The longitudinal use of EmPHasis-10 and CAMPHOR questionnaire health-related quality of life scores in patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension.

BACKGROUND: Health-related quality of life (HRQoL) is impaired in patients with pulmonary hypertension (PH). The EmPHasis-10 and CAMPHOR questionnaires are developed to evaluate HRQoL specifically in patients with PH. Data on the longitudinal use of both questionnaires are still limited. We evaluated the longitudinal value of both questionnaires and established minimal clinically important differences (MCID). METHODS: Sixty-one treatment naïve pulmonary arterial hypertension or chronic thromboembolic patients were prospectively included. Patients were treated according to the current ESC/ERS guidelines. We compared EmPHasis-10 and CAMPHOR scores between baseline, 6 and 12 months of follow-up and evaluated the correlation between these scores and a 5-scale symptom severity score, 5-scale overall health score, NYHA-classification, 6 min walk test distance (6MWD), NT-proBNP and echocardiographic parameters. RESULTS: After one year of treatment a significant reduction in EmPHasis-10 score and CAMPHOR QoL and symptoms domain score was observed. Moderate to good correlations were observed between the questionnaires and the overall-health and symptom severity score and 6MWD. No relevant correlations were seen between the questionnaires and NT-pro-BNP and echocardiographic parameters. EmPHasis-10 scores showed strong correlations with all CAMPHOR domains. The MCID for the EmPHasis-10 questionnaire was -8. The MCIDs for the CAMPHOR domains were: activity -3, symptoms -4, QoL -3. CONCLUSION: The EmPHasis-10 and CAMPHOR questionnaires are valid tools for the longitudinal measurement of HRQoL in patients with PH. The much shorter EmPHasis-10 correlates well with the CAMPHOR domain scores and with the clinical endpoints and it may be easier to use in daily practice.

The effect of the walk-bike on quality of life and exercise capacity in patients with idiopathic pulmonary fibrosis: a feasibility study.

Idiopathic pulmonary fibrosis (IPF) is characterized by progressive loss of pulmonary function and exercise capacity, leading to loss of quality of life and often social isolation. A new walking aid, the walk-bike, showed an improvement in exercise performance in COPD patients. Aims of this pilot study were to evaluate feasibility of a homebased walk-bike intervention study in IPF patients and to explore the effect of the walk-bike on quality of life (QoL) and exercise capacity. Twenty-three patients with IPF were included in a randomized multicenter crossover study with 8 weeks of standard care and 8 weeks of walk-bike use at home. Ten patients completed both study phases. Study barriers included reluctance to participate and external factors (e.g. weather and road conditions) that hampered adherence. Patients' satisfaction and experience with the walk-bike varied greatly. After training with the walk-bike, health-related QoL (St. George's Respiratory and King's Brief Interstitial Lung Disease questionnaires) demonstrated a tendency towards improvement, exercise capacity did not. A clinically important difference was found between 6-minute walk test with the walk-bike and the standard test; median (range) respectively 602 m (358-684) and 486 m (382-510). Conclusions: Due to practical barriers a larger study with the walk-bike in patients with IPF seems not feasible. Individual patients may benefit from the use of a walk-bike as it improved action radius and showed a tendency towards improvement in QoL. No effect on exercise capacity was observed. (Sarcoidosis Vasc Diffuse Lung Dis 2020; 37 (2): 192-202).

The Effects of a 10-wk Outpatient Pulmonary Rehabilitation Program on Exercise Performance, Muscle Strength, Soluble Biomarkers, and Quality of Life in Patients With Pulmonary Hypertension.

PURPOSE: Pulmonary arterial hypertension (PAH) is characterized by right ventricular failure, leading to exertional dyspnea, skeletal muscle weakness, and poor quality of life (QOL). Apart from treatment with PAH-specific drugs, guidelines recommend pulmonary rehabilitation (PR). Clinical PR programs have shown improvement in functional capacity and QOL. However, little is known about the effectiveness of an outpatient PR program. The aim of our study was to assess effectiveness of a multidisciplinary outpatient PR program. METHODS: Patients with PAH or chronic thromboembolic pulmonary hypertension (CTEPH), who were in a stable condition on optimized drug therapy, followed a 10-wk outpatient program in a rehabilitation center. The PR program was designed to improve exercise capacity and health status by means of low load cycling, walking, and muscle training twice a week combined with psychological counseling. QOL was measured by the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) questionnaire. RESULTS: Twenty-one patients (13 women) with PAH (n = 16) or CTEPH (n = 5) completed the study. All patients were in New York Heart Association (NYHA) functional class III, and their mean age was 45 ± 16 yr. After PR, the mean cycling endurance time increased by 4.4 min (P < .001), 6-min walk distance by 12.2 m (P < .05), and maximum inspiratory pressure by 5.8 cm H2O (P = .01). Skeletal muscle function increased significantly. The CAMPHOR questionnaire demonstrated significant decrease in symptoms and improvement in QOL. Soluble biomarkers did not show any change before and after PR. CONCLUSIONS: Outpatient PR could be an effective instrument to improve exercise capacity and health status in patients with PAH or CTEPH.

