RESUMO
BACKGROUND: Patients with severe-to-profound hearing loss may benefit from management with cochlear implants. These patients need a referral to a cochlear implant team for further assessment and possible surgery. The referral pathway may result in varied access to hearing healthcare. This study aimed to explore referral patterns and whether there were any socioeconomic or ethnic associations with the likelihood of referral. The primary outcome was to determine factors influencing referral for implant assessment. The secondary outcome was to identify factors impacting whether healthcare professionals had discussed the option of referral. METHODS AND FINDINGS: A multicentre multidisciplinary observational study was conducted in secondary care Otolaryngology and Audiology units in Great Britain. Adults fulfilling NICE (2019) audiometric criteria for implant assessment were identified over a 6-month period between 1 July and 31 December 2021. Patient- and site-specific characteristics were extracted. Multivariable binary logistic regression was employed to compare a range of factors influencing the likelihood of implant discussion and referral including patient-specific (demographics, past medical history, and degree of hearing loss) and site-specific factors (cochlear implant champion and whether the hospital performed implants). Hospitals across all 4 devolved nations of the UK were invited to participate, with data submitted from 36 urban hospitals across England, Scotland, and Wales. Nine hospitals (25%) conducted cochlear implant assessments. The majority of patients lived in England (n = 5,587, 86.2%); the rest lived in Wales (n = 419, 6.5%) and Scotland (n = 233, 3.6%). The mean patient age was 72 ± 19 years (mean ± standard deviation); 54% were male, and 75·3% of participants were white, 6·3% were Asian, 1·5% were black, 0·05% were mixed, and 4·6% were self-defined as a different ethnicity. Of 6,482 submitted patients meeting pure tone audiometric thresholds for cochlear implantation, 311 already had a cochlear implant. Of the remaining 6,171, 35.7% were informed they were eligible for an implant, but only 9.7% were referred for assessment. When adjusted for site- and patient-specific factors, stand-out findings included that adults were less likely to be referred if they lived in more deprived area decile within Indices of Multiple Deprivation (4th (odds ratio (OR): 2·19; 95% confidence interval (CI): [1·31, 3·66]; p = 0·002), 5th (2·02; [1·21, 3·38]; p = 0·05), 6th (2·32; [1·41, 3·83]; p = 0.05), and 8th (2·07; [1·25, 3·42]; p = 0·004)), lived in London (0·40; [0·29, 0·57]; p < 0·001), were male (females 1·52; [1·27, 1·81]; p < 0·001), or were older (0·97; [0·96, 0·97]; p < 0·001). They were less likely to be informed of their potential eligibility if they lived in more deprived areas (4th (1·99; [1·49, 2·66]; p < 0·001), 5th (1·75; [1·31, 2·33], p < 0·001), 6th (1·85; [1·39, 2·45]; p < 0·001), 7th (1·66; [1·25, 2·21]; p < 0·001), and 8th (1·74; [1·31, 2·31]; p < 0·001) deciles), the North of England or London (North 0·74; [0·62, 0·89]; p = 0·001; London 0·44; [0·35, 0·56]; p < 0·001), were of Asian or black ethnic backgrounds compared to white patients (Asian 0·58; [0·43, 0·79]; p < 0·001; black 0·56; [0·34, 0·92]; p = 0·021), were male (females 1·46; [1·31, 1·62]; p < 0·001), or were older (0·98; [0·98, 0·98]; p < 0·001). The study methodology was limited by its observational nature, reliance on accurate documentation of the referring service, and potential underrepresentation of certain demographic groups. CONCLUSIONS: The majority of adults meeting pure tone audiometric threshold criteria for cochlear implantation are currently not appropriately referred for assessment. There is scope to target underrepresented patient groups to improve referral rates. Future research should engage stakeholders to explore the reasons behind the disparities. Implementing straightforward measures, such as educational initiatives and automated pop-up tools for immediate identification, can help streamline the referral process.
