Conclusion
For children with relapsed MDS with a good performance status and
absence of uncontrolled infections, GvHD, and other treatment related
toxicities, a second HCT should be considered if disease control can be
achieved and if aligned with the family’s goals. Acknowledging that
early second HCT is associated with increased
TRM16,18,21, temporizing disease control with less
myelosuppressive agents, like hypomethylating agents in tandem with DLI,
may be beneficial. Individualized treatment approaches that utilize
targeted therapies with less risk for TRM like Bcl-2 inhibition
(e.g.venetoclax)38 or immunotherapy (e.g. magrolimab)
should be further studied in pediatric MDS. Consolidation strategies in
the event of relapse after second HCT are not standardized; selected
novel treatments might provide therapeutic benefit with minimal toxicity
and therefore warrant consideration.