Impact on patients of the coronovirus disease 2019 pandemic and postponement of cochlear implant surgery: a qualitative study.

OBJECTIVE: This study aimed to explore the impact of the coronavirus disease 2019 pandemic and postponement of elective surgical procedures for profoundly deaf patients awaiting cochlear implantation. METHOD: Open-ended questionnaires were sent to all adult patients awaiting cochlear implantation surgery. Qualitative analysis was performed using a grounded theory approach. RESULTS: Participants described a primarily negative impact on wellbeing from the surgery delay, expressing feelings of isolation or loneliness. Low mood, depression or hopelessness were commonly expressed by elderly participants; frustration and anxiety were described by young adults. Participants described a negative impact on their general daily life, describing difficulties communicating with facemasks and struggles with reliance on telephone communication because of social distancing. Despite these significant psychosocial challenges, only a minority described adaptive coping strategies. DISCUSSION: Profoundly deaf patients may be at greater psychosocial risk because of unique challenges from their hearing disability. Our findings can be used to develop evidence-driven strategies to improve communication, wellbeing and quality of life.

Core outcome set for surgical trials in gastric cancer (GASTROS study): international patient and healthcare professional consensus.

BACKGROUND: Surgery is the primary treatment that can offer potential cure for gastric cancer, but is associated with significant risks. Identifying optimal surgical approaches should be based on comparing outcomes from well designed trials. Currently, trials report different outcomes, making synthesis of evidence difficult. To address this, the aim of this study was to develop a core outcome set (COS)-a standardized group of outcomes important to key international stakeholders-that should be reported by future trials in this field. METHODS: Stage 1 of the study involved identifying potentially important outcomes from previous trials and a series of patient interviews. Stage 2 involved patients and healthcare professionals prioritizing outcomes using a multilanguage international Delphi survey that informed an international consensus meeting at which the COS was finalized. RESULTS: Some 498 outcomes were identified from previously reported trials and patient interviews, and rationalized into 56 items presented in the Delphi survey. A total of 952 patients, surgeons, and nurses enrolled in round 1 of the survey, and 662 (70 per cent) completed round 2. Following the consensus meeting, eight outcomes were included in the COS: disease-free survival, disease-specific survival, surgery-related death, recurrence, completeness of tumour removal, overall quality of life, nutritional effects, and 'serious' adverse events. CONCLUSION: A COS for surgical trials in gastric cancer has been developed with international patients and healthcare professionals. This is a minimum set of outcomes that is recommended to be used in all future trials in this field to improve trial design and synthesis of evidence.

The role of virtual reality in the changing landscape of surgical training.

BACKGROUND: The current coronavirus disease 2019 pandemic has caused unprecedented challenges to surgical training across the world. With the widespread cancellations of clinical and academic activities, educators are looking to technological advancements to help 'bridge the gap' and continue medical education. SOLUTIONS: Simulation-based training as the 'gold standard' for medical education has limitations that prevent widespread adoption outside suitably resourced centres. Virtual reality has the potential to surmount these barriers, whilst fulfilling the fundamental aim of simulation-based training to provide a safe, effective and realistic learning environment. CURRENT LIMITATIONS AND INSIGHTS FOR FUTURE: The main limitations of virtual reality technology include comfort and the restrictive power of mobile processors. There exists a clear developmental path to address these restrictions. Continued developments of the hardware and software set to deepen immersion and widen the possibilities within surgical education. CONCLUSION: In the post coronavirus disease 2019 educational landscape, virtual, augmented and mixed reality technology may prove invaluable in the training of the next generation of surgeons.

Validation of the Manchester spoken language development scale (MSLDS).

Introduction: Standardized outcome measures are importantfor accurately monitoring the language development of pre-lingually deaf children receiving auditory implants. Current commonly used outcome measures are time-consuming,limiting the practicality of regular testing. To address these limitations, the Manchester Spoken Language Development Scale (MSLDS) was developed as a quick and easily applicable interim measurement. This is an 11-point scale designed to provide a streamlined overview of a child's expressive language development. This study describes the MSLDS, evaluates its ease of use and inter-rater reliability, and outlines its application in the paediatric auditory implant population. Methods: Sixteen speech therapists and teachers for the deaf reviewed videos of paediatric cochlear implant assessmentsand rehabilitation sessions at a UK auditory implant centre. Twenty-five videos from fourteen children were used in this validation study. Reviewers were asked to evaluate a child's language development using the MSLDS by assigning a score for each video and to evaluate the ease of use of the scale. Each video wasrated by three different reviewers. Results: MSLDS scores showed a high degree of consistency between raters for each child. 8/25 (32%) videos demonstrated perfect agreement on the MSLDS. In 15/25 (60%) videos, there was a one-point difference between MSLDS scores. The remaining 2/25 (8%) videos varied by 2 points. Statistical analysis demonstrated an intra-class correlation coefficient (ICC) of 0.987, indicating a high level of agreement between users of the scale. Qualitative feedback from the raters suggested further modifications which have been incorporated into the scale. Conclusion: The high inter-rater agreement reflects the potential for the MSLDS to be a reliable tool for monitoring language development in the paediatric auditory implant population.

