Table 3 Recommendations for selection of initial Fabry disease-specific therapy and treatment conversion
From: A review and recommendations for oral chaperone therapy in adult patients with Fabry disease
Initiate migalastat | Amenable mutation and eGFR > 30 ml/min/1.73 m2 |
Age 12 years or more and weight for adolescents of at least 45 kg | |
Compliance with every-other-day oral drug administration | |
No intention by female patients to become pregnant, | |
Use of medications that may show drug interactions with ERT affecting its efficacy (e.g., chloroquine, hydroxychloroquine, or amiodarone) | |
Patient’s preference | |
Initiate ERT | All mutation types, |
Age 8 years or more, Chronic kidney disease with | |
eGFR < 30 ml/min/1.73 m2 | |
Compliance with intravenous every other week infusion | |
Intention by female patients to become pregnant | |
Patient’s preference | |
ERT to migalastat switch | Only amenable mutations, |
Age 12 years or more and weight for adolescents of at least 45 kg | |
Use of medications that are not allowed in patients receiving ERT, such as chloroquine, hydroxychloroquine, or amiodarone | |
Persistent severe or moderate infusion-associated reactions that do not respond to prophylaxis | |
The presence of IgE antibody against agalsidase; may be associated with anaphylaxis | |
Anti-drug antibodies presence (against agalsidases), particularly if clinical symptoms worsen | |
Patient’s preference | |
Migalastat to ERT switch | No compliance with every-other-day oral drug administration |
eGFR < 30 ml/min/1.73 m2 | |
Side effects of migalastat therapy such as severe recurrent or persistent headache, dizziness and gastrointestinal symptoms, fatigue, muscle pain, and skin changes | |
Women considering pregnancy | |
Patient’s preference | |
Withdrawal of disease-specific therapy (ERT or chaperon) | No response to long-term treatment |
Patient’s request | |
Life expectancy less than one year due to severe comorbid illness or due to severe Fabry disease with end-stage heart failure; if not a candidate for heart transplantation | |
The permanent severe neurocognitive decline of any cause | |
Severe reduction in quality of life and functional status despite disease-specific therapy | |
Severe, life-threatening infusion-related adverse reactions despite prophylactic treatment and amenable mutation | |
Poor patient adherence to disease-specific therapy (e.g., less than 80% of drug doses taken) |