Adolescent XLH Patient Experience Data from Burosumab Study

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, NCT05181839, was conducted at multiple sites and collected data on symptom severity scores for pain, stiffness, and fatigue on a 0–10 scale. The dataset is 5.5 KB in size, stored in an XLS file format.

Use Cases

• Analyze patient-reported symptom severity scores for pain, stiffness, and fatigue to understand symptom burden in adolescents with XLH.
• Correlate data from wearable devices with patient-reported outcomes to assess the impact of physical activity on symptoms.
• Examine interview data for emotional concerns related to XLH and treatment transition to inform patient support strategies.
• Investigate the relationship between symptom triggers (e.g., physical activity) and interference with daily life as reported by patients.

Strengths

• Data originates from a prospective, multicentre clinical study (NCT05181839), providing a structured research context.
• Includes mixed-methods data: patient-reported outcomes, wearable device metrics, and qualitative interview insights.
• The dataset is openly licensed under CC BY 4.0, facilitating reuse and sharing.

Limitations

• The dataset is very small at 5.5 KB, indicating a limited scope and likely a small sample size of adolescent patients.
• The specific number of rows, columns, and patient records is unknown, limiting precise assessment of analytical potential.
• The tags are auto-generated and contain fragmented phrases, which may not accurately represent the dataset's core content.

Provenance

Source

Author Vrinda Saraff, published on the figshare platform.

Collection Method

Data was gathered via a prospective, multicentre, mixed-methods clinical study involving patient-reported outcomes, wearable devices, and interviews.

Time Range

null

Freshness

The dataset was last updated on March 20, 2026, indicating it is current or forward-dated.

Geography

Multicentre study, but specific geographic coverage is not stated.