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Table 1 Assumptions and questions related to impact of vosoritide on growth velocity, height, and body proportions reaching ≥ 75% panel agreement

From: Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study

Assumption

% Agreementa

Can’t judge (%)

It is likely that long-term treatment with vosoritide increases growth velocity until final height is reached in individuals with achondroplasia starting treatment between 2 years of age and puberty (Tanner stage > 1)

92%

8

It is likely that long-term treatment with vosoritide results in a greater final height in those starting at an earlier age than in those starting later

100%

8

A clinically meaningful positive impact of vosoritide on abnormal upper-to-lower body segment ratio is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting treatment later

92%

8

Question

% Likely + Very likelyb

 

How likely do you consider long-term treatment with vosoritide to result in a clinically meaningful improvement in upper-to-lower body segment ratio in individuals with ACH starting between 2 years of age and puberty (Tanner stage > 1)?

85%

0

  1. a% of panel members (N = 13) agreeing or strongly agreeing with the assumption, excluding “can’t judge” votes
  2. b% of panel members voting “likely” or “very likely”, excluding “Can’t judge” votes