Advertisement

Social/economic costs and health-related quality of life in patients with histiocytosis in Europe

  • Georgi IskrovEmail author
  • Itziar Astigarraga
  • Rumen Stefanov
  • Julio López-Bastida
  • Renata Linertová
  • Juan Oliva-Moreno
  • Pedro Serrano-Aguilar
  • Manuel Posada-de-la-Paz
  • Arrigo Schieppati
  • Domenica Taruscio
  • Márta Péntek
  • Johann Matthias Graf von der Schulenburg
  • Panos Kanavos
  • Karine Chevreul
  • Ulf Persson
  • Giovanni Fattore
  • BURQOL-RD Research Network
Original Paper

Abstract

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with histiocytosis in Europe.

Methods

We conducted a cross-sectional study of patients with histiocytosis from France, Germany, Italy, Spain, Bulgaria, the UK, and Sweden. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.

Results

A total of 134 patients (35 France, 32 Germany, 30 Italy, 24 Spain, 7 Bulgaria, 4 UK and 2 Sweden) completed the questionnaire. The average annual costs ranged from € 6832 to € 33,283 between countries, the year of reference being 2012. Estimated direct healthcare costs ranged from € 1698 to € 18,213; direct nonhealthcare costs ranged from € 2936 to € 17,622 and labour productivity losses ranged from € 1 to € 8855. The mean EQ-5D score for adult histiocytosis patients was estimated at between 0.32 and 0.85, and the mean EQ-5D visual analogue scale score was estimated at between 50.00 and 66.50.

Conclusion

The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of histiocytosis patients from a broad perspective (societal costs). This type of analysis is very scarce in international literature for rare diseases in comparison with other illnesses. We conclude that histiocytosis patients incur considerable societal costs and experience substantial deterioration in HRQOL.

Keywords

Histiocytosis Cost-of-illness Social cost Health-related quality of life Europe 

Notes

Acknowledgments

The authors wish to thank: National Alliance of People with Rare Diseases (NAPRD), Bulgaria; Alliance Maladies Rares, France; ACHSE, Germany; Hungarian Federation of People with Rare and Congenital Diseases (RIROSZ), Hungary; Federazione Italiana Malattie Rare (UNIAMO), Italy; Consulta Nazionale delle Malattie Rare, Italy; AIRI LCH Onlus - Associazione Italiana Ricerca Istiocitos, Italyi; Rare Diseases Sweden; Federación Española de Efermedades Raras (FEDER), Spain; Rare Disease UK; Euro-Histio-Net and Rare Diseases Europe (EURORDIS); Euro-Histio-Net; Association Histiocytose France; Histiozytosehilfe e.V., Germany; AIRI LCH Onlus– Associazione Italiana Ricerca Istiocitosi, Italy; Föräldraföreningen för barn med Histiocytos, Sweden; Asociación Española contra la Histiocitosis, Spain.

Compliance with ethical standards

Funding

Supported by the Social Economic Burden and Health-Related Quality of Life in Patients with Rare Diseases in Europe Project, which has received funding from the European Union in the framework of the Health Programme [grant A101205]. The Executive Agency of the European Union is not responsible for any use that may be made of the information contained here.

Conflict of interest

The authors declare that they have no conflicts of interest.

Supplementary material

10198_2016_790_MOESM1_ESM.docx (16 kb)
Supplementary material 1 (DOCX 16 kb)
10198_2016_790_MOESM2_ESM.doc (41 kb)
Supplementary material 2 (DOC 41 kb)

