Fig. 1

Conceptual diagram illustrating comparison of pairs of participants in the win ratio analysis. As a first step in win ratio calculation, each participant’s response for the two endpoints was classified as a meaningful improvement, no meaningful change, or a meaningful decline based on the midpoints of published minimal clinically important differences for FVC and 6MWT for chronic respiratory diseases as applied in previous studies in Pompe disease (see Table 1). In this conceptual diagram, the comparison of FVC % predicted is tied for the first pair of participants, as both participants had a meaningful improvement; the comparison therefore moves to the 6MWT, which results in a win for AVA because the participant from this group had meaningful improvement, while the participant on ALG had no meaningful change. In the second pair, the participant on AVA had a meaningful improvement in FVC % predicted while the participant on ALG had no meaningful change on this endpoint; this results in a win for AVA, and 6MWT is not considered. The third pair ends in a loss for AVA because the other participant in the pair had a better response on FVC % predicted. The fourth pair ends in a tie because responses were in the same category for both FVC % predicted and 6MWT. Abbreviations: 6MWT, 6-min walk test; ALG, alglucosidase alfa; AVA, avalglucosidase alfa; FVC, forced vital capacity