Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome.

ABSTRACT:

Objectives

Sanfilippo syndrome is a rare multisystem disease with no approved treatments. This study explores caregiver perspectives on the most impactful symptoms and patient-relevant clinical outcomes assessments. The pediatric onset and progressive neurodegenerative nature of Sanfilippo limits use of self-report in clinical research. This study obtains Sanfilippo caregiver data to support the selection of fit-for-purpose and patient-relevant clinical outcome assessments (COAs).

Methods

We conducted an asynchronous online focus group (n = 11) followed by individual interviews with caregivers (n = 19) of children with Sanfilippo syndrome. All participants reported on the impact of disease symptoms and level of unmet treatment need across Sanfilippo symptom domains. Focus gr

SUBMITTER: Porter KA 

PROVIDER: S-EPMC9038975 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature