HIGHLIGHTS
SUMMARY
Recognizing a genetic cause of BMF is the crucial step in preventing inappropriate administration of immunosuppressive therapy (IST), timely initiation of hematopoietic stem_cell transplantation (HSCT), the selection of unaffected HLA-matched sibling donors, and importantly adapting the conditioning regimen to avoid toxicity arising from underlying genetic defects. To the classical IBMFS, indirect damage to the hematopoietic stem_cells due to constitutional gene defects such as CTLA4 or DADA2 mutations, can also cause pancytopenia. The authors have developed a renewed diagnostic protocol for all pediatric patients suspected of BMF in which genetic and functional diagnostics including . . .
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