Long‐term outcome after allogeneic stem cell transplantation for <scp>GATA2</scp> deficiency: An analysis of 67 adults and children from France and Belgium

Modalities and timing of haematopoietic stem cell transplant (HSCT) in patients with GATA2 deficiency are still subject to debate. On June 2022, 67 patients (median age 20.6 years) underwent a first allogeneic HSCT among 21 centres. Indications for HSCT were myelodysplastic syndrome (MDS) ≤5% blasts ± immunodeficiency (66%), MDS >5% blasts (15%), acute myeloid leukaemia (19%). Conditioning regimen was myeloablative in 85% and anti-thymocyte globulins were used in 67%. The cumulative incidence (CInc) of acute graft versus host disease (GvHD) grade II-IV and III-IV at day 100 were 42% and 13%, and CInc of chronic and extensive chronic GvHD at 2 years were 42% and 23%. CInc of relapses was 3% and 11% at 1 and 5 years. Overall survival (OS) at 1 and 5 years was 83% and 72% (median follow-up 5.6 years). The factors associated with worse OS in multivariable analysis were the year of HSCT, a history of excess blasts before transplant and peripheral blood stem cell (PBSC) grafts. Age at HSCT, non-myeloablative conditioning and PBSC grafts were associated with increased non-relapse mortality. In conclusion, bone marrow monitoring to identify clonal evolution and perform HSCT before the appearance of excess blast is mandatory.

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Publication Details

Journal
British Journal of Haematology
Published
2024-08-19
DOI
https://doi.org/10.1111/bjh.19691
Citations
3
Primary Topic
Congenital heart defects research
Type
article
Field-Weighted Citation Impact
0.70
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article

Long‐term outcome after allogeneic stem cell transplantation for GATA2 deficiency: An analysis of 67 adults and children from France and Belgium

Bruno Lioure, Bénédicte Neven, Sylvain Thépot, Jean Donadieu et al.
3 citations
British Journal of Haematology
Congenital heart defects research
0.70
article

Long‐term outcome after allogeneic stem cell transplantation for GATA2 deficiency: An analysis of 67 adults and children from France and Belgium

Bruno Lioure, Bénédicte Neven, Sylvain Thépot, Jean Donadieu, Marlène Pasquet, Xavier Poiré, Stéphanie Nguyen‐Quoc, Alice Garnier, Florian Chevillon, Sylvie Chevret, Virginie Gandemer, Laure Vincent, Flore Sicre de Fontbrune, Régis Peffault de Latour, Arthur Sterin, Ambroise Marçais, Édouard Forcade, Suzanne Tavitian, Matthieu Resche Riggon, Kristell Desseaux, Mony Fahd
article en
3 citations

Abstract

Modalities and timing of haematopoietic stem cell transplant (HSCT) in patients with GATA2 deficiency are still subject to debate. On June 2022, 67 patients (median age 20.6 years) underwent a first allogeneic HSCT among 21 centres. Indications for HSCT were myelodysplastic syndrome (MDS) ≤5% blasts ± immunodeficiency (66%), MDS >5% blasts (15%), acute myeloid leukaemia (19%). Conditioning regimen was myeloablative in 85% and anti-thymocyte globulins were used in 67%. The cumulative incidence (CInc) of acute graft versus host disease (GvHD) grade II-IV and III-IV at day 100 were 42% and 13%, and CInc of chronic and extensive chronic GvHD at 2 years were 42% and 23%. CInc of relapses was 3% and 11% at 1 and 5 years. Overall survival (OS) at 1 and 5 years was 83% and 72% (median follow-up 5.6 years). The factors associated with worse OS in multivariable analysis were the year of HSCT, a history of excess blasts before transplant and peripheral blood stem cell (PBSC) grafts. Age at HSCT, non-myeloablative conditioning and PBSC grafts were associated with increased non-relapse mortality. In conclusion, bone marrow monitoring to identify clonal evolution and perform HSCT before the appearance of excess blast is mandatory.

British Journal of HaematologyVol. 208(1)
Hôpital Necker-Enfants Malades (FR), Cliniques Universitaires Saint-Luc (BE), Inserm (FR), Centre Hospitalier Universitaire de Bordeaux (FR), Centre Hospitalier Universitaire de Nantes (FR), Sorbonne Université (FR), Centre Hospitalier Universitaire de Montpellier (FR), Assistance Publique – Hôpitaux de Paris (FR), Centre Hospitalier Universitaire d'Angers (FR), Hôpital Nord (FR), Pitié-Salpêtrière Hospital (FR), Hôpital Robert-Debré (FR), Hôpital Purpan (FR), Hôpital Saint-Louis (FR), Hôpitaux Universitaires de Strasbourg (FR), Centre Hospitalier Universitaire de Rennes (FR), Hôpital de la Timone (FR)
Good health and well-being
Openalex Percentile: Top 30%
Congenital heart defects research
0.70
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