Table 2.
Results of the round 1 and 2 modified Delphi consensus process relating to α-Gal A measurement in patients receiving migalastat.
| ID | Statement | Rank,* n | Verdict | Mean consensus score† | ||
|---|---|---|---|---|---|---|
| Agreement | Neutral | Disagreement | ||||
| 1A | Migalastat should be continued in female patients with amenable mutations who are showing a stabilization or improvement in Fabry-related symptoms, regardless of the change in α-Gal A activity | 10 | 2 | 1 | Consensus (agreement) | 1.2 |
| 1B | Measurement of α-Gal A activity in female patients should not be used to make decisions about whether to continue migalastat treatment | 12 | 0 | 1 | Consensus (agreement) | 1.1 |
| 1C | In male patients receiving migalastat, α-Gal A enzyme activity in leukocytes should be measured before initiation of migalastat and as part of routine follow-up | 9 | 3 | 1 | Consensus (agreement) | 1.0 |
| 1D | Migalastat should be continued in male patients with amenable mutations who are showing a stabilization or improvement in Fabry-related symptoms, regardless of the change in α-Gal A activity | 10 | 1 | 2 | Consensus (agreement) | 1.0 |
| 1E | Migalastat should be continued in both male and female patients with stable or improved organ function, regardless of any change in the α-Gal A activity | 10 | 0 | 3 | Consensus (agreement) | 0.8 |
| 1F | α-Gal A activity in leukocytes should be routinely measured from migalastat initiation to inform an assessment of the biological activity of migalastat | 9 | 1 | 3 | Consensus (agreement) | 0.6 |
| 1G | In male patients receiving migalastat, an increase in α-Gal A activity to 10–20% of normal levels indicates biological activity of migalastat and supports a clinical decision to continue treatment | 8 | 2 | 3 | Criteria not reached | 0.5 |
| 1H | An increase in α-Gal A activity by 20–30% from baseline indicates biological activity of migalastat and supports a clinical decision to continue treatment | 5 | 3 | 5 | Disagreement | 0.1 |
| 1I | In female patients receiving migalastat, α-Gal A enzyme activity in leukocytes should be measured before initiation of migalastat and as part of routine follow-up | 1 | 6 | 6 | Disagreement | −0.4 |
*
The number of panelists with a Likert scale rank of agreement (“agree” or “strongly agree”), neutral (“neither agree nor disagree”), or disagreement (“disagree” or “strongly disagree”) for each statement is presented;
†
Strongest agreement = 2, strongest disagreement = –2.
α-Gal A, α-galactosidase A.