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Case report shows fostamatinib improves autoimmune hepatitis and cytopenia in a pediatric patient post-HSCT, indicating its potential therapeutic role.
To describe the effectiveness and safety of fostamatinib off-label use in a pediatric patient with autoimmune cytopenia and autoimmune hepatitis following allogeneic hematopoietic stem cell transplantation (HSCT). We report the detailed changes in clinical, histopathological, and cytokine profiling in a pediatric HSCT recipient during treatment with fostamatinib. Fostamatinib administration was well-tolerated with no adverse events observed, leading to sustained hematological recovery and a significant improvement in liver enzyme levels. Histological features confirmed the complete remission of autoimmune hepatitis. Cytokine profiling demonstrated substantial normalization of pro-inflammatory markers, downregulated at onset. Fostamatinib demonstrated a dual benefit in resolving autoimmune cytopenia and immune-mediated liver inflammation, supporting its potential as a therapeutic option for immune dysregulation following allogeneic HSCT.
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Tessitore et al. (2026) studied this question.
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