AI Article Synopsis
- The study examines outcomes of 67 patients (average age 20.6 years) who underwent their first allogeneic hematopoietic stem cell transplant (HSCT) due to GATA2 deficiency across 21 centers in June 2022, showing varied indications for the transplant.
- The findings reveal significant rates of acute graft versus host disease (GvHD) and chronic GvHD; the incidence of relapses was low, with overall survival rates at 1 and 5 years being 83% and 72%, respectively.
- The analysis highlights that monitoring bone marrow for clonal evolution is crucial to initiate HSCT before the development of excessive blasts, noting that factors like earlier years of HSCT and certain
Article 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.
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| http://dx.doi.org/10.1111/bjh.19691 | DOI Listing |
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