Science Direct, 11/09/2026
A new retrospective study published in Transplantation and Cellular Therapy (September 2026) brings positive news for patients with high-risk acute myeloid leukemia (AML). The study confirms that cord blood (CB) stem cell transplantation significantly reduces relapse rates and improves progression-free survival (PFS) compared to other donor sources.
Context & Background
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potentially curative treatment for high-risk AML (such as cases with adverse cytogenetics, myelodysplasia-related AML, or poor treatment response). Although an HLA-matched sibling donor (MSD) is typically the preferred choice, finding a suitable matched donor remains a major challenge. Cord blood stands out due to its potent graft-versus-leukemia (GVL) effect, yet direct comparative data against other donor sources in poor-prognosis populations have been limited.
Study Design & Methodology
- Study Population: 488 patients with high-risk AML undergoing their first stem cell transplant (2008–2024).
- Donor Stratification:
- Cord Blood (CB): 394 patients (80.7%).
- Other Donors: 94 patients (19.3%) — including HLA-matched sibling donors (MSD), haploidentical family donors (HID), and matched/mismatched unrelated donors.
- Analytical Approach: Multivariable analysis and propensity score matching (PSM with 94 matched pairs) were conducted to adjust for baseline confounders.
| Clinical Outcome (5-Year) | Cord Blood (CB) | Other Donor Sources | Statistical Significance |
| Progression-Free Survival (PFS) | 49.8% | 35.4% | Significantly improved ($P = 0.04$) |
| Cumulative Incidence of Relapse | 24.5% | 42.8% | Markedly lower ($P < 0.01$) |
| Overall Survival (OS) | 56.2% | 44.4% | Comparable ($P = 0.16$) |
| Non-Relapse Mortality (NRM) | 25.7% | 21.8% | Comparable ($P = 0.75$) |
Key Takeaway: Despite CB recipients being older (median age 56 vs. 48 years) and having lower complete remission (CR) rates prior to transplant (15.0% vs. 35.1%), this group still achieved superior long-term disease control.
Conclusion & Clinical Implications
The robust GVL effect mediated by cord blood T cells effectively suppresses leukemia relapse without increasing the risk of non-relapse mortality (NRM). These findings highlight cord blood as a highly favorable and viable alternative donor option, offering higher survival prospects for high-risk AML patients even when conventional donor options are available.
Source: Science Direct
Link: https://www.sciencedirect.com/science/article/abs/pii/S2666636726003532?via%3Dihub
