Adolescent and Parent Preferences for Hypodontia: Discrete Choice Experiment.

Choosing hypodontia treatment requires young people and parents to consider a large amount of information, identify what is important to them, and make trade-offs between options. This study aimed to examine young people and parents' preferences for hypodontia treatment using discrete choice experiment (DCE). This was a cross-sectional survey of young people (12-16 y) with hypodontia of any severity, at any stage of treatment, and their parents. Participants were recruited from NHS Hospitals in England and Wales. A bespoke DCE questionnaire was developed to measure preferences for 6 attributes of hypodontia treatment (waiting time, treatment time, problems during treatment, discomfort during treatment, bite, appearance). The questionnaire was completed 1) online by young people and parents, individually or together, and 2) by child-parent dyads under observation. Preferences were analyzed using regression models. In total, 204 participants (122 young people, 56 parents, 26 dyads) completed the online questionnaire and 15 child-parent dyads completed the questionnaire under observation. The most important attribute in hypodontia treatment was improvement in appearance, but significant heterogeneity was found in preferences. Four distinct groups of participants were found: group 1 (39%): severe discomfort and problems were most important; group 2 (31%): most concerned about improvement in appearance of teeth and improvement in bite; group 3 (22%): appearance 3 times more important than any other attribute; and group 4 (9%): preferences difficult to interpret. There was variation in how child-parent dyads approached decision-making, with some negotiating joint preferences, while for others, one individual dominated. Making trade-offs in DCE tasks helped some people think about treatment and identify their preferences. Appearance is an important outcome from hypodontia treatment, but preferences vary and potential risks and functional outcome are also important to some people. There is a notable level of uncertainty in decision-making, which suggests further shared decision support would be valuable.

Patient perspective on decisions to switch disease-modifying treatments in relapsing-remitting multiple sclerosis.

BACKGROUND: There are now large cohorts of people with relapsing-remitting multiple sclerosis (pwRRMS) who have taken several Disease-Modifying Treatments (DMTs). Studies about switching DMTs mostly focus on clinical outcomes rather than patients' decision-making. Neurologists are now required to support decisions at various times during the relapsing disease course and they do so with concerns about DMTs risks. This qualitative study investigates how pwRRMS weigh up the pros and cons of DMTs, focusing on perceptions of effectiveness and risks when new treatments are considered. OBJECTIVE: To increase understanding of people's experiences of decision-making when switching DMTs. METHODS: 30 semi-structured interviews were conducted with pwRRMS in England. 16 participants had switched DMT and their experiences were compared with those who had only taken one DMT. Interviews were analysed thematically to answer: what main factors influence people's decision-making to switch DMTs and why? RESULTS: Of the 16 participants with experience of switching DMT, eight had taken two or more DMTs; eight had taken three or more. Two was the DMT median. This study demonstrated that despite the term "switching" implying that similar treatments are inter-changeable, for pwRRMS taking new treatments involves different emotions, routines, risks, prognosis and communication experiences. Two meta themes identified were: 1) A distinctive, rapid and emotional decision-making process where old emotions related to MS prognosis are revisited. 2) Switching has a different impact on communication for escalation or de-escalation processes. CONCLUSION: Switching DMT involves different routines, risks, prognosis and communication experiences. These decisions are emotionally difficult because of the fear about transitioning to secondary progressive MS, and DMT effectiveness uncertainty. Patient centred decision aids should include information about first and consecutive treatment decisions.

Laparoscopic versus open colorectal surgery in the acute setting (LaCeS trial): a multicentre randomized feasibility trial.

BACKGROUND: Approximately 30 000 people undergo major emergency abdominal gastrointestinal surgery annually, and 36 per cent of these procedures (around 10 800) are carried out for emergency colorectal pathology. Some 14 per cent of all patients requiring emergency surgery have a laparoscopic procedure. The aims of the LaCeS (laparoscopic versus open colorectal surgery in the acute setting) feasibility trial were to assess the feasibility, safety and acceptability of performing a large-scale definitive phase III RCT, with a comparison of emergency laparoscopic versus open surgery for acute colorectal pathology. METHODS: LaCeS was designed as a prospective, multicentre, single-blind, parallel-group, pragmatic feasibility RCT with an integrated qualitative study. Randomization was undertaken centrally, with patients randomized on a 1 : 1 basis between laparoscopic or open surgery. RESULTS: A total of 64 patients were recruited across five centres. The overall mean steady-state recruitment rate was 1·2 patients per month per site. Baseline compliance for clinical and health-related quality-of-life data was 99·8 and 93·8 per cent respectively. The conversion rate from laparoscopic to open surgery was 39 (95 per cent c.i. 23 to 58) per cent. The 30-day postoperative complication rate was 27 (13 to 46) per cent in the laparoscopic arm and 42 (25 to 61) per cent in the open arm. CONCLUSION: Laparoscopic emergency colorectal surgery may have an acceptable safety profile. Registration number: ISRCTN15681041 ( http://www.controlled-trials.com).

