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Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis.
Clin Ther. 2009 Sep; 31(9):1966-76.CT

Abstract

BACKGROUND

Fabry disease is an X-linked disease caused by a deficiency in the activity of the lysosomal enzyme alpha-galactosidase A. Affected individuals typically develop left ventricular hypertrophy (LVH) among other pathologies.

OBJECTIVE

The purpose of the present study was to investigate the effect of >or=12 months of enzyme replacement therapy (ERT) with agalsidase alfa on LV mass (LVM) in men and women with Fabry disease.

METHODS

This was a retrospective, blinded, pooled analysis of data from several studies assessing the effect of ERT with agalsidase alfa on LVM in patients with Fabry disease with baseline LVH. Men and women aged >or=18 years with a confirmed diagnosis of Fabry disease who had received >or=36 months of ERT with agalsidase alfa were eligible, provided they had a baseline echocardiogram and a follow-up echocardiogram at 12 and/or 36 months. Data from 4 studies were included in the present analysis. LVM was normalized to height (in meters) to the 2.7 power (LVM/h = LVM/m(2.7)).

RESULTS

In total, 45 adult patients (34 men and 11 women) with a confirmed diagnosis of Fabry disease and serial echocardiograms obtained at baseline and after 12 and/or 36 months of treatment were included. The mean (SD) age of this cohort was 39.8 (10.4) years (range, 18.9-67.2 years), and the mean weight was 72.5 (13.4) kg (range, 46.7-102.9 kg). Forty-two patients were white, 2 were Hispanic, and 1 was classified as other. At baseline, 14 patients had LVH (mean LVM/h = 55.4 [5.7] g/m(2.7)). After 12 months of ERT with agalsidase alfa, LVM/h decreased significantly by 9.2 (7.9) g/m(2.7) in 9 patients (P = 0.008), and after 36 months, LVM/h decreased significantly by 5.1 (7.5) g/m(2.7) in 10 patients (P = 0.037). In patients without baseline LVH (n = 31), a significant increase in LVM/h was observed after 12 months of treatment (3.6 [5.7] g/m(2.7); P = 0.002). After 36 months of treatment, however, there was no significant change from baseline in 10 patients (2.1 [7.9] g/m(2.7); P = NS).

CONCLUSION

Treatment with agalsidase alfa for 12 or 36 months was associated with reduced LVM in these patients with Fabry disease with baseline LVH, and it appeared to stabilize LVM in these patients without baseline LVH.

Authors+Show Affiliations

Division of Cardiology, University Children's Hospital, Mainz, Germany. christoph.kampmann@uni-mainz.deNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, N.I.H., Intramural
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

19843486

Citation

Kampmann, Christoph, et al. "Effect of Agalsidase Alfa Replacement Therapy On Fabry Disease-related Hypertrophic Cardiomyopathy: a 12- to 36-month, Retrospective, Blinded Echocardiographic Pooled Analysis." Clinical Therapeutics, vol. 31, no. 9, 2009, pp. 1966-76.
Kampmann C, Linhart A, Devereux RB, et al. Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis. Clin Ther. 2009;31(9):1966-76.
Kampmann, C., Linhart, A., Devereux, R. B., & Schiffmann, R. (2009). Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis. Clinical Therapeutics, 31(9), 1966-76. https://doi.org/10.1016/j.clinthera.2009.09.008
Kampmann C, et al. Effect of Agalsidase Alfa Replacement Therapy On Fabry Disease-related Hypertrophic Cardiomyopathy: a 12- to 36-month, Retrospective, Blinded Echocardiographic Pooled Analysis. Clin Ther. 2009;31(9):1966-76. PubMed PMID: 19843486.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Effect of agalsidase alfa replacement therapy on Fabry disease-related hypertrophic cardiomyopathy: a 12- to 36-month, retrospective, blinded echocardiographic pooled analysis. AU - Kampmann,Christoph, AU - Linhart,Ales, AU - Devereux,Richard B, AU - Schiffmann,Raphael, PY - 2009/06/17/accepted PY - 2009/10/22/entrez PY - 2009/10/22/pubmed PY - 2010/1/19/medline SP - 1966 EP - 76 JF - Clinical therapeutics JO - Clin Ther VL - 31 IS - 9 N2 - BACKGROUND: Fabry disease is an X-linked disease caused by a deficiency in the activity of the lysosomal enzyme alpha-galactosidase A. Affected individuals typically develop left ventricular hypertrophy (LVH) among other pathologies. OBJECTIVE: The purpose of the present study was to investigate the effect of >or=12 months of enzyme replacement therapy (ERT) with agalsidase alfa on LV mass (LVM) in men and women with Fabry disease. METHODS: This was a retrospective, blinded, pooled analysis of data from several studies assessing the effect of ERT with agalsidase alfa on LVM in patients with Fabry disease with baseline LVH. Men and women aged >or=18 years with a confirmed diagnosis of Fabry disease who had received >or=36 months of ERT with agalsidase alfa were eligible, provided they had a baseline echocardiogram and a follow-up echocardiogram at 12 and/or 36 months. Data from 4 studies were included in the present analysis. LVM was normalized to height (in meters) to the 2.7 power (LVM/h = LVM/m(2.7)). RESULTS: In total, 45 adult patients (34 men and 11 women) with a confirmed diagnosis of Fabry disease and serial echocardiograms obtained at baseline and after 12 and/or 36 months of treatment were included. The mean (SD) age of this cohort was 39.8 (10.4) years (range, 18.9-67.2 years), and the mean weight was 72.5 (13.4) kg (range, 46.7-102.9 kg). Forty-two patients were white, 2 were Hispanic, and 1 was classified as other. At baseline, 14 patients had LVH (mean LVM/h = 55.4 [5.7] g/m(2.7)). After 12 months of ERT with agalsidase alfa, LVM/h decreased significantly by 9.2 (7.9) g/m(2.7) in 9 patients (P = 0.008), and after 36 months, LVM/h decreased significantly by 5.1 (7.5) g/m(2.7) in 10 patients (P = 0.037). In patients without baseline LVH (n = 31), a significant increase in LVM/h was observed after 12 months of treatment (3.6 [5.7] g/m(2.7); P = 0.002). After 36 months of treatment, however, there was no significant change from baseline in 10 patients (2.1 [7.9] g/m(2.7); P = NS). CONCLUSION: Treatment with agalsidase alfa for 12 or 36 months was associated with reduced LVM in these patients with Fabry disease with baseline LVH, and it appeared to stabilize LVM in these patients without baseline LVH. SN - 1879-114X UR - https://www.unboundmedicine.com/medline/citation/19843486/Effect_of_agalsidase_alfa_replacement_therapy_on_Fabry_disease_related_hypertrophic_cardiomyopathy:_a_12__to_36_month_retrospective_blinded_echocardiographic_pooled_analysis_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0149-2918(09)00337-3 DB - PRIME DP - Unbound Medicine ER -