameters of the neck and swallowing function after radiotherapy (RT) in head and neck cancer (HNC) patients. The fourty patients with HNC have been evaluated before, 1 and 3 months after RT. The posture, normal joint movement, muscle strength and endurance of the neck were evaluated as artrokinematic parameters. The clinical and radiological swallowing function evaluations were also done. Mean age of patients were 53,22 +/- 10,92 years. Head anterior tilt, kyphosis and shoulder protraction were more in the 3rd month after RT in posture analysis (p<0.001). The neck flexion, lateral flexion and rotation limitations have increased (p<0.001), neck muscle strength and endurance have gradually decreased (p<0,001) after RT. At the same time, swallowing phases have been adversely affected after AT with all these side effects (p<0.001) and the severity of aspiration in all consistencies has increased gradually (p<0.05). As a result, RT starts to show its negative effects from the early period of cancer treatment. Unlike other studies, we concluded that RT affects head and neck structures negatively and these describes how RT affects swallowing function. So it is highly important to include head and neck structure mobility, strength and endurance assessments and exercises in swallowing rehabilitation.WoSScopu

Reliability and validity of the turkish version of the feeding/swallowing impact survey

Inal, Ozgu (Trakya author)The purpose of this study was to translate the Feeding/Swallowing Impact Survey (FS-IS) into Turkish and to test its reliability and validity in caregivers of children with cerebral palsy (CP). Caregivers accompanying the 117 children with CP who were referred for an instrumental swallowing evaluation were included in the study. The FS-IS was translated from English into Turkish by using the forward, backward, forward translation method. Parents completed the Turkish version of the Feeding/Swallowing Impact Survey (T-FS-IS) which has 3 subscales including daily activities, worry and feeding difficulties. Swallowing function was assessed with Videofluoroscopic Swallowing Study, and penetration and aspiration severity was determined according to the Penetration-Aspiration Scale (PAS). The internal consistency, test-retest reliability, construct validity, and discriminant validity of the T-FS-IS were investigated. Internal consistency was excellent with Cronbach alphas all above 0.8 (Total score=0.99, daily activities=0.98, worries=0.98, and feeding difficulties=0.96). The Intraclass correlation coefficient was 0.93, demonstrating excellent test-retest reliability. All three subscales of the T-FS-IS including daily activities, worry, and feeding difficulties as well as the total score significantly correlated with the PAS scores. Caregivers whose children had aspiration reported worse scores in the T-FS-IS total and its subscales compared with caregivers whose children without airway aspiration according to PAS (p<0.01). The T-FS-IS is a reliable and valid instrument for measuring the impact of swallowing disorders on caregivers of children with CP. It can be used in clinical practice and research. Clinical trial number: NCT03005093

Mapping the differences in care for 5,000 Spinal Muscular Atrophy patients, a survey of 24 national registries in North America, Australasia and Europe

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder characterised by the degeneration of motor neurons and progressive muscle weakness. It is caused by homozygous deletions in the survival motor neuron gene on chromosome 5. SMA shows a wide range of clinical severity, with SMA type I patients often dying before 2\ua0years of age, whereas type III patients experience less severe clinical manifestations and can have a normal life span. Here, we describe the design, setup and utilisation of the TREAT-NMD national SMA patient registries characterised by a small, but fully standardised set of registry items and by genetic confirmation in all patients. We analyse a selection of clinical items from the SMA registries in order to provide a snapshot of the clinical data stratified by SMA subtype, and compare these results with published recommendations on standards of care. Our study included 5,068 SMA patients in 25 countries. A total of 615 patients were ventilated, either invasively (178) or non-invasively (437), 439 received tube feeding and 455 had had scoliosis surgery. Some of these interventions were not available to patients in all countries, but differences were also noted among high-income countries with comparable wealth and health care systems. This study provides the basis for further research, such as quality of life in ventilated SMA patients, and will inform clinical trial planning

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Background: Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy. Methods: In this phase 3, randomised, double-blind, placebo-controlled study, patients aged 2–25 years with confirmed 5q autosomal recessive type 2 or type 3 spinal muscular atrophy were recruited from 42 hospitals in 14 countries across Europe, North America, South America, and Asia. Participants were eligible if they were non-ambulant, could sit independently, and had a score of at least 2 in entry item A of the Revised Upper Limb Module. Patients were stratified by age and randomly assigned (2:1) to receive either daily oral risdiplam, at a dose of 5·00 mg (for individuals weighing ≥20 kg) or 0·25 mg/kg (for individuals weighing &lt;20 kg), or daily oral placebo (matched to risdiplam in colour and taste). Randomisation was conducted by permutated block randomisation with a computerised system run by an external party. Patients, investigators, and all individuals in direct contact with patients were masked to treatment assignment. The primary endpoint was the change from baseline in the 32-item Motor Function Measure total score at month 12. All individuals who were randomly assigned to risdiplam or placebo, and who did not meet the prespecified missing item criteria for exclusion, were included in the primary efficacy analysis. Individuals who received at least one dose of risdiplam or placebo were included in the safety analysis. SUNFISH is registered with ClinicalTrials.gov, NCT02908685. Recruitment is closed; the study is ongoing. Findings: Between Oct 9, 2017, and Sept 4, 2018, 180 patients were randomly assigned to receive risdiplam (n=120) or placebo (n=60). For analysis of the primary endpoint, 115 patients from the risdiplam group and 59 patients from the placebo group were included. At month 12, the least squares mean change from baseline in 32-item Motor Function Measure was 1·36 (95% CI 0·61 to 2·11) in the risdiplam group and –0·19 (–1·22 to 0·84) in the placebo group, with a treatment difference of 1·55 (0·30 to 2·81, p=0·016) in favour of risdiplam. 120 patients who received risdiplam and 60 who received placebo were included in safety analyses. Adverse events that were reported in at least 5% more patients who received risdiplam than those who received placebo were pyrexia (25 [21%] of 120 patients who received risdiplam vs ten [17%] of 60 patients who received placebo), diarrhoea (20 [17%] vs five [8%]), rash (20 [17%] vs one [2%]), mouth and aphthous ulcers (eight [7%] vs 0), urinary tract infection (eight [7%] vs 0), and arthralgias (six [5%] vs 0). The incidence of serious adverse events was similar between treatment groups (24 [20%] of 120 patients in the risdiplam group; 11 [18%] of 60 patients in the placebo group), with the exception of pneumonia (nine [8%] in the risdiplam group; one [2%] in the placebo group). Interpretation: Risdiplam resulted in a significant improvement in motor function compared with placebo in patients aged 2–25 years with type 2 or non-ambulant type 3 spinal muscular atrophy. Our exploratory subgroup analyses showed that motor function was generally improved in younger individuals and stabilised in older individuals, which requires confirmation in further studies. SUNFISH part 2 is ongoing and will provide additional evidence regarding the long-term safety and efficacy of risdiplam. Funding: F Hoffmann-La Roche