Adolescent XLH Patient Activity Diary and Symptom Reports

Featuring patient-reported outcomes, wearable device data, and interview data from a prospective, mixed-methods study of adolescents with X-linked hypophosphataemia (XLH) treated with burosumab. The study measured symptom severity scores for pain, stiffness, and fatigue on a 0–10 scale. It was authored by Vrinda Saraff and is available under a CC BY 4.0 license.

Use Cases

• Analyze the relationship between physical activity triggers and reported symptom severity scores for pain, stiffness, and fatigue.
• Investigate emotional concerns related to XLH and treatment transition using qualitative interview data.
• Examine the low median symptom severity scores (e.g., pain 0.00, stiffness 0.00) in the context of patient-reported outcomes.
• Study the lived experience of adolescents with XLH at the end of skeletal growth using mixed-methods data.

Strengths

• Data was collected via a prospective, multicentre study design (NCT05181839).
• Includes multiple data types: patient-reported outcomes, wearable device data, and interviews.
• Symptom severity is measured on a standardized 0–10 scale for pain, stiffness, and fatigue.

Limitations

• The dataset is very small at 5.5 KB, indicating a limited number of observations or variables.
• Focus is on a specific, rare genetic disorder (XLH) and a specific treatment (burosumab), limiting generalizability.
• The sample is restricted to adolescents at the end of skeletal growth, not representing the broader XLH population.

Provenance

Source

figshare, authored by Vrinda Saraff.

Collection Method

Prospective, multicentre, mixed-methods study using patient-reported outcomes, wearable devices, and interviews.

Freshness

Last updated March 20, 2026.