Enzyme replacement therapy and renal function in 201 patients with Fabry disease

A. Schwarting; F. Dehout; S. Feriozzi; M. Beck; A. Mehta; Gere Sunder-Plassmann; O. Bodamer; A. C. Hauser; J. Kleinert; C. Binder; P. Kotanko; T. Kroepfl; B. Plecko; G. Clerbaux; B. Georges; M. C. Nassogne; Y. Pirson; D. Roland; L. Van Maldergem; P. Goyens; F. Eyskens; J. Bultas; D. Karetová; A. Linhart; J. C. Lubanda; S. Magage; G. Choukroun; J. Berthelot; S. Carey Reomonnay; D. Lacombe; S. Benziane; E. Hachulla; B. Dussol; P. Jaeger; D. Germain; O. Lidove; R. Jaussaud; D. Caraman; A. von Arnim-Baas; J. Hennermann; B. Hoffmann; H. P.H. Neumann; A. Das; S. Illsinger; K. Baron; S. Delgado-Sanchez; R. Hartung; C. Kampmann; C. Whybra; B. Koletzko; T. Böttcher; A. Rolfs; O. Gabrielli; I. F. Salvatori; D. Concolino; P. Strisciuglio; G. Vega; W. Borsini; S. Buchner; R. Parini; R. Ravaglia; S. Santus; R. Di Vito; A. Burlina; G. Tognana; D. Antuzzi; N. Castorina; M. Di Lillo; S. Feriozzi; R. Ricci; G. Houge; L. M. Lægreid; N. Strømsvik; E. Svarstad; C. Tøndel; A. Skarbøvik; A. B. Tafjord; M. A. Barba; E. Gómez Huertas; J. Herrera; J. Ara; J. Bonal; E. Larrousse; G. Pintos; J. Ballarin; R. Torra; J. Torras; V. Torregrosa; J. González; M. Garcia; C. Herrera; I. Martin; J. Rodriguez; F. J. Barbado; J. Garcia-Consuegra; J. García de Lorenzo; M. López; J. Paniagua; S. Hernández

Enzyme replacement therapy and renal function in 201 patients with Fabry disease

A. Schwarting, F. Dehout, S. Feriozzi, M. Beck, A. Mehta, Gere Sunder-Plassmann, O. Bodamer, A. C. Hauser, J. Kleinert, C. Binder, P. Kotanko, T. Kroepfl, B. Plecko, G. Clerbaux, B. Georges, M. C. Nassogne, Y. Pirson, D. Roland, L. Van Maldergem, P. GoyensF. Eyskens, J. Bultas, D. Karetová, A. Linhart, J. C. Lubanda, S. Magage, G. Choukroun, J. Berthelot, S. Carey Reomonnay, D. Lacombe, S. Benziane, E. Hachulla, B. Dussol, P. Jaeger, D. Germain, O. Lidove, R. Jaussaud, D. Caraman, A. von Arnim-Baas, J. Hennermann, B. Hoffmann, H. P.H. Neumann, A. Das, S. Illsinger, K. Baron, S. Delgado-Sanchez, R. Hartung, C. Kampmann, C. Whybra, B. Koletzko, T. Böttcher, A. Rolfs, O. Gabrielli, I. F. Salvatori, D. Concolino, P. Strisciuglio, G. Vega, W. Borsini, S. Buchner, R. Parini, R. Ravaglia, S. Santus, R. Di Vito, A. Burlina, G. Tognana, D. Antuzzi, N. Castorina, M. Di Lillo, S. Feriozzi, R. Ricci, G. Houge, L. M. Lægreid, N. Strømsvik, E. Svarstad, C. Tøndel, A. Skarbøvik, A. B. Tafjord, M. A. Barba, E. Gómez Huertas, J. Herrera, J. Ara, J. Bonal, E. Larrousse, G. Pintos, J. Ballarin, R. Torra, J. Torras, V. Torregrosa, J. González, M. Garcia, C. Herrera, I. Martin, J. Rodriguez, F. J. Barbado, J. Garcia-Consuegra, J. García de Lorenzo, M. López, J. Paniagua, S. Hernández

Human Genetics

Research output: Contribution to journal › Article

74 Scopus citations

Abstract

Aim: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of α-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in end-stage renal disease. We examined the effects of enzyme replacement therapy (ERT) with A galsidase-α on renal function using data from a large international database, the Fabry Outcome Survey (FOS). Methods: This analysis was based on 1,040 serum creatinine measurements in 201 patients with Fabry disease, aged 20 - 60 years, with serum creatinine concentrations of less than 2 mg/dl and duration of ERT of up to 4.7 years. Both pretreatment and treatment data were used to examine independent predictors of changes in serum creatinine. In a second approach longitudinal serum creatinine measurements from 1 year before treatment, at baseline and 1 and 2 years after the start of treatment were analyzed in 20 patients with chronic kidney disease (CKD) Stage 2 and 3. Results: We found an independent negative association between serum creatinine and time on Agalsidase-α treatment (p < 0.05). Renal function declined significantly (p < 0.05) in the year before treatment. After 1 year of treatment, however, the decline in estimated glomerular filtration rate had been halted, and renal function was preserved for up to 2 years. Conclusions: In conclusion, ERT with Agalsidase-α is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.

