Successful Management of Enzyme Replacement Therapy in Related Fabry Disease Patients with Severe Adverse Events by Switching from Agalsidase Beta (Fabrazyme(Â®)) to Agalsidase Alfa (Replagal (Â®)).

[fabry disease]

Background : Enzyme replacement therapy (ERT ) is the only approved therapy for Fabry disease . In June 2009 , there was a worldwide shortage of agalsidase beta , necessitating dose reductions or switching to agalsidase alfa in some patients . Case presentation : We present two cases of Fabry disease (a parent and a child ) who received agalsidase beta for 27 months at the licensed dose and 10 months at a reduced dose , followed by a switch to agalsidase alfa for 28 months . Case 1 , a 26 -year -old male had severe coughing and fatigue during ERT with agalsidase beta requiring antitussive and asthmatic drug therapy . After switching to agalsidase alfa , the coughing gradually resolved completely . Case 2 , a 62 -year -old female had advanced cardiac manifestations at the time of diagnosis . Despite receiving ERT with the approved dose of agalsidase beta , she experienced aggravation of congestive heart failure and was hospitalized . After switching to agalsidase alfa with standard care in heart disease , BNP level , echocardiographic parameters , eGFR rate and lyso-Gb 3 levels were improved or stabilized . Conclusions : We report on two Fabry disease patients who experienced severe adverse events while on approved and /or reduced doses of agalsidase beta . Switching to agalsidase alfa associated with standard care in heart disease led to resolution or improvement in the cardiorespiratory status . And reduction in dose associated with standard care in respiratory disease was useful for decrease in cough and fatigue . Plasma BNP level was useful for monitoring heart failure and the effects of ERT .

Diseases
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Diseases presenting "severe adverse events while on approved" symptom

fabry disease

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