ANTECEDENTES: Aproximadamente 30.000 personas se someten cada año una operación de cirugía mayor urgente gastrointestinal de las cuales el 36% (~ 10.800) se realizan por patología colorrectal urgente. Aproximadamente el 14% de todos los pacientes que requieren cirugía urgente son operados mediante abordaje laparoscópico. Los objetivos del ensayo de factibilidad LaCeS (Laparoscopic versus Open Colorectal Surgery in the Acute Setting; Cirugía Colorrectal Laparoscópica versus Abierta en Urgencias) fueron evaluar la factibilidad, seguridad y aceptabilidad de realizar un ensayo clínico aleatorizado definitivo a gran escala de fase III comparando la cirugía colorrectal urgente por vía laparoscópica con el abordaje abierto. MÉTODOS: LaCeS se diseñó como un ensayo clínico prospectivo, multicéntrico, simple ciego, de grupos paralelos, pragmático, aleatorizado (factibilidad) con un estudio cualitativo integrado. La asignación al azar se realizó de forma centralizada y los pacientes se asignaron al azar en proporción 1:1 a cirugía laparoscópica o abierta. RESULTADOS: Un total de 64 pacientes fueron reclutados en 5 centros. La tasa media global estable de reclutamiento fue de 1,2 pacientes/mes. El cumplimiento inicial de los datos clínicos y de calidad de vida (HRQoL) fue del 99,8% y del 93,8%, respectivamente. La tasa de conversión de la cirugía laparoscópica a cirugía abierta fue del 39,4% (i.c. del 95%: 22,9% a 57,9%). La tasa de complicaciones postoperatorias a los 30 días fue del 27,3% (i.c. del 95%: 13,3-45,5) para la cirugía laparoscópica y del 41,9% (i.c. del 95%: 24,6-60,9) para la cirugía abierta. CONCLUSIÓN: La cirugía colorrectal urgente por vía laparoscópica puede tener un perfil de seguridad aceptable.

Eliciting Preferences in Dentistry with Multiattribute Stated Preference Methods: A Systematic Review.

BACKGROUND: Preference experiments are used to understand how patients and stakeholders value aspects of health care. These methods are gaining popularity in dentistry, but quality and breadth of use have not been evaluated. OBJECTIVES: To describe multiattribute stated preference experiment use in dentistry through illustration and critique of existing studies. DATA SOURCES: Systematic literature search of PubMed, Econlit and Ovid for Medline, Embase, PsychINFO, PsychARTICLES, and All EBM Reviews, as well as gray literature. STUDY ELIGIBILITY: Multiattribute stated preference experiments eliciting preferences for dental service delivery, treatments, and oral health states from the perspective of patients, the public, and dental professionals. Outcomes of interest were preference weights and marginal rates of substitution. Study selection was independently performed by 2 reviewers. APPRAISAL: Ten-point checklist published by the International Society of Pharmacoeconomics and Outcomes Research was used for quality assessment. SYNTHESIS: Descriptive analysis. RESULTS: Searches identified 12 records published between 1999 and 2015, mostly in nondental academic journals. Studies were undertaken in high-income countries in Europe and the United States. The studies aimed to elicit preference for service delivery, treatment, or oral health states from the perspective of the patients, dentists, or the public via discrete choice experiment methods. The quality scores for the studies ranged from 53% to 100%. LIMITATIONS: A detailed description and critique of stated preference methods are provided, but it was not possible to provide synthesized preference data. CONCLUSIONS: Multiattribute stated preference experiments are increasingly popular, but understanding the methods and outputs is essential for designing and interpreting preference studies to improve patient care. Patient preferences highlight important considerations for decision making during treatment planning. Valuation of health states and estimation of willingness-to-pay are important for resource planning and allocation and economic evaluation. Preference estimates and relative value of attributes for interventions and service delivery inform development and selection of treatments and services (PROSPERO 21.3.17: CRD42017059859). KNOWLEDGE TRANSFER STATEMENT: Understanding patient, professional, and public preferences is fundamental for evidence-based decision making and treatment delivery. Preference elicitation methods can be used to estimate the value given to health states, service delivery, individual treatments, and health outcomes. By describing and appraising the methodology and application of multiattribute stated preference experiments in dentistry, this review provides an essential first step to wider use of well-designed, high-quality preference elicitation methods.