Assuntos
Implante Coclear , Implantes Cocleares , Perda Auditiva Neurossensorial , Perda Auditiva , Adulto , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Idoso , Idoso de 80 Anos ou mais , Perda Auditiva Neurossensorial/cirurgia , Perda Auditiva/cirurgia , EscolaridadeRESUMO
OBJECTIVES: Chronic suppurative otitis media (CSOM) is defined as persistent discharge through a tympanic membrane perforation for greater than 2 weeks. It is associated with a significant disease burden, including hearing loss, and reducing its incidence could significantly improve short- and long-term health. We aimed to identify risk factors associated with the development of CSOM in children. DESIGN AND SETTING: Systematic review and meta-analysis of studies set in community, primary and secondary care settings, identified from Medline, Embase and Cochrane databases from 2000 to 2022. PARTICIPANTS: Children 16 years old and below. MAIN OUTCOME MEASURES: Clinical diagnosis of CSOM. RESULTS: In total, 739 papers were screened, with 12 deemed eligible for inclusion in the systematic review, of which, 10 were included in the meta-analysis. Risk factors examined included perinatal, patient, dietary, environmental and parental factors. Meta-analysis results indicate that atopy (RR = 1.18, 95% CI [1.01-1.37], p = .04, 2 studies); and birth weight <2500 g (RR = 1.79 [1.27-2.50], p < .01, 2 studies) are associated with an increased risk of CSOM development. Factors not associated were male sex (RR = 0.96 [0.82-1.13], p = .62, 8 studies); exposure to passive smoking (RR = 1.27 [0.81-2.01], p = .30, 3 studies); and parental history of otitis media (RR = 1.14 [0.59-2.20], p = .69, 2 studies). CONCLUSION: Optimal management of risk factors associated with CSOM development will help reduce the burden of disease and prevent disease progression or recurrence. The current quality of evidence in the literature is variable and heterogeneous. Future studies should aim to use standardised classification systems to define risk factors to allow meta-analysis.
Assuntos
Perda Auditiva , Otite Média Supurativa , Otite Média , Criança , Humanos , Masculino , Adolescente , Feminino , Otite Média Supurativa/complicações , Otite Média Supurativa/epidemiologia , Doença Crônica , Otite Média/complicações , Perda Auditiva/etiologia , Fatores de RiscoRESUMO
OBJECTIVES: Tonsillectomy is the most common operation performed by otolaryngologists in the UK, despite this we have a poor understanding of the post-operative recovery. We aimed to investigate post-operative bleeding and pain following paediatric tonsillectomy using a patient diary. DESIGN: Prospective observational cohort study. SETTING: Multi-centre study involving 12 secondary and tertiary otolaryngology units across the North of England. Patients were recruited from 1st March 2020 to 30th June 2022. Multilevel ordered logistic regression model statistics were performed. PARTICIPANTS: Children (≥4 years, ≤16 years) undergoing tonsillectomy (with or without adenoidectomy) for benign pathology. MAIN OUTCOME MEASURES: Frequency and severity of post-operative bleeding. Intensity and pattern of post-operative pain. RESULTS: In total 297 children were recruited, with 91 (30.6%) diaries eligible for analysis. Post-operative bleeding occurred in 44% of children. Most frequently blood in the saliva was reported (82.9%). Increasing age significantly increased bleeding odds by 17% per year (p = .001). Bleeding frequency decreased with higher surgeon grade (p = .003) and when performing intracapsular coblation tonsillectomy (p = .02) compared with other techniques. Lower age and intracapsular coblation tonsillectomy, against other techniques, significantly reduced rates of pain post-operatively (p < .0001 and p = .0008). CONCLUSION: A high level of low-level post-operative bleeding was observed. Pain scores remained high for 5 days post-operatively then gradually reduce to normal by day 13. Intracapsular coblation tonsillectomy appears to be superior to all other techniques in terms of reducing post-operative bleeding and pain. These findings should be used to guide patients in the consent process to inform them of the expected nature of post-surgical recovery.
Assuntos
Tonsilectomia , Criança , Humanos , Tonsilectomia/efeitos adversos , Tonsilectomia/métodos , Estudos de Coortes , Estudos Prospectivos , Adenoidectomia/efeitos adversos , Adenoidectomia/métodos , Hemorragia Pós-Operatória/epidemiologia , Hemorragia Pós-Operatória/etiologia , Dor Pós-Operatória/epidemiologia , Dor Pós-Operatória/etiologiaRESUMO
OBJECTIVES: Our primary aim was to validate the Liverpool Peritonsillar abscess Score (LPS) externally in a new patient cohort. Our secondary aim was to modify the LPS in the light of the COVID-19 pandemic to produce a no-examination variant for use in this instance. DESIGN: Prospective multicentre external validation study. SETTING: Six different secondary care institutions across the United Kingdom. PARTICIPANTS: Patients over 16 years old who were referred to ENT with any uncomplicated sore throat such a tonsillitis or peritonsillar abscess (PTA). MAIN OUTCOME MEASURES: Sensitivity, specificity, positive predictive value and negative predictive value for both the original LPS model and the modified model for COVID-19. RESULTS: The LPS model had sensitivity and specificity calculated at 98% and 79%, respectively. The LPS has a high negative predictive value (NPV) of 99%. The positive predictive value (PPV) was slightly lower at 63%. Receiver operating characteristic (ROC) curve, including the area under the curve (AUROC), was 0.888 which indicates very good accuracy. CONCLUSIONS: External validation of the LPS against an independent geographically diverse population yields high NPV. This may support non-specialist colleagues who may have concerns about mis-diagnosing a PTA. The COVID-19 modification of the LPS has a similar NPV, which may be of use where routine oral examination is to be avoided during the COVID-19 pandemic.
