Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data.
Details
Serval ID
serval:BIB_10B71DA221AE
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data.
Journal
Lancet
Working group(s)
Fabry Outcome Survey investigators
Contributor(s)
Rozenfeld P., Nicholls K., Plecko B., Kotanko P., Binder C., Brunner-Krainz M., Sunder-Plassmann G., Kleinert J., Kristoferitsch W., Schreiber W., Eyskens F., Nassogne MC., Pirson Y., Goyens P., Libert M., Verellen-Dumoulin C., Dehout F., Roland D., Van Maldergem L., Mazoin N., Giugliani R., Casey R., Chan A., West M., Bichet D., Drouin R., Clarke J., Linhart A., Dostalova G., Choukroun G., Drobacheff MC., Goizet C., Bataille P., Aaron L., Boudet R., Benzaine S., Vuillemet F., Lorcerie B., Thevenot C., Hachulla E., Fouilhoux A., Dussol B., Reade R., Kaminsky P., Kuster A., Lino M., Ghafari T., Lidove O., Germain DP., Ouali N., Gaborieau V., Jaussaud R., Hardy P., Richalet B., Klotz V., Andres E., Labarthe F., Perrichot R., von Armin-Baas A., Hennermann J., Stolz S., Hoffmann B., Neumann HP., Gal A., Muschol N., Das A., Illsinger S., Haase C., Beck M., Schueuermann R., Koletzko B., Rolfs A., Rimmele F., Weis S., Wössner R., Wanner C., Maródi L., Altarescu G., Gabrielli O., Concolino D., Zoli G., Parini R., Di Vito R., Burlina A., Perticoni G., Feliciani C., Feriozzi S., Komatsuda A., Yabe I., Furusyo F., Katafuchi R., Maruyama S., Sato T., Kai Y., Furujyo M., Okada S., Mori T., Sugi Y., Ootake A., Satou K., Komamura K., Mori T., Furusyo F., Eto Y., Ueno Y., Hollak C., Wijburg F., Houge G., Vujkovac B., Zerjav-Tansek M., Barba MA., Franco A., Climent V., Arenas M., Del Pino MD. , Herrera J., Huertas£££E Gómez£££ EG. , Pintos G., Ibernon M., Torra R., Torregrosa V., González J., Mora A., Valverde V., Martin J., Del Bosque E., Pineda M., Prados MD., Torras J., Martin I., Hernández S., Barbado FJ., Rodriguez ML., Bosch T., Ruiz MA., de Toro JM. , Paniagua J., Tamargo LM., Fernández V., García AP., Febrer MI., Rivera A., Cabadés F., Ferrari P., Bonny O., Theytaz J., Huynh-Do Y., Barbey F., Theytaz J., Teta D., Ruggieri F., Magage S., Lubanda JC., Hendriksz C., Deegan P., Ramaswami U., Mehta A., Hughes D., Schiffmann R., Pastores G.
ISSN
1474-547X (Electronic)
ISSN-L
0140-6736
Publication state
Published
Issued date
2009
Peer-reviewed
Oui
Volume
374
Number
9706
Pages
1986-1996
Language
english
Notes
Publication types: Journal Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Publication Status: ppublish
Abstract
BACKGROUND: We analysed 5-year treatment with agalsidase alfa enzyme replacement therapy in patients with Fabry's disease who were enrolled in the Fabry Outcome Survey observational database (FOS).
METHODS: Baseline and 5-year data were available for up to 181 adults (126 men) in FOS. Serial data for cardiac mass and function, renal function, pain, and quality of life were assessed. Safety and sensitivity analyses were done in patients with baseline and at least one relevant follow-up measurement during the 5 years (n=555 and n=475, respectively).
FINDINGS: In patients with baseline cardiac hypertrophy, treatment resulted in a sustained reduction in left ventricular mass (LVM) index after 5 years (from 71.4 [SD 22.5] g/m(2.7) to 64.1 [18.7] g/m(2.7), p=0.0111) and a significant increase in midwall fractional shortening (MFS) from 14.3% (2.3) to 16.0% (3.8) after 3 years (p=0.02). In patients without baseline hypertrophy, LVM index and MFS remained stable. Mean yearly fall in estimated glomerular filtration rate versus baseline after 5 years of enzyme replacement therapy was -3.17 mL/min per 1.73 m(2) for men and -0.89 mL/min per 1.73 m(2) for women. Average pain, measured by Brief Pain Inventory score, improved significantly, from 3.7 (2.3) at baseline to 2.5 (2.4) after 5 years (p=0.0023). Quality of life, measured by deviation scores from normal EuroQol values, improved significantly, from -0.24 (0.3) at baseline to -0.17 (0.3) after 5 years (p=0.0483). Findings were confirmed by sensitivity analysis. No unexpected safety concerns were identified.
INTERPRETATION: By comparison with historical natural history data for patients with Fabry's disease who were not treated with enzyme replacement therapy, long-term treatment with agalsidase alfa leads to substantial and sustained clinical benefits.
FUNDING: Shire Human Genetic Therapies AB.
METHODS: Baseline and 5-year data were available for up to 181 adults (126 men) in FOS. Serial data for cardiac mass and function, renal function, pain, and quality of life were assessed. Safety and sensitivity analyses were done in patients with baseline and at least one relevant follow-up measurement during the 5 years (n=555 and n=475, respectively).
FINDINGS: In patients with baseline cardiac hypertrophy, treatment resulted in a sustained reduction in left ventricular mass (LVM) index after 5 years (from 71.4 [SD 22.5] g/m(2.7) to 64.1 [18.7] g/m(2.7), p=0.0111) and a significant increase in midwall fractional shortening (MFS) from 14.3% (2.3) to 16.0% (3.8) after 3 years (p=0.02). In patients without baseline hypertrophy, LVM index and MFS remained stable. Mean yearly fall in estimated glomerular filtration rate versus baseline after 5 years of enzyme replacement therapy was -3.17 mL/min per 1.73 m(2) for men and -0.89 mL/min per 1.73 m(2) for women. Average pain, measured by Brief Pain Inventory score, improved significantly, from 3.7 (2.3) at baseline to 2.5 (2.4) after 5 years (p=0.0023). Quality of life, measured by deviation scores from normal EuroQol values, improved significantly, from -0.24 (0.3) at baseline to -0.17 (0.3) after 5 years (p=0.0483). Findings were confirmed by sensitivity analysis. No unexpected safety concerns were identified.
INTERPRETATION: By comparison with historical natural history data for patients with Fabry's disease who were not treated with enzyme replacement therapy, long-term treatment with agalsidase alfa leads to substantial and sustained clinical benefits.
FUNDING: Shire Human Genetic Therapies AB.
Keywords
Adult, Fabry Disease/drug therapy, Fabry Disease/physiopathology, Female, Heart Function Tests, Humans, Isoenzymes/therapeutic use, Kidney Function Tests, Male, Pain Measurement, Quality of Life, Questionnaires, Registries, Treatment Outcome, alpha-Galactosidase/therapeutic use
Pubmed
Web of science
Create date
05/02/2010 11:53
Last modification date
23/11/2020 12:06
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