Cost-effectiveness of outpatient parenteral antibiotic therapy: a simulation modelling approach.

Objectives: In the UK, patients who require intravenous antimicrobial (IVA) treatment may receive this in the community through outpatient parenteral antimicrobial therapy (OPAT) services. Services include: IVA administration at a hospital outpatient clinic (HO); IVA administration at home by a general nurse (GN) or a specialist nurse (SN); or patient self-administered (SA) IVA administration following training. There is uncertainty regarding which OPAT services represent value for money; this study aimed to estimate their cost-effectiveness. Methods: A cost-effectiveness decision-analytic model was developed using a simulation technique utilizing data from hospital records and a systematic review of the literature. The model estimates cost per QALY gained from the National Health Service (NHS) perspective for short- and long-term treatment of infections and service combinations across these. Results: In short-term treatments, HO was estimated as the most effective (0.7239 QALYs), but at the highest cost (£973). SN was the least costly (£710), producing 0.7228 QALYs. The combination between SN and HO was estimated to produce 0.7235 QALYs at a cost of £841. For long-term treatments, SN was the most effective (0.677 QALYs), costing £2379, while SA was the least costly at £1883, producing 0.666 QALYs. A combination of SA and SN was estimated to produce 0.672 QALYs at a cost of £2128. Conclusions: SN and SA are cost-effective for short- and long-term treatment of infections, while combining services may represent the second-best alternative for OPAT in the UK.

Clinical and cost-effectiveness, safety and acceptability of community intravenous antibiotic service models: CIVAS systematic review.

OBJECTIVE: Evaluate evidence of the efficacy, safety, acceptability and cost-effectiveness of outpatient parenteral antimicrobial therapy (OPAT) models. DESIGN: A systematic review. DATA SOURCES: MEDLINE, EMBASE, CINAHL, Cochrane Library, National Health Service (NHS) Economic Evaluation Database (EED), Research Papers in Economics (RePEc), Tufts Cost-Effectiveness Analysis (CEA) Registry, Health Business Elite, Health Information Management Consortium (HMIC), Web of Science Proceedings, International Pharmaceutical Abstracts, British Society for Antimicrobial Chemotherapy website. Searches were undertaken from 1993 to 2015. STUDY SELECTION: All studies, except case reports, considering adult patients or practitioners involved in the delivery of OPAT were included. Studies combining outcomes for adults and children or non-intravenous (IV) and IV antibiotic groups were excluded, as were those focused on process of delivery or clinical effectiveness of 1 antibiotic over another. Titles/abstracts were screened by 1 reviewer (20% verified). 2 authors independently screened studies for inclusion. RESULTS: 128 studies involving >60 000 OPAT episodes were included. 22 studies (17%) did not indicate the OPAT model used; only 29 involved a comparator (23%). There was little difference in duration of OPAT treatment compared with inpatient therapy, and overall OPAT appeared to produce superior cure/improvement rates. However, when models were considered individually, outpatient delivery appeared to be less effective, and self-administration and specialist nurse delivery more effective. Drug side effects, deaths and hospital readmissions were similar to those for inpatient treatment, but there were more line-related complications. Patient satisfaction was high, with advantages seen in being able to resume daily activities and having greater freedom and control. However, most professionals perceived challenges in providing OPAT. CONCLUSIONS: There were no systematic differences related to the impact of OPAT on treatment duration or adverse events. However, evidence of its clinical benefit compared with traditional inpatient treatment is lacking, primarily due to the dearth of good quality comparative studies. There was high patient satisfaction with OPAT use but the few studies considering practitioner acceptability highlighted organisational and logistic barriers to its delivery.

Shaping dental contract reform: a clinical and cost-effective analysis of incentive-driven commissioning for improved oral health in primary dental care.

