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Outcomes of patients treated through the Canadian Fabry disease initiative.
Mol Genet Metab. 2014 Apr; 111(4):499-506.MG

Abstract

BACKGROUND

The Canadian Fabry disease initiative (CFDI) tracks outcomes of subjects with Fabry disease treated enzyme replacement therapy (ERT) given to subjects who meet evidence-based treatment guidelines and cardiovascular risk factor modification.

METHODS

We report 5 year follow-up data on 362 subjects for a composite endpoint (death, neurologic or cardiovascular events, development of end-stage renal disease or sustained increase in serum creatinine of 50% from baseline).

RESULTS

At enrollment, 86 subjects had previously received ERT (Cohort 1a) and 67 subjects were newly started (Cohort 1b) and randomized to agalsidase alfa or agalsidase beta. 209 subjects did not initially meet ERT criteria (Cohort 1c), 25 of whom met ERT criteria in follow-up and were moved to Cohort 1b (total N=178 ERT treated subjects). Use of supportive therapies such as aspirin (78%), renin-angiotensin blockade (59%), and statins (55%) was common in ERT treated subjects. In Cohort 1a, 32 subjects met the composite endpoint with 8 deaths. In Cohort 1b, 16 subjects met the composite endpoint with 1 death. Cohort 1b had fewer clinical events than Cohort 1a (p=0.039) suggesting that the treatment protocol was effective in targeting subjects at an earlier stage. 19.4% of Cohort 1b subjects on agalsidase alfa and 13.3% on agalsidase beta had a clinical event (p=0.57). 10 Cohort 1c subjects had clinical events, none of which would have been prevented by earlier use of ERT.

CONCLUSIONS

Cardiovascular risk factor modification and targeted use of ERT reduce the risk of adverse outcomes related to Fabry disease.

Authors+Show Affiliations

Department of Medicine University of British Columbia, Canada. Electronic address: Sandra.Sirrs@vch.ca.Department of Medicine University of Montreal, Canada.Department of Pediatrics University of Calgary, Canada.Department of Pediatrics, Hospital for Sick Children and Centre Hospitalier Universitaire de Sherbrooke, Canada.Department of Pediatrics, Capital District Health Authority, Canada.Department of Community Health and Epidemiology, Dalhousie University, Halifax, Nova Scotia, Canada.Department of Medicine Dalhousie University, Canada.No affiliation info available

Pub Type(s)

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

Language

eng

PubMed ID

24534763

Citation

Sirrs, S M., et al. "Outcomes of Patients Treated Through the Canadian Fabry Disease Initiative." Molecular Genetics and Metabolism, vol. 111, no. 4, 2014, pp. 499-506.
Sirrs SM, Bichet DG, Casey R, et al. Outcomes of patients treated through the Canadian Fabry disease initiative. Mol Genet Metab. 2014;111(4):499-506.
Sirrs, S. M., Bichet, D. G., Casey, R., Clarke, J. T., Lemoine, K., Doucette, S., & West, M. L. (2014). Outcomes of patients treated through the Canadian Fabry disease initiative. Molecular Genetics and Metabolism, 111(4), 499-506. https://doi.org/10.1016/j.ymgme.2014.01.014
Sirrs SM, et al. Outcomes of Patients Treated Through the Canadian Fabry Disease Initiative. Mol Genet Metab. 2014;111(4):499-506. PubMed PMID: 24534763.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Outcomes of patients treated through the Canadian Fabry disease initiative. AU - Sirrs,S M, AU - Bichet,D G, AU - Casey,R, AU - Clarke,J T R, AU - Lemoine,K, AU - Doucette,S, AU - West,M L, AU - ,, Y1 - 2014/02/02/ PY - 2013/12/12/received PY - 2014/01/28/revised PY - 2014/01/28/accepted PY - 2014/2/19/entrez PY - 2014/2/19/pubmed PY - 2014/12/15/medline KW - Agalsidase KW - Enzyme replacement therapy KW - Fabry disease KW - Outcomes SP - 499 EP - 506 JF - Molecular genetics and metabolism JO - Mol. Genet. Metab. VL - 111 IS - 4 N2 - BACKGROUND: The Canadian Fabry disease initiative (CFDI) tracks outcomes of subjects with Fabry disease treated enzyme replacement therapy (ERT) given to subjects who meet evidence-based treatment guidelines and cardiovascular risk factor modification. METHODS: We report 5 year follow-up data on 362 subjects for a composite endpoint (death, neurologic or cardiovascular events, development of end-stage renal disease or sustained increase in serum creatinine of 50% from baseline). RESULTS: At enrollment, 86 subjects had previously received ERT (Cohort 1a) and 67 subjects were newly started (Cohort 1b) and randomized to agalsidase alfa or agalsidase beta. 209 subjects did not initially meet ERT criteria (Cohort 1c), 25 of whom met ERT criteria in follow-up and were moved to Cohort 1b (total N=178 ERT treated subjects). Use of supportive therapies such as aspirin (78%), renin-angiotensin blockade (59%), and statins (55%) was common in ERT treated subjects. In Cohort 1a, 32 subjects met the composite endpoint with 8 deaths. In Cohort 1b, 16 subjects met the composite endpoint with 1 death. Cohort 1b had fewer clinical events than Cohort 1a (p=0.039) suggesting that the treatment protocol was effective in targeting subjects at an earlier stage. 19.4% of Cohort 1b subjects on agalsidase alfa and 13.3% on agalsidase beta had a clinical event (p=0.57). 10 Cohort 1c subjects had clinical events, none of which would have been prevented by earlier use of ERT. CONCLUSIONS: Cardiovascular risk factor modification and targeted use of ERT reduce the risk of adverse outcomes related to Fabry disease. SN - 1096-7206 UR - https://www.unboundmedicine.com/medline/citation/24534763/Outcomes_of_patients_treated_through_the_Canadian_Fabry_disease_initiative_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S1096-7192(14)00040-7 DB - PRIME DP - Unbound Medicine ER -