METHODS: Twenty genetically confirmed ATTRv amyloidosis patients (nine symptomatic, 11 pre-symptomatic) were enrolled prospectively between January to March 2023. Muscle ultrasound was performed on six muscles at standardized locations. QMUS parameters included muscle thickness (MT) and muscle echo intensity (EI). Twenty-five age- and sex-matched healthy controls were recruited for comparison. Significant QMUS parameters were correlated with clinical outcome measures.
RESULTS: Muscle volume of first dorsal interosseus (FDI) muscle [measured as cross-sectional area (CSA)] was significantly lower in symptomatic patients compared to healthy controls and pre-symptomatic carriers (98.3 ± 58.0 vs. 184.4 ± 42.5 vs. 198.3 ± 56.8, p
METHODS: A user profile and topic list were established based on previous needs analysis studies. Content was developed for each identified topic. An expert panel assessed the content validity of both informational content and activity sections. Subsequently, the contents were presented via a learning management system to parents of children newly diagnosed with leukemia or lymphoma. Parents evaluated the quality of e-HOPE using the Website Evaluation Questionnaire (WEQ) after an 8-week period.
RESULTS: The scale content validity index (S-CVI/Ave) achieved 0.996 for informational content and 0.991 for the activity section. Sixteen parents provided evaluations of e-HOPE after an 8-week usage period. Mean WEQ scores for various dimensions ranged from 4.23 for completeness to 4.88 for relevance.
CONCLUSIONS: E-HOPE was meticulously designed and developed to offer Malaysian parents a Malay-language resource complementing current caregiver education practices. It exhibited strong content validity and received positive user ratings for quality. Further assessment is warranted to evaluate its effectiveness in supporting parents of children with leukemia or lymphoma. The resource is anticipated to enhance information accessibility and support for Malaysian parents facing hematological cancers in their children.
TRIAL REGISTRATION: Clinicaltrials.govNCT05455268.