OBJECTIVE: To evaluate the clinical and cost-effectiveness of a new blended dental contract incentivising improved oral health compared with a traditional dental contract based on units of dental activity (UDAs). DESIGN: Non-randomised controlled study. SETTING: Six UK primary care dental practices, three working under a new blended dental contract; three matched practices under a traditional contract. PARTICIPANTS: 550 new adult patients. INTERVENTIONS: A new blended/incentive-driven primary care dentistry contract and service delivery model versus the traditional contract based on UDAs. MAIN OUTCOME MEASURES: Primary outcome was as follows: percentage of sites with gingival bleeding on probing. Secondary outcomes were as follows: extracted and filled teeth (%), caries (International Caries Detection and Assessment System (ICDAS)), oral health-related quality of life (Oral Health Impact Profile-14 (OHIP-14)). Incremental cost-effective ratios used OHIP-14 and quality adjusted life years (QALYs) derived from the EQ-5D-3L. RESULTS: At 24 months, 291/550 (53%) patients returned for final assessment; those lost to follow-up attended 6.46 appointments on average (SD 4.80). The primary outcome favoured patients in the blended contract group. Extractions and fillings were more frequent in this group. Blended contracts were financially attractive for the dental provider but carried a higher cost for the service commissioner. Differences in generic health-related quality of life were negligible. Positive changes over time in oral health-related quality of life in both groups were statistically significant. CONCLUSIONS: This is the first UK study to assess the clinical and cost-effectiveness of a blended contract in primary care dentistry. Although the primary outcome favoured the blended contract, the results are limited because 47% patients did not attend at 24 months. This is consistent with 39% of adults not being regular attenders and 27% only visiting their dentist when they have a problem. Promotion of appropriate attendance, especially among those with high need, necessitates being factored into recruitment strategies of future studies.

Community IntraVenous Antibiotic Study (CIVAS): protocol for an evaluation of patient preferences for and cost-effectiveness of community intravenous antibiotic services.

INTRODUCTION: Outpatient parenteral antimicrobial therapy (OPAT) is used to treat a wide range of infections, and is common practice in countries such as the USA and Australia. In the UK, national guidelines (standards of care) for OPAT services have been developed to act as a benchmark for clinical monitoring and quality. However, the availability of OPAT services in the UK is still patchy and until quite recently was available only in specialist centres. Over time, National Health Service (NHS) Trusts have developed OPAT services in response to local needs, which has resulted in different service configurations and models of care. However, there has been no robust examination comparing the cost-effectiveness of each service type, or any systematic examination of patient preferences for services on which to base any business case decision. METHODS AND ANALYSIS: The study will use a mixed methods approach, to evaluate patient preferences for and the cost-effectiveness of OPAT service models. The study includes seven NHS Trusts located in four counties. There are five inter-related work packages: a systematic review of the published research on the safety, efficacy and cost-effectiveness of intravenous antibiotic delivery services; a qualitative study to explore existing OPAT services and perceived barriers to future development; an economic model to estimate the comparative value of four different community intravenous antibiotic services; a discrete choice experiment to assess patient preferences for services, and an expert panel to agree which service models may constitute the optimal service model(s) of community intravenous antibiotics delivery. ETHICS AND DISSEMINATION: The study has been approved by the NRES Committee, South West-Frenchay using the Proportionate Review Service (ref 13/SW/0060). The results of the study will be disseminated at national and international conferences, and in international journals.

Costs of cancer care for use in economic evaluation: a UK analysis of patient-level routine health system data.

BACKGROUND: The rising financial burden of cancer on health-care systems worldwide has led to the increased demand for evidence-based research on which to base reimbursement decisions. Economic evaluations are an integral component of this necessary research. Ascertainment of reliable health-care cost and quality-of-life estimates to inform such studies has historically been challenging, but recent advances in informatics in the United Kingdom provide new opportunities. METHODS: The costs of hospital care for breast, colorectal and prostate cancer disease-free survivors were calculated over 15 months from initial diagnosis of cancer using routinely collected data within a UK National Health Service (NHS) Hospital Trust. Costs were linked at patient level to patient-reported outcomes and registry-derived sociodemographic factors. Predictors of cost and the relationship between costs and patient-reported utility were examined. RESULTS: The study population included 223 breast cancer patients, 145 colorectal and 104 prostate cancer patients. The mean 15-month cumulative health-care costs were £12 595 (95% CI £11 517-£13 722), £12 643 (£11 282-£14 102) and £3722 (£3263-£4208), per-patient respectively. The majority of costs occurred within the first 6 months from diagnosis. Clinical stage was the most important predictor of costs for all cancer types. EQ-5D score was predictive of costs in colorectal cancer but not in breast or prostate cancer. CONCLUSION: It is now possible to evaluate health-care cost using routine NHS data sets. Such methods can be utilised in future retrospective and prospective studies to efficiently collect economic data.

