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Case Reports
. 2025 Jul 1;47(5):e199-e202.
doi: 10.1097/MPH.0000000000003051. Epub 2025 May 12.

Successful Unmanipulated Haploidentical Hematopoietic Stem Cell Transplant With Post-transplant Cyclophosphamide in a Child With Down Syndrome and Myelodysplastic Syndrome

Chane Choed-Amphai  1   2   3 , Yigal Dror  4 , Michaela Cada  4 , Tal Schechter  1 , Joerg Krueger  1 , Muhammad Ali  1 , Yogi Chopra  1
Affiliations
Case Reports

Successful Unmanipulated Haploidentical Hematopoietic Stem Cell Transplant With Post-transplant Cyclophosphamide in a Child With Down Syndrome and Myelodysplastic Syndrome

Chane Choed-Amphai et al. J Pediatr Hematol Oncol. .

Abstract

Allogeneic hematopoietic stem cell transplant (HSCT) in children with Down syndrome and hematologic malignancies is challenging and is reserved for those who develop relapsed/refractory disease due to concerns regarding transplant-related mortality. Haploidentical HSCT, although performed in limited cases using graft manipulation methods to prevent graft-versus-host disease (GVHD), often results in dismal outcomes. Herein, we report a case of a 12-year-old boy with Down syndrome and myelodysplastic syndrome who underwent unmanipulated haploidentical HSCT using a reduced toxicity treosulfan-based conditioning and in vivo T-cell depletion with post-transplant cyclophosphamide. At the 1-year follow-up, he is alive with complete donor chimerism and no chronic GVHD.

Keywords: Down syndrome; haploidentical transplant; myelodysplastic syndrome; pediatric.

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Conflict of interest statement

The authors declare no conflict of interest.

References

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