References

  1. 1.
    Hervier, B., Haroche, J., Arnaud, L., Charlotte, F., Donadieu, J., et al.: Association of both Langerhans cell histiocytosis and Erdheim-Chester disease linked to the BRAFV600E mutation. Blood 124, 1119–1126 (2014)CrossRefPubMedGoogle Scholar
  2. 2.
    Histiocytosis Association: LCH in Children. http://www.histio.org/lchinchildren#.VJkyssgA. Accessed 23 December 2014
  3. 3.
    Stålemark, H., Laurencikas, E., Karis, J., Gavhed, D., Fadeel, B., et al.: Incidence of Langerhans cell histiocytosis in children: a population-based study. Pediatr. Blood Cancer 51, 76–81 (2008)CrossRefPubMedGoogle Scholar
  4. 4.
    Mataix, J., Betlloch, I.: Langerhans cell histiocytosis: an update. G. Ital. Dermatol. Venereol. 144, 119–134 (2009)PubMedGoogle Scholar
  5. 5.
    Morimoto, A., Oh, Y., Shioda, Y., Kudo, K., Imamura, T.: Recent advances in Langerhans cell histiocytosis. Pediatr. Int. 56, 451–461 (2014)CrossRefPubMedGoogle Scholar
  6. 6.
    Wilejto, M., Abla, O.: Langerhans cell histiocytosis and Erdheim-Chester disease. Curr. Opin. Rheumatol. 24, 90–96 (2012)CrossRefPubMedGoogle Scholar
  7. 7.
    DiCaprio, M.R., Roberts, T.T.: Diagnosis and Management of Langerhans Cell Histiocytosis. J. Am. Acad. Orthop. Surg. 22, 643–652 (2014)CrossRefPubMedGoogle Scholar
  8. 8.
    Martin, A., Macmillan, S., Murphy, D., Carachi, R.: Langerhans cell histiocytosis: 23 years’ paediatric experience highlights severe long-term sequelae. Scott. Med. J. 59, 149–157 (2014)CrossRefPubMedGoogle Scholar
  9. 9.
    Aricò, M., Girschikofsky, M., Généreau, T., Klersy, C., McClain, K., et al.: Langerhans cell histiocytosis in adults. Report from the International Registry of the Histiocyte Society. Eur. J. Cancer 39, 2341–2348 (2003)CrossRefPubMedGoogle Scholar
  10. 10.
    Girschikofsky, M., Arico, M., Castillo, D., Chu, A., Doberauer, C., et al.: Management of adult patients with Langerhans cell histiocytosis: recommendations from an expert panel on behalf of Euro-Histio-Net. Orphanet. J. Rare Dis. (2013). doi: 10.1186/1750-1172-8-72 PubMedPubMedCentralGoogle Scholar
  11. 11.
    Linertová, R., Serrano-Aguilar, P., Posada de la Paz, M., Hens-Pérez, M., Kanavos, P., et al.: Delphi approach to select rare diseases for a European representative survey. The BURQOL-RD study. Health Policy 108, 19–26 (2012)CrossRefPubMedGoogle Scholar
  12. 12.
    Brooks, R.: EuroQol: the current state of play. Health Policy 37, 53–72 (1996)CrossRefPubMedGoogle Scholar
  13. 13.
    Drummond, M.F., O’Brien, B., Stoddart, G.L., Torrance, G.W.: Methods for the economic evaluation of health care programmes, 2nd edn. Oxford University Press, Oxford (1997)Google Scholar
  14. 14.
    McDaid, D.: Estimating the costs of informal care for people with Alzheimer’s disease: methodological and practical challenges. Int. J. Geriatr. Psychiatry 16, 400–405 (2001)CrossRefPubMedGoogle Scholar
  15. 15.
    Van den Berg, B., Brouwer, W., Koopmanschap, M.: Economic valuation of informal care: an overview of methods and applications. Eur. J. Health Econ 5, 36–45 (2004)CrossRefPubMedGoogle Scholar
  16. 16.
    Hodgson, T.A., Meiners, M.R.: Cost-of-illness methodology: a guide to assessment practices and procedures. Milbank Mem Fund Q 60, 429–491 (1982)CrossRefGoogle Scholar
  17. 17.
    Dolan, P.: Modeling valuations for EuroQol health states. Med. Care 35, 1095–1108 (1997)CrossRefPubMedGoogle Scholar
  18. 18.
    Mahoney, F.I., Barthel, D.W.: Functional evaluation: the Barthel Index. Md State Med 14, 61–65 (1965)Google Scholar
  19. 19.
    Shah, S., Vanclay, F., Cooper, B.: Improving the sensitivity of the Barthel Index for stroke rehabilitation. J. Clin. Epidemiol. 42, 703–709 (1989)CrossRefPubMedGoogle Scholar
  20. 20.
    Hérbert, R., Bravo, G., Préville, M.: Reliability, validity, and reference values of the Zarit Burden Interview for assessing informal caregivers of community-dwelling older persons with dementia. Can J Aging 19, 494–507 (2000)CrossRefGoogle Scholar
  21. 21.
    Imrie, J., Galani, C., Gairy, K., Lock, K., Hunsche, E.: Cost of illness associated with Niemann-Pick disease type C in the UK. J Med. Econ 12, 219–229 (2009)CrossRefPubMedGoogle Scholar
  22. 22.
    Klein, A.D., Alvarez, A., Zanlungo, S.: The unique case of the Niemann-Pick type C cholesterol storage disorder. Pediatr. Endocrinol. Rev. 12, 166–175 (2014)PubMedGoogle Scholar
