American journal of hematology · 2025
Sensitivity of Pediatric Myelodysplastic Syndromes With Excess of Blasts With UBTF -TD to Venetoclax/Azacitidine.
Клинический случайПисьмо в редакциюПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
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История · было → стало
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Merli Pietro, Masetti Riccardo, Pigazzi Martina, Girardi Katia, Miele Evelina, Bresolin Silvia, Baccelli Francesco, Peplinski Jack H, Becilli Marco, Paganelli Valeria, Strocchio Luisa, Buldini Barbara, Pagliara Daria, Meshinchi Soheil, Locatelli Franco
Аннотация
UBTF-TD has been reported in a significant percentage of childhood MDS-EB and has been associated with inferior survival compared to that of patients with the wild-type gene. We treated three consecutive pediatric patients affected by UBTF-TD MDS-EB with venetoclax and azacitidine (ven/aza) in combination as 28-day cycles on a compassionate use basis three consecutive pediatric patients affected by UBTF-TD MDS-EB as a bridge to allogeneic HSCT. Treatment with ven/aza was well-tolerated, and all patients responded to the ven/aza course, achieving CR with flow-cytometry negativity. All three patients were bridged to myeloablative HSCT. All patients are disease-free and graft-versus-host disease-free at last follow-up. Comprehensive biological characterization of the disease showed (i) high expression of the BCL2 gene, paralleled by a low expression of BCL2A1 and MCL1; (ii) overexpression of both HOXA and HOXB; and (iii) a distinct methylation signature of patients with UBTF-TD myeloid neoplasms.