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Table 4 Responses to statement included in Round 3 (19 respondents)

From: International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study

Question Round 3
Agree/neutral Disagree
1. The 5-domain NPCCSS scale is the first choice for assessing clinical severity of NPC in routine clinical practice 68% (13) 32% (6)
2. The 17-domain NPCCSS scale is the first choice for assessing clinical severity of NPC in clinical trial settings, prioritising the domains in the 5-domain scale (e.g. as primary endpoints) 74% (14) 26% (5)
3. There is no need for a new universal scale for all settings to be developed 74% (14) 26% (5)
4. Resources/training on how to apply the NPCCSS (17- and 5-domains) should be developed and provided to clinicians working in NPC 89% (17) 11% (2)
5. The consensus paper is reviewed periodically to ensure that its recommendations remain accurate 100% (19) 0% (0)
6. The timescale for periodic review of the consensus paper should be every 5 years 84% (16) 16% (3)
  1. Numbers highlighted in bold indicate questions/statements for which consensus was achieved (greater than or equal to 70% agreement of neutrality)