Shwetha Bindhu, Anunita Nattam, Catherine Xu, Tripura Vithala, Tiffany Grant, Jacinda K Dariotis, Hexuan Liu, Danny T Y Wu
Roles of Health Literacy in Relation to Social Determinants of Health and Recommendations for Informatics-Based Interventions: Systematic Review
Online journal of public health informatics
approximately twothirds patients mucopolysaccharidosis ii mps ii severe neuronopathic phenotype characterized somatic cognitive behavioral issues current standard care treatment mps ii enzyme replacement therapy intravenous recombinant human iduronate2sulfatase idursulfase target cognitive manifestations mps ii idursulfase formulated intrathecal administration cerebrospinal fluid idursulfaseit accordance recommendations patientfocused drug development semistructured interviews conducted assess caregiver experiences observations 52week phase 23 trial idursulfaseit addition intravenous idursulfase pediatric patients neuronopathic mps ii substudy enrolled patients younger 3 years old received idursulfaseit overall 46 caregivers providing care 50 children mean range age 79 317 years interview took part single 60min exit interview six children participated substudy qualitative quantitative data obtained demonstrating burden mps ii experienced caregivers families following participation trials 39 78 children reported caregivers experienced improvements symptoms impact disease improvements 37 95 experienced cognitive improvements 26 67 experienced emotionalbehavioral improvements overall 43 children 86 rated caregivers moderate severe symptoms trials trials 28 children 56 considered mild symptoms six children participated substudy proportions 83 100 respectively caregivers qualitative descriptions trial experiences suggested improvements childrens verbal nonverbal functioning spatial motor skills well positive impact family life study revealed caregiverreported improvements childrens mps ii symptoms impact disease patients families trend cognitive improvement reduction severity mps ii symptoms many years extensive review regulatory discussions idursulfaseit clinical trial data found insufficient meet evidentiary standard support regulatory filings