Dermatophytes and skin dermatophytoses in Southeast Asia-First epidemiological survey from Cambodia.

BACKGROUND: Dermatomycoses count to the most frequent dermatoses in Cambodia. OBJECTIVES: The aim of this survey was to investigate the occurrence of dermatophytes in this Southeast Asian country. METHODS: From June 2017 to July 2018, skin scrapings were taken from 67 patients with superficial dermatophytosis for mycological diagnostics. Identification of dermatophytes was confirmed by sequencing of the 'internal transcribed spacer'-(ITS) region of the rDNA, and the gene of the Translation Elongation Factor (TEF)-1α. RESULTS: Patients were suffering from tinea corporis and tinea inguinalis/cruris 42/67 (63%), tinea capitis/faciei 14/67 (21%), tinea corporis/capitis/faciei 6/67 (9%), tinea manuum/pedis 2/67 (3%), tinea pedis 2/67 (3%) and tinea manuum 1/67 (1%). Both, by culture and/or PCR, a dermatophyte was detected in 52 (78%) out of 67 samples. Culture positive were 42 (81%) of 52, PCR positive were 50 (96%). The following dermatophytes were found: Trichophyton (T.) rubrum, 36/52 strains (69%, 29 by culture), T. mentagrophytes/T. interdigitale (TM/TI) 9/52 (17%, six by culture) and Microsporum (M.) canis 5/52 strains (10%, by culture). One strain of Nannizzia (N.) incurvata 1/52 (2%) and N. nana 1/52 (2%) was isolated. Based on sequencing, we demonstrated that two T. mentagrophytes strains out of the nine TM/TI represented the new ITS genotype XXV Cambodia. We found one T. mentagrophytes strain genotype VIII (now, reclassified as T. indotineae). This isolate was terbinafine resistant, and it exhibited the amino acid substitution Phe397Leu in the squalene epoxidase. Three strains of T. interdigitale genotype II* were isolated. CONCLUSION: This is the first survey on epidemiology of dermatophytes in Cambodia. Currently, T. rubrum represents the most frequent species in Cambodia. One Indian strain genotype VIII T. mentagrophytes was found. A highlight was the first description of the new T. mentagrophytes genotype XXV Cambodia.

[Zoophilic dermatophytes during coronavirus pandemic in Germany]. / Zoophile Dermatophyten während der Corona-Pandemie in Deutschland.

During the coronavirus pandemic, significantly more pets were probably bought and kept. This study focuses on whether more zoophilic dermatophytes have subsequently been isolated and which species predominate. In the 1­year period from March 2020 through February 2021, all zoophilic dermatophytes from all submissions to the Mölbis laboratory were recorded. Both the cultural and the molecular evidence of fungal detection from skin scrapings, hair roots, and, in single cases, from nails, were considered. For dermatophyte DNA (Deoxyribonucleic acid) detection, an in-house polymerase chain reaction (PCR) - enzyme-linked immunosorbent assay (ELISA) was used. In distinct cases, identification of dermatophytes was confirmed by sequencing of the internal transcribed spacer (ITS) region of the rDNA, and of the gene of the translation elongation factor (TEF)-1α. In 579 (2.56%) of 22,575 samples studied in the year 2020/2021, zoophilic dermatophytes were detectable with PCR-ELISA and/or by cultivation. In comparison, the proportion of zoophilic dermatophytes was 2.03% in the 1­year period 2014/2015, and only 1.6% in 2018/2019. The 579 zoophilic dermatophytes were identified as follows: Trichophyton (T.) benhamiae 186 (32.1%), T. mentagrophytes 173 (29.9%), T. quinckeanum 110 (19.0%), Microsporum (M.) canis 78 (13.5%), T. verrucosum 22 (3.8%), Nannizzia (N.) persicolor 8 (1.4%), T. erinacei 1 (0.2%), and T. equinum 1 (0.2%). T. benhamiae had the highest prevalence from June to September 2020, then again in December. T. quinckeanum is associated with a sharp increase in the mice population in Germany in 2020; a significant increase was found in the months September 2020 to January 2021. T. mentagrophytes had a conspicuous peak in September. Compered with that M. canis in November. Up to 50% of the dermatophytoses caused by T. mentagrophytes, T. quinckeanum, and M. canis affected children and adolescents, while in the case of T. benhamiae it was as much as two thirds. Tinea corporis was the most common, followed by tinea faciei and tinea capitis. M. canis infections affected the capillitium more frequently than the face. Zoophilic dermatophytes were increasingly isolated during the coronavirus pandemic in Germany when compared to previous year periods. In first place, the dermatophyte T. benhamiae from guinea pigs was found in children and adolescents. A significant proportion of dermatophytoses concerned adults. T. quinckeanum is an emerging pathogen in Germany with unprecedented high infection rates in 2020.

[Sustainable reimbursement of the B­centres for rare diseases in Germany-status quo and solution approaches]. / Nachhaltige Vergütung der B­Zentren für Seltene Erkrankungen in Deutschland ­ Status quo und Lösungsansätze.

BACKGROUND: To ensure specialized care of patients with rare diseases, numerous centres for rare diseases were funded over the past few years. The reimbursement of patients' ambulatory care in hospitals, however, is characterized by a plurality of forms of care and payment. There is some evidence of deficits in the reimbursement of care of patients suffering from a rare disease from studies on individual rare diseases. OBJECTIVES: To investigate current forms of care provision and reimbursement of centres for rare diseases and to develop future approaches for sustainable compensation. MATERIALS AND METHODS: Initially, centres for rare diseases in Germany were asked to provide information about their forms of care and reimbursement using questionnaires. Subsequently, two focus group interviews and one expert interview with representatives from centres for rare diseases, health insurance, health politics and patients were conducted to discuss current and future meritocratic forms of care provision and reimbursement. The data were evaluated using content analysis. RESULTS AND CONCLUSIONS: Thirty-nine centres for rare diseases participated in the questionnaire survey. Of those, 38% receive a flat fee/allowance for university outpatient departments, the amount of which varies notably, and 41% obtain a mixed payment comprising an allowance for university outpatient departments and other forms of reimbursement. An under-recovery of costs in centres for rare diseases and its impact on patient care were mentioned in the interviews. In this context, a need to further develop forms of care provision and reimbursement has been identified. Participants prefer a special flat fee/allowance for rare diseases that covers the time-consuming care for patients with rare diseases.

The State of Care for Persons With a Diagnosis of Depression.

BACKGROUND: Depression is one of the most common mental illnesses. There are well-known deficiencies in the care of patients with depression, particularly at the interfaces between health service areas or sectors. The aim of this study was to analyze the characteristics and state of care of persons with a diagnosis of depression. METHODS: The analysis was based on diagnosis and billing data obtained from a statutory health insurance carrier in Germany. Patients carrying a diagnosis of depression in 2018 (ICD-10 F32, F33, F34.1) were identified and studied with respect to their utilization of care services for depression. Sociodemographic characteristics and the degree of severity of depression were also taken into account. RESULTS: More than 285 000 individuals with a diagnosis of depression were included in the analysis. Their mean age was 57.5 years, and 67.5% were female. About three-quarters had at least one comorbid mental disorder. Primary care physicians were involved in the treatment of depression for 82.8% of patients with severe depression; 41.1% of such patients had contact with a medical specialist, and 9.2% with a psychologist-psychotherapist. Antidepressants were prescribed for 60.3% of patients with severe depression, and 10.2% were receiving psychotherapeutic services in accordance with the psychotherapy directive. CONCLUSION: Our findings point to existing deficiencies in the care of patients with depression, particularly in the establishment of the diagnosis and in the assessment of disease severity. These are essential matters for the initiation of guideline-oriented therapy and therefore deserve to be more strongly emphasized in continuing medical education.

Nannizzia incurvata as a rare cause of favus and tinea corporis in Cambodia and Vietnam.

Nannizzia (N.) incurvata (formerly Microsporum incurvatum) represents a geophilic dermatophyte which has been previously classified as belonging to the species complex of N. gypsea (formerly Microsporum gypseum). A 42-year-old Vietnamese female from Saxony, Germany, suffered from tinea corporis of the right buttock after she returned from a 2-week-visit to her homeland Vietnam. From skin scrapings of lesions, N. incurvata grew on Sabouraud's dextrose agar. Treatment by ciclopirox olamine cream twice daily for 4 weeks was successful. A 6-year-old Cambodian boy living near river Mekong with contact history to chicken, dogs and cattle suffered from tinea faciei and capitis. Symptoms of the favus-like tinea capitis and tinea faciei were erythema and scaly patches with areas of alopecia. N. incurvata grew on Sabouraud's dextrose agar. The boy was treated with oral terbinafine 125 mg daily, topical miconazole cream and ketoconazole shampoo. The symptoms healed within 4 weeks of treatment. Cultivation of the samples revealed growth of N. incurvata. For confirmation of species identification, the isolates were subject to sequencing of ITS (internal transcribed spacer) region of the rDNA, and addition of the "translation elongation factor 1 α" (TEF 1 α) gene. Sequencing of the ITS region showed 100% accordance with the sequence of N. incurvata deposited at the NCBI database under the accession number MF415405. N. incurvata is a rare, or might be underdiagnosed geophilic dermatophyte described in Sri Lanka and Vietnam until now. This is the first isolation of N. incurvata in Cambodia, and the first description of favus in a child due to this dermatophyte.

Spread of Terbinafine-Resistant Trichophyton mentagrophytes Type VIII (India) in Germany-"The Tip of the Iceberg?"

Chronic recalcitrant dermatophytoses, due to Trichophyton (T.) mentagrophytes Type VIII are on the rise in India and are noteworthy for their predominance. It would not be wrong to assume that travel and migration would be responsible for the spread of T. mentagrophytes Type VIII from India, with many strains resistant to terbinafine, to other parts of the world. From September 2016 until March 2020, a total of 29 strains of T. mentagrophytes Type VIII (India) were isolated. All patients were residents of Germany: 12 females, 15 males and the gender of the remaining two was not assignable. Patients originated from India (11), Pakistan (two), Bangladesh (one), Iraq (two), Bahrain (one), Libya (one) and other unspecified countries (10). At least two patients were German-born residents. Most samples (21) were collected in 2019 and 2020. All 29 T. mentagrophytes isolates were sequenced (internal transcribed spacer (ITS) and translation elongation factor 1-α gene (TEF1-α)). All were identified as genotype VIII (India) of T. mentagrophytes. In vitro resistance testing revealed 13/29 strains (45%) to be terbinafine-resistant with minimum inhibitory concentration (MIC) breakpoints ≥0.2 µg/mL. The remaining 16 strains (55%) were terbinafine-sensitive. Point mutation analysis revealed that 10/13 resistant strains exhibited Phe397Leu amino acid substitution of squalene epoxidase (SQLE), indicative for in vitro resistance to terbinafine. Two resistant strains showed combined Phe397Leu and Ala448Thr amino acid substitutions, and one strain a single Leu393Phe amino acid substitution. Out of 16 terbinafine-sensitive strains, in eight Ala448Thr, and in one Ala448Thr +, new Val444 Ile amino acid substitutions were detected. Resistance to both itraconazole and voriconazole was observed in three out of 13 analyzed strains. Treatment included topical ciclopirox olamine plus topical miconazole or sertaconazole. Oral itraconazole 200 mg twice daily for four to eight weeks was found to be adequate. Terbinafine-resistant T. mentagrophytes Type VIII are being increasingly isolated. In Germany, transmission of T. mentagrophytes Type VIII from the Indian subcontinent to Europe should be viewed as a significant public health issue.

[Microsporum ferrugineum-an anthropophilic dermatophyte in Germany : Case report and review of the literature]. / Microsporum ferrugineum ­ ein anthropophiler Dermatophyt in Deutschland : Patientenbeschreibung und Übersicht über die Literatur.

Three boys from the same city, treated by the same dermatologist, developed tinea capitis. Two of them, 4 and 8 years old, underwent mycological diagnostic workup. However, no pathogens familiar in this country, such as Microsporum (M.) canis or Trichophyton (T.) tonsurans, were isolated, but instead that of a dermatophyte that has not been found in Germany for decades. Both dermatophyte isolates showed white-beige-brownish colonies with a flat, radiating edge and a central, verrucous curvature. The sequencing of the internal transcribed spacer (ITS) region of the rDNA confirmed the suspicion of M. ferrugineum already expressed based on the morphological picture. The anthropophilic dermatophyte occurs in the Middle East, Asia, Eastern Europe and Africa and is considered to be the cause of tinea capitis or tinea corporis in children and adolescents. In 2016, M. ferrugineum has again been isolated in Germany, probably as a result of migration movements. The fungus is strikingly isolated to martial arts, especially wrestlers. It mainly affects children and adolescents, some with a Russian-German background. The anthropophilic dermatophyte is transmitted directly from person to person, especially in the case of tinea capitis. An indirect transmission, for example, via mats in martial arts is likely.

Alarming India-wide phenomenon of antifungal resistance in dermatophytes: A multicentre study.

BACKGROUND: An alarming increase in recalcitrant dermatophytosis has been witnessed in India over the past decade. Drug resistance may play a major role in this scenario. OBJECTIVES: The aim of the present study was to determine the prevalence of in vitro resistance to terbinafine, itraconazole and voriconazole in dermatophytes, and to identify underlying mutations in the fungal squalene epoxidase (SQLE) gene. PATIENTS/METHODS: We analysed skin samples from 402 patients originating from eight locations in India. Fungi were identified by microbiological and molecular methods, tested for antifungal susceptibility (terbinafine, itraconazole, voriconazole), and investigated for missense mutations in SQLE. RESULTS: Trichophyton (T.) mentagrophytes internal transcribed spacer (ITS) Type VIII was found in 314 (78%) samples. Eighteen (5%) samples harboured species identified up to the T interdigitale/mentagrophytes complex, and T rubrum was detected in 19 (5%) samples. 71% of isolates were resistant to terbinafine. The amino acid substitution Phe397Leu in the squalene epoxidase of resistant T mentagrophytes was highly prevalent (91%). Two novel substitutions in resistant Trichophyton strains, Ser395Pro and Ser443Pro, were discovered. The substitution Ala448Thr was found in terbinafine-sensitive and terbinafine-resistant isolates but was associated with increased MICs of itraconazole and voriconazole. CONCLUSIONS: The high frequencies of terbinafine resistance in dermatophytes are worrisome and demand monitoring and further research. Squalene epoxidase substitutions between Leu393 and Ser443 could serve as markers of resistance in the future.

The current Indian epidemic of superficial dermatophytosis due to Trichophyton mentagrophytes-A molecular study.

The disease burden of chronic-relapsing and therapy-refractory superficial dermatophytosis dramatically increased in India within the past 5-6 years. In order to evaluate the prevalence of this trend, 201 skin scrapings were collected from patients from all parts of India and were tested for dermatophytes using both fungal culture and a PCR-ELISA directly performed with native skin scrapings. Fungal culture material was identified by genomic Sanger sequencing of the internal transcribed spacer (ITS) region and the translation elongation factor (TEF)-1α gene. In total, 149 (74.13%) out of the 201 samples showed a dermatophyte-positive culture result. Out of this, 138 (92.62%) samples were identified as Trichophyton (T.) mentagrophytes and 11 (7.38%) as Trichophyton rubrum. The PCR-ELISA revealed similar results: 162 out of 201 (80.56%) samples were dermatophyte-positive showing 151 (93.21%) T mentagrophytes- and 11 (6.79%) T rubrum-positive samples. In this study, we show for the first time a dramatic Indian-wide switch from T rubrum to T mentagrophytes. Additionally, sequencing revealed a solely occurring T mentagrophytes "Indian ITS genotype" that might be disseminated Indian-wide due to the widespread abuse of topical clobetasol and other steroid molecules mixed with antifungal and antibacterial agents.

Dermatomycoses Due to Nannizzia praecox (Formerly Microsporum praecox) in Germany: Case Reports and Review of the Literature.

Nannizzia praecox, formerly known as Microsporum praecox, is a geophilic dermatophyte. Up to now 31 cases of human tinea have been reported in the literature, most of them with an inflammatory course. Three recent cases diagnosed in Germany within 1 year suggest that the fungus might be a more common cause of human dermatophytosis than reported so far. This might be based on the fact that N. praecox is often found in an equine environment and that horse riding is becoming more popular recently.

Pressure on oxygen levels of Jakarta Bay.

Jakarta Bay is subject to inputs of large amounts of dissolved inorganic nutrients and organic matter deriving from the Jakarta Metropolitan Area and its hinterland. As a consequence, inorganic and organic nutrient concentrations in the nearshore area are considerably increased. The hydrographic pattern inside the bay fosters phytoplankton bloom formation due to pronounced vertical density gradients. High Biomass Blooms (HBB) of phytoplankton are observed recurrently, representing an additional source for oxygen consuming degradable organic matter. An oxygen deficiency area of 20km(2) can be identified in the eastern part of Jakarta Bay where oxygen levels decrease to 60% saturation, equaling a noxiously low concentration of 4.0mg·dm(-3) O2. The absolute minimum detected was 51% O2 or 3.2mg·dm(-3) O2. The observed oxygen concentrations fail Indonesian quality standards for seawater.

