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Practice Guideline
. 2024 Sep;71(9):e31141.
doi: 10.1002/pbc.31141. Epub 2024 Jul 4.

Guideline for treating relapsed or refractory myeloid leukemia in children with Down syndrome

Milica Miladinovic  1 , Dirk Reinhardt  2 , Henrik Hasle  3 , Bianca F Goemans  4 , Daisuke Tomizawa  5 , Johann Hitzler  6 , Jan-Henning Klusmann  1
Affiliations
Practice Guideline

Guideline for treating relapsed or refractory myeloid leukemia in children with Down syndrome

Milica Miladinovic et al. Pediatr Blood Cancer. 2024 Sep.

Abstract

Treatment of relapsed and refractory myeloid leukemia in Down syndrome (r/r ML-DS) poses significant challenges, as prognosis is dire and there is no established standard treatment. This guideline provides treatment recommendations based on a literature review and collection of expert opinions, aiming to improve overall and event-free survival of patients. Treatment options include fludarabine and cytarabine (FLA) ± gemtuzumab ozogamicin (GO), azacytidine (AZA) ± panobinostat, and hematopoietic stem cell transplantation (HSCT). Preferred approaches are AZA ± panobinostat for cases with low blast count or FLA ± GO for cases with high blast count, followed by HSCT after remission. Further research is crucial for the investigation of targeted therapies (e.g., BH3 mimetics, LSD1, JAK inhibitors).

Keywords: Down syndrome; acute myeloid leukemia; guideline; trisomy 21.

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References

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