Real-World Outcomes of CPX-351 For High-Risk AML Patients

CPX-351 Shows Durable Results in Real-World Patients with High-Risk AML

For people newly diagnosed with high-risk acute myeloid leukemia (AML), treatment decisions can feel urgent and uncertain. Two specific types, therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC), are known for their aggressive behavior and complex genetic backgrounds. A newer treatment called CPX-351 (Vyxeos, Jazz Pharmaceuticals), already approved based on earlier clinical trials, was evaluated again in a large U.S. real-world study. This research offers patients and families a clearer view of what to expect outside of a clinical trial setting.

What is CPX-351 and how does it treat secondary AML 

CPX-351 is a specialized chemotherapy. It is a liposomal combination of two commonly used chemotherapies called daunorubicin and cytarabine. The combination is delivered via liposomes, which are tiny, bubble-like carriers that are given through an IV.   

The U.S. Federal Drug Administration (FDA) approved CPX-351 for adults with newly diagnosed secondary AML in 2017. This includes t-AML and AML-MRC. In 2021, the approval was expanded to include children 1 year and older. The approval was based on a phase 3 trial that showed better response rates and survival compared to standard chemotherapy.

Treatment responses and survival outcomes in the real world

While clinical trials offer controlled data, real-world studies can confirm how well a therapy works in everyday settings. Real-world studies often include people who may not be typically represented in clinical trials. The V-RULES study looked at how well CPX-351 treated a broad group of patients with newly diagnosed t-AML or AML-MRC at multiple U.S. centers.

The study included 161 people with secondary AML. Most received only one cycle of CPX-351. One in three received additional cycles as consolidation therapy. Consolidation therapy is therapy that is given after the first treatment to destroy any remaining cancer cells. Nearly half the participants were under age 60. Among the 149 patients evaluable for response: 

• 63% achieved a complete remission or similar outcomes.
• Patients with t-AML responded more (85%) than those with AML-MRC (53%).
• The median overall survival was 12.9 months. This was longer than what had been observed in the original trial, which reported 9.56 months. 
• Patients under age 60 had a longer median survival (17.8 months) compared to those 60 or older (10.6 months).

Read more about the role of CPX-351 uses in pediatric AML here. 

The Role of Genetic Features in Survival

The study also looked at how survival differed based on genetic features. A quarter of patients had a TP53 mutation, a high-risk gene change linked to shorter survival. These patients had a median survival of 5.3 months. On the other hand, patients with myelodysplasia-related mutations, found in 63% of tested patients, lived longer, with a median survival of 17.8 months.

Understanding these genetic factors may help patients and their care teams make more informed decisions about their treatment plans and additional therapies, such as clinical trials or transplant.

Hematopoietic Cell Transplant Improved Long-Term Outcomes

Among patients who went on to receive a hematopoietic cell transplant (sometimes called a stem cell transplant) after CPX-351, median survival increased to 45.6 months. This suggests that CPX-351 can serve as an effective bridge to transplant, which may be an important goal for eligible patients.

If a transplant is being considered, this data supports CPX-351 as a potential first step to help improve long-term survival.

Side Effects and Recovery

Treatment with CPX-351 came with expected side effects. The most common serious issues were infections (52%) and febrile neutropenia—fever due to low white blood cell counts (42%). Recovery of white blood cells and platelets took about 35 to 36 days after the first cycle of treatment. Two patients had serious heart-related events.

Knowing what to expect during recovery can help patients prepare and coordinate with their care teams for infection prevention and supportive care needs.

Summary

The V-RULES study reinforces that CPX-351 remains an effective and safe treatment option for patients with newly diagnosed t-AML or AML-MRC, even outside of clinical trial settings. Younger patients and those who receive a transplant may see particularly favorable outcomes. Genetic testing may help tailor treatment approaches further.

Stay tuned for more treatment advances updates with the HealthTree News site! 

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Source: 

• V-RULES: Real-world effectiveness and safety of CPX-351 in patients with secondary acute myeloid leukemia (AML).