Fabry's disease medical therapy: Difference between revisions
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==Medical Therapy== | ==Medical Therapy== | ||
===Specific | ===Specific Treatments=== | ||
*Enzyme: alpha-galactosidase A enzyme | *Enzyme: [[Fabry's disease|alpha-galactosidase A]] enzyme | ||
**Enzyme replacement therapy: recombinant human enzymes | **Enzyme replacement therapy ([[ERT]]): recombinant human enzymes | ||
***Agalsidase alfa( Replagal) | ***[[Agalsidase alfa]]( Replagal) | ||
***Agalsidase beta (Fabrazyme) | ***[[Agalsidase beta]] (Fabrazyme) | ||
**Increase the enzyme activity if GLA gene positive: Migalastat | **Increase the enzyme activity if [[GLA gene]] positive: [[Migalastat]] | ||
** | |||
=== Symptom and Complication Treatments === | |||
==== Kidney disease <ref name="pmid17699807">{{cite journal| author=Wanner C, Breunig F| title=Fabry nephropathy and the case for adjunctive renal therapy. | journal=J Am Soc Nephrol | year= 2007 | volume= 18 | issue= 9 | pages= 2426-8 | pmid=17699807 | doi=10.1681/ASN.2007070783 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17699807 }}</ref> ==== | |||
* [[ACE inhibitor|ACE inhibitors]] and [[ARBs]]: can reduce [[proteinuria]] and stable [[Glomerular filtration rate|GFR]] in patients with [[hypertension]] and Fabry's disease. | |||
* [[Dialysis]]: in [[ESRD]] | |||
==== Cardiovascular disease ==== | |||
* [[Anti-anginal]] medications<ref name="pmid20109602">{{cite journal| author=O'Mahony C, Elliott P| title=Anderson-Fabry disease and the heart. | journal=Prog Cardiovasc Dis | year= 2010 | volume= 52 | issue= 4 | pages= 326-35 | pmid=20109602 | doi=10.1016/j.pcad.2009.11.002 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=20109602 }}</ref> | |||
* [[Antiarrhythmic drug|Antiarrhythmic]] medications<ref name="pmid27265676">{{cite journal| author=Weidemann F, Maier SK, Störk S, Brunner T, Liu D, Hu K | display-authors=etal| title=Usefulness of an Implantable Loop Recorder to Detect Clinically Relevant Arrhythmias in Patients With Advanced Fabry Cardiomyopathy. | journal=Am J Cardiol | year= 2016 | volume= 118 | issue= 2 | pages= 264-74 | pmid=27265676 | doi=10.1016/j.amjcard.2016.04.033 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=27265676 }}</ref> | |||
* [[Heart failure]] Medications<ref name="pmid23741058">{{cite journal| author=WRITING COMMITTEE MEMBERS. Yancy CW, Jessup M, Bozkurt B, Butler J, Casey DE | display-authors=etal| title=2013 ACCF/AHA guideline for the management of heart failure: a report of the American College of Cardiology Foundation/American Heart Association Task Force on practice guidelines. | journal=Circulation | year= 2013 | volume= 128 | issue= 16 | pages= e240-327 | pmid=23741058 | doi=10.1161/CIR.0b013e31829e8776 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=23741058 }}</ref> | |||
==== Neurological disease ==== | |||
* Neuropathic pain<ref name="pmid26141332">{{cite journal| author=Watson JC, Dyck PJ| title=Peripheral Neuropathy: A Practical Approach to Diagnosis and Symptom Management. | journal=Mayo Clin Proc | year= 2015 | volume= 90 | issue= 7 | pages= 940-51 | pmid=26141332 | doi=10.1016/j.mayocp.2015.05.004 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=26141332 }}</ref> | |||
** Reduce by ERT | |||
** Gabapentin | |||
** Anti-convulsant drugs | |||
* Reduce the risk of stroke<ref name="pmid17362993">{{cite journal| author=Moore DF, Kaneski CR, Askari H, Schiffmann R| title=The cerebral vasculopathy of Fabry disease. | journal=J Neurol Sci | year= 2007 | volume= 257 | issue= 1-2 | pages= 258-63 | pmid=17362993 | doi=10.1016/j.jns.2007.01.053 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=17362993 }}</ref> | |||
** Antiplatelet (primary and secondary prevention) | |||
* | |||
==References== | ==References== | ||
Revision as of 17:53, 15 April 2022
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Medical Therapy
Specific Treatments
- Enzyme: alpha-galactosidase A enzyme
- Enzyme replacement therapy (ERT): recombinant human enzymes
- Agalsidase alfa( Replagal)
- Agalsidase beta (Fabrazyme)
- Increase the enzyme activity if GLA gene positive: Migalastat
- Enzyme replacement therapy (ERT): recombinant human enzymes
Symptom and Complication Treatments
Kidney disease [1]
- ACE inhibitors and ARBs: can reduce proteinuria and stable GFR in patients with hypertension and Fabry's disease.
- Dialysis: in ESRD
Cardiovascular disease
- Anti-anginal medications[2]
- Antiarrhythmic medications[3]
- Heart failure Medications[4]
Neurological disease
- Neuropathic pain[5]
- Reduce by ERT
- Gabapentin
- Anti-convulsant drugs
- Reduce the risk of stroke[6]
- Antiplatelet (primary and secondary prevention)
References
- ↑ Wanner C, Breunig F (2007). "Fabry nephropathy and the case for adjunctive renal therapy". J Am Soc Nephrol. 18 (9): 2426–8. doi:10.1681/ASN.2007070783. PMID 17699807.
- ↑ O'Mahony C, Elliott P (2010). "Anderson-Fabry disease and the heart". Prog Cardiovasc Dis. 52 (4): 326–35. doi:10.1016/j.pcad.2009.11.002. PMID 20109602.
- ↑ Weidemann F, Maier SK, Störk S, Brunner T, Liu D, Hu K; et al. (2016). "Usefulness of an Implantable Loop Recorder to Detect Clinically Relevant Arrhythmias in Patients With Advanced Fabry Cardiomyopathy". Am J Cardiol. 118 (2): 264–74. doi:10.1016/j.amjcard.2016.04.033. PMID 27265676.
- ↑ WRITING COMMITTEE MEMBERS. Yancy CW, Jessup M, Bozkurt B, Butler J, Casey DE; et al. (2013). "2013 ACCF/AHA guideline for the management of heart failure: a report of the American College of Cardiology Foundation/American Heart Association Task Force on practice guidelines". Circulation. 128 (16): e240–327. doi:10.1161/CIR.0b013e31829e8776. PMID 23741058.
- ↑ Watson JC, Dyck PJ (2015). "Peripheral Neuropathy: A Practical Approach to Diagnosis and Symptom Management". Mayo Clin Proc. 90 (7): 940–51. doi:10.1016/j.mayocp.2015.05.004. PMID 26141332.
- ↑ Moore DF, Kaneski CR, Askari H, Schiffmann R (2007). "The cerebral vasculopathy of Fabry disease". J Neurol Sci. 257 (1–2): 258–63. doi:10.1016/j.jns.2007.01.053. PMID 17362993.