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Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study.
Kidney Int. 2004 Apr; 65(4):1381-5.KI

Abstract

BACKGROUND

We sought to assess the safety and efficacy of enzyme replacement therapy (ERT) with recombinant human-alpha-galactosidase A (rh-alpha-Gal A) in kidney transplant recipients with Fabry disease, a previously unstudied population.

METHODS

Three male kidney transplant recipients with biochemically, genetically, and histologically confirmed Fabry disease and documented Fabry myocardiopathy received the rh-alpha-Gal A, agalsidase beta, 1 mg/kg of body weight every 2 weeks by intravenous infusion and were monitored biochemically, clinically, and electrocardiographically and echocardiographically for 18 months.

RESULTS

Patients showed biochemical, clinical/functional, and morphologic response to ERT. Plasma globotriaosylceramide decreased 23% to 50%. Extremity pain resolved within 2 months in the patient with this manifestation. On echocardiography, left ventricular mass, end diastolic diameter (EDD), and cardiac contractility, shown by ejection fraction (EF), improved in 2 of the 3 patients receiving essentially all planned infusions. EDD and EF remained basically stable, but cardiac morphologic abnormalities progressed in the other patient, who had a 5-month interruption in ERT after the initial month. Mild mitral insufficiency persisted in all patients, as did atrial fibrillation in the affected individual. After a combined total of 116 infusions, no treatment-related adverse event, intolerance, or seroconversion was seen. Renal function remained stable and the immunosuppression regimen unchanged in all patients.

CONCLUSION

Our pilot study provides preliminary evidence that ERT with agalsidase beta, 1 mg/kg every 2 weeks, is safe and often effective against extra-renal manifestations in kidney transplant patients with Fabry disease. Studies with longer courses of this and higher doses of ERT are merited in this population.

Authors+Show Affiliations

Department of Nephrology and Dialysis, Infermi Hospital, Rimini, Italy. rmignani@auslrn.netNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

15086478

Citation

Mignani, Renzo, et al. "Enzyme Replacement Therapy With Agalsidase Beta in Kidney Transplant Patients With Fabry Disease: a Pilot Study." Kidney International, vol. 65, no. 4, 2004, pp. 1381-5.
Mignani R, Panichi V, Giudicissi A, et al. Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study. Kidney Int. 2004;65(4):1381-5.
Mignani, R., Panichi, V., Giudicissi, A., Taccola, D., Boscaro, F., Feletti, C., Moneti, G., & Cagnoli, L. (2004). Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study. Kidney International, 65(4), 1381-5.
Mignani R, et al. Enzyme Replacement Therapy With Agalsidase Beta in Kidney Transplant Patients With Fabry Disease: a Pilot Study. Kidney Int. 2004;65(4):1381-5. PubMed PMID: 15086478.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study. AU - Mignani,Renzo, AU - Panichi,Vincenzo, AU - Giudicissi,Antonio, AU - Taccola,Daniele, AU - Boscaro,Francesca, AU - Feletti,Carlo, AU - Moneti,Gloriano, AU - Cagnoli,Leonardo, PY - 2004/4/17/pubmed PY - 2004/10/22/medline PY - 2004/4/17/entrez SP - 1381 EP - 5 JF - Kidney international JO - Kidney Int. VL - 65 IS - 4 N2 - BACKGROUND: We sought to assess the safety and efficacy of enzyme replacement therapy (ERT) with recombinant human-alpha-galactosidase A (rh-alpha-Gal A) in kidney transplant recipients with Fabry disease, a previously unstudied population. METHODS: Three male kidney transplant recipients with biochemically, genetically, and histologically confirmed Fabry disease and documented Fabry myocardiopathy received the rh-alpha-Gal A, agalsidase beta, 1 mg/kg of body weight every 2 weeks by intravenous infusion and were monitored biochemically, clinically, and electrocardiographically and echocardiographically for 18 months. RESULTS: Patients showed biochemical, clinical/functional, and morphologic response to ERT. Plasma globotriaosylceramide decreased 23% to 50%. Extremity pain resolved within 2 months in the patient with this manifestation. On echocardiography, left ventricular mass, end diastolic diameter (EDD), and cardiac contractility, shown by ejection fraction (EF), improved in 2 of the 3 patients receiving essentially all planned infusions. EDD and EF remained basically stable, but cardiac morphologic abnormalities progressed in the other patient, who had a 5-month interruption in ERT after the initial month. Mild mitral insufficiency persisted in all patients, as did atrial fibrillation in the affected individual. After a combined total of 116 infusions, no treatment-related adverse event, intolerance, or seroconversion was seen. Renal function remained stable and the immunosuppression regimen unchanged in all patients. CONCLUSION: Our pilot study provides preliminary evidence that ERT with agalsidase beta, 1 mg/kg every 2 weeks, is safe and often effective against extra-renal manifestations in kidney transplant patients with Fabry disease. Studies with longer courses of this and higher doses of ERT are merited in this population. SN - 0085-2538 UR - https://www.unboundmedicine.com/medline/citation/15086478/Enzyme_replacement_therapy_with_agalsidase_beta_in_kidney_transplant_patients_with_Fabry_disease:_a_pilot_study_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0085-2538(15)49847-3 DB - PRIME DP - Unbound Medicine ER -