Assuntos
COVID-19/epidemiologia , Gerenciamento Clínico , Pandemias , Abscesso Peritonsilar/diagnóstico , SARS-CoV-2 , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Comorbidade , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Abscesso Peritonsilar/epidemiologia , Estudos Prospectivos , Curva ROC , Reino Unido/epidemiologia , Adulto JovemRESUMO
BACKGROUND: Acute otitis media with discharge (AOMd) results from a tympanic membrane perforation secondary to a middle ear infection. Currently, the impact of AOMd on children and young people (CYP) and their families is not well understood. There is also a need to explore the experience of healthcare professionals in treating AOMd. Interviews with CYP and their parents, and focus groups with medical professionals, were conducted to explore these objectives. METHODS: A total of 26 parents of CYP (age range: 7 months to 15 years) with a history of AOMd (within the last year) and 28 medical professionals were recruited across the UK between August 2023 and March 2024. Healthcare professionals were from primary care (n=17), ear, nose and throat (ENT) (n=7) and emergency medicine (n=4) backgrounds. Thematic analysis was performed independently by three reviewers. RESULTS: The majority of CYP (n=25/26) (96.2%) had suffered from multiple episodes of AOMd. AOMd has a physical, psychological, educational, financial and social impact on CYP and their parents. Parents found accessing healthcare services and information difficult, which increased parental anxiety. Antibiotic overuse was also a concern among parents. The majority of general practitioners and emergency care staff described using oral amoxicillin, compared with ENT doctors who predominantly prescribed topical antibiotics. CONCLUSIONS: AOMd has a significant impact on CYP and their parent's daily lives. Need for clear, easily accessible patient information was identified as a priority by the parents of CYP with AOMd. Evidence-based management guidelines should be developed once high-quality evidence is available. TRIAL REGISTRATION NUMBER: ISCTRN43760.
Assuntos
Pais , Pesquisa Qualitativa , Humanos , Criança , Pré-Escolar , Masculino , Feminino , Reino Unido/epidemiologia , Adolescente , Lactente , Pais/psicologia , Doença Aguda , Efeitos Psicossociais da Doença , Antibacterianos/uso terapêutico , Otite Média/tratamento farmacológico , Grupos Focais , Otite Média com DerrameRESUMO
INTRODUCTION: Paediatric otorrhoea (PO) describes a middle ear infection that results in a perforation of the tympanic membrane and ear discharge, in children and young people (CYP). Prolonged infection may be associated with hearing loss and developmental delay. The current management of paediatric otorrhoea is variable, including non-invasive treatments (conservative, oral antibiotics, topical antibiotics) and surgery, reflecting the lack of a sufficiently strong evidence base. Outcome reporting is fundamental to producing reliable and meaningful evidence to inform best practice. OBJECTIVES: Primary objective: to determine which outcome measures are currently used to evaluate treatment success in studies of non-surgical treatments for paediatric otorrhoea. SECONDARY OBJECTIVES: to identify outcome measurement instruments used in the literature and assess their applicability for use in clinical trials of PO. METHODS: This systematic review was registered with PROSPERO (CRD42023407976). Database searches of EMBASE, MEDLINE and Cochrane was performed on June 6, 2023, covering from Jan 1995 to May 2023. Randomised controlled trials or study protocols involving CYP with PO were included following PRISMA guidelines. Risk of bias was assessed with Cochrane's tool. RESULTS: Of the 377 papers identified, six were included in the systematic review. The primary outcome of five of the studies related to otorrhoea cessation; both time to cessation and proportion recovered at various time points were used as measures. Two measurement instruments were identified: Otitis Media-6 Questionnaire and the Institute for Medical Technology Assessment Productivity Cost Questionnaire. Both were shown to be applicable measurement instruments when used in clinical trials of PO. CONCLUSIONS: To promote homogeneity and facilitate meaningful comparison and combination of studies, we propose that time to cessation of otorrhoea from onset of otorrhoea should be used as the primary outcome in future studies. Further research is needed to establish if this is the most important outcome to children and their caregivers.
