Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa

Ozlem Goker-Alpan, Khan Nedd, Suma Shankar, Yeong Hau H. Lien, Neal Weinreb, Anna Wijatyk, Peter Chang, Rick Martin

Research output: Chapter in Book/Report/Conference proceedingChapter

7 Citations (Scopus)

Abstract

Objectives: In a multicenter, open-label, treatment protocol (HGT-REP-059; NCT01031173), clinical effects and tolerability of agalsidase alfa (agalα; 0.2 mg/kg every other week) were evaluated in patients with Fabry disease who were treatment naïve or switched from agalsidase beta (switch). Over 24 months, data were collected on the safety profile; renal and cardiac parameters were assessed using estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMI), and midwall fractional shortening (MFS). Results: Enrolled patients included 71 switch (median [range] age, 46.6 [5–84] years; male to female [M:F], 40:31) and 29 treatment naïve (38.7 [12–74] years; M:F, 14:15). Adverse events (AEs) were consistent with the known safety profile of agalα. Two switch patients had hospitalization due to possibly/probably drug-related serious AEs (one with transient ischemic attack, one with infusion-related AEs). One switch and two treatment-naïve patients discontinued treatment because of AEs. Three patients (one each switch, treatment naïve, and previous agalα) died; no deaths were considered drug-related. There was no significant change from baseline in LVMI or MFS in either group. Similarly, eGFR remained stable; mean ± standard error annualized change in eGFR (mL/min/1.73 m2) was −2.40 ± 1.04 in switch and −1.68 ± 2.21 in treatment-naïve patients. Conclusions: This is the largest cohort of patients with Fabry disease who were started on or switched to agalα in an FDA-accepted protocol during a worldwide supply shortage of agalsidase beta. Because this protocol was primarily designed to provide access to agalα, there were limitations, including not having stringent selection criteria and the lack of a placebo group.

Original languageEnglish (US)
Title of host publicationJIMD Reports
PublisherSpringer
Pages7-15
Number of pages9
DOIs
StatePublished - Jan 1 2015
Externally publishedYes

Publication series

NameJIMD Reports
Volume23
ISSN (Print)2192-8304
ISSN (Electronic)2192-8312

Fingerprint

Fabry Disease
Switches
Patient treatment
Glomerular Filtration Rate
Therapeutics
Safety
Transient Ischemic Attack
Pharmaceutical Preparations
Clinical Protocols
agalsidase beta
agalsidase alfa
Labels
Patient Selection
Hospitalization
Placebos
Kidney

Keywords

  • Chronic kidney disease stage
  • Enzyme replacement therapy
  • Fabry disease
  • Left ventricular hypertrophy
  • Left ventricular mass index

ASJC Scopus subject areas

  • Internal Medicine
  • Endocrinology, Diabetes and Metabolism
  • Biochemistry, Genetics and Molecular Biology (miscellaneous)

Cite this

Goker-Alpan, O., Nedd, K., Shankar, S., Lien, Y. H. H., Weinreb, N., Wijatyk, A., ... Martin, R. (2015). Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa. In JIMD Reports (pp. 7-15). (JIMD Reports; Vol. 23). Springer. https://doi.org/10.1007/8904_2015_422

Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa. / Goker-Alpan, Ozlem; Nedd, Khan; Shankar, Suma; Lien, Yeong Hau H.; Weinreb, Neal; Wijatyk, Anna; Chang, Peter; Martin, Rick.

JIMD Reports. Springer, 2015. p. 7-15 (JIMD Reports; Vol. 23).

Research output: Chapter in Book/Report/Conference proceedingChapter

Goker-Alpan, O, Nedd, K, Shankar, S, Lien, YHH, Weinreb, N, Wijatyk, A, Chang, P & Martin, R 2015, Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa. in JIMD Reports. JIMD Reports, vol. 23, Springer, pp. 7-15. https://doi.org/10.1007/8904_2015_422
Goker-Alpan, Ozlem ; Nedd, Khan ; Shankar, Suma ; Lien, Yeong Hau H. ; Weinreb, Neal ; Wijatyk, Anna ; Chang, Peter ; Martin, Rick. / Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa. JIMD Reports. Springer, 2015. pp. 7-15 (JIMD Reports).
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abstract = "Objectives: In a multicenter, open-label, treatment protocol (HGT-REP-059; NCT01031173), clinical effects and tolerability of agalsidase alfa (agalα; 0.2 mg/kg every other week) were evaluated in patients with Fabry disease who were treatment na{\"i}ve or switched from agalsidase beta (switch). Over 24 months, data were collected on the safety profile; renal and cardiac parameters were assessed using estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMI), and midwall fractional shortening (MFS). Results: Enrolled patients included 71 switch (median [range] age, 46.6 [5–84] years; male to female [M:F], 40:31) and 29 treatment na{\"i}ve (38.7 [12–74] years; M:F, 14:15). Adverse events (AEs) were consistent with the known safety profile of agalα. Two switch patients had hospitalization due to possibly/probably drug-related serious AEs (one with transient ischemic attack, one with infusion-related AEs). One switch and two treatment-na{\"i}ve patients discontinued treatment because of AEs. Three patients (one each switch, treatment na{\"i}ve, and previous agalα) died; no deaths were considered drug-related. There was no significant change from baseline in LVMI or MFS in either group. Similarly, eGFR remained stable; mean ± standard error annualized change in eGFR (mL/min/1.73 m2) was −2.40 ± 1.04 in switch and −1.68 ± 2.21 in treatment-na{\"i}ve patients. Conclusions: This is the largest cohort of patients with Fabry disease who were started on or switched to agalα in an FDA-accepted protocol during a worldwide supply shortage of agalsidase beta. Because this protocol was primarily designed to provide access to agalα, there were limitations, including not having stringent selection criteria and the lack of a placebo group.",
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AU - Lien, Yeong Hau H.

AU - Weinreb, Neal

AU - Wijatyk, Anna

AU - Chang, Peter

AU - Martin, Rick

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N2 - Objectives: In a multicenter, open-label, treatment protocol (HGT-REP-059; NCT01031173), clinical effects and tolerability of agalsidase alfa (agalα; 0.2 mg/kg every other week) were evaluated in patients with Fabry disease who were treatment naïve or switched from agalsidase beta (switch). Over 24 months, data were collected on the safety profile; renal and cardiac parameters were assessed using estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMI), and midwall fractional shortening (MFS). Results: Enrolled patients included 71 switch (median [range] age, 46.6 [5–84] years; male to female [M:F], 40:31) and 29 treatment naïve (38.7 [12–74] years; M:F, 14:15). Adverse events (AEs) were consistent with the known safety profile of agalα. Two switch patients had hospitalization due to possibly/probably drug-related serious AEs (one with transient ischemic attack, one with infusion-related AEs). One switch and two treatment-naïve patients discontinued treatment because of AEs. Three patients (one each switch, treatment naïve, and previous agalα) died; no deaths were considered drug-related. There was no significant change from baseline in LVMI or MFS in either group. Similarly, eGFR remained stable; mean ± standard error annualized change in eGFR (mL/min/1.73 m2) was −2.40 ± 1.04 in switch and −1.68 ± 2.21 in treatment-naïve patients. Conclusions: This is the largest cohort of patients with Fabry disease who were started on or switched to agalα in an FDA-accepted protocol during a worldwide supply shortage of agalsidase beta. Because this protocol was primarily designed to provide access to agalα, there were limitations, including not having stringent selection criteria and the lack of a placebo group.

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KW - Enzyme replacement therapy

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KW - Left ventricular hypertrophy

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