No evidence found for an association between prednisone dose and FVC change in newly-treated pulmonary sarcoidosis.

BACKGROUND: Prednisone is used as first-line therapy for pulmonary sarcoidosis. What dosing strategy has the best balance between effect and side-effects is largely unknown. We analyzed change in forced vital capacity (FVC) and weight during different prednisone doses used in daily practice for treatment naïve pulmonary sarcoidosis patients. METHODS: Multilevel models were used to describe FVC and weight change over time. Correlations were calculated using linear regression models. RESULTS: Fifty-four patients were included. FVC changed over time (p < 0.001), with an average increase of 9.6% predicted (95% CI: 7.2 to 12.1) at 12 months. Weight changed significantly over time (p < 0.001), with an average increase of 4.3 kg (95% CI: 3.0 to 5.6) at 12 months. Although FVC and weight changed significantly over time, there was little correlation between prednisone dose and FVC change, while weight increase correlated significantly with cumulative prednisone dose at 24 months. In patients treated with a high cumulative prednisone dose, baseline FVC was on average lower (p = 0.001) compared to low dose treated patients, while no significant differences were observed in need for second/third-line therapy or number of exacerbations. A strategy leading to a low cumulative dose at 12 months was defined by rapid dose tapering to 10 mg/day within 3.5 months. CONCLUSIONS: These results suggest that prednisone therapy aimed at improving or preserving FVC in newly- treated pulmonary sarcoidosis can often be reduced in dose, using a treatment regimen that is characterized by early dose tapering.

Translation and validation of the King's Brief Interstitial Lung Disease (K-BILD) questionnaire in French, Italian, Swedish, and Dutch.

No disease-specific instruments exist in Dutch, French, Italian, and Swedish to measure health status in idiopathic pulmonary fibrosis (IPF) and other interstitial lung diseases (ILDs). The King's Brief Interstitial Lung Disease (K-BILD) is a 15-item validated questionnaire assessing health status in patients with ILD. The aim of this study was to translate and validate the K-BILD to French, Italian, Swedish, and Dutch versions. The K-BILD was translated following a forward-backward multistep procedure and tested in structured patient interviews. Subsequently, 195 outpatients with ILD were asked to complete K-BILD, St. George's Respiratory Questionnaire (SGRQ), and Euroqol EQ-5D-5L (EQ5D), twice, 2 weeks apart. Internal consistency, concurrent validity, and repeatability were determined. No major difficulties occurred in the translation processes. The K-BILD was considered comprehensible and relevant by patients. One hundred seventy-six patients (108 IPF and 68 other ILDs) completed the translated K-BILD. Internal consistency was good for all K-BILD modules (Cronbach's α 0.70-0.93). Concurrent validity of K-BILD was strong compared with SGRQ ( r = -0.86) and EQ5D ( r = 0.68), low with transfer capacity of the lung for carbon monoxide corrected for hemoglobin ( r = 0.33) and with forced vital capacity ( r = 0.35). The K-BILD and its domains were repeatable over 2 weeks; intraclass correlation coefficients were 0.86-0.93 ( n = 159). Known groups validity showed K-BILD was able to discriminate between patients based on severity of disease. K-BILD's validity and reliability for patients with IPF was similar to that of other ILDs. The French, Italian, Swedish, and Dutch translated K-BILD questionnaires were well-received by patients and demonstrated excellent validity comparable to the original English K-BILD.

Validation of the King's Sarcoidosis Questionnaire (KSQ) in a Dutch sarcoidosis population.

BACKGROUND: The King's Sarcoidosis Questionnaire (KSQ) is a brief questionnaire assessing health status using five modules (General Health Status, Lung, Eyes, Skin, Medication) in patients with sarcoidosis. The KSQ was only validated in one English sarcoidosis cohort. OBJECTIVE: The aim of this study was to validate the KSQ in a Dutch sarcoidosis population. METHODS: The KSQ was translated according to international guidelines and tested in interviews with patients. Consecutive outpatients completed multiple questionnaires twice, two weeks apart. Construct validity, internal consistency and repeatability were determined. RESULTS: Of the 98 patients included 85 had lung, 22 skin and 24 eye disease. There was good construct validity of the KSQ General Health Status module against the World Health Organization Quality of Life-BREF questionnaire. The Medication module correlated weak to moderate with most questionnaires. The correlations with organ-specific questionnaires varied from strong for Eyes (r=0.75), Skin (r=-0.62) to moderate for Lung (r=-0.45 with MRC breathlessness scale). Internal consistency was good for all KSQ modules (Cronbach's α 0.72-0.93). Intraclass correlation coefficients (0.70-0.90) and Bland-Altman plots showed good repeatability of the KSQ. CONCLUSION: The Dutch KSQ is the first translation of the English KSQ, validated in a Dutch sarcoidosis population.