Assuntos
Surdez , Otopatias , Otite Média , Criança , Humanos , Adolescente , Otite Média/tratamento farmacológico , Antibacterianos/uso terapêutico , Otopatias/tratamento farmacológico , Resultado do TratamentoRESUMO
Background Paediatric otorrhoea (PO) is a symptom-based diagnosis encompassing acute and chronic ear infections which cause otorrhoea in children and young people (CYP). Aim To understand the burden of PO on primary care services. Design and Setting A longitudinal population study in UK primary care. Methods Data from the Clinical Practice Research Datalink (CPRD Aurum), January 2005 to December 2019, was analysed. CYP under 17 years of age with otorrhoea were included. Standardised annual incidence and presentation rates were estimated. Poisson regression modelling was used to determine risk ratios comparing sex, age and IMD. A probabilistic simulation scaled-up estimates for the UK population. Results The cohort included 6,605,193 CYP, observed over 32,942,594 person-years. There were 80,454 incident cases and 106,318 presentations of PO during the 15-year period, equating to standardised annual incidence and presentation rates per 1000 patient-years of 2.42 (95% CI: 2.40-2.44) and 3.15 (3.13-3.17) respectively. In the UK this equates to 41,141 primary care appointments per year. Incidence was higher in males, those aged 0-2 years, and those living in the least deprived quintile. Treatment involved oral antibiotics (57.1%), no prescription (28.1%), topical antibiotics (9.7%), or combination (4.9%). The cost to NHS primary care is estimated at £1.97 million per year. Conclusions This is the first longitudinal population-based study investigating PO which demonstrates the burden on primary care. Antimicrobial prescribing predominantly follows NICE guidelines using oral amoxicillin. Aminoglycosides are the most frequently prescribed topical antibiotic despite the concern of ototoxicity.
RESUMO
INTRODUCTION: Paediatric otorrhoea (PO) refers to the leakage of fluid through a perforation in the ear drum, resulting from an infection of the middle ear of a child or young person (CYP). PO frequently results in hearing loss which may lead to developmental delay, restricted communication and reduced educational attainment.Epidemiological information for PO is largely derived from low-income countries. The aim of this study will be to establish the incidence of PO within the UK and to understand the impact of PO on CYP and their families' everyday lives. It will build the foundations for a randomised controlled trial investigating the best antibiotic treatment for PO. METHODS AND ANALYSIS: The study will consist of two work packages. (1) Data from the Clinical Practice Research Datalink (CPRD), January 2005 to July 2021, will be used to determine the incidence of patient presentations with PO to primary care in the UK. It will also explore the current antimicrobial prescribing practice for PO in primary care. (2) Thirty semi-structured interviews will be conducted from 13 July to 31 October 2023 with CYP and their parents/carers to help identify the impact of PO on everyday life, the patient journey and how service users define treatment success. Three medical professional focus groups will be used to understand the current management practice, how treatment success is measured and acceptability to randomise patients. Thematic analysis will be used. ETHICS AND DISSEMINATION: The Health Research Authority, The Health and Social Care Research Ethics Committee (23/NI/0082) and the CPRD's research data governance panel (22_002508) reviewed this study. Results will be disseminated at medical conferences, in peer-reviewed journals and via social media. The study will cocreate a webpage on healthtalk.org, with the Dipex Charity, about PO to ensure members of the public can learn more about the condition. TRIAL REGISTRATION NUMBER: ISRCTN46071200.
