Validation of Polish-Language Questionnaires for Assessing the Quality of Life of Patients with Primary Ciliary Dyskinesia (PCD-QOL).

In recent years, questionnaires were published in English to assess the quality of life of patients with PCD (Primary Ciliary Diskinesia) for adults, adolescents aged 13-17 years, and children aged 6-12 years and their caregivers. This study aimed to prepare Polish versions of the questionnaires and validate them in specific age groups with the participation of Polish patients with PCD. The individual questionnaires were translated and discussed with the involvement of the creator of the original questionnaire in English. Patients completed the questionnaires according to their affiliation with one of the groups. Validation was based on internal consistency analysis (Cronbach's alpha coefficient and split-half reliability) and test-retest reliability (intraclass correlation coefficient-ICC). The internal consistency of all questionnaires was from moderate to very good (Cronbach's alpha 0.67-0.91, split-half reliability 0.53-0.95). The consistency of the measurements showed excellent repeatability (ICC 0.67-0.91). The surveyed Polish PCD patients rated their quality of life quite well (63-77%). QOL questionnaires for patients with PCD can be used routinely during each medical check-up as a simple tool to provide the doctor with an indication of the effectiveness of treatment and the impact of the disease on the patient's quality of life.

A Human Right-Based Approach to Dealing with Adverse Events in Residential Care Facilities.

Managing residential care facilities (RCFs) includes the ability to manage adverse events while maintaining a human rights-based approach to care and support. Literature investigating rights-based approaches in RCFs is scarce; therefore, an investigation of the current approach in RCFs will inform improvements. This study sought to identify whether RCFs in Ireland upheld a rights-based approach during the course of adverse events by analyzing notifications of adverse events from 2021 taken from the Database of Statutory Notifications from Social Care in Ireland. Data analysis was conducted independently by two researchers. Notifications of adverse events were coded according to whether the human rights principles of fairness, respect, equality, dignity, and autonomy were upheld or violated during the adverse event and its subsequent management. There was some evidence of violations, including staff violations during adverse events and their management, as well as residents violating fellow residents' autonomy, respect, and dignity in notifications of "serious injury" and "allegations of abuse." However, overall, good practice was identified, with residents' human rights upheld by staff. Our findings indicate that a rights-based approach to care and support is being upheld during adverse events and their management, which may indicate that such an approach to care and support has been adopted.

Mortality in residential care facilities for people with disability: a descriptive cross-sectional analysis of statutory notifications in Ireland.

OBJECTIVES: To determine incidence of death in residential care facilities for people with disability in Ireland, primary cause of death, associations of facility characteristics and deaths, and to compare characteristics of deaths reported as expected and unexpected. DESIGN: Descriptive cross-sectional study. SETTING: All residential care facilities for people with disability operational in Ireland in 2019 and 2020 (n=1356). PARTICIPANTS: n=9483 beds. MAIN OUTCOME MEASURES: All expected and unexpected deaths notified to the social services regulator. Cause of death as reported by the facility. RESULTS: 395 death notifications were received in 2019 (n=189) and 2020 (n=206). 45% (n=178) were for unexpected deaths. Incidence of death per 1000 beds per year was 20.83 for all, 11.44 for expected and 9.39 for unexpected deaths. Respiratory disease was the most common cause of death, accounting for 38% (n=151) of all deaths. In adjusted negative binomial regression analysis, congregated settings versus non-congregated (incidence rate ratio (95% CI): 2.59 (1.80 to 3.73)) and higher bed numbers (highest vs lowest quartile) (4.02 (2.19 to 7.40)) were positively associated with mortality. There was also a positive n-shaped association with category of nursing staff-to-resident ratio when compared with zero nurses. Emergency services were contacted for 6% of expected deaths. Of the deaths reported as unexpected, 29% were receiving palliative care and 10.8% had a terminal illness. CONCLUSION: Although incidence of death was low, residents of congregated and larger settings had higher incidence of death than residents of other settings. This should be a consideration for practice and policy. Given the high contribution of respiratory diseases to deaths and the potential avoidability of these, there is a need for improved respiratory health management in this population. Nearly half of all deaths were reported as unexpected; however, overlap in the characteristics of expected and unexpected deaths highlights the need for clearer definitions.