Data in support of the longitudinal characterization of pulmonary function in children with Mucopolysaccharidoses IVA.

Mucopolysaccharidoses type IVA (Morquio disease) is a rare, autosomal recessive lysosomal storage disease that causes both obstructive and restrictive airway pathology, with respiratory failure being the primary cause of death. This article provides original data on the longitudinal characterization of pulmonary function changes in children with Mucopolysaccharidoses (MPS) IVA by presenting the data and nuanced trends of changes from sequential spirometry and oximetry. The sample size included 16 subjects, 13 had undergone enzyme replacement therapy (ERT), three had not undergone ERT treatment. A total of 180 individual plots are presented for spirometry variables (FEV1, FEV1 [%Pred] FVC, FVC [%Pred] and FEV1/FVC), 6MWT and oximetry variables (median %Spo2, ODI 3%, mean nadir 3%, ODI 4%, mean nadir 4% and min dip SpO2 [%]); over a nine-year period at a single quaternary paediatric metabolic centre. This data has been made public and has utility to clinicians and researchers due to the following: [1,2] by providing the first comprehensive report of detailed changes in pulmonary function in children with MPS IVA, with and without ERT; [1-3] as well as changes in pulmonary function following the institution of non-invasive ventilation (NIV) and adenotonsillectomy. The data presented is related to the research article by Kenth et al. "The Characterization of Pulmonary Function in Patients with Mucopolysaccharidoses IVA: A Longitudinal Analysis".

The characterisation of pulmonary function in patients with mucopolysaccharidoses IVA: A longitudinal analysis.

INTRODUCTION: Mucopolysaccharidosis (MPS) type IVA is a rare, autosomal recessive lysosomal storage disease causing substrate accumulation in various organs and tissues. MPS IVA is associated with both obstructive and restrictive airway disease, with the former often resulting in sleep disordered breathing (SDB). Respiratory failure is a primary cause of death in this condition. The aim of this study was to characterise and catalogue the long-term respiratory changes in patients with MPS IVA treated with, or without, enzyme replacement therapy (ERT). METHODS: In this retrospective, longitudinal, repeated-measures cohort study, descriptive statistics and non-parametric correlation were performed for demographic, respiratory function and oximetry variables over a study period from January 2009 to December 2018. Composite clinical endpoints used in this study for evaluating pulmonary function included spirometry variables (FEV1, FEV1 [%Pred] FVC, FVC [%Pred] and FEV1/FVC), oximetry variables (median %Spo2, ODI 3%, mean nadir 3%, ODI 4%, mean nadir 4% and min dip SpO2 [%]) and 6MWT to assess functional exercise capacity and thus integrated cardiopulmonary function. RESULTS: Sequential spirometry and oximetry values were collected from 16 patients, of which 13/16 were ERT treated. In general, during the study period there was a global reduction in static spirometry values in all subjects, as well as cardiorespiratory function as assessed by the 6MWT, with the decline being delayed in the ERT group. Oximetry changed to a minor degree over time in the ERT group, whereas it declined in the non-ERT group. FEV1, FVC [%predicted] and ODI 3% exhibited a strong, combined positive correlation (r 0.74-95% CI 0.61 to 0.83; p < .0001). Non-invasive ventilation (NIV) and adenotonsillectomy appeared more effective in the ERT group, either improving pulmonary function or attenuating deterioration. CONCLUSIONS: Whilst spirometry values showed a gradual decline across all groups, oximetry showed modest improvement in respiratory function. The amalgamation of FEV1, FVC [%predicted] and ODI 3% appeared predictive of changes in respiratory function in this study, suggestive as being composite endpoints for monitoring disease progression as well as guiding response to ERT in MPS IVA patients.

