Journal: Blood cancer journal
This single-center retrospective study evaluated outcomes of allogeneic stem cell transplantation in 2045 patients with newly diagnosed higher-risk MDS (IPSS-R >3.5) treated between 2000 and 2023; 427 patients (21%) proceeded to transplant.
Key findings:
- Post-transplant outcomes for the overall transplanted cohort:
- Median overall survival (OS): 23.9 months
- Median progression-free survival (PFS): 14.4 months
- 5-year cumulative incidence of relapse: 37%
- 5-year treatment-related mortality (TRM): 25%
- Age did not significantly affect OS, PFS, relapse risk, or TRM among transplanted patients.
- Survival after transplant improved over time:
- Median OS 2000–2010: 11.8 months
- Median OS 2011–2016: 28.2 months
- Median OS 2017–2023: 40.2 months (p = 0.01 for trend)
The most powerful determinant of post-transplant survival was TP53 status:
- TP53–wild type:
- 5-year OS: 69%
- Transplant markedly reduced risk of death: HR 0.32, p < 0.001, indicating substantial long-term benefit.
- TP53-mutated disease:
- Monoallelic TP53: median OS 9.1 months; no statistically significant survival benefit from transplant (HR 0.88, p = 0.69).
- Biallelic TP53: median OS 6.8 months; no clear significant benefit (HR 0.76, p = 0.14).
In summary, allogeneic transplantation offers excellent long-term survival in higher-risk MDS when TP53 is wild type, with clear improvements over recent decades. In contrast, outcomes for TP53-mutated MDS—especially biallelic—remain poor and show no clear survival advantage from transplant in this analysis.