Definitions of serious injury in long-term residential care: a systematic review protocol.

Background: Evidence indicates that the reporting of serious injury in long-term residential care has increased substantially over the past decade. However, what constitutes a serious injury in residential care is poorly and inconsistently defined. This may result in incidences being unnecessarily reported as a serious injury. It is therefore, crucial to develop a consistent definition of serious injury to reduce reporting burden and to facilitate comparison between different residential care settings and across jurisdictions. This protocol describes the methods for a systematic review of existing definitions from the literature to inform the development of a consistent definition of serious injury in long-term residential care. Methods: A wide range of published peer-reviewed and grey literature will be sought for this review, including guidance and policy documents. Searches will be conducted of databases including MEDLINE, CINAHL, SocINDEX, Academic Search Ultimate, and Westlaw International. Grey literature database searches will include Trip and Social Care Online. Country specific searches of government and health and social care websites will be conducted. Quality appraisal will be facilitated using the Quality Assessment for Diverse Studies (QuADS) tool and Tyndall's checklist. The level of confidence in the findings will be assessed using the GRADE CERQual approach. A customised data extraction form will be used to extract data to reduce the risk of bias. Conceptual content analysis of data will facilitate identification of definitions of serious injury and their frequency within texts. Conclusions: The findings will inform the development of a consistent definition of serious injury in long-term residential care that will reduce reporting burden, facilitate the accuracy of data collected and allow for comparison across jurisdictions. A more universal and consistent definition will enable regulators, policy makers, service providers and researchers to develop policy and practical interventions to prevent the occurrence of serious injury in long-term residential care.

The Impact on Parents of Diagnosing PCD in Young Children.

Primary ciliary dyskinesia (PCD) is an incurable, rare, inherited, chronic condition. Treatment includes the regular clearing of airway mucus, aggressive treatment of infections and management of hearing loss. Caregiver burden has not been explored, hence we interviewed 18 mothers and 6 fathers of children under 6 years to understand the impact of diagnostic testing and implications of a positive diagnosis. Interviews were transcribed and thematically analysed and five key themes were identified. These included the parents' experiences following child's diagnosis, impact of child's treatment regimen on parent, impact of child's health status on parent, parent's coping strategies, and parental concerns for the future. Parents described their diagnostic journey, with the findings revealing how a lack of awareness among clinicians of the PCD symptom pattern can lead to a delayed diagnosis. Parents discussed the emotional and practical impact of a PCD diagnosis and the coping strategies employed to deal with challenges arising following a diagnosis. Parents use a variety of different lifestyle changes to accommodate their child's treatment regimen and to cope with disruptive life events such as the COVID-19 pandemic. This study provides valuable insights into parental adjustment and adaptation to a PCD diagnosis and management regimen. Going forward, this research highlights the need for integrated social care for PCD patients and their families.

Translation of the quality of life questionnaire for primary ciliary dyskinesia (QOL-PCD) into Hebrew: The Israeli experience.

BACKGROUND: Patients with primary ciliary dyskinesia (PCD) have chronic morbidities affecting their quality of life (QOL). Health-related QOL (HRQOL) has been recognized as an important tool for assessing the burden of PCD on patients and their families. A PCD-specific HRQOL questionnaire (QOL-PCD) was developed and validated for the English-speaking populations. Still, it has not yet been translated into Hebrew or adapted for Israeli PCD patients. METHODS: This describes our translation of the original English version of QOL-PCD into a Hebrew version. The process involved forward translation by independent translators, construction of a consensus version, back-translation into the original English version, analysis by the expert committee, and a pretest. The pretest was administered to 20 participants with PCD (the patients and the parents of minor patients) to assess the feasibility and practicality of the tool. Scale and item indices (scale-level content validity index [S-CVI] and item-level content validity index [I-CVI]) were calculated for content validity. RESULTS: All study participants understood the final Hebrew version that the expert committee had approved with no difficulty. The instrument had satisfactory (>0.80) content validity, with S-CVI indexes of 0.99 for the adult version, 0.86 for the parental version, 0.95 for the adolescent version, and 1.00 for the child version. CONCLUSION: The English version of a QOL-PCD was translated into Hebrew and adapted in Israel. This translation is a valid instrument to assess HRQOL in Hebrew-speaking patients with PCD and their family members. It may be helpful for PCD patient management and research in Israel.

Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review.

OBJECTIVES: Disease-specific, well-defined and validated clinical outcome measures are essential in designing research studies. Poorly defined outcome measures hamper pooling of data and comparisons between studies. We aimed to identify and describe pulmonary outcome measures that could be used for follow-up of patients with primary ciliary dyskinesia (PCD). METHODS: We conducted a scoping review by systematically searching MEDLINE, Embase and the Cochrane Database of Systematic Reviews online databases for studies published from 1996 to 2020 that included ≥10 PCD adult and/or paediatric patients. RESULTS: We included 102 studies (7289 patients). 83 studies reported on spirometry, 11 on body plethysmography, 15 on multiple-breath washout, 36 on high-resolution computed tomography (HRCT), 57 on microbiology and 17 on health-related quality of life. Measurement and reporting of outcomes varied considerably between studies (e.g. different scoring systems for chest HRCT scans). Additionally, definitions of outcome measures varied (e.g. definition of chronic colonisation by respiratory pathogen), impeding direct comparisons of results. CONCLUSIONS: This review highlights the need for standardisation of measurements and reporting of outcome measures to enable comparisons between studies. Defining a core set of clinical outcome measures is necessary to ensure reproducibility of results and for use in future trials and prospective cohorts.

Teachers' views and experiences of student mental health and well-being programmes: A systematic review.

Background: In schools, teachers are often tasked with implementing mental health and well-being programmes. However, little is known about teachers' views on and experiences with implementing these programmes.Aim: The aim of this systematic review was to explore teachers' views and experiences of mental health and well-being intervention programmes developed to promote and protect student mental health.Methods: A systematic review of the empirical literature was conducted using the following databases: Academic Search Complete, APA PsycArticles, APA PsycInfo, British Education Index, Education Full Text (H.W. Wilson), ERIC, Social Sciences Full Text (H.W. Wilson), and SocINDEX with Full Text.Findings: Seven papers met the inclusion criteria. Teachers reported several challenges to the successful implementation of mental health and well-being programmes, including a lack of time allotted in the curriculum, insufficient training, and inadequate interagency support. There was evidence of conflicting opinions regarding the role of teachers in supporting students.Conclusion: It is recommended that mental health and well-being are viewed as central to schools' ethos and that teachers are adequately prepared to implement programmes.

Health related quality of life in adult primary Ciliary dyskinesia patients in Cyprus: development and validation of the Greek version of the QOL-PCD questionnaire.

BACKGROUND: The QOL-PCD questionnaire is a recently developed Health Related Quality of Life (HRQoL) instrument for Primary Ciliary Dyskinesia. The aim of this study was to translate the adult QOL-PCD questionnaire into Greek language and to conduct psychometric validation to assess its performance. METHODS: Forward translations to Greek and backward translation to English were performed, followed by cognitive interviews in 12 adult PCD patients. The finalized translated version was administered to a consecutive sample of 31 adult, Greek speaking PCD patients in Cyprus for psychometric validation, which included assessment of internal consistency, test-retest reliability, construct and convergent validity. Internal consistency was assessed by Cronbach's alpha test in terms of the overall and sub-scales. Test-retest reliability was assessed by repeat administration of the questionnaire within 2 weeks and calculation of the intra-class correlation (ICC). Construct validity was assessed by comparing different groups of patients based on a-priori hypotheses and convergent validity was evaluated by examining associations between the QOL-PCD and SF-36 questionnaires. RESULTS: Moderate to good internal consistency was observed (Cronbach's α: 0.46-0.88 across sub-scales) and test-retest reliability assessment demonstrated good repeatability for most scales (ICC: 0.67-0.91 across subscales). Patients of female gender, older age and lower lung function exhibited lower QOL-PCD scores in general, while high correlations for most QOL-PCD scales with corresponding SF-36 scales were observed, in particular for physical functioning (r = 0.78, p < 0.05). CONCLUSION: The adult version of QoL-PCD questionnaire has been translated according to international guidelines resulting to a cross-culturally validated Greek version which exhibited moderate to good metric properties in terms of internal consistency, stability, known-group and convergent validity.

