Development of ATAQ-LAM: a tool to assess quality of life in Lymphangioleiomyomatosis.

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a progressive lung disease that impairs health-related quality of life (HRQL). OBJECTIVE: To develop and conduct initial testing of ATAQ-LAM (A Tool to Assess Quality of Life in LAM). METHODS: A pilot version of the questionnaire was administered to respondents with LAM. We used a deletion algorithm to retain items and then applied multi-trait scaling to place retained items into appropriate domains, thus generating the ATAQ-LAM. Rasch analysis was used to assess item fit to a unidimensional model of HRQL. We determined internal consistency (IC) and floor and ceiling effects of ATAQ-LAM scores and conducted analyses aimed at supporting the validity of ATAQ-LAM. RESULTS: Sixty-nine LAM patients provided response data. Thirty-two items survived the deletion algorithm. Scaling suggested ATAQ-LAM should have a four-domain structure (Exertional dyspnea, IC = 0.94; Cough, IC = 0.91; Fatigue, IC = 0.91; Emotional Well-Being, IC = 0.89). All items fit the Rasch model. Among 17 respondents with spirometry within three months of questionnaire completion, three of five ATAQ-LAM scores correlated with FEV1% (Exertional Dyspnea: r = -0.72, p = 0.001; Fatigue: r = -0.62, p = 0.007 and total: r = -0.53, p = 0.02). Compared with those in the highest tertile of FEV1%, subjects in the lowest tertile had greater ATAQ-LAM total (121.8 ± 14.3 vs. 79.8 ± 13.1, p = 0.04), Exertional Dyspnea (54.4 ± 6.3 vs. 25.5 ± 5.8, p = 0.005) and Fatigue (2.8 ± 2.4 vs. 14.8 ± 2.3, p = 0.03) scores, indicating greater impairment in HRQL. CONCLUSIONS: ATAQ-LAM is a disease-specific instrument designed to assess HRQL in LAM patients. Additional studies are needed to generate data in support of its validity as an instrument capable of assessing HRQL over time in LAM patients.

Physical functional capacity in idiopathic pulmonary fibrosis: performance characteristics of the continuous-scale physical function performance test.

BACKGROUND: Physical functional capacity is impaired in idiopathic pulmonary fibrosis (IPF). There is no tool to measure this key clinical outcome. The continuous-scale physical function performance (CS-PFP) test is one that assesses activities of daily living, but it has never been used in IPF. METHODS: We determined internal consistency of the CS-PFP. We used correlations to assess the strength of association between CS-PFP scores and various parameters of IPF severity, and compared the CS-PFP scores between patients with IPF and published values from a healthy control group. RESULTS: Sixteen subjects completed the test and retest. Test-retest reliability (0.84, p = 0.003) and internal consistency (Cronbach's α = 0.91) were excellent. Subjects with IPF had significantly worse CS-PFP scores than controls (46.0 ± 11.1 vs 58.7 ± 12.5, p = 0.001). In IPF, the CS-PFP scores correlated moderately to very strongly with several disease severity variables. CONCLUSION: The CS-PFP is a reliable and valid tool in IPF.

A qualitative study of informal caregivers' perspectives on the effects of idiopathic pulmonary fibrosis.

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a life-shortening lung disease that leads to significant morbidity in patients. The devastation IPF imposes extends beyond patients: it affects their spouses, loved ones and any other person who might take on the role of informal caregiver (IC) to the patient. OBJECTIVE: The aim of this study was to capture ICs' perspectives on how they are affected by having a loved one with IPF. Given ICs' vantage, data were also collected on their perceptions of how IPF impacted their patient-loved ones over the course of the disease. METHODS: Reflexive team analysis was used to analyse the transcripts from semistructured focus groups conducted with ICs of patients with IPF. Based on the analyses, a conceptual framework of the IC's journey with a patient with IPF was developed and includes suggestions for interventions that might ease the burdens ICs endure while caring for their patient-loved ones. RESULTS: 14 ICs included in this study experienced several hardships throughout the course of their loved ones' illness, from emotional devastation at the time of diagnosis to living with an 'impatient,' 'cranky' loved one and being forced to exist in a 'smaller world' because of the physical limitations IPF imposed on their partners. The threat of patients needing supplemental oxygen was central to creating angst among patients and ICs, and supplemental oxygen use by patients prohibited them and their ICs from living the 'normal', carefree lives they desired. CONCLUSIONS: Being an IC to a patient with IPF is extremely challenging (as 1 IC put it: "…harder on the spouse than the patient in some ways"). As patients attempt to adapt to the 'sick person' role, ICs face a struggle between performing their duties as caregiver and maintaining their own identities and independence.

