Four Adolescent Cases of Refractory Immune Cytopenia Treated with Mycophenolate Mofetil

A 2026 case series from figshare authored by Cristina Cifaldi describes four pediatric/adolescent patients with chromosome 22q11.2 deletion syndrome and refractory immune thrombocytopenia. The document details a treatment regimen using low-dose Mycophenolate Mofetil over more than 24 months, accompanied by longitudinal immunological monitoring via flow cytometry. It reports on treatment outcomes, including platelet stabilization and changes in specific immune cell populations.

Use Cases

• Analyzing treatment response to Mycophenolate Mofetil based on longitudinal platelet count data.
• Studying immunological biomarkers like Tfh and Treg cell frequencies in a rare disease cohort.
• Investigating the association between specific immune cell profiles and disease remission in refractory immune cytopenia.

Strengths

• Longitudinal data collection spanning more than 24 months of treatment.
• Includes multiparametric flow cytometry analysis of specific immune cell subsets.
• Released under a permissive CC-BY-4.0 license for reuse.

Limitations

• Dataset is very small, consisting of only four patient cases.
• Column-level documentation is absent; field semantics must be inferred after download.
• Data is presented in a DOCX document, requiring manual extraction for analysis.

Provenance

Source

figshare

Collection Method

Clinical case series with longitudinal immunological investigations.

Time Range

Treatment and monitoring period of more than 24 months.

Freshness

Last updated 2026-05-13 05:02:42; freshness should be verified.