Assuntos
Sucesso Acadêmico , Surdez , Humanos , Criança , Adolescente , Instituições de Caridade , Comunicação , Reino Unido , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
OBJECTIVE: Evidence for the management of acute otitis externa (AOE) is limited, with unclear diagnostic criteria and variably reported outcome measures that may not reflect key stakeholder priorities. We aimed to develop 1) a definition, 2) diagnostic criteria and 3) a core outcome set (COS) for AOE. STUDY DESIGN: COS development according to Core Outcome Measures in Effectiveness Trials (COMET) methodology and parallel consensus selection of diagnostic criteria/definition. SETTING: Stakeholders from the United Kingdom. SUBJECTS AND METHODS: Comprehensive literature review identified candidate items for the COS, definition and diagnostic criteria. Nine individuals with past AOE generated further patient-centred candidate items. Candidate items were rated for importance by patient and professional (ENT doctors, general practitioners, microbiologists, nurses, audiologists) stakeholders in a three-round online Delphi exercise. Consensus items were grouped to form the COS, diagnostic criteria, and definition. RESULTS: Candidate COS items from patients (n = 28) and literature (n = 25) were deduplicated and amalgamated to a final candidate list (n = 46). Patients emphasised quality-of-life and the impact on daily activities/work. Via the Delphi process, stakeholders agreed on 31 candidate items. The final COS covered six outcomes: pain; disease severity; impact on quality-of-life and daily activities; patient satisfaction; treatment-related outcome; and microbiology. 14 candidate diagnostic criteria were identified, 8 reaching inclusion consensus. The final definition for AOE was 'diffuse inflammation of the ear canal skin of less than 6 weeks duration'. CONCLUSION: The development and adoption of a consensus definition, diagnostic criteria and a COS will help to standardise future research in AOE, facilitating meta-analysis. Consulting former patients throughout development highlighted deficiencies in the outcomes adopted previously, in particular concerning the impact of AOE on daily life.
Assuntos
Orelha Externa/patologia , Otite Externa/diagnóstico , Otite Externa/patologia , Dor/diagnóstico , Atividades Cotidianas , Técnica Delphi , Humanos , Otite Externa/terapia , Avaliação de Resultados em Cuidados de Saúde , Qualidade de Vida , Resultado do TratamentoRESUMO
OBJECTIVES: The coronavirus outbreak has triggered the implementation of nationwide social distancing measures. We aimed to investigate the impact on patients with recurrent tonsillitis and parental perceptions towards tonsillectomy during the COVID-19 pandemic. METHODS: A telephone questionnaire was conducted for all children awaiting tonsillectomy for recurrent tonsillitis after social distancing for 2 months at our centre. The COVID-19 lockdown period was compared with the 2 months prior to lockdown. RESULTS: Forty-four children had been social distancing at home during lockdown. There was a significant reduction in tonsillitis episodes during the 2-month lockdown period in comparison with 2 months prior to lockdown (p = 0.0001). In 70% (n = 31) of cases parents wanted their child's tonsillectomy during the coronavirus outbreak. CONCLUSION: These findings demonstrate that viral exposure is a key factor in the pathophysiology of recurrent tonsillitis and that social distancing measures can reduce the frequency of recurrent tonsillitis. Despite the overall reduction in tonsillitis frequency during the lockdown period, the majority of parents wanted their child's tonsillectomy during the coronavirus outbreak. This demonstrates the impact tonsillitis has on the patient and their family's quality of life.
Assuntos
Atitude Frente a Saúde , COVID-19 , Pais/psicologia , Distanciamento Físico , Tonsilectomia/psicologia , Tonsilite/cirurgia , Adolescente , COVID-19/epidemiologia , COVID-19/prevenção & controle , COVID-19/psicologia , Criança , Pré-Escolar , Estudos Transversais , Feminino , Pesquisas sobre Atenção à Saúde , Humanos , Lactente , Masculino , Pandemias , Percepção , Fatores de Proteção , Qualidade de Vida , Recidiva , Estudos Retrospectivos , Fatores de Risco , Tonsilite/etiologia , Tonsilite/prevenção & controle , Reino Unido/epidemiologiaRESUMO
Background Recent evidence surrounding the use of venovenous extracorporeal membrane oxygenation in treating acute respiratory failure has led to the expansion of extracorporeal membrane oxygenation services worldwide. The high rate of complications related to venovenous extracorporeal membrane oxygenation often requires intervention by specialist thoracic surgeons. This study aimed to investigate the role of specialist thoracic surgeons within the multidisciplinary team managing these high-risk patients. Methods We retrospectively reviewed 90 patients who received venovenous extracorporeal membrane oxygenation at our tertiary referral center between December 2011 and May 2015. Four patients who underwent lung transplantation were excluded. Results We found that 29.1% (25/86) of patients on venovenous extracorporeal membrane oxygenation had undergone a thoracic intervention. A total of 82 interventions were performed: 11 thoracotomies, 49 chest drains, 13 rigid bronchoscopies, 4 flexible bronchoscopies, 4 temporary endobronchial blockers, and 1 sternotomy. Of the 11 thoracotomies, 3 were reexplorations. Survival to discharge for patients who underwent thoracic surgical interventions was 72% (18/25). Conclusions Our experience has demonstrated that a large proportion of patients receiving venovenous extracorporeal membrane oxygenation require a thoracic intervention, many of which are major intraoperative procedures. Patients on venovenous extracorporeal membrane oxygenation have benefited from rapid on-site access to thoracic surgical services to manage these challenging life-threatening complications.
