Chapter title |
Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa
|
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Chapter number | 422 |
Book title |
JIMD Reports, Volume 23
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Published in |
JIMD Reports, January 2015
|
DOI | 10.1007/8904_2015_422 |
Pubmed ID | |
Book ISBNs |
978-3-66-247466-2, 978-3-66-247467-9
|
Authors |
Ozlem Goker-Alpan, Khan Nedd, Suma P. Shankar, Yeong-Hau H. Lien, Neal Weinreb, Anna Wijatyk, Peter Chang, Rick Martin, Goker-Alpan, Ozlem, Nedd, Khan, Shankar, Suma P., Lien, Yeong-Hau H., Weinreb, Neal, Wijatyk, Anna, Chang, Peter, Martin, Rick |
Abstract |
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). 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 m(2)) was -2.40 ± 1.04 in switch and -1.68 ± 2.21 in treatment-naïve patients. 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. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 23 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 6 | 26% |
Student > Bachelor | 5 | 22% |
Student > Master | 2 | 9% |
Student > Doctoral Student | 1 | 4% |
Other | 1 | 4% |
Other | 1 | 4% |
Unknown | 7 | 30% |
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Economics, Econometrics and Finance | 1 | 4% |
Other | 1 | 4% |
Unknown | 7 | 30% |