Prevalence and course of disease after lung resection in primary ciliary dyskinesia: a cohort & nested case-control study.

BACKGROUND: Lung resection is a controversial and understudied therapeutic modality in Primary Ciliary Dyskinesia (PCD). We assessed the prevalence of lung resection in PCD across countries and compared disease course in lobectomised and non-lobectomised patients. METHODS: In the international iPCD cohort, we identified lobectomised and non-lobectomised age and sex-matched PCD patients and compared their characteristics, lung function and BMI cross-sectionally and longitudinally. RESULTS: Among 2896 patients in the iPCD cohort, 163 from 20 centers (15 countries) underwent lung resection (5.6%). Among adult patients, prevalence of lung resection was 8.9%, demonstrating wide variation among countries. Compared to the rest of the iPCD cohort, lobectomised patients were more often females, older at diagnosis, and more often had situs solitus. In about half of the cases (45.6%) lung resection was performed before presentation to specialized PCD centers for diagnostic work-up. Compared to controls (n = 197), lobectomised patients had lower FVC z-scores (- 2.41 vs - 1.35, p = 0.0001) and FEV1 z-scores (- 2.79 vs - 1.99, p = 0.003) at their first post-lung resection assessment. After surgery, lung function continued to decline at a faster rate in lobectomised patients compared to controls (FVC z-score slope: - 0.037/year Vs - 0.009/year, p = 0.047 and FEV1 z-score slope: - 0.052/year Vs - 0.033/year, p = 0.235), although difference did not reach statistical significance for FEV1. Within cases, females and patients with multiple lobe resections had lower lung function. CONCLUSIONS: Prevalence of lung resection in PCD varies widely between countries, is often performed before PCD diagnosis and overall is more frequent in patients with delayed diagnosis. After lung resection, compared to controls most lobectomised patients have poorer and continuing decline of lung function despite lung resection. Further studies benefiting from prospective data collection are needed to confirm these findings.

Validation of pediatric health-related quality of life instruments for primary ciliary dyskinesia (QOL-PCD).

RATIONALE: Having developed the first disease-specific, health-related quality of life (HRQoL) instruments for children with primary ciliary dyskinesia (PCD), we aimed to assess the psychometric performance of quality of life (QOL)-PCD child, adolescent, and parent-proxy versions in terms of reliability and validity across cross-cultural settings and caring for patients with this rare disease. METHODS: Children (n = 71), adolescents (n = 85), and parents (n = 68) from multiple centers in the UK and North America completed age-appropriate QOL-PCD and generic QOL measures: pediatric QOL inventory, COPD assessment test (CAT), and Sino-Nasal Outcome Test 20. Total of 13 children, 13 parents, and 17 adolescents repeated QOL-PCD 10 to 14 days later to assess test-retest reliability. Multitrait analysis evaluated how the items loaded to hypothesized scales: physical, emotional & social functioning, treatment burden, role, vitality, upper and lower respiratory symptoms, and ears and hearing symptoms. Examination of item-to-total correlations led to removal of three, five, and six items, respectively in the prototype child, adolescent and parent-proxy versions; the validated measures now comprise between 34 and 38 items. RESULTS: The QOL-PCD scales had good internal consistency; Cronbach's α for QOL-PCD parent-proxy ranged 0.62 to 0.86. Test-retest reliability demonstrated stability across all scales; for example QOL-PCD adolescent intraclass correlation coefficients ranged 0.71 to 0.89. Significant relationships were found between QOL-PCD scales and similar constructs on generic questionnaires, for example, QOL-PCD adolescent lower respiratory symptoms and the CAT score (r = .64, P < .01); weaker correlations were found between different constructs. CONCLUSION: Age-specific QOL-PCD demonstrated good internal consistency, test-retest reliability, and validity. QOL-PCD offers promising outcome measures for multicenter clinical trials, as well as monitoring symptoms, functioning, and QOL during routine care.

Translation of the quality-of-life measure for adults with primary ciliary dyskinesia and its application in patients in Brazil.

Primary ciliary dyskinesia (PCD) is a genetic disorder that is typically inherited in an autosomal recessive manner. It is clinically characterized by recurrent respiratory infections. However, its repercussions for patient quality of life should not be overlooked. Studies have shown that PCD has a significant impact on the lives of patients, although there are as yet no PCD-specific markers of quality of life. To address that problem, researchers in the United Kingdom developed a quality-of-life questionnaire for patients with PCD. The present communication focuses on the process of translating that questionnaire into Brazilian Portuguese, through a partnership between researchers in Brazil and those in the United Kingdom, as well as its subsequent application in patients in Brazil.

Translation of the quality-of-life measure for adults with primary ciliary dyskinesia and its application in patients in Brazil / Tradução do questionário de qualidade de vida para pacientes adultos com discinesia ciliar primária no Brasil

ABSTRACT Primary ciliary dyskinesia (PCD) is a genetic disorder that is typically inherited in an autosomal recessive manner. It is clinically characterized by recurrent respiratory infections. However, its repercussions for patient quality of life should not be overlooked. Studies have shown that PCD has a significant impact on the lives of patients, although there are as yet no PCD-specific markers of quality of life. To address that problem, researchers in the United Kingdom developed a quality-of-life questionnaire for patients with PCD. The present communication focuses on the process of translating that questionnaire into Brazilian Portuguese, through a partnership between researchers in Brazil and those in the United Kingdom, as well as its subsequent application in patients in Brazil.

RESUMO A discinesia ciliar primária (DCP) é uma doença genética de origem comumente autossômica recessiva. Caracteriza-se clinicamente por infecções respiratórias de repetição; porém, a repercussão na qualidade de vida desses pacientes deve ser levada em consideração. Estudos têm demonstrado um importante impacto da doença nesse quesito, mas ainda faltam marcadores de qualidade de vida específicos para DCP. Nesse sentido, foi desenvolvido o questionário de qualidade de vida em pacientes com DCP. O presente comunicado versa sobre o processo de tradução do questionário desenvolvido no Reino Unido para o português falado no Brasil através de uma parceria entre pesquisadores do Brasil e Reino Unido e sua posterior aplicação a pacientes brasileiros.

Nurses' Use of Appropriate Needle Sizes When Administering Intramuscular Injections.

BACKGROUND: In cases where patients are overweight or obese, administration of intramuscular medications can be ineffective due to inappropriate sizes of needles used. This study investigated whether the size of needles used to administer intramuscular injections is appropriate for patients on the basis of their weight or body mass index. METHOD: This retrospective review examined 100 instances of intramuscular injection on a 50-bed ward. RESULTS: In most instances, intramuscular medication was inappropriately administered. Needle sizes were not determined based on body mass index, and the possibility of true intramuscular penetration was minimal. Appropriate needle sizes were more likely to be chosen when instructions were provided with medications. CONCLUSION: Current available needle sizes may be inappropriate for certain patients. Nursing staff require further education to assist them in making correct needle choices. It is also important that health care settings have evidence-based policies in place and the necessary resources provided to ensure safe and correct administration of medication. J Contin Educ Nurs. 2018;49(11):519-525.

Lung function in patients with primary ciliary dyskinesia: an iPCD Cohort study.

