Pediatric HLH Treatment Outcomes with Ruxolitinib Versus HLH-94

67 pediatric hemophagocytic lymphohistiocytosis patients were analyzed in a retrospective study comparing a ruxolitinib-based regimen to the HLH-94 protocol. The dataset contains clinical outcomes for 40 control patients treated from 2022-2023 and 27 treatment patients from 2024. It was authored by Na Song and shared under a CC BY 4.0 license.

Use Cases

• Compare 12-month overall survival (OS) and event-free survival (EFS) rates between the two treatment groups.
• Analyze complete response (CR) rates at weeks 2, 4, and 8 across patient cohorts.
• Model the relationship between key inflammatory markers like ferritin, IFN-γ, and IL-10 and early treatment response.
• Assess the distribution and cumulative exposure to glucocorticoids and etoposide during the first 8 weeks of therapy.

Strengths

• Directly compares two treatment protocols for a rare pediatric condition across 67 patient records.
• Includes specific clinical endpoints: 12-month OS, EFS, and week 2/4/8 complete response rates with statistical significance values.
• Reports detailed metrics on chemotherapy exposure, including median glucocorticoid and etoposide doses with interquartile ranges.

Limitations

• Small sample size of 67 patients limits statistical power for subgroup analyses.
• Single-center, retrospective historical control design introduces potential selection bias.
• Dataset scope is limited to 11.1 KB, indicating it likely contains summary results, not raw patient-level data.

Provenance

Source

figshare, authored by Na Song.

Collection Method

Single-center, retrospective historical control study.

Time Range

Patient data from 2022 to 2024.

Freshness

Last updated March 2026.

Geography

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