  23. 23.
    Kirchhoff, A.C., Fluchel, M.N., Wright, J., Ying, J., Sweeney, C., et al.: Risk of hospitalization for survivors of childhood and adolescent cancer. Cancer Epidemiol Biomarkers Prev 23, 1280–1289 (2014)CrossRefPubMedGoogle Scholar
  24. 24.
    Audino, A.N., Yeager, N.D., Asti, L., Miao, Y., O’Brien, S.H.: Length of stay and treatment-related complications are similar in pediatric and AYA patients with bone sarcoma in United States children’s hospitals. Pediatr. Blood Cancer 60, 415–419 (2012)CrossRefPubMedGoogle Scholar
  25. 25.
    Warner, E.L., Kirchhoff, A.C., Nam, G.E., Fluchel, M.: Financial Burden of Pediatric Cancer for Patients and Their Families. J Oncol Pract (2014)Google Scholar
  26. 26.
    Wakefield, C.E., McLoone, J.K., Evans, N.T., Ellis, S.J., Cohn, R.J.: It’s more than dollars and cents: the impact of childhood cancer on parents’ occupational and financial health. J. Psychosoc. Oncol. 32, 602–621 (2014)CrossRefPubMedGoogle Scholar
  27. 27.
    Fluchel, M.N., Kirchhoff, A.C., Bodson, J., Sweeney, C., Edwards, S.L.: Geography and the burden of care in pediatric cancers. Pediatr. Blood Cancer 61, 1918–1924 (2014)CrossRefPubMedPubMedCentralGoogle Scholar
  28. 28.
    Kirchhoff, A.C., Lyles, C.R., Fluchel, M., Wright, J., Leisenring, W.: Limitations in health care access and utilization among long-term survivors of adolescent and young adult cancer. Cancer 118, 5964–5972 (2012)CrossRefPubMedGoogle Scholar
  29. 29.
    Dussel, V., Bona, K., Heath, J.A., Hilden, J.M., Weeks, J.C., et al.: Unmeasured costs of a child’s death: perceived financial burden, work disruptions, and economic coping strategies used by American and Australian families who lost children to cancer. J. Clin. Oncol. 29, 1007–1013 (2011)CrossRefPubMedGoogle Scholar
  30. 30.
    Vrijmoet-Wiersma, C.M., Kooloos, V.M., Koopman, H.M., Kolk, A.M., van der Laan, I., et al.: Health-related quality of life, cognitive functioning and behaviour problems in children with Langerhans cell histiocytosis. Pediatr. Blood Cancer 52, 116–122 (2009)CrossRefPubMedGoogle Scholar
  31. 31.
    Nanduri, V.R., Pritchard, J., Levitt, G., Glaser, A.W.: Long term morbidity and health related quality of life after multi-system Langerhans cell histiocytosis. Eur. J. Cancer 42, 2563–2569 (2006)CrossRefPubMedGoogle Scholar
  32. 32.
    Lau, L.M., Stuurman, K., Weitzman, S.: Skeletal Langerhans cell histiocytosis in children: permanent consequences and health-related quality of life in long-term survivors. Pediatr. Blood Cancer 50, 607–612 (2008)CrossRefPubMedGoogle Scholar
  33. 33.
    Laurencikas, E., Gavhed, D., Stålemark, H., van’t Hooft, I., Prayer, D., et al.: Incidence and pattern of radiological central nervous system Langerhans cell histiocytosis in children: a population based study. Pediatr. Blood Cancer 56, 250–257 (2011)CrossRefPubMedGoogle Scholar
  34. 34.
    Vassallo, R., Ryu, J.H., Schroeder, D.R., Decker, P.A., Limper, A.H.: Clinical outcomes of pulmonary Langerhans’-cell histiocytosis in adults. N. Engl. J. Med. 346, 484–490 (2002)CrossRefPubMedGoogle Scholar
  35. 35.
    Koopman, H.M., Koetsier, J.A., Taminiau, A.H., Hijnen, K.E., Bresters, D., et al.: Health-related quality of life and coping strategies of children after treatment of a malignant bone tumor: a 5-year follow-up study. Pediatr. Blood Cancer 45, 694–699 (2005)CrossRefPubMedGoogle Scholar
  36. 36.
    Simms, S., Warner, N.J.: A framework for understanding and responding to the psychosocial needs of children with Langerhans cell histiocytosis and their families. Hematol. Oncol. Clin. North Am. 12, 359–367 (1998)CrossRefPubMedGoogle Scholar
  37. 37.
    Mason, R.H., Foley, N.M., Branley, H.M., Adamali, H.I., Hetzel, M., et al.: Pulmonary Langerhans cell histiocytosis (PLCH): a new UK register. Thorax 69, 766–767 (2014)CrossRefPubMedGoogle Scholar
  38. 38.
    Taruscio, D., Gentile, A.E., Evangelista, T., Frazzica, R.G., Bushby, K., et al.: Centres of Expertise and European Reference Networks: key issues in the field of rare diseases. The EUCERD Recommendations. Blood Transfus Suppl 3, 621–625 (2014)Google Scholar
  39. 39.
    Suri, H.S., Yi, E.S., Nowakowski, G.S., Vassallo, R.: Pulmonary langerhans cell histiocytosis. Orphanet. J. Rare Dis (2012). doi: 10.1186/1750-1172-7-16 PubMedPubMedCentralGoogle Scholar

