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Initial psychometric evaluation of the adult PKU-SSIS using an interim data cut from the OPAL study

Publication summary

Description

OPAL is an ongoing, Phase 4, multicenter, observational study of real-world safety profile and efficacy of pegvaliase in adults with PKU. This study is an interim data cut (December 2022) of OPAL to assess the initial psychometric performance of the adult PKU-SSIS.

Results showed that PKU-SSIS accurately reflects variability in symptom severity and impacts reported and showed moderate to high inter-item correlation. PKU-SSIS is demonstrated to be a valid, robust and reliable tool to assess PKU QoL.

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References:

Karimi M, Gibson A, Rowe A, Oluboyede Y, Khattab S, Lindstrom K, Sazova O. Genet Med Open. 2025;3,102873.