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

Fiche publication

Date publication

août 2024

Journal

British journal of haematology

Auteurs

Membres identifiés du Cancéropôle Est :
Dr LIOURE Bruno

Tous les auteurs :
Sicre de Fontbrune F, Chevillon F, Fahd M, Desseaux K, Poiré X, Forcade E, Sterin A, Neven B, Gandemer V, Thepot S, Garnier A, Lioure B, Marcais A, Nguyen-Quoc S, Tavitian S, Vincent L, Donadieu J, Resche Riggon M, Chevret S, Pasquet M, Peffault de Latour R

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/39159950

Résumé

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.