Respiratory complications of metabolic disease in the paediatric population: A review of presentation, diagnosis and therapeutic options.

Inborn errors of metabolism (IEMs) whilst individually rare, as a group constitute a field which is increasingly demands on pulmonologists. With the advent of new therapies such as enzyme replacement and gene therapy, early diagnosis and treatment of these conditions can impact on long term outcome, making their timely recognition and appropriate investigation increasingly important. Conversely, with improved treatment, survival of these patients is increasing, with the emergence of previously unknown respiratory phenotypes. It is thus important that pulmonologists are aware of and appropriately monitor and manage these complications. This review aims to highlight the respiratory manifestations which can occur. It isdivided into conditions resulting primarily in obstructive airway and lung disease, restrictive lung disease such as interstitial lung disease or pulmonary alveolar proteinosis and pulmonary hypertension, whilst acknowledging that some diseases have the potential to cause all three. The review focuses on general phenotypes of IEMs, their known respiratory complications and the basic metabolic investigations which should be performed where an IEM is suspected.

Improved speech and language development after unilateral cochlear implantation in children with a potentially useable contralateral ear.

OBJECTIVES: Increasingly, children are considered for a unilateral cochlear implant (CI), even if the contralateral ear falls outside current audiological guidelines, especially if they are not considered to be reaching their educational potential. Here we present the outcomes of CI in children with potentially useable hearing in the contralateral ear. METHODS: A retrospective case note review was performed for a total of 57 patients. Primary outcome was speech and language (SaL) development, as measured by the Manchester Speech and Language Development Scale (MSLDS) and SaL age equivalent. Secondary outcomes were auditory perception, perceived parental benefit and compliance; respectively measured by Categories of Auditory Performance (CAP), Brief Assessment of Parental Perception (BAPP) and reported use. RESULTS: SaL development improved after CI with a mean pre-operative MSLDS score of 5.8 to a postoperative score of 8.0 (n = 57) and a mean SaL age equivalent of 14 months in a one-year period (n = 14). Furthermore, CAP scores improved from 4.9 to 7.0 (n = 57). Analysis of BAPP scores showed improved quality of life in 18/19 patients (94.7%). With regards to compliance, 50/57 (87.7%) are fulltime users of both their CI and their HA. CONCLUSION: The present study indicates that despite one ear having potentially useable hearing outside national audiological criteria, the majority of participants received benefit from a CI in the poorer hearing ear. We suggest that assessment of each ear separately and treatment with the most appropriate amplification device, has given these children a benefit they may not otherwise have acquired if they only had bilateral HA.

Multidisciplinary guidelines for the management of paediatric tracheostomy emergencies.

Temporary and permanent tracheostomies are required in children to manage actual or anticipated long-term ventilatory support, to aid secretion management or to manage fixed upper airway obstruction. Tracheostomies may be required from the first few moments of life, with the majority performed in children < 4 years of age. Although similarities with adult tracheostomies are apparent, there are key differences when managing the routine and emergency care of children with tracheostomies. The National Tracheostomy Safety Project identified the need for structured guidelines to aid multidisciplinary clinical decision making during paediatric tracheostomy emergencies. These guidelines describe the development of a bespoke emergency management algorithm and supporting resources. Our aim is to reduce the frequency, nature and severity of paediatric tracheostomy emergencies through preparation and education of staff, parents, carers and patients.

Peptide hydrogel in vitro non-inflammatory potential.

Peptide-based hydrogels have attracted significant interest in recent years as these soft, highly hydrated materials can be engineered to mimic the cell niche with significant potential applications in the biomedical field. Their potential use in vivo in particular is dependent on their biocompatibility, including their potential to cause an inflammatory response. In this work, we investigated in vitro the inflammatory potential of a ß-sheet forming peptide (FEFEFKFK; F: phenylalanine, E: glutamic acid; K: lysine) hydrogel by encapsulating murine monocytes within it (3D culture) and using the production of cytokines, IL-ß, IL-6 and TNFα, as markers of inflammatory response. No statistically significant release of cytokines in our test sample (media + gel + cells) was observed after 48 or 72 h of culture showing that our hydrogels do not incite a pro-inflammatory response in vitro. These results show the potential biocompatibility of these hydrogels and therefore their potential for in vivo use. The work also highlighted the difference in monocyte behaviour, proliferation and morphology changes when cultured in 2D vs. 3D. © 2016 The Authors Journal of Peptide Science published by European Peptide Society and John Wiley & Sons Ltd.

Mucopolysaccharidosis I; Parental beliefs about the impact of disease on the quality of life of their children.

BACKGROUND: Hematopoietic stem cell transplants, alongside enzyme replacement therapy and good multi-disciplinary care, have dramatically improved the life expectancy in children with Mucopolysaccharidosis (MPS) I, with better objective and functional outcomes. Despite these improvements, children with both the attenuated (non-Hurler) and severe (Hurler) variants of the disease have marked residual morbidity. Children with MPS I suffer with head and neck disease including obstructive sleep apnoea and hearing loss. The impact of these on quality of life has been poorly researched and no previous work has been published looking at patients' perception of their own health, an important domain when considering the impact of treatment. METHODS: This exploratory qualitative study aimed to discover the effect of head and neck disease, alongside that of MPS I as a whole, on the quality of life of affected children. A grounded theory approach was used to conduct this study. Children and their parents were invited to participate in semi-structured interviews. The transcribed interviews were coded and emergent themes explored until saturation occurred. RESULTS: The families of eleven children with MPS I were interviewed, five with Hurler's and six with the attenuated non-Hurler's. Important themes to emerge were- the fear of dying associated with obstructive sleep apnoea, difficulties communicating at school due to the delayed acquisition of language, chronic pain and restricted mobility, physical differences and restricted participation in social activities such as sports secondary to the musculoskeletal disease burden. The overall theme running through the analysis was the desire to fit in with ones peers. CONCLUSION: Parents and children with MPS 1 worry about 'fitting-in' with broader society. The presence of airway disease has a profound impact on the emotional well being of parents whilst language delay and musculoskeletal disease have the biggest impact on the quality of life of the children themselves. It is important to understand the impact of MPS I on the quality of life of children and their families so that we may improve future treatment and management of this sub-group of children who have an increasing life span.

Unilateral cochlear implantation in children with a potentially useable contralateral ear.

Increasingly, children are considered for a unilateral CI, even if the contralateral ear falls outside current audiological guidelines, especially if they are not considered to be reaching their educational potential. The primary aim was to investigate the benefit of unilateral CI in children currently outside UK [National Institute for Health and Care Excellence Technology Appraisal Guidance. 2009. Cochlear implants for children and adults with severe to profound deafness. NICE technology appraisal guidance [TAG166]. Available January 29, 2016 from http://www.nice.org.uk/ta166 ] audiological guidelines in the contralateral ear. The secondary aim was to measure compliance. A retrospective case review with standard demographic data was performed. Forty-seven children were identified as having received a unilateral CI with the contralateral ear falling outside of current UK audiological criteria. These children were allocated to two groups; with hearing between 50 and 70 dB, and 70 and 90 dB at 2 and 4 kHz in the contralateral ear, respectively. Categories of auditory performance (CAP) were assessed. Pre- and post-operative CAP scores demonstrated a statistically significant improvement in auditory perception. We would suggest that assessing candidacy in individual ears and subsequent unilateral CI, has given these children a benefit they may not otherwise have acquired if they only had bilateral hearing aid.

The management of children with Down syndrome and profound hearing loss.

INTRODUCTION: Although, the association between Down syndrome (DS) and conductive hearing loss is well recognized, the fact that a small proportion of these children may have a severe to profound sensorineural hearing loss that could benefit from cochlear implantation (CI) is less well understood. The management of significant co-morbidities in children with DS can delay initial diagnosis of hearing impairment and assessment of suitability for CI can likewise be challenging, due to difficulties conditioning to behavioural hearing tests. METHODS: We performed a retrospective case note review of three children with DS referred to the Manchester Cochlear Implant Programme. RESULTS: Three illustrative cases are described including CI in a 4 years old. Using conventional outcome measurement instruments, the outcome could be considered to be suboptimal with a Categories of Auditory Performance score of 4 at 6 months post-op and at last follow up. In part, this is likely to reflect the delay in implantation, but the role of cognitive impairment must be considered. The cases described emphasize the importance of comprehensive radiological and audiological assessment in children with DS being considered for CI. CONCLUSION: The influence of cognitive impairment upon outcome of CI must be taken into account, but should not be considered a contra-indication to implantation in children with DS. Benefit that might be considered limited when quantified using existing general outcome measurement instruments, may have a significant impact upon psychosocial development and quality of life in children with significant cognitive impairment, or other additional needs.

Infantile haemangiopericytoma: a rare congenital cervical tumour.