Original language	English (US)
Pages (from-to)	77-84
Number of pages	8
Journal	Clinical Nephrology
Volume	66
Issue number	2
State	Published - Aug 2006

Keywords

Chronic kidney disease
Creatinine
Enzyme replacement therapy
Fabry disease
Fabry Outcome Survery
Glomerular filtration rate

ASJC Scopus subject areas

Nephrology

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Schwarting, A., Dehout, F., Feriozzi, S., Beck, M., Mehta, A., Sunder-Plassmann, G., Bodamer, O., Hauser, A. C., Kleinert, J., Binder, C., Kotanko, P., Kroepfl, T., Plecko, B., Clerbaux, G., Georges, B., Nassogne, M. C., Pirson, Y., Roland, D., Van Maldergem, L., ... Hernández, S. (2006). Enzyme replacement therapy and renal function in 201 patients with Fabry disease. Clinical Nephrology, 66(2), 77-84.

Enzyme replacement therapy and renal function in 201 patients with Fabry disease. / Schwarting, A.; Dehout, F.; Feriozzi, S.; Beck, M.; Mehta, A.; Sunder-Plassmann, Gere; Bodamer, O.; Hauser, A. C.; Kleinert, J.; Binder, C.; Kotanko, P.; Kroepfl, T.; Plecko, B.; Clerbaux, G.; Georges, B.; Nassogne, M. C.; Pirson, Y.; Roland, D.; Van Maldergem, L.; Goyens, P.; Eyskens, F.; Bultas, J.; Karetová, D.; Linhart, A.; Lubanda, J. C.; Magage, S.; Choukroun, G.; Berthelot, J.; Carey Reomonnay, S.; Lacombe, D.; Benziane, S.; Hachulla, E.; Dussol, B.; Jaeger, P.; Germain, D.; Lidove, O.; Jaussaud, R.; Caraman, D.; von Arnim-Baas, A.; Hennermann, J.; Hoffmann, B.; Neumann, H. P.H.; Das, A.; Illsinger, S.; Baron, K.; Delgado-Sanchez, S.; Hartung, R.; Kampmann, C.; Whybra, C.; Koletzko, B.; Böttcher, T.; Rolfs, A.; Gabrielli, O.; Salvatori, I. F.; Concolino, D.; Strisciuglio, P.; Vega, G.; Borsini, W.; Buchner, S.; Parini, R.; Ravaglia, R.; Santus, S.; Di Vito, R.; Burlina, A.; Tognana, G.; Antuzzi, D.; Castorina, N.; Di Lillo, M.; Feriozzi, S.; Ricci, R.; Houge, G.; Lægreid, L. M.; Strømsvik, N.; Svarstad, E.; Tøndel, C.; Skarbøvik, A.; Tafjord, A. B.; Barba, M. A.; Gómez Huertas, E.; Herrera, J.; Ara, J.; Bonal, J.; Larrousse, E.; Pintos, G.; Ballarin, J.; Torra, R.; Torras, J.; Torregrosa, V.; González, J.; Garcia, M.; Herrera, C.; Martin, I.; Rodriguez, J.; Barbado, F. J.; Garcia-Consuegra, J.; García de Lorenzo, J.; López, M.; Paniagua, J.; Hernández, S.

In: Clinical Nephrology, Vol. 66, No. 2, 08.2006, p. 77-84.