Expression of KIT in the ovary, and the role of somatic precursor cells.

KIT is a type III receptor protein tyrosine kinase, and KITL its cognate ligand. KIT can mediate its effects via several intracellular signalling pathways, or by formation of a cell-cell anchor with its ligand. Through these mechanisms, KIT controls fundamental cellular processes, including migration, proliferation, differentiation and survival. These cellular processes are modulated by soluble KIT, a cleavage product of KIT, generated at the cell membrane. A cell-retained KIT cleavage fragment also arises from this cleavage event. This cleavage fragment must be distinguished from truncated KIT (trKIT), which originates through cryptic promoter usage. The expression of trKIT is highly restricted to postmeiotic germ cells in the testis. In contrast, KIT, together with its cleavage products, is present in somatic cells and germ cells in the gonads of both sexes. A functional KITL/KIT system is mandatory for normal population of the gonads by germ cells. Signalling via the KITL/KIT system promotes the growth, maturation, and survival of germ cells within the gonads, and prevents meiotic entry and progression. In addition to its importance in germ cell biology, the KITL/KIT system is crucial for gonadal stromal differentiation. During foetal life, KIT is expressed by testicular stromal precursor cells, which develop into Leydig cells. In the ovary, stromal cell KIT expression accompanies theca layer development around advanced follicles. After ovulation, KIT-immunopositive cells translocate from the theca layer to the luteal ganulosa where they contribute to a delicate cellular network that extends between the fully luteinised large luteal cells. In the outer regions of the developing corpus luteum, a highly conspicuous subpopulation of KIT/CD14-double-immunopositive cells can be observed. KIT/CD14-double-immunopositive cells are also seen in the haematopoietic-like colonies of long-term granulosa cultures established from late antral follicles. These cultures demonstrate expression of pluripotency marker genes such as octamer binding transcription factor-3/4 and sex determining region Y-box 2. The KIT/CD14-double-immunopositive cells can be purified and enriched by KIT-immunopositive magnetic cell sorting. Subsequent exposure of the KIT-expressing cells to the hanging drop culture method, combined with haematopoietic differentiation medium, provides the signals necessary for their differentiation into endothelial and steroidogenic cells. This suggests that monocyte-derived multipotent cells are involved in ovarian tissue remodelling. In summary, multicelluar KITL/KIT signalling organizes the stroma in the ovary and testis; monocyte-derived multipotent cells may be involved.

Resource usage in outpatient care and reimbursement for cystic fibrosis in Germany.

OBJECTIVE: Objective of this study is to assess and evaluate resource use in outpatient treatment in Germany and to compare it with remuneration. METHODS: Outpatient treatment was evaluated in seven different centers for pediatric and adult CF patients. Data were recorded during one representative month in 2006. A micro-costing approach was used to value resource use data. RESULTS: For outpatient treatment mean costs (excluding drugs) of 488 € per patient per quarter occurred. Correlation analyses identified significant cost drivers including age and co-morbidities (pancreatic insufficiency, hepatobiliary complications, lung function capacity, or bacterial lung colonization). Remuneration covered only 51% of the total costs (252 € per patient/quarter). CONCLUSIONS: As the human resources available to these centers today are already below the requirements set by the European consensus for standards of CF care it will be important for a high level of patient care to reach a cost-covering remuneration scheme.

Quality of life results of balloon kyphoplasty versus non surgical management for osteoporotic vertebral fractures in Germany.

BACKGROUND: To compare improvement in quality of life (QoL) and symptoms' relief in vertebral compression fractures (VCF) due to osteoporosis for patients undergoing balloon kyphoplasty (BKP) to those undergoing non-surgical management (NSM) in a real-life setting. METHODS: In this prospective, comparative study, quality-of-life was evaluated in eight centres in Germany between 2005 and 2008, for 82 patients, with the EQ-5D questionnaire, and the Roland Morris Disability Questionnaire (RMDQ). RESULTS: BKP patients demonstrated a statistical and clinical significant higher improvement in EQ-5D than NSM patients, 0.44 and 0.25 from baseline to 12 months, respectively. Moreover, BKP patients showed a clinically relevant improvement in the RMDQ by 6.25 from baseline to 12 months, whereas NSM patients had no significant improvement in the RMDQ. CONCLUSIONS: This study demonstrates for VCF patients that in real-life quality of life for BKP patients improves more than for NSM patients; confirming the results of a large randomized clinical trial.