Copyright information

© Springer-Verlag Berlin Heidelberg 2016

Authors and Affiliations

  • Georgi Iskrov
    • 1
    • 17
    Email author
  • Itziar Astigarraga
    • 2
  • Rumen Stefanov
    • 1
    • 17
  • Julio López-Bastida
    • 3
    • 4
  • Renata Linertová
    • 4
    • 5
  • Juan Oliva-Moreno
    • 4
    • 6
  • Pedro Serrano-Aguilar
    • 4
    • 7
  • Manuel Posada-de-la-Paz
    • 8
  • Arrigo Schieppati
    • 9
  • Domenica Taruscio
    • 10
  • Márta Péntek
    • 11
  • Johann Matthias Graf von der Schulenburg
    • 12
  • Panos Kanavos
    • 13
  • Karine Chevreul
    • 14
    • 18
    • 19
  • Ulf Persson
    • 15
  • Giovanni Fattore
    • 16
  • BURQOL-RD Research Network
  1. 1.Institute for Rare DiseasesPlovdivBulgaria
  2. 2.Department of Pediatrics, BioCruces Health Research InstituteHospital Universitario CrucesBizkaiaSpain
  3. 3.University of Castilla-La ManchaTalavera de la Reina, ToledoSpain
  4. 4.Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)MadridSpain
  5. 5.Fundación Canaria de Investigación Sanitaria (FUNCANIS)Las Palmas de Gran CanariaSpain
  6. 6.University of Castilla-La ManchaToledoSpain
  7. 7.Evaluation and Planning Service at Canary Islands Health ServiceSanta Cruz de TenerifeSpain
  8. 8.Institute of Rare Diseases ResearchISCIII, SpainRDR & CIBERERMadridSpain
  9. 9.Centro di Ricerche Cliniche per Malattie Rare Aldo e Cele DaccòIstituto di Ricerche Farmacologiche Mario NegriBergamoItaly
  10. 10.National Centre for Rare DiseasesIstituto Superiore di Sanità (ISS)RomeItaly
  11. 11.Department of Health EconomicsCorvinus University of BudapestBudapestHungary
  12. 12.Centre for Health Economics Research Hannover (CHERH)Leibniz Universität HannoverHannoverGermany
  13. 13.Department of Social Policy and LSE HealthLondon School of Economics and Political ScienceLondonUK
  14. 14.URC Eco Ile de FranceAP-HP, Hôtel DieuParisFrance
  15. 15.The Swedish Institute for Health EconomicsLundSweden
  16. 16.Centre for Research on Health and Social Care Management (CERGAS)Bocconi UniversityMilanItaly
  17. 17.Department of Social Medicine and Public Health, Faculty of Public HealthMedical University of PlovdivPlovdivBulgaria
  18. 18.Université Paris Diderot, Sorbonne Paris Cité, ECEVE, UMRS 1123ParisFrance
  19. 19.INSERM, ECEVE, U1123ParisFrance

Personalised recommendations