What is the overall response rate for CPX-351 in therapy-related Acute Myeloid Leukemia?

CPX-351 is a liposomal formulation of cytarabine and daunorubicin approved for therapy-related acute myeloid leukemia (t-AML) and AML with myelodysplasia-related changes. The overall response rate (ORR) includes both complete remission (CR) and CR with incomplete blood count recovery (CRi). Studies report ORR ranging from 47% to 61% in t-AML and related subtypes, though rates vary based on patient characteristics and study design.

What the research says

A retrospective study of 60 patients treated with first-line CPX-351 for t-AML or AML with myelodysplastic-related changes found an overall response rate of 61%, with 58% achieving complete remission ⁴. A larger real-world analysis of 188 patients (29% with t-AML) reported a CR/CRi rate of 47% after induction, with 64% of evaluable patients achieving measurable residual disease negativity ⁹. Another retrospective study of 169 patients with AML-MRC or t-AML showed an ORR of 53.3% across all ages, with similar rates in younger (<60 years, 47.2%) and older (≥60 years, 54.9%) patients ¹¹. The phase 3 trial that led to CPX-351 approval demonstrated improved survival in AML with myelodysplasia-related changes compared to standard 7+3 chemotherapy, though response rates were not separately reported for t-AML in that analysis ². Factors associated with lower response include complex karyotype ⁴⁹ and prior treatment with hypomethylating agents ⁹.

What to ask your doctor

• What is my expected overall response rate with CPX-351 based on my specific AML subtype and genetic profile?
• How does my age and overall health affect the likelihood of response to CPX-351?
• If I achieve remission with CPX-351, what are the next steps, including the possibility of stem cell transplant?
• What are the common side effects of CPX-351, and how are they managed?
• Are there alternative treatments if CPX-351 is not effective for me?