Outpatient medication costs of patients with cystic fibrosis in Germany.

BACKGROUND: Cystic fibrosis (CF) patients need specialized long-term treatment. In order to support lung function, pharmaceuticals such as bronchodilators, mucolytic agents or anti-inflammatory drugs have to be used. Oral, inhaled or intravenous antibacterial therapy is of special importance for patients who have problems with chronic bacterial colonization of the lung and airways. In case of pancreatic insufficiency, digestive enzymes have to be substituted with every meal. Furthermore, patients often need additional supplements of vitamins as well as high caloric food. All of these aspects lead to high medication use in CF patients. OBJECTIVE: To analyse outpatient medication costs for CF in Germany from a sickness funds perspective (plus some out-of-pocket payments by patients). METHODS: Medication data were evaluated from seven different outpatient CF centres. Data were recorded via medication lists by the physicians, reporting name of medication, dosage and pharmaceutical form. As the medications are mostly used long term, resource use was valued using the largest available package sizes. Prices were taken from the German 'Rote Liste' with year 2006 values. Annual and daily medication costs were analysed for different age groups. In addition, cost-influencing factors were analysed via correlation analyses. RESULTS: A total of 3150 pharmaceutical records from 301 CF patients were collected. Mean annual costs for medication were €21,603 per patient (range €69-104,477). Correlation analyses showed significant correlations between costs of medication and age, co-morbidities (such as pancreatic insufficiency and diabetes mellitus) and clinical parameters such as bacterial colonization of the lung, as well as functional parameters (percent of vital capacity, forced expiratory volume in 1 second, maximal expiratory flow at 25% of forced vital capacity). For example, mean annual costs for medication were €23,815 and €14,884 for patients with and without bacterial colonization of the lung, respectively. Other correlation factors yielded similar cost dispersions between patients with and without the factors. CONCLUSIONS: Costs of outpatient medication for CF patients significantly depend on age, co-morbidities and other clinical parameters. Hence, non-optimal treatment could lead to a significantly higher burden for the healthcare system.

Cross-sectional validity of the EQ-5D-Y as a generic health outcome instrument in children and adolescents with cystic fibrosis in Germany.

BACKGROUND: Quality of life is recognized as an important additional outcome measure in clinical trials and health economic evaluations. The EQ-5D is an important generic health outcome instrument often used for economic evaluations as a complement with disease-specific outcome measures. In this study quality of life data was assessed using the EQ-5D-Y (new EQ-5D version for children and adolescents) and the Cystic Fibrosis Questionnaire (CFQ). The objective of the study is to evaluate the cross-sectional validity of the EQ-5D-Y as a generic health outcome instrument in children and adolescents with cystic fibrosis in Germany. METHODS: In 2006 a multi-centre study was conducted in four cystic fibrosis centres in Germany. Quality of life data from 96 patients between eight and seventeen years was collected using the EQ-5D-Y as a generic outcome instrument and the Cystic Fibrosis Questionnaire (CFQ) as a disease-specific instrument. Results of both instruments were compared by statistical analyses using Spearman's rank correlations. RESULTS: 44.6% of the patients stated that they had no problems in any of the EQ-5D-Y dimensions. Several low to high correlations between separate dimensions and the visual analogue scale of the EQ-5D-Y and the different scales of the CFQ for children, their parents and adolescents can be presented in this paper. Looking at the five EQ-5D-Y dimensions the highest correlation (rS = 0.625, p = 0.01) was found between the dimension 'mobility' and the CFQ scale 'physical functioning' in adolescent patients. The overall highest correlation was found between the 'subjective health perception' and the visual analogue scale (rS = 0.744, p = 0.01) in adolescent patients older than 13 years. CONCLUSION: The EQ-5D-Y can be considered a cross-sectional valid generic health outcome instrument which reflects differences in health according to the progression of the life-long chronic disease cystic fibrosis.