Research output: Contribution to journal › Article

Schwarting, A, Dehout, F, Feriozzi, S, Beck, M, Mehta, A, Sunder-Plassmann, G, Bodamer, O, Hauser, AC, Kleinert, J, Binder, C, Kotanko, P, Kroepfl, T, Plecko, B, Clerbaux, G, Georges, B, Nassogne, MC, Pirson, Y, Roland, D, Van Maldergem, L, Goyens, P, Eyskens, F, Bultas, J, Karetová, D, Linhart, A, Lubanda, JC, Magage, S, Choukroun, G, Berthelot, J, Carey Reomonnay, S, Lacombe, D, Benziane, S, Hachulla, E, Dussol, B, Jaeger, P, Germain, D, Lidove, O, Jaussaud, R, Caraman, D, von Arnim-Baas, A, Hennermann, J, Hoffmann, B, Neumann, HPH, Das, A, Illsinger, S, Baron, K, Delgado-Sanchez, S, Hartung, R, Kampmann, C, Whybra, C, Koletzko, B, Böttcher, T, Rolfs, A, Gabrielli, O, Salvatori, IF, Concolino, D, Strisciuglio, P, Vega, G, Borsini, W, Buchner, S, Parini, R, Ravaglia, R, Santus, S, Di Vito, R, Burlina, A, Tognana, G, Antuzzi, D, Castorina, N, Di Lillo, M, Feriozzi, S, Ricci, R, Houge, G, Lægreid, LM, Strømsvik, N, Svarstad, E, Tøndel, C, Skarbøvik, A, Tafjord, AB, Barba, MA, Gómez Huertas, E, Herrera, J, Ara, J, Bonal, J, Larrousse, E, Pintos, G, Ballarin, J, Torra, R, Torras, J, Torregrosa, V, González, J, Garcia, M, Herrera, C, Martin, I, Rodriguez, J, Barbado, FJ, Garcia-Consuegra, J, García de Lorenzo, J, López, M, Paniagua, J & Hernández, S 2006, 'Enzyme replacement therapy and renal function in 201 patients with Fabry disease', Clinical Nephrology, vol. 66, no. 2, pp. 77-84.

Schwarting, A. ; Dehout, F. ; Feriozzi, S. ; Beck, M. ; Mehta, A. ; Sunder-Plassmann, Gere ; Bodamer, O. ; Hauser, A. C. ; Kleinert, J. ; Binder, C. ; Kotanko, P. ; Kroepfl, T. ; Plecko, B. ; Clerbaux, G. ; Georges, B. ; Nassogne, M. C. ; Pirson, Y. ; Roland, D. ; Van Maldergem, L. ; Goyens, P. ; Eyskens, F. ; Bultas, J. ; Karetová, D. ; Linhart, A. ; Lubanda, J. C. ; Magage, S. ; Choukroun, G. ; Berthelot, J. ; Carey Reomonnay, S. ; Lacombe, D. ; Benziane, S. ; Hachulla, E. ; Dussol, B. ; Jaeger, P. ; Germain, D. ; Lidove, O. ; Jaussaud, R. ; Caraman, D. ; von Arnim-Baas, A. ; Hennermann, J. ; Hoffmann, B. ; Neumann, H. P.H. ; Das, A. ; Illsinger, S. ; Baron, K. ; Delgado-Sanchez, S. ; Hartung, R. ; Kampmann, C. ; Whybra, C. ; Koletzko, B. ; Böttcher, T. ; Rolfs, A. ; Gabrielli, O. ; Salvatori, I. F. ; Concolino, D. ; Strisciuglio, P. ; Vega, G. ; Borsini, W. ; Buchner, S. ; Parini, R. ; Ravaglia, R. ; Santus, S. ; Di Vito, R. ; Burlina, A. ; Tognana, G. ; Antuzzi, D. ; Castorina, N. ; Di Lillo, M. ; Feriozzi, S. ; Ricci, R. ; Houge, G. ; Lægreid, L. M. ; Strømsvik, N. ; Svarstad, E. ; Tøndel, C. ; Skarbøvik, A. ; Tafjord, A. B. ; Barba, M. A. ; Gómez Huertas, E. ; Herrera, J. ; Ara, J. ; Bonal, J. ; Larrousse, E. ; Pintos, G. ; Ballarin, J. ; Torra, R. ; Torras, J. ; Torregrosa, V. ; González, J. ; Garcia, M. ; Herrera, C. ; Martin, I. ; Rodriguez, J. ; Barbado, F. J. ; Garcia-Consuegra, J. ; García de Lorenzo, J. ; López, M. ; Paniagua, J. ; Hernández, S. / Enzyme replacement therapy and renal function in 201 patients with Fabry disease. In: Clinical Nephrology. 2006 ; Vol. 66, No. 2. pp. 77-84.