Cross-Atlantic modification and validation of the A Tool to Assess Quality of Life in Idiopathic Pulmonary Fibrosis (ATAQ-IPF-cA).

RATIONALE: The A Tool to Assess Quality of Life in Idiopathic Pulmonary Fibrosis (ATAQ-IPF) was developed in the USA to assess health-related quality of life in patients with IPF. It is likely that some of the original ATAQ-IPF items perform differently when applied in different countries. This paper reports results of a study conducted to identify the need to refine the content of the ATAQ-IPF to minimise cross-country bias between the USA and the UK. METHODS: The ATAQ-IPF and other study measures were completed by patients attending specialist IPF clinics in the USA and UK. Rasch analysis was used to determine which items performed differently across countries (USA vs UK) and refine the original ATAQ-IPF to an instrument without cross-country bias (ATAQ-IPF-cA). Preliminary validation of the modified instrument was examined by assessing correlations between ATAQ-IPF-cA scores and scores from dyspnoea-specific patient-reported outcome (PRO) measures. RESULTS: 139 patients with IPF (USA=74; UK=65) participated in the study. A total of 41 items and 4 domains were removed from the original, 86-item instrument to yield the 43 items and 10 domains of the ATAQ-IPF-cA. Each domain had good fit to the Rasch model, internal consistency was comparable to the corresponding domains for the original ATAQ-IPF, and validity was supported by significant correlations between its scores and scores from dyspnoea-specific PROs. CONCLUSIONS: The reliability and validity of the substantially shortened ATAQ-IPF-cA are acceptable and comparable to the original instrument. We recommend use of the ATAQ-IPF-cA in IPF studies in which participants are enrolled from the USA and UK.

Protocol for a mixed-methods study of supplemental oxygen in pulmonary fibrosis.

BACKGROUND: Little is known about whether or how supplemental oxygen affects patients with pulmonary fibrosis. METHODS/DESIGN: A mixed-methods study is described. Patients with pulmonary fibrosis, informal caregivers of pulmonary fibrosis patients and practitioners who prescribe supplemental oxygen will be interviewed to gather data on perceptions of how supplemental oxygen impacts patients. In addition, three hundred pulmonary fibrosis patients who do not use daytime supplemental oxygen will be recruited to participate in a longitudinal, pre-/post- study in which patient-reported outcome (PRO) and activity data will be collected at baseline, immediately before daytime supplemental oxygen is initiated, and then once and again 9-12 months later. Activity data will be collected using accelerometers and portable GPS data recorders. The primary outcome is change in dyspnea from before to one month after supplemental oxygen is initiated. Secondary outcomes include scores from PROs to assess cough, fatigue and quality of life as well as the activity data. In exploratory analyses, we will use longitudinal data analytic techniques to assess the trajectories of outcomes over time while controlling for potentially influential variables. DISCUSSION: Throughout the study and at its completion, results will be posted on the website for our research program (the Participation Program for Pulmonary Fibrosis or P3F) at http://www.pulmonaryfibrosisresearch.org.

"Getting stuck with LAM": patients perspectives on living with lymphangioleiomyomatosis.