OBJECTIVE: Infantile haemangiopericytoma is a rare childhood vascular tumour of borderline malignancy and unpredictable clinical course. It can present a diagnostic challenge due to indeterminate clinical, radiological and pathological features. This report presents the case of a large congenital haemangiopericytoma of the neck in a neonate, and discusses diagnosis, imaging, pathology and surgical management. CLINICAL PRESENTATION: A full-term neonate presented with a large posterior neck mass at birth. Pre-operative radiological appearances were suggestive of teratoma, but following surgical excision the diagnosis of infantile haemangiopericytoma was confirmed on histological analysis. There were no signs of recurrence at 12-month follow up. CONCLUSION: Haemangiopericytoma can follow an aggressive course in adults, including local recurrence and metastasis. The infantile variant is rare but typically follows a distinct clinical course, and is associated with more benign behaviour compared with similar tumours in adults and children over one year. Congenital haemangiopericytoma can be effectively treated with surgery, without requiring adjuvant therapy.

The role of stability measurements of the Baha® system in children.

OBJECTIVE: Percutaneous bone conduction hearing aids are an established treatment for selected children unable to use conventional hearing aids. Currently in children, loading the implant is delayed for 3-6 months following fixture placement, due to concerns regarding bone quality, bone thickness and subsequent implant stability. Traditionally, such concerns led to children undergoing 2-stage Baha(®) surgery, with a second operation to attach the abutment after 3-6 months. Bone conduction implant stability can be objectively measured using resonance-frequency analysis (RFA) to generate Implant Stability Quotients (ISQs). We aimed to assess implant stability in children undergoing 1-stage surgery using RFA measurements and investigate the possible implications for earlier loading following surgery. METHODS: We report a case series of consecutive children undergoing Baha(®) at our tertiary paediatric hospital. The interval to implant loading remained 3-6 months for the duration of this study. RFA measurements were taken peri-operatively, 1 week post-surgery, within 3 months of surgery and then subsequently at follow up appointments. RFA measurements were also measured at loading of the hearing processor and at follow up appointments after loading. RESULTS: Nine children received 10 Cochlear™ Baha(®) BI300 implants (8 unilateral and 1 bilateral) with a mean age of 9 years 4 months (4 years 9 months to 13 years 5 months). The mean time to loading of the hearing processor was 3.3 months (3 to 5 months, n=9) and mean follow up was 9 months (12 weeks to 23 months). Eight children had ISQs that were potentially sufficient to have loaded the implant earlier than 3 months. Implant stability was maintained after loading with the hearing processor. CONCLUSION: This study supports the potential use of RFA measurements to either, guide implant loading following 1-stage surgery in children, or to enable a larger prospective study of early loading (4-6 weeks) in children.

Feasibility of cochlear implantation in Mucopolysaccharidosis.

UNLABELLED: Mucopolysaccharidoses (MPS) are a group of rare inherited metabolic disorders resulting from deficiencies of particular enzymes involved in the breakdown of glycosaminoglycans. Amongst the manifestations of MPS within the head and neck patients may develop conductive, mixed or sensorineural hearing loss. OBJECTIVE: The main objective of this paper is to describe the management of profound sensorineural hearing loss in children with Mucopolysaccaridosis. The primary outcome measures for this case series were improvement in auditory performance and speech perception scores following cochlear implantation. Secondary outcome measures included surgical complications. METHODS: We carried out a casenote review of the first two cases of cochlear implantation (CI) to rehabilitate profound sensory neural hearing loss in Mucopolysaccharidoses. Improvement in auditory performance was measured by categories of auditory performance (CAP) score, speech reception score (SRS) and the IHR McCormick toy discrimination test. RESULTS: Both patients with MPS had demonstrable benefit from CI in terms of auditory performance and speech perception. The first patient improved from pre-operatively only managing to recognise environmental sounds to understanding conversation without lip-reading with a familiar talker. Following CI, the second patient can discriminate speech in noisy environments to a degree, without lip-reading. No peri-operative complications were noted in either patient. CONCLUSION: As the medical management of the MPS has progressed there is likely to be a corresponding increase in survival. This increased life-expectancy will likely lead to greater numbers of patients with MPS surviving long enough to develop profound hearing loss. Likewise, when considering the risks and benefits of quality of life interventions such as CI in patients with MPS, it is more likely that the risks of surgery and general anaesthesia will be considered acceptable. Clinicians managing such patients will need to be aware of these developments.