TY - JOUR

T1 - Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage

AU - Linthorst, Gabor E

AU - Burlina, Alessandro P

AU - Cecchi, Franco

AU - Cox, Timothy M

AU - Fletcher, Janice M

AU - Feldt-Rasmussen, Ulla

AU - Giugliani, Roberto

AU - Hollak, Carla E M

AU - Houge, Gunnar

AU - Hughes, Derralynn

AU - Kantola, Iikka

AU - Lachmann, Robin

AU - Lopez, Monica

AU - Ortiz, Alberto

AU - Parini, Rossella

AU - Rivera, Alberto

AU - Rolfs, Arndt

AU - Ramaswami, Uma

AU - Svarstad, Einar

AU - Tondel, Camilla

AU - Tylki-Szymanska, Anna

AU - Vujkovac, Bojan

AU - Waldek, Steven

AU - West, Michael

AU - Weidemann, F

AU - Mehta, Atul

PY - 2012

Y1 - 2012

N2 - The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.

AB - The interruption of the manufacturing process of agalsidase beta has led to a worldwide shortage of this drug. In the EU, nearly all patients initially reduced their agalsidase beta dose, and many of these switched to agalsidase alfa (Replagal Shire HGT). The clinical consequences of this period of drug shortage need to be further evaluated. A gradual increase of agalsidase beta supply is now expected. This implies that patients could resume or even commence agalsidase beta treatment. Guidance for prioritization of patients is needed to support equitable distribution of agalsidase beta to EU member states. To achieve this, in absence of level I clinical evidence, a draft consensus proposal was initiated and distributed. No full consensus was achieved, as there is disagreement regarding the indications for switching patients from agalsidase alfa to agalsidase beta. Some physicians support the concept that the 1.0 mg/kg EOW dose of agalsidase beta is more effective than agalsidase alfa at 0.2 mg/kg EOW, while others believe that at recommended dose, the preparations are equivalent. In light of these difficulties and the uncertainties with respect to supply of agalsidase beta, recommendations were agreed upon by a subgroup of physicians. These current recommendations focus on prioritization of criteria indicative of disease progression.

U2 - 10.1007/8904_2012_160

DO - 10.1007/8904_2012_160

M3 - Journal article

C2 - 23430520

VL - 8

SP - 51

EP - 56

JO - JIMD Reports

JF - JIMD Reports

SN - 2192-8304

ER -