Limits...
Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment.

Kampmann C, Perrin A, Beck M - Orphanet J Rare Dis (2015)

Bottom Line: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients.During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

View Article: PubMed Central - PubMed

Affiliation: Section Head for Congenital Heart Defects, Center for Pediatric and Adolescent Medicine, University Medical Center, University of Mainz, Langenbeckstr. 1, Mainz, DE-55101, Germany. kampmann@mail.uni-mainz.de.

ABSTRACT

Background: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults.

Methods: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment.

Results: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.

Conclusions: Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

No MeSH data available.


Related in: MedlinePlus

a Cluster analysis of NYHA heart failure classification. b Cluster analysis of CCS grading of angina pectoris scores before and after approximately 10 years of agalsidase alfa enzyme replacement therapy. Values in the boxes indicate the numbers (and percentages) of patients in that category. Arrows indicate directions of changes in classifications or scores, and numbers adjacent to arrows show the number of patients with that change. CCS: Canadian Cardiovascular Society; NYHA: New York Heart Association
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

License 1 - License 2
getmorefigures.php?uid=PMC4587871&req=5

Fig1: a Cluster analysis of NYHA heart failure classification. b Cluster analysis of CCS grading of angina pectoris scores before and after approximately 10 years of agalsidase alfa enzyme replacement therapy. Values in the boxes indicate the numbers (and percentages) of patients in that category. Arrows indicate directions of changes in classifications or scores, and numbers adjacent to arrows show the number of patients with that change. CCS: Canadian Cardiovascular Society; NYHA: New York Heart Association

Mentions: At treatment start, heart failure symptoms (NYHA class ≥ II) were present in 31 % and anginal symptoms (CCS score ≥ 2) were present in 24 % of patients (Fig. 1). After 10 years of agalsidase alfa treatment, NYHA classification had improved by at least 1 class in 22/42 patients. Classifications were unchanged in 19 patients; only 1 patient had a worse classification, and no patients were classified as NYHA class III, compared with 9 before treatment (Fig. 1). Fifteen of 42 patients had an improved CCS score and 26 had a stable score after 10 years of ERT, with only 1 patient showing deterioration, and none having scores of 2, 3, or 4, compared with 11 patients before ERT (Fig. 1).Fig. 1


Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment.

Kampmann C, Perrin A, Beck M - Orphanet J Rare Dis (2015)

a Cluster analysis of NYHA heart failure classification. b Cluster analysis of CCS grading of angina pectoris scores before and after approximately 10 years of agalsidase alfa enzyme replacement therapy. Values in the boxes indicate the numbers (and percentages) of patients in that category. Arrows indicate directions of changes in classifications or scores, and numbers adjacent to arrows show the number of patients with that change. CCS: Canadian Cardiovascular Society; NYHA: New York Heart Association
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4587871&req=5

Fig1: a Cluster analysis of NYHA heart failure classification. b Cluster analysis of CCS grading of angina pectoris scores before and after approximately 10 years of agalsidase alfa enzyme replacement therapy. Values in the boxes indicate the numbers (and percentages) of patients in that category. Arrows indicate directions of changes in classifications or scores, and numbers adjacent to arrows show the number of patients with that change. CCS: Canadian Cardiovascular Society; NYHA: New York Heart Association
Mentions: At treatment start, heart failure symptoms (NYHA class ≥ II) were present in 31 % and anginal symptoms (CCS score ≥ 2) were present in 24 % of patients (Fig. 1). After 10 years of agalsidase alfa treatment, NYHA classification had improved by at least 1 class in 22/42 patients. Classifications were unchanged in 19 patients; only 1 patient had a worse classification, and no patients were classified as NYHA class III, compared with 9 before treatment (Fig. 1). Fifteen of 42 patients had an improved CCS score and 26 had a stable score after 10 years of ERT, with only 1 patient showing deterioration, and none having scores of 2, 3, or 4, compared with 11 patients before ERT (Fig. 1).Fig. 1

Bottom Line: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients.During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

View Article: PubMed Central - PubMed

Affiliation: Section Head for Congenital Heart Defects, Center for Pediatric and Adolescent Medicine, University Medical Center, University of Mainz, Langenbeckstr. 1, Mainz, DE-55101, Germany. kampmann@mail.uni-mainz.de.

ABSTRACT

Background: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults.

Methods: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment.

Results: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.

Conclusions: Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

No MeSH data available.


Related in: MedlinePlus