Adolescent XLH Patient Experience and Treatment Data

Patient-reported outcomes, wearable device data, and interview insights from a prospective, multicenter study of adolescents with X-linked hypophosphataemia (XLH) treated with burosumab. The study, registered as NCT05181839, collected data on symptom severity, activity triggers, and emotional concerns related to treatment transition. The dataset is 9.5 KB in size, but the specific row and column counts are unknown.

Use Cases

• Analyze patient-reported symptom severity scores for pain, stiffness, and fatigue on a 0–10 scale to understand treatment impact.
• Correlate symptom triggers, such as physical activity, with data from wearable devices to assess daily life interference.
• Examine interview data on emotional concerns related to XLH and treatment transition to inform patient support strategies.
• Investigate the relationship between symptom severity and the timing of treatment relative to skeletal growth ends.

Strengths

• Data originates from a prospective, multicenter clinical study (NCT05181839), providing structured research context.
• Includes mixed-methods data: patient-reported outcomes, wearable device metrics, and qualitative interview insights.
• Focuses on a specific, understudied population: adolescents with XLH at the end of skeletal growth.

Limitations

• The dataset is very small at 9.5 KB, indicating limited scope and likely a small sample size.
• Column names and sample data are unavailable, hindering precise understanding of the data structure.
• The study focuses on a rare genetic disorder, limiting generalizability to broader populations.

Provenance

Source

Author Vrinda Saraff, published on figshare.

Collection Method

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

Freshness

Last updated on 2026-03-20.