Primary ciliary dyskinesia (PCD) has been considered a relatively mild disease, especially compared to cystic fibrosis (CF), but studies on lung function in PCD patients have been few and small.This study compared lung function from spirometry of PCD patients to normal reference values and to published data from CF patients. We calculated z-scores and % predicted values for forced expiratory volume in 1 s (FEV1) and forced vital capacity (FVC) using the Global Lung Function Initiative 2012 values for 991 patients from the international PCD Cohort. We then assessed associations with age, sex, country, diagnostic certainty, organ laterality, body mass index and age at diagnosis in linear regression models. Lung function in PCD patients was reduced compared to reference values in both sexes and all age groups. Children aged 6-9 years had the smallest impairment (FEV1 z-score -0.84 (-1.03 to -0.65), FVC z-score -0.31 (-0.51 to -0.11)). Compared to CF patients, FEV1 was similarly reduced in children (age 6-9 years PCD 91% (88-93%); CF 90% (88-91%)), but less impaired in young adults (age 18-21 years PCD 79% (76-82%); CF 66% (65-68%)). The results suggest that PCD affects lung function from early in life, which emphasises the importance of early standardised care for all patients.

Depressive Symptoms and Suicidal Ideation among Irish Undergraduate College Students.

This study examined the prevalence of depressive symptoms and suicidal ideation among 220 first year undergraduate university students in Ireland. Data were collected using the Centre for Epidemiological Studies Depression Scale and the Suicidal Behaviours Questionnaire. Findings indicated that 59% of participants experienced depressive symptoms and 28.5% had suicidal ideation. Financial stress and poor relationships with both parents, increased the odds of experiencing depressive symptoms. Sexual orientation, financial stress, and poor relationships with fathers, increased the odds of experiencing suicidal ideation. Suicidal ideation was also higher in those who had sought help in the past from mental health professionals. Nurses need to be aware of the factors associated with mental ill-health in this population.

Growth and nutritional status, and their association with lung function: a study from the international Primary Ciliary Dyskinesia Cohort.

Chronic respiratory disease can affect growth and nutrition, which can influence lung function. We investigated height, body mass index (BMI), and lung function in patients with primary ciliary dyskinesia (PCD).In this study, based on the international PCD (iPCD) Cohort, we calculated z-scores for height and BMI using World Health Organization (WHO) and national growth references, and assessed associations with age, sex, country, diagnostic certainty, age at diagnosis, organ laterality and lung function in multilevel regression models that accounted for repeated measurements.We analysed 6402 measurements from 1609 iPCD Cohort patients. Height was reduced compared to WHO (z-score -0.12, 95% CI -0.17 to -0.06) and national references (z-score -0.27, 95% CI -0.33 to -0.21) in male and female patients in all age groups, with variation between countries. Height and BMI were higher in patients diagnosed earlier in life (p=0.026 and p<0.001, respectively) and closely associated with forced expiratory volume in 1 s and forced vital capacity z-scores (p<0.001).Our study indicates that both growth and nutrition are affected adversely in PCD patients from early life and are both strongly associated with lung function. If supported by longitudinal studies, these findings suggest that early diagnosis with multidisciplinary management and nutritional advice could improve growth and delay disease progression and lung function impairment in PCD.

The patient's experience of primary ciliary dyskinesia: a systematic review.