@article{ef94a17061c1401aafd9c368fe950784,

title = "Enzyme replacement therapy and renal function in 201 patients with Fabry disease",

abstract = "Aim: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of α-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in end-stage renal disease. We examined the effects of enzyme replacement therapy (ERT) with A galsidase-α on renal function using data from a large international database, the Fabry Outcome Survey (FOS). Methods: This analysis was based on 1,040 serum creatinine measurements in 201 patients with Fabry disease, aged 20 - 60 years, with serum creatinine concentrations of less than 2 mg/dl and duration of ERT of up to 4.7 years. Both pretreatment and treatment data were used to examine independent predictors of changes in serum creatinine. In a second approach longitudinal serum creatinine measurements from 1 year before treatment, at baseline and 1 and 2 years after the start of treatment were analyzed in 20 patients with chronic kidney disease (CKD) Stage 2 and 3. Results: We found an independent negative association between serum creatinine and time on Agalsidase-α treatment (p < 0.05). Renal function declined significantly (p < 0.05) in the year before treatment. After 1 year of treatment, however, the decline in estimated glomerular filtration rate had been halted, and renal function was preserved for up to 2 years. Conclusions: In conclusion, ERT with Agalsidase-α is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.",

keywords = "Chronic kidney disease, Creatinine, Enzyme replacement therapy, Fabry disease, Fabry Outcome Survery, Glomerular filtration rate",

author = "A. Schwarting and F. Dehout and S. Feriozzi and M. Beck and A. Mehta and Gere Sunder-Plassmann and O. Bodamer and Hauser, {A. C.} and J. Kleinert and C. Binder and P. Kotanko and T. Kroepfl and B. Plecko and G. Clerbaux and B. Georges and Nassogne, {M. C.} and Y. Pirson and D. Roland and {Van Maldergem}, L. and P. Goyens and F. Eyskens and J. Bultas and D. Karetov{\'a} and A. Linhart and Lubanda, {J. C.} and S. Magage and G. Choukroun and J. Berthelot and {Carey Reomonnay}, S. and D. Lacombe and S. Benziane and E. Hachulla and B. Dussol and P. Jaeger and D. Germain and O. Lidove and R. Jaussaud and D. Caraman and {von Arnim-Baas}, A. and J. Hennermann and B. Hoffmann and Neumann, {H. P.H.} and A. Das and S. Illsinger and K. Baron and S. Delgado-Sanchez and R. Hartung and C. Kampmann and C. Whybra and B. Koletzko and T. B{\"o}ttcher and A. Rolfs and O. Gabrielli and Salvatori, {I. F.} and D. Concolino and P. Strisciuglio and G. Vega and W. Borsini and S. Buchner and R. Parini and R. Ravaglia and S. Santus and {Di Vito}, R. and A. Burlina and G. Tognana and D. Antuzzi and N. Castorina and {Di Lillo}, M. and S. Feriozzi and R. Ricci and G. Houge and L{\ae}greid, {L. M.} and N. Str{\o}msvik and E. Svarstad and C. T{\o}ndel and A. Skarb{\o}vik and Tafjord, {A. B.} and Barba, {M. A.} and {G{\'o}mez Huertas}, E. and J. Herrera and J. Ara and J. Bonal and E. Larrousse and G. Pintos and J. Ballarin and R. Torra and J. Torras and V. Torregrosa and J. Gonz{\'a}lez and M. Garcia and C. Herrera and I. Martin and J. Rodriguez and Barbado, {F. J.} and J. Garcia-Consuegra and {Garc{\'i}a de Lorenzo}, J. and M. L{\'o}pez and J. Paniagua and S. Hern{\'a}ndez",

year = "2006",

month = aug,

language = "English (US)",

volume = "66",

pages = "77--84",

journal = "Clinical Nephrology",

issn = "0301-0430",

publisher = "Dustri-Verlag Dr. Karl Feistle",

number = "2",

}

TY - JOUR

T1 - Enzyme replacement therapy and renal function in 201 patients with Fabry disease

AU - Schwarting, A.

AU - Dehout, F.

AU - Feriozzi, S.

AU - Beck, M.

AU - Mehta, A.

AU - Sunder-Plassmann, Gere

AU - Bodamer, O.

AU - Hauser, A. C.

AU - Kleinert, J.

AU - Binder, C.

AU - Kotanko, P.

AU - Kroepfl, T.

AU - Plecko, B.

AU - Clerbaux, G.

AU - Georges, B.

AU - Nassogne, M. C.

AU - Pirson, Y.

AU - Roland, D.

AU - Van Maldergem, L.

AU - Goyens, P.

AU - Eyskens, F.

AU - Bultas, J.

AU - Karetová, D.

AU - Linhart, A.

AU - Lubanda, J. C.

AU - Magage, S.

AU - Choukroun, G.

AU - Berthelot, J.

AU - Carey Reomonnay, S.

AU - Lacombe, D.

AU - Benziane, S.

AU - Hachulla, E.

AU - Dussol, B.

AU - Jaeger, P.

AU - Germain, D.

AU - Lidove, O.

AU - Jaussaud, R.

AU - Caraman, D.

AU - von Arnim-Baas, A.

AU - Hennermann, J.

AU - Hoffmann, B.

AU - Neumann, H. P.H.

AU - Das, A.

AU - Illsinger, S.

AU - Baron, K.

AU - Delgado-Sanchez, S.

AU - Hartung, R.

AU - Kampmann, C.

AU - Whybra, C.

AU - Koletzko, B.

AU - Böttcher, T.

AU - Rolfs, A.

AU - Gabrielli, O.

AU - Salvatori, I. F.

AU - Concolino, D.

AU - Strisciuglio, P.

AU - Vega, G.

AU - Borsini, W.

AU - Buchner, S.

AU - Parini, R.

AU - Ravaglia, R.

AU - Santus, S.

AU - Di Vito, R.

AU - Burlina, A.

AU - Tognana, G.

AU - Antuzzi, D.

AU - Castorina, N.

AU - Di Lillo, M.

AU - Feriozzi, S.

AU - Ricci, R.

AU - Houge, G.

AU - Lægreid, L. M.

AU - Strømsvik, N.

AU - Svarstad, E.

AU - Tøndel, C.

AU - Skarbøvik, A.

AU - Tafjord, A. B.

AU - Barba, M. A.

AU - Gómez Huertas, E.

AU - Herrera, J.

AU - Ara, J.

AU - Bonal, J.

AU - Larrousse, E.

AU - Pintos, G.

AU - Ballarin, J.

AU - Torra, R.

AU - Torras, J.

AU - Torregrosa, V.

AU - González, J.

AU - Garcia, M.

AU - Herrera, C.

AU - Martin, I.

AU - Rodriguez, J.

AU - Barbado, F. J.

AU - Garcia-Consuegra, J.

AU - García de Lorenzo, J.

AU - López, M.

AU - Paniagua, J.

AU - Hernández, S.

PY - 2006/8

Y1 - 2006/8

N2 - Aim: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of α-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in end-stage renal disease. We examined the effects of enzyme replacement therapy (ERT) with A galsidase-α on renal function using data from a large international database, the Fabry Outcome Survey (FOS). Methods: This analysis was based on 1,040 serum creatinine measurements in 201 patients with Fabry disease, aged 20 - 60 years, with serum creatinine concentrations of less than 2 mg/dl and duration of ERT of up to 4.7 years. Both pretreatment and treatment data were used to examine independent predictors of changes in serum creatinine. In a second approach longitudinal serum creatinine measurements from 1 year before treatment, at baseline and 1 and 2 years after the start of treatment were analyzed in 20 patients with chronic kidney disease (CKD) Stage 2 and 3. Results: We found an independent negative association between serum creatinine and time on Agalsidase-α treatment (p < 0.05). Renal function declined significantly (p < 0.05) in the year before treatment. After 1 year of treatment, however, the decline in estimated glomerular filtration rate had been halted, and renal function was preserved for up to 2 years. Conclusions: In conclusion, ERT with Agalsidase-α is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.

AB - Aim: Fabry disease is a rare lysosomal storage disorder caused by deficient activity of α-galactosidase A, resulting in progressive cellular accumulation of glycolipids, which may ultimately result in end-stage renal disease. We examined the effects of enzyme replacement therapy (ERT) with A galsidase-α on renal function using data from a large international database, the Fabry Outcome Survey (FOS). Methods: This analysis was based on 1,040 serum creatinine measurements in 201 patients with Fabry disease, aged 20 - 60 years, with serum creatinine concentrations of less than 2 mg/dl and duration of ERT of up to 4.7 years. Both pretreatment and treatment data were used to examine independent predictors of changes in serum creatinine. In a second approach longitudinal serum creatinine measurements from 1 year before treatment, at baseline and 1 and 2 years after the start of treatment were analyzed in 20 patients with chronic kidney disease (CKD) Stage 2 and 3. Results: We found an independent negative association between serum creatinine and time on Agalsidase-α treatment (p < 0.05). Renal function declined significantly (p < 0.05) in the year before treatment. After 1 year of treatment, however, the decline in estimated glomerular filtration rate had been halted, and renal function was preserved for up to 2 years. Conclusions: In conclusion, ERT with Agalsidase-α is associated with decrease of serum creatinine and may prevent the deterioration of renal function in patients with Fabry disease.

KW - Chronic kidney disease

KW - Creatinine

KW - Enzyme replacement therapy

KW - Fabry disease

KW - Fabry Outcome Survery

KW - Glomerular filtration rate

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JF - Clinical Nephrology

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