Efficacy and Safety of Matched Unrelated Donor Hematopoietic Stem Cell Transplantation for Non–Down syndrome Acute Megakaryoblastic Leukemia: A Long-Term Follow-Up Study DOI

Fei Pan,

Long Jing,

Xingyu Cao

et al.

Research Square (Research Square), Journal Year: 2025, Volume and Issue: unknown

Published: May 19, 2025

Abstract Non-Down syndrome acute megakaryoblastic leukemia (non-DS-AMKL) is a rare and aggressive subtype of pediatric myeloid (AML) with heterogeneous genetic landscape poor prognosis. Matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) potentially curative approach, yet data on its long-term efficacy safety remain limited. We retrospective analysis 12 non-DS-AMKL patients undergoing first MUD-HSCT (2016–2025) outcomes—overall survival (OS), leukemia‑free (LFS), non‑relapse mortality (NRM), cumulative incidence relapse (CIR), transplant-related complications—estimated by Kaplan–Meier competing-risk models. Among (median age: 2 years, range: 1–7), profiling revealed fusion genes in 50% patients—predominantly KMT2A rearrangements—and complex cytogenetics 50%. All 5 deceased had adverse or alterations. Engraftment was achieved 100% patients, median neutrophil platelet recovery times 15 10 days, respectively. The follow-up 32 months (range, 2.0–105.3), 7/12 (58.3%) remained alive 53.9 28.1–105.3). Day-100 Transplant-Related Mortality (TRM) rate 16.7%, 5-year OS LFS rates 58.3% (95% CI, 30.4–86.2%) CIR as well NRM 25% 9.4–66.6%). Additionally, grade 3–4 GVHD occurred chronic while viral reactivation for CMV EBV were 66.7% 33.3%, This represents the decade-long study non-DS-AMKL. feasible non-DS-AMKL, rapid engraftment durable remission CR patients. Precision risk stratification post-transplant surveillance strategies are essential to improve outcomes this population.

Language: Английский

Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients DOI
Pasquale Niscola

Expert Review of Hematology, Journal Year: 2025, Volume and Issue: unknown

Published: March 31, 2025

Introduction Older patients with acute myeloid leukemia (AML) are often unsuitable for standard treatments and traditionally have a dismal prognosis. For 20 years, hypomethylating agents (HMAs), as single recently backbone venetoclax, been used in this setting. The oral combination of decitabine cedazuridine (C-DEC), which is therapeutically pharmacologically equivalent to the intravenous (IV) formulation (IV-DEC), has expanded therapeutic arsenal AML, allowing better convenience administration. This review provides an overview C-DEC, current clinical applications, ongoing studies, highlighting its potential role managing AML older patients.

Language: Английский

Citations

0
Efficacy and Safety of Matched Unrelated Donor Hematopoietic Stem Cell Transplantation for Non–Down syndrome Acute Megakaryoblastic Leukemia: A Long-Term Follow-Up Study DOI

Fei Pan,

Long Jing,

Xingyu Cao

et al.

Research Square (Research Square), Journal Year: 2025, Volume and Issue: unknown

Published: May 19, 2025

Abstract Non-Down syndrome acute megakaryoblastic leukemia (non-DS-AMKL) is a rare and aggressive subtype of pediatric myeloid (AML) with heterogeneous genetic landscape poor prognosis. Matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) potentially curative approach, yet data on its long-term efficacy safety remain limited. We retrospective analysis 12 non-DS-AMKL patients undergoing first MUD-HSCT (2016–2025) outcomes—overall survival (OS), leukemia‑free (LFS), non‑relapse mortality (NRM), cumulative incidence relapse (CIR), transplant-related complications—estimated by Kaplan–Meier competing-risk models. Among (median age: 2 years, range: 1–7), profiling revealed fusion genes in 50% patients—predominantly KMT2A rearrangements—and complex cytogenetics 50%. All 5 deceased had adverse or alterations. Engraftment was achieved 100% patients, median neutrophil platelet recovery times 15 10 days, respectively. The follow-up 32 months (range, 2.0–105.3), 7/12 (58.3%) remained alive 53.9 28.1–105.3). Day-100 Transplant-Related Mortality (TRM) rate 16.7%, 5-year OS LFS rates 58.3% (95% CI, 30.4–86.2%) CIR as well NRM 25% 9.4–66.6%). Additionally, grade 3–4 GVHD occurred chronic while viral reactivation for CMV EBV were 66.7% 33.3%, This represents the decade-long study non-DS-AMKL. feasible non-DS-AMKL, rapid engraftment durable remission CR patients. Precision risk stratification post-transplant surveillance strategies are essential to improve outcomes this population.

Language: Английский

Citations

0