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Disease burden of spinal muscular atrophy in Germany


Klug, Constanze ; Schreiber-Katz, Olivia ; Thiele, Simone ; Schorling, Elisabeth ; Zowe, Janet ; Reilich, Peter ; Walter, Maggie C. ; Nagels, Klaus:
Disease burden of spinal muscular atrophy in Germany.
In: Orphanet Journal of Rare Diseases. Bd. 11 (2016) Heft 1 . - S. 58.
ISSN 1750-1172
DOI: https://doi.org/10.1186/s13023-016-0424-0


This study aimed at analyzing the economic burden and disease-specific health-related quality of life (HRQOL) of patients with spinal muscular atrophy (SMA) in Germany. SMA is a so far non-curable neuromuscular disease of the anterior nerve cells that causes high rates of morbidity and mortality.
In a cross-sectional study we analyzed the cost of illness (COI) and factors that influence the direct, indirect and informal care costs of affected patients and their families by using standardized, self-developed questionnaires. We used the PedsQL™(©) Measurement Model to analyze the disease-specific HRQOL of patients.
One hundred eighty nine patients with SMA types I to III aged <1 to 73 years were enrolled. The average annual COI was estimated at €70,566 per patient in 2013. The highest cost resulted in SMA I with significant lower costs for the milder phenotypes. Inversely, the self-estimated HRQOL increased from SMA I to SMA III. Major cost drivers were informal care cost and indirect cost incurred by patients and their caregivers.
Although SMA requires high standards of care, there has been a distinct lack of health services research on SMA. Accordingly, our results significantly contribute to a more comprehensive insight into the current burden of SMA and quality of life status as related to SMA health services in Germany. In the light of innovative therapeutic interventions, our results suggest a notable potential for a reduction in overall COI and improvement of HRQOL if the therapeutic intervention leads to a less severe course of the disease.

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Publikationsform: Artikel in einer Zeitschrift
Begutachteter Beitrag: Ja
Keywords: Cost of illness (COI); Direct cost; Health care burden; Health services research; Health-related quality of life (HRQOL); Indirect cost; Informal care cost; Neuromuscular disease; Spinal muscular atrophy (SMA)
Institutionen der Universität: Fakultäten > Rechts- und Wirtschaftswissenschaftliche Fakultät > Lehrstuhl Medizinmanagement und Versorgungsforschung > Lehrstuhl Medizinmanagement und Versorgungsforschung - Univ.-Prof. Dr. Klaus Nagels
Fakultäten > Rechts- und Wirtschaftswissenschaftliche Fakultät
Fakultäten > Rechts- und Wirtschaftswissenschaftliche Fakultät > Lehrstuhl Medizinmanagement und Versorgungsforschung
Titel an der UBT entstanden: Ja
Themengebiete aus DDC: 300 Sozialwissenschaften > 300 Sozialwissenschaften, Soziologie
300 Sozialwissenschaften > 330 Wirtschaft
600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
Eingestellt am: 08 Sep 2017 08:11
Letzte Änderung: 25 Sep 2017 14:59
URI: https://eref.uni-bayreuth.de/id/eprint/39406