Secondary haematological dysplasia after <scp>CAR</scp>‐T‐cell therapy for acute lymphoblastic leukaemia in children

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.

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Publication Details

Journal
British Journal of Haematology
Published
2024-10-28
DOI
https://doi.org/10.1111/bjh.19862
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
0.00

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article

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

Alexandre Théron, Jaime Verdú‐Amorós, Ignacio Isola, Albert Català et al.
British Journal of Haematology
CAR-T cell therapy research
article

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

Alexandre Théron, Jaime Verdú‐Amorós, Ignacio Isola, Albert Català, Susana Rives, Monica López‐Duarte, Edurne Sarrate, Nazaret Sanchez‐Sierra, Cristina Díaz de Heredia, Núria Conde, José Luís Dapena, Anna Alonso‐Saladrigues, Mireia Camós, Elena Esperanza‐Cebollada, Esther Cuatrecasas, Montserrat Torrebadell, Sandra Andreu
article en

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.

British Journal of HaematologyVol. 206(1)
Hospital Sant Joan de Déu Barcelona (ES), Université de Montpellier (FR), Instituto de Salud Carlos III (ES), Marqués de Valdecilla University Hospital (ES), Vall d'Hebron Institut de Recerca (ES), Centre for Biomedical Network Research on Rare Diseases (ES), Hôpital Arnaud de Villeneuve (FR), INCLIVA Health Research Institute (ES), Instituto de Investigación de Enfermedades Raras (ES), Universitat de Barcelona (ES)
Fundació la Marató de TV3, Instituto de Salud Carlos III
Good health and well-being
Openalex Percentile: Top 66%
CAR-T cell therapy research
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