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Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study.
Heart. 2009 Jul; 95(13):1103-7.H

Abstract

BACKGROUND

Anderson-Fabry disease is a multisystem X linked disorder of lipid metabolism frequently associated with cardiac symptoms, including left ventricular (LV) hypertrophy gradually impairing cardiac function. Evidence showing that enzyme-replacement therapy (ERT) can be effective in reducing LV hypertrophy and improving myocardial function in the long term is limited.

OBJECTIVE

This study aimed to assess the long-term effects of ERT with recombinant alpha-galactosidase A (agalsidase beta, Fabrazyme) on LV function and myocardial signal intensity in 11 patients with Anderson-Fabry disease.

PATIENTS

Eleven patients (eight males, three females) with varying stages of genetically confirmed Anderson-Fabry disease were examined by means of physical examination and magnetic resonance imaging before ERT with agalsidase beta at 1 mg/kg every other week (study 1) and after a mean treatment duration of 45 months (study 2).

RESULTS

At 45 months of treatment, LV mass and LV wall thickness had significantly reduced: 188 (SD 60) g versus 153 (47) g, and 16 (4) mm versus 14 (4) mm, respectively. Furthermore, a significant reduction in myocardial T2 relaxation times was noted in all myocardial regions, that is, interventricular septum 80 (5) ms versus 66 (8) ms, apex 79 (10) ms versus 64 (10) ms, and lateral wall 80 (8) ms versus 65 (16) ms. Changes in LV ejection fraction were not significant. Amelioration of clinical symptoms was observed in all patients.

CONCLUSIONS

Long-term therapy with agalsidase beta at 1 mg/kg every 2 weeks was effective in significantly reducing LV hypertrophy, improving overall cardiac performance and ameliorating clinical symptoms in patients with Anderson-Fabry disease.

Authors+Show Affiliations

Department of Biomorphological and Functional Sciences, University Federico II, Naples, Italy. mimbriaco@hotmail.comNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article

Language

eng

PubMed ID

19372091

Citation

Imbriaco, M, et al. "Effects of Enzyme-replacement Therapy in Patients With Anderson-Fabry Disease: a Prospective Long-term Cardiac Magnetic Resonance Imaging Study." Heart (British Cardiac Society), vol. 95, no. 13, 2009, pp. 1103-7.
Imbriaco M, Pisani A, Spinelli L, et al. Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study. Heart. 2009;95(13):1103-7.
Imbriaco, M., Pisani, A., Spinelli, L., Cuocolo, A., Messalli, G., Capuano, E., Marmo, M., Liuzzi, R., Visciano, B., Cianciaruso, B., & Salvatore, M. (2009). Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study. Heart (British Cardiac Society), 95(13), 1103-7. https://doi.org/10.1136/hrt.2008.162800
Imbriaco M, et al. Effects of Enzyme-replacement Therapy in Patients With Anderson-Fabry Disease: a Prospective Long-term Cardiac Magnetic Resonance Imaging Study. Heart. 2009;95(13):1103-7. PubMed PMID: 19372091.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study. AU - Imbriaco,M, AU - Pisani,A, AU - Spinelli,L, AU - Cuocolo,A, AU - Messalli,G, AU - Capuano,E, AU - Marmo,M, AU - Liuzzi,R, AU - Visciano,B, AU - Cianciaruso,B, AU - Salvatore,M, Y1 - 2009/04/15/ PY - 2009/4/18/entrez PY - 2009/4/18/pubmed PY - 2009/7/8/medline SP - 1103 EP - 7 JF - Heart (British Cardiac Society) JO - Heart VL - 95 IS - 13 N2 - BACKGROUND: Anderson-Fabry disease is a multisystem X linked disorder of lipid metabolism frequently associated with cardiac symptoms, including left ventricular (LV) hypertrophy gradually impairing cardiac function. Evidence showing that enzyme-replacement therapy (ERT) can be effective in reducing LV hypertrophy and improving myocardial function in the long term is limited. OBJECTIVE: This study aimed to assess the long-term effects of ERT with recombinant alpha-galactosidase A (agalsidase beta, Fabrazyme) on LV function and myocardial signal intensity in 11 patients with Anderson-Fabry disease. PATIENTS: Eleven patients (eight males, three females) with varying stages of genetically confirmed Anderson-Fabry disease were examined by means of physical examination and magnetic resonance imaging before ERT with agalsidase beta at 1 mg/kg every other week (study 1) and after a mean treatment duration of 45 months (study 2). RESULTS: At 45 months of treatment, LV mass and LV wall thickness had significantly reduced: 188 (SD 60) g versus 153 (47) g, and 16 (4) mm versus 14 (4) mm, respectively. Furthermore, a significant reduction in myocardial T2 relaxation times was noted in all myocardial regions, that is, interventricular septum 80 (5) ms versus 66 (8) ms, apex 79 (10) ms versus 64 (10) ms, and lateral wall 80 (8) ms versus 65 (16) ms. Changes in LV ejection fraction were not significant. Amelioration of clinical symptoms was observed in all patients. CONCLUSIONS: Long-term therapy with agalsidase beta at 1 mg/kg every 2 weeks was effective in significantly reducing LV hypertrophy, improving overall cardiac performance and ameliorating clinical symptoms in patients with Anderson-Fabry disease. SN - 1468-201X UR - https://www.unboundmedicine.com/medline/citation/19372091/Effects_of_enzyme_replacement_therapy_in_patients_with_Anderson_Fabry_disease:_a_prospective_long_term_cardiac_magnetic_resonance_imaging_study_ L2 - http://heart.bmj.com/cgi/pmidlookup?view=long&pmid=19372091 DB - PRIME DP - Unbound Medicine ER -