Assuntos
Oxigenação por Membrana Extracorpórea/efeitos adversos , Complicações Pós-Operatórias/cirurgia , Insuficiência Respiratória/cirurgia , Procedimentos Cirúrgicos Torácicos , Doença Aguda , Adolescente , Adulto , Idoso , Oxigenação por Membrana Extracorpórea/mortalidade , Feminino , Mortalidade Hospitalar , Humanos , Masculino , Pessoa de Meia-Idade , Equipe de Assistência ao Paciente , Papel do Médico , Complicações Pós-Operatórias/etiologia , Complicações Pós-Operatórias/mortalidade , Insuficiência Respiratória/diagnóstico , Insuficiência Respiratória/mortalidade , Insuficiência Respiratória/fisiopatologia , Estudos Retrospectivos , Fatores de Risco , Especialização , Cirurgiões , Centros de Atenção Terciária , Procedimentos Cirúrgicos Torácicos/efeitos adversos , Procedimentos Cirúrgicos Torácicos/mortalidade , Fatores de Tempo , Resultado do Tratamento , Adulto JovemRESUMO
Advances in extracorporeal membrane oxygenation have enabled the facilitation of surgical intervention in patients who may otherwise be considered inoperable using conventional ventilation-for example, the resection of an early stage metachronous lung cancer in a patient who has previously undergone a pneumonectomy for a prior lung cancer. We present one such case: a 52-year-old man who underwent right lower lobe segmentectomy for a second primary lung cancer, 3 years after left pneumonectomy, using extracorporeal membrane oxygenation. To our knowledge, there are no published case reports describing the use of extracorporeal membrane oxygenation in resection of metachronous lung cancer after pneumonectomy.
Assuntos
Carcinoma de Células Escamosas/cirurgia , Oxigenação por Membrana Extracorpórea/métodos , Neoplasias Pulmonares/cirurgia , Segunda Neoplasia Primária/diagnóstico por imagem , Segunda Neoplasia Primária/terapia , Pneumonectomia/métodos , Biópsia por Agulha , Broncoscopia/métodos , Carcinoma de Células Escamosas/patologia , Terapia Combinada , Seguimentos , Humanos , Imuno-Histoquímica , Neoplasias Pulmonares/patologia , Masculino , Pessoa de Meia-Idade , Segunda Neoplasia Primária/patologia , Medição de Risco , Tomografia Computadorizada por Raios X/métodos , Resultado do TratamentoRESUMO
BACKGROUND: Patients with pulmonary arterial hypertension (PAH) are managed according to evidence-based treatment guidelines. METHODS AND RESULTS: In this single-centre retrospective analysis, we examined outcomes of patients with PAH caused by congenital heart disease (PAH-CHD) with respect to exercise capacity and survival of adults treated with either bosentan or sildenafil monotherapy or bosentan-sildenafil dual therapy between January 2007 and January 2014. Of the 82 patients analysed, 29 had Down syndrome; 54 (65.8%) received bosentan monotherapy, 16 (19.5%) sildenafil monotherapy and 12 (14.6%) dual therapy. Mean treatment duration was 2.5â years for all patients and 4.1â years for 38 patients treated for ≥2â years. Pooled patient and treatment data showed initial improvement followed by stabilisation in mean 6â min walk distance (6MWD). For Down and non-Down patients, mean 6MWD increased and then stabilised on bosentan monotherapy. Mean 6MWD of patients on dual therapy at the time of analysis was 246.3â m before PAH-specific therapy initiation, 211.9â m immediately prior to addition of a second therapy and 214.4â m at last visit while on dual therapy. 1, 2 and 3-year survival rates for all patients from time of treatment initiation were 96%, 87% and 80%, respectively. CONCLUSIONS: For the majority of patients, monotherapy with a PAH-specific medication provided improved and sustained exercise benefits. For the small percentage of patients who required it, add-on therapy appeared to prevent further deterioration in exercise capacity but did not improve 6MWD.