Barriers to HSCT and outcomes post-HSCT in responding patients with TP53-mutated AML

Outcomes in patients with TP53-mutated (TP53m) AML are particularly poor, with a median survival of ~6–9 months.¹ Although HSCT is currently the only strategy with demonstrated potential to offer durable survival in this population, a significant proportion of patients are unable to proceed to HSCT despite achieving appropriate response and having a potential donor.¹

Senapati et al. published a retrospective analysis in Bone Marrow Transplantation, of barriers to allogeneic HSCT (allo-HSCT) and outcomes following allo-HSCT, in patients with TP53m AML (N = 373).¹ Outcomes included cCR, OR, RFS, OS, and MRD status, and barriers were evaluated by individual patient chart review.¹ Given that short DoR is an established common barrier to HSCT, patients with DoR <2 months were excluded from the final analysis.¹

Key learnings

Among 278 patients aged <75 years, 41.4% achieved OS and 33.1% achieved cCR. Of patients with DoR >2 months (n = 76), median treatment cycle to best response was 1 and most patients achieved best response within 2 cycles (92.1%).

Patients treated with HSCT (n = 43) had significantly improved median RFS (9.3 months vs 4.5 months; p < 0.001) and median OS (13.6 months vs 7.6 months; p < 0.001), vs patients without HSCT (n = 33).

Among 33 non-transplanted patients, common barriers to HSCT included infection with/without poor PS or other organ dysfunction (51.5% of patients), concurrent other cancer (13.1%), and relapse <3 months post-remission (12.1%). Fourteen patients had >1 barrier to HSCT.

Favorable outcomes following HSCT were similar regardless of pre-transplant OR, cCR, or MRD negativity. Findings highlight HSCT as the only treatment currently available with the potential for meaningfully improved survival in patients with TP53m AML.