Cost of care and clinical condition in paediatric cystic fibrosis patients

Baumann, Ulrich; Stocklossa, Christiane; Greiner, Wolfgang; Schulenburg, Johann-Matthias Graf von der; Hardt, H. von der

doi:10.1016/s1569-1993(03)00024-9

Cited by 72 publications

(68 citation statements)

References 12 publications

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“…The study raises awareness about the lack of research on the socio-economic impact of rare diseases, which can be substantial, as well as methodological issues related to the comparability of the available evidence across borders which need to be addressed in future research. Prevalence 1:2,500 -3,600 newborns (13)(14) 1:3,600 male infants (18) 1:3,600 males; 1:4,000 -6,000 females (20) A: 1:5,000 -10,000 males B: 1:20,000 -34,000 males (21) 8-150:100,00 children (22) Type I 1:100,000; Type II 1:250,000; Type III 1:50,000 to 1:280,000; Type IV 1:75,000; Type VI 1:250,000 (23)(24)(25)(26)(27) 74:100,000 women; 13:100,000 men (30) 1:22,000 newborns (32) 1:150,000 (NMD) (35) 5:100,000 (37) Prevalence (per 100,000 population) (16) A c c e p t e d M a n u s c r i p t 33 A c c e p t e d M a n u s c r i p t 34 9-57% (14)(15)(50)(51)(52)(53)(54)(55)(56)(57)(58)(59)(60)(61) n/a 15-81% (15,(50)(51)(52)(53)(54)(55)(56)(57)(58)(59)(60)(61) DMD 541,593 (65) n/a n/a 1...…”

Section: Discussionmentioning

confidence: 99%

“…A c c e p t e d M a n u s c r i p t 8 CF is the most frequent pediatric autosomal recessive disease in Caucasian populations (13)(14)(15), occurring in 1 in 2,500 to 3,600 births, with a European Union (EU) prevalence of 12.6:100,000 population (16). CF causes abnormalities in chloride ion transportation resulting in increased viscosity of mucus secretions within numerous organ systems.…”

Section: Disease Characteristics and Prevalence In Europementioning

confidence: 99%

“…A c c e p t e d M a n u s c r i p t Patients with P. aeruginosa infection can incur >2-3x higher costs (15,48) (c)…”

Section: Page 35 Of 43mentioning

confidence: 99%

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Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

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A c c e p t e d M a n u s c r i p t 2 Highlights  We systematically reviewed the socioeconomic cost literature for 10 rare diseases. Direct and indirect costs incurred by patients, carers and society were searched. 77 studies were included with an unequal distribution of studies over disease types. Level of existing evidence is highest for diseases with available drug treatments. Indirect costs are in most cases of similar or higher magnitude than direct costs. found to range between 1-2 per 100,000 population (PWS, a sub-type of Histiocytosis, and EB) up to 42 per 100,000 population (Scleroderma). Overall, cost evidence on rare diseases appears to be very scarce (a total of 77 studies were identified across all diseases), with CF (n=29) and Haemophilia (n=22) being relatively well studied, compared to the other conditions, where very limited cost of illness information was available. In terms of data availability, total lifetime cost figures were found only across four diseases, and total annual costs (including indirect costs) across five diseases.Overall, data availability was found to correlate with the existence of a pharmaceutical treatment and indirect costs tended to account for a significant proportion of total costs.Although methodological variations prevent any detailed comparison between conditions, most of the rare diseases examined are associated with significant economic burden, both direct and indirect.

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Section: Discussionmentioning

confidence: 99%

Section: Disease Characteristics and Prevalence In Europementioning

confidence: 99%

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Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

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“…These 3 methods, which are combined in most CF centers, are successful, cost-efficient and have completely changed the epidemiology of chronic P. aeruginosa lung infections in CF patients from being very common in CF children to being predominantly a problem for adult patients. No problems of resistance to the antibiotics have been recorded in intermittently colonized CF patients [34,[44][45]. Early aggressive eradication therapy has also been shown to be efficient in a CF animal model of P. aeruginosa infection [46].…”

Section: Figurementioning

confidence: 99%

The clinical impact of bacterial biofilms

Høiby¹,

Ciofu²,

Johansen³

et al. 2011

Int J Oral Sci

735

536

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Bacteria survive in nature by forming biofilms on surfaces and probably most, if not all, bacteria (and fungi) are capable of forming biofilms. A biofilm is a structured consortium of bacteria embedded in a self-produced polymer matrix consisting of polysaccharide, protein and extracellular DNA. Bacterial biofilms are resistant to antibiotics, disinfectant chemicals and to phagocytosis and other components of the innate and adaptive inflammatory defense system of the body. It is known, for example, that persistence of staphylococcal infections related to foreign bodies is due to biofilm formation. Likewise, chronic Pseudomonas aeruginosa lung infections in cystic fibrosis patients are caused by biofilm growing mucoid strains. Gradients of nutrients and oxygen exist from the top to the bottom of biofilms and the bacterial cells located in nutrient poor areas have decreased metabolic activity and increased doubling times. These more or less dormant cells are therefore responsible for some of the tolerance to antibiotics. Biofilm growth is associated with an increased level of mutations. Bacteria in biofilms communicate by means of molecules, which activates certain genes responsible for production of virulence factors and, to some extent, biofilm structure. This phenomenon is called quorum sensing and depends upon the concentration of the quorum sensing molecules in a certain niche, which depends on the number of the bacteria. Biofilms can be prevented by antibiotic prophylaxis or early aggressive antibiotic therapy and they can be treated by chronic suppressive antibiotic therapy. Promising strategies may include the use of compounds which can dissolve the biofilm matrix and quorum sensing inhibitors, which increases biofilm susceptibility to antibiotics and phagocytosis.

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“…Chronic PA colonisation is associated with a lower FEV 1 in childhood , an accelerated rate of FEV 1 decline (Pamukcu et al, 1995;Kosorok et al, 2001;Emerson et al, 2002), a shorter median life expectancy (CFF Registry, 1996;Emerson et al, 2002) and much higher treatment costs (Baumann et al, 2003). Preventing this colonisation is considered the most important challenge for the CF clinician, as it frequently determines the patient's future quality of life and long-term survival (CF Trust 2002;Koch, 2002).…”

Section: Challenge 1: Postpone Chronic Colonisation By Pseudomonas Aementioning

confidence: 99%

The Prognosis of Cystic Fibrosis - A Clinician's Perspective

Lebecque¹

2012

Cystic Fibrosis - Renewed Hopes Through Research

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Cost of care and clinical condition in paediatric cystic fibrosis patients

Abstract: Health care expenditures for patients with CF vary with the clinical course. The variation can be explained to a large extend by clinical parameters.

Cited by 72 publications

References 12 publications

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

The clinical impact of bacterial biofilms

The Prognosis of Cystic Fibrosis - A Clinician's Perspective

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