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. 2025 Jan;206(1):186-194.
doi: 10.1111/bjh.19862. Epub 2024 Oct 28.

Secondary haematological dysplasia after CAR-T-cell therapy for acute lymphoblastic leukaemia in children

Alexandre Theron  1   2 , Anna Alonso-Saladrigues  1   3   4 , Jose-Luis Dapena  1   4 , Mónica López-Duarte  5 , Cristina Diaz de Heredia  6   7 , Jaime Verdú-Amorós  7   8 , Edurne Sarrate  9 , Elena Esperanza-Cebollada  4 , Esther Cuatrecasas  10 , Sandra Andreu  10 , Nuria Conde  1 , Nazaret Sanchez-Sierra  1 , Ignacio Isola  4   9 , Mireia Camós  4   7   9 , Montse Torrebadell  4   7   9 , Susana Rives  1   4   7 , Albert Català  1   4   7
Affiliations

Secondary haematological dysplasia after CAR-T-cell therapy for acute lymphoblastic leukaemia in children

Alexandre Theron et al. Br J Haematol. 2025 Jan.

Abstract

The use of CAR-T is becoming more widespread in the treatment of haematological malignancies. In adults, secondary myelodysplastic syndromes (MDS) after CAR-T have been described. However, there are currently no data on the risk of MDS following CAR-T in children treated for acute lymphoblastic leukaemia (ALL). We studied all children treated with CAR-T cells at Hospital Sant Joan de Déu in Barcelona and those with persistent cytopenias were evaluated at the cytological, cytogenetic, and molecular levels to look for MDS. A total of 106 patients received CAR-T for ALL. Among 40 patients without early relapse or subsequent therapy after CAR-T, four fulfilled the WHO criteria for myelodysplasia. These four patients had received a haematopoietic stem cell transplantation (HSCT) prior to CAR-T and presented cytopenias with severe dysplastic changes in bone marrow after CAR-T. One patient had clonal MDS with high-risk cytogenetics arising from the host cells requiring a HSCT. Three patients had non-progressive dysplasia arising from the donor cells. Two are alive in complete remission with stable cytopenias and one succumbed to ALL relapse. This is the first description of post-CAR-T MDS and haematological dysplasia in children and highlights the need to monitor children with persistent post-CAR-T cytopenias.

Keywords: CAR‐T cells; children; cytopenia; dysplasia; leukaemia; myelodysplastic syndrome.

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References

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