Sustained Remission in Acute Promyelocytic Leukemia with ATO-ATRA and Gemtuzumab Ozogamicin: Long-Term Phase 2 Outcomes

Highlight

  • The combination of ATO-ATRA with gemtuzumab ozogamicin (GO) achieves a 94% cure rate in both standard- and high-risk acute promyelocytic leukemia (APL) patients.
  • Five-year event-free, disease-free, and overall survival rates exceed 92%, with median follow-up of over 5 years.
  • The regimen is well tolerated with low induction mortality (2.7%) and no veno-occlusive disease observed.
  • Negative measurable residual disease was achieved in over 97% of patients in complete remission, supporting deep molecular responses.

Study Background and Disease Burden

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