BACKGROUND: Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by progressive sinopulmonary disease, with symptoms starting soon after birth. The aim of this study is to critically review, analyse, and synthesise the literature in order to understand the experiences of patients with primary ciliary dyskinesia (PCD) and the impact on health-related quality of life. METHOD: MEDLINE, EBSCO, Cumulative Index to Nursing and Allied Health Literature (CINAHL), PsycINFO and EMBASE were searched according to the inclusion criteria. A qualitative analysis of 14 studies was conducted. RESULTS: Fourteen studies were included in the review, five with qualitative methodologies. Studies originated from the UK, USA, Italy, Denmark and Belgium, one study included a survey distributed internationally. Significant relationships were found between age and worsening of respiratory symptoms, physical, and mental domains of health-related quality of life, with a greater decline compared with reference populations. Variations between the UK and Italy were found for health-related quality of life and its correlation with time since diagnosis. PCD was found to have a physical impact in all age groups: patients found it difficult to keep up with others, and found energy levels were easily depleted compared to family or peers. In terms of social impact, symptoms lead to embarrassment and a sense of isolation, with patients concealing symptoms and/or their diagnosis. In turn, isolation was also linked with the lack of public and medical knowledge. In relation to emotional impact, anxiety was reported in a number of qualitative studies; patients were anxious about getting sick or when thinking about their future health. The burden of treatment and factors influencing adherence were also discussed in depth. CONCLUSION: Health-related quality of life decreases with age in patients with PCD. For all age groups, PCD was found to greatly impact physical, emotional, social functioning, and treatment burden. More research is needed on the psychosocial impact of the illness, disease burden and its effect on quality of life.

Validation of a health-related quality of life instrument for primary ciliary dyskinesia (QOL-PCD).

BACKGROUND: Quality of life (QOL)-primary ciliary dyskinesia (PCD) is the first disease-specific, health-related QOL instrument for PCD. Psychometric validation of QOL-PCD assesses the performance of this measure in adults, including its reliability, validity and responsiveness to change. METHODS: Seventy-two adults (mean (range) age: 33 years (18-79 years); mean (range) FEV1% predicted: 68 (26-115)) with PCD completed the 49-item QOL-PCD and generic QOL measures: Short-Form 36 Health Survey, Sino-Nasal Outcome Test 20 (SNOT-20) and St George Respiratory Questionnaire (SGRQ)-C. Thirty-five participants repeated QOL-PCD 10-14 days later to measure stability or reproducibility of the measure. RESULTS: Multitrait analysis was used to evaluate how the items loaded on 10 hypothesised scales: physical, emotional, role and social functioning, treatment burden, vitality, health perceptions, upper respiratory symptoms, lower respiratory symptoms and ears and hearing symptoms. This analysis of item-to-total correlations led to 9 items being dropped; the validated measure now comprises 40 items. Each scale had excellent internal consistency (Cronbach's α: 0.74 to 0.94). Two-week test-retest demonstrated stability for all scales (intraclass coefficients 0.73 to 0.96). Significant correlations were obtained between QOL-PCD scores and age and FEV1. Strong relationships were also found between QOL-PCD scales and similar constructs on generic questionnaires, for example, lower respiratory symptoms and SGRQ-C (r=0.72, p<0.001), while weak correlations were found between measures of different constructs. CONCLUSIONS: QOL-PCD has demonstrated good internal consistency, test-retest reliability, convergent and divergent validity. QOL-PCD offers a promising tool for evaluating new therapies and for measuring symptoms, functioning and QOL during routine care.

The international primary ciliary dyskinesia cohort (iPCD Cohort): methods and first results.

Data on primary ciliary dyskinesia (PCD) epidemiology is scarce and published studies are characterised by low numbers. In the framework of the European Union project BESTCILIA we aimed to combine all available datasets in a retrospective international PCD cohort (iPCD Cohort).We identified eligible datasets by performing a systematic review of published studies containing clinical information on PCD, and by contacting members of past and current European Respiratory Society Task Forces on PCD. We compared the contents of the datasets, clarified definitions and pooled them in a standardised format.As of April 2016 the iPCD Cohort includes data on 3013 patients from 18 countries. It includes data on diagnostic evaluations, symptoms, lung function, growth and treatments. Longitudinal data are currently available for 542 patients. The extent of clinical details per patient varies between centres. More than 50% of patients have a definite PCD diagnosis based on recent guidelines. Children aged 10-19 years are the largest age group, followed by younger children (≤9 years) and young adults (20-29 years).This is the largest observational PCD dataset available to date. It will allow us to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments, and to investigate genotype-phenotype correlations.