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a rare, progressive lung disease that affects almost exclusively women and is most often diagnosed before menopause. The main symptom of LAM is shortness of breath. LAM patients' perceptions of how the disease impacts their lives is largely unknown, but such information could be useful to generate patient reported outcome measures for use in drug trials (or other research studies) and to formulate interventions aimed at easing the burdens LAM imposes on patients. OBJECTIVE: To capture patients' perceptions of how LAM affects their lives. METHODS: We used reflexive team analysis to analyze transcripts from semi-structured focus groups conducted with LAM patients at LAMposium 2013. We sought to determine what patients perceive as the primary symptoms of LAM and how the disease affects them in their daily lives. RESULTS: The 37 participants described seven primary symptoms of LAM and five common psychological experiences from living with the disease. Shortness of breath and low energy (or fatigue) dominated the symptomatic picture; cough, sensations in the chest, difficulty sleeping, gastrointestinal issues, and mild cognitive difficulties were less common. The common psychological experiences participants reported included frustration, worry, loss of identity, embarrassment, and in some participants, a healthy defiance against the disease. CONCLUSIONS: Patients perceive the physical symptoms from LAM to be intrusive and limiting. Women living with LAM are frustrated by their physical limitations, and they worry about what the future will be like if the disease progresses. Therapeutic interventions should take aim at improving these perceptions.

Assessing exertional dyspnea in patients with idiopathic pulmonary fibrosis.

BACKGROUND: Dyspnea is a hallmark symptom of idiopathic pulmonary fibrosis (IPF), and dyspnea induced physical activity limitation is a prominent driver of quality of life impairment among IPF patients. METHODS: We examined response data for the 21 physical activity items (the first 21 of 24) from the University of California San Diego Shortness of Breath Questionnaire (UCSD) collected at baseline in a recently conducted IPF trial. We used Rasch analysis and hypothesis testing with conventional statistical methodology to achieve three objectives: 1) to examine the items to identify the one characteristic that distinguishes one from another; 2) to asses these items for their ability to measure dyspnea severity in IPF; 3) to use the items to develop a dyspnea ruler. RESULTS: The sample comprised 178 subjects. The 21 items fit the Rasch model. There was very strong correlation between Rasch item severity and their metabolic equivalents (METS) values (r = -0.86, p < 0.0001). With the sample stratified on scores from the 21 items, there were significant between group differences in FVC%, DLCO% and distance walked during the six-minute walk test. The dyspnea ruler can be used to put dyspnea levels in a more easily understood clinical context. CONCLUSIONS: The first 21 items from the UCSD compose a unidimensional dyspnea-with-activity scale and are both sensibly ordered and distinguished from each other by their METS values. These 21 items can be used confidently to formulate clinically-relevant inferences about IPF patients and should be considered for use as a meaningful endpoint in IPF research.

Patient expectations and experiences in idiopathic pulmonary fibrosis: implications of patient surveys for improved care.

For patients with idiopathic pulmonary fibrosis (IPF), prognosis is extremely poor and treatment options are limited. An improved understanding of the experiences and expectations of IPF patients could lead to better clinical management and patient satisfaction. A review of the literature found that diagnosis of IPF typically involved a protracted course including multiple evaluations. Patients felt that educational resources were inadequate and inaccessible. Overall, patients had reasonable expectations for IPF-specific therapies, and were enthusiastic about trying promising new therapies. Numerous domains of health and functioning identified by patients as important were impaired because of IPF. Existing patient-reported outcome instruments (including assessments of health-related quality of life) do not capture many of these domains or are irrelevant to patients with IPF, highlighting the need for an IPF-specific instrument. Patients treated in centers of excellence expressed greater satisfaction with quality of care and treatments, and also valued the opportunity to interact with other IPF patients.

Health-related quality of life in idiopathic pulmonary fibrosis: where are we now?

PURPOSE OF REVIEW: To summarize current knowledge of health-related quality of life (HRQL) assessment in idiopathic pulmonary fibrosis (IPF), with an emphasis on reviewing available data on HRQL instruments, operationalizing HRQL assessment in IPF research and interventions that show promise for improving HRQL in patients with IPF. RECENT FINDINGS: Findings from several studies support the validity of the Medical Outcomes Study Short-Form 36-item Questionnaire and St George's Respiratory Questionnaire for assessing HRQL in IPF. IPF-specific HRQL instruments are in development, but additional research is needed before their broad implementation. Pulmonary rehabilitation and IPF disease management programs appear to be promising interventions for improving HRQL in IPF patients. SUMMARY: IPF is a devastating disease. Because there is no cure, an improved understanding of how this disease affects patients' lives is needed. Rigorously developed and carefully administered instruments are needed to assess those effects and to measure the impact of interventions aimed at improving the lives of patients with IPF.