The economic burden of bronchiectasis - known and unknown:a systematic review

Abstract Background The increasing prevalence and recognition of bronchiectasis in clinical practice necessitates a better understanding of the economic disease burden to improve the management and achieve better clinical and economic outcomes. This study aimed to assess the economic burden of bronchiectasis based on a review of published literature. Methods A systematic literature review was conducted using MEDLINE, Embase, EconLit and Cochrane databases to identify publications (1 January 2001 to 31 December 2016) on the economic burden of bronchiectasis in adults. Results A total of 26 publications were identified that reported resource use and costs associated with management of bronchiectasis. Two US studies reported annual incremental costs of bronchiectasis versus matched controls of US$5681 and US$2319 per patient. Twenty-four studies reported on hospitalization rates or duration of hospitalization for patients with bronchiectasis. Mean annual hospitalization rates per patient, reported in six studies, ranged from 0.3–1.3, while mean annual age-adjusted hospitalization rates, reported in four studies, ranged from 1.8–25.7 per 100,000 population. The average duration of hospitalization, reported in 12 studies, ranged from 2 to 17 days. Eight publications reported management costs of bronchiectasis. Total annual management costs of €3515 and €4672 per patient were reported in two Spanish studies. Two US studies reported total costs of approximately US$26,000 in patients without exacerbations, increasing to US$36,00–37,000 in patients with exacerbations. Similarly, a Spanish study reported higher total annual costs for patients with > 2 exacerbations per year (€7520) compared with those without exacerbations (€3892). P. aeruginosa infection increased management costs by US$31,551 to US$56,499, as reported in two US studies, with hospitalization being the main cost driver. Conclusions The current literature suggests that the economic burden of bronchiectasis in society is significant. Hospitalization costs are the major driver behind these costs, especially in patients with frequent exacerbations. However, the true economic burden of bronchiectasis is likely to be underestimated because most studies were retrospective, used ICD-9-CM coding to identify patients, and often ignored outpatient burden and cost. We present a conceptual framework to facilitate a more comprehensive assessment of the true burden of bronchiectasis for individuals, healthcare systems and society

Muscle MRI findings in childhood/adult onset Pompe disease correlate with muscle function

Enzyme replacement therapy has shown to be effective for childhood/adult onset Pompe disease (AOPD). The discovery of biomarkers useful for monitoring disease progression is one of the priority research topics in Pompe disease. Muscle MRI could be one possible test but the correlation between muscle MRI and muscle strength and function has been only partially addressed so far.We studied 34 AOPD patients using functional scales (Manual Research Council scale, hand held myometry, 6 minutes walking test, timed to up and go test, time to climb up and down 4 steps, time to walk 10 meters and Motor Function Measure 20 Scale), respiratory tests (Forced Vital Capacity seated and lying, Maximun Inspiratory Pressure and Maximum Expiratory Pressure), daily live activities scales (Activlim) and quality of life scales (Short Form-36 and Individualized Neuromuscular Quality of Life questionnaire). We performed a whole body muscle MRI using T1w and 3-point Dixon imaging centered on thighs and lower trunk region.T1w whole body muscle MRI showed a homogeneous pattern of muscle involvement that could also be found in pre-symptomatic individuals. We found a strong correlation between muscle strength, muscle functional scales and the degree of muscle fatty replacement in muscle MRI analyzed using T1w and 3-point Dixon imaging studies. Moreover, muscle MRI detected mild degree of fatty replacement in paraspinal muscles in pre-symptomatic patients.Based on our findings, we consider that muscle MRI correlates with muscle function in patients with AOPD and could be useful for diagnosis and follow-up in pre-symptomatic and symptomatic patients under treatment.Muscle MRI correlates with muscle function in patients with AOPD and could be useful to follow-up patients in daily clinic

Gastrointestinal pirfenidone adverse events in idiopathic pulmonary fibrosis depending on diet: the MADIET clinical trial

Individuals with IPF who follow a MUFA diet report a lower incidence of pirfenidone gastrointestinal adverse events than those that follow a SFA diet, which could explain the different prevalence in GI pirfenidone AEs reported by countries in IPF cohorts https://bit.ly/3LuzAUJ Idiopathic pulmonary fibrosis (IPF) is a chronic and lethal interstitial lung disease (ILD) [1, 2]. Antifibrotic medications such as pirfenidone have been a turning point in the management of IPF, slowing of disease progression and improving survival [1–5]. eng research projects or scientific advice from Esteve-Teijin, Roche, Boehringer Ingelheim and Chiesi. V. Vicens-Zygmunt received fees for scientific advice from Boehringer Ingelheim. P. Rivera-Ortega declares speaker and consultation fees from Boehringer Ingelheim and Hoffmann-La Roche, and fees received for research projects from Boehringer Ingelheim, Hoffmann-La Roche, CSL Behring, FibroGen, Vicore Pharma AB, Gilead Sciences and Galecto; all research fees were paid to her institution. F. Bonella declares speaker and consultation fees from Boehringer Ingelheim, Hoffman La Roche and Fibrogene, outside the submitted work. E. Renzoni reports grants, lecture fees and advisory board fees from Boehringer Ingelheim, lecture fees from Roche and Chiesi, research grants from Raynaud's and Scleroderma, and support for attending meetings from Boehringer Ingelheim, outside the submitted work; all grants and fees were paid to her institution. A-M. Russell declares speaker and consultation fees from Boehringer Ingelheim and Hoffman-La Roche. T.M. Maher reports consultancy fees from AstraZeneca, Bayer, Blade Therapeutics, Boehringer Ingelheim, Bristol Myers Squibb, Galapagos, Galecto, GlaxoSmithKline, IQVIA, Pliant, Respivant Sciences, Roche/Genentech, Theravance Biopharma and Veracyte, and fees for presentations from Boehringer Ingelheim and Roche/Genentech. G. Suarez-Cuartin has received grants from Grifols, travel grants from Chiesi, Menarini and Boehringer Ingelheim, a speaker fee from Insmed, and advisory board fees from Insmed Inc. and Zambon. M. Wijsenbeek has received grants from Boehringer Ingelheim, The Netherlands Organisation for Health Research and Development, The Dutch Lung Foundation, Sarcoidose.nl and The Dutch Pulmonary Society, consulting fees from Boehringer Ingelheim, Galapagos, Bristol Myers Squibb, Galecto, Respivant, NeRRe Therapeutics, Horizon Therapeutics, PureTech health, Kinevant Sciences, Molecure and CLS Behring, speaker fees from Boehringer Ingelheim, Hoffman-La Roche, Novartis and CLS Behring, support for attending meetings from Boehringer Ingelheim, Galapagos and Hoffman-La Roche, and has participated in advisory boards of different patient associations (unpaid); all grants and fees were paid to her institution. C. Vancheri served on advisory committees of InterMune, Roche, AstraZeneca, Sanofi, Insmed, Zambon and Boehringer Ingelheim, and received lecture fees and nongovernmental research support from InterMune, Roche, Boehringer Ingelheim, Novartis, Chiesi, Menarini, AstraZeneca, GSK, Sanofi and Insmed. The rest of the authors have no relevant relationships to disclose.Published version, accepted version (12 month embargo), submitted versionThe article is available via Open Access. Click on the 'Additional link' above to access the full-text