The cost-effectiveness of primary care referral to a UK commercial weight loss programme.

Primary care referral to commercial weight loss programmes that follow best practice is included in current UK guidance on the management of adult obesity. This study investigated whether such a programme was cost-effective compared with usual care. A decision-analytical Markov model was developed to estimate the lifetime costs and benefits of the referral programme compared with usual care and enable a cost-utility analysis. The model cohort transited between body mass index classifications and type 2 diabetes, stroke and myocardial infarction (MI) with risk, cost and effect parameter values taken from published literature. The cost per incremental quality-adjusted life year (QALY) was calculated. Extensive deterministic and scenario sensitivity analyses and probabilistic sensitivity analyses (PSA) were conducted. At 12 months, the incremental cost-effectiveness ratio was £6906, indicating that programme referral was cost-effective. Over a lifetime, referral to the commercial programme was dominant as it led to a cost saving of £924 and conferred incremental benefit (0.22 QALY) over usual care. Model simulations estimated lower lifetime rates of type 2 diabetes, stroke and MI as a result of the weight loss achieved. The results were robust to extensive sensitivity analyses. The PSA indicated that programme referral had a 68% chance of being cost-effective at a willingness to pay per incremental QALY threshold of £20,000. Referral to the programme dominated usual care, being both cheaper and more effective. These results compare favourably with economic evaluations of other obesity interventions and add to a growing evidence base on the cost-effectiveness of commercial weight loss providers and practices.

The development and validation of the Unidimensional Fatigue Impact Scale (U-FIS).

BACKGROUND: The multidimensional assessment of fatigue is complicated by the interrelation of its multiple causes and effects. OBJECTIVE: The purpose of the research was to develop a unidimensional assessment of fatigue (U-FIS). METHODS: Data collected with the Fatigue Impact Scale (FIS) were subjected to Rasch analysis to identify potential problems with the scale. Additional items for the U-FIS were generated from interviews with UK MS patients. The U-FIS was tested for face and content validity in patient interviews and included in a validation survey to determine dimensionality (Rasch model), reliability and validity. RESULTS: The original FIS was not unidimensional when subscale items were combined. The modification of the FIS and addition of a number of items allowed the development of a 22-item unidimensional scale (U-FIS) that was reliable (Cronbach Alpha = 0.96; test-retest = 0.86,) and valid given correlations with the Nottingham Health Profile and ability to distinguish between MS severity groups. There was no significant difference in U-FIS scores according to MS type. CONCLUSION: It is valid to conceptualize the functional impact of fatigue as unidimensional. The U-FIS is a reliable and valid questionnaire that will allow the measurement of this construct in clinical studies.

The development of patient-reported outcome indices for multiple sclerosis (PRIMUS).

BACKGROUND: Complex diseases such as multiple sclerosis (MS) present dilemmas over the choice of patient-reported outcome measures as no single scale can inform on all types of MS impact from the patient's perspective. OBJECTIVE: To develop an outcome tool, the Patient-Reported Indices for Multiple Sclerosis (PRIMUS), to assess MS symptoms, activities, and quality of life. METHODS: PRIMUS content was derived from qualitative interviews with UK MS patients and checked by clinical experts. Semi-structured cognitive debriefing interviews assessed scale face and content validity. PRIMUS scaling properties, reliability, and construct validity were assessed by a test-retest postal survey. RESULTS: Cognitive debriefing interviews (n = 15) demonstrated scale clarity, relevance, and comprehensiveness. The postal survey was completed by 135 patients with MS. After removal of misfitting items and those exhibiting differential item functioning, all scales fitted the Rasch model, confirming unidimensionality. For all scales, test-retest reliability exceeded 0.80. Scale scores were related to perceived MS severity, general health, and symptoms of depression. Moderate correlations were observed between PRIMUS and Nottingham Health Profile scores. CONCLUSIONS: Clinicians and researchers can have confidence in scores obtained by respondents on the PRIMUS. The PRIMUS will aid the assessment of the impact of MS from the patient's perspective.

The responsiveness and validity of the CAMPHOR Utility Index.

The aim of the present study was to validate and determine the minimal important difference (MID) and responsiveness of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) Utility Index, a new tool enabling cost utility analyses. CAMPHOR, 6-min walking test (6MWT) and New York Heart Association (NYHA) data for 869 pulmonary hypertension patients (545 (63%) female; mean+/-SD age 56.6+/-15.4 yrs) from three centres were analysed. Utility was correlated with 6MWT data and calculated by NYHA class to assess validity. Effect sizes were calculated for those with two CAMPHOR assessments. Distribution and anchor-based MIDs were calculated. Analyses were carried out in patients receiving bosentan in order to determine whether or not those remaining in NYHA class III following treatment improved. The Utility Index distinguished between adjacent NYHA classes and correlated with 6MWT results. CAMPHOR subscales and utility were as responsive as the 6MWT (effect sizes ranged 0.31-0.69 for the CAMPHOR and 0.16-0.34 for the 6MWT). The within-group MID for the Utility Index was estimated to be approximately 0.09. Patients remaining in NYHA class III experienced, on average, a significant improvement (CAMPHOR Utility Index and functioning), which exceeded the MID. The CAMPHOR Utility Index is valid and responsive to change. Patients can experience significant and important improvements even if they do not improve on the basis of traditional outcomes, such as NYHA functional class.

Adaptation of the RAQoL for use in Australia.

Quality of life is an important patient-reported outcome of rheumatoid arthritis (RA) in addition to structural and functional outcomes. The RAQoL (Rheumatoid Arthritis Quality of Life questionnaire) was developed in the UK and the Netherlands as a disease-specific tool. It was adapted for use in the Australian social context and the reliability and validity was tested. A lay panel assessed the UK version and adapted the wording for use within Australia. Reliability and validity were assessed by a postal survey of the RAQoL and comparator questionnaires to 100 patients with RA. The RAQoL was easily adapted into Australian-English. Test-retest reliability was high with a Spearman rank correlation coefficient of 0.93. RAQoL scores correlated well with patient-perceived disease activity and severity--indicating good validity. The Australian version of the RAQoL is a valid and reliable tool for the assessment of quality of life. It is practical, easy to administer and has good potential for use in clinical settings and trials in Australia.

The Rheumatoid Arthritis Quality of Life (RAQoL) for Sweden: adaptation and validation.

OBJECTIVE: To produce and evaluate the official Swedish language version of the Rheumatoid Arthritis Quality of Life instrument (RAQoL). METHODS: The UK RAQoL was translated into Swedish by a bilingual translation panel. A separate lay panel was conducted to consider the appropriateness and comprehensibility of the items in Swedish. Interviews were conducted with 15 Swedish RA patients to assess face and content validity. Responses to a postal survey were used to calculate reproducibility and construct validity. RESULTS: Few difficulties arose in the translation process and the new language version was well received by the lay panel and field-test participants. One hundred and sixty-five RA patients participated in the postal survey (69% female; mean age 62.7 years, SD 11.3, RA duration range 1-62 years). Cronbach's alpha for the Swedish RAQoL was 0.91 and test-retest reliability was 0.95, indicating that the instrument has adequate inter-relatedness of items and very low inherent random measurement error. A high correlation with the Health Assessment Questionnaire (HAQ) was observed; this was expected because RA has significant physical manifestations that are in turn a significant determinant of QoL. The Swedish RAQoL was able to distinguish between patients who differed according to their self-perceived RA severity, general health, and rating of their RA today, in addition to whether or not the patient was experiencing a flare of RA. CONCLUSIONS: The official Swedish RAQoL was well received by RA patients. The psychometric quality of the adapted questionnaire means that it is suitable for inclusion in clinical trials involving patients with RA.

The Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR): a measure of health-related quality of life and quality of life for patients with pulmonary hypertension.

OBJECTIVE: No outcome measures specific to pulmonary hypertension (PH) currently exist. The aim of the study was to develop health-related quality of life (symptoms and functioning) scales and a quality of life scale that would allow comprehensive, accurate and valid patient-reported outcome assessment in clinical studies. METHODS: The content of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) was derived from qualitative interviews conducted with 35 patients. Item reduction was based on the analysis of responses to a postal survey (n=75) and patient interviews (n=15) designed to determine face and content validity. A final postal validation study (n=91) was performed to determine reproducibility and construct validity. RESULTS: The questionnaire was well received by participants who found it to be relevant, comprehensible and quick and easy to complete. Rasch and factor analyses were conducted to ensure unidimensionality of the final CAMPHOR scales; Overall symptoms (made up of Energy, Breathlessness and Mood subscales), Functioning and Quality of life. The CAMPHOR scales had good internal consistency (alpha=0.90-0.92) and reproducibility (test-retest correlations=0.86-0.92). They also exhibited convergent, divergent and known groups validity. CONCLUSIONS: The CAMPHOR is a valuable new instrument for assessing patient-reported outcome in PH clinical trials and routine practice.