Combining New and Traditional Therapies for AML and MDS Treatment

Medical researchers at MD Anderson Cancer Center are conducting a significant clinical trial exploring an innovative strategy: combining a newer targeted therapy, venetoclax (Venclexta, AbbVie), with traditional chemotherapy medications in younger patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). With nearly 100 participants enrolled so far, this ongoing Phase II trial set out to answer a crucial question: could this combination approach improve outcomes for patients who historically faced challenging prospects? 

The interim results, which include both short-term responses and early long-term survival data, offer valuable insights for patients and healthcare providers considering their treatment options. The findings summarized below are particularly relevant for those who might be candidates for subsequent stem cell transplantation, a potentially curative procedure for these blood cancers.

Ongoing Study Shows High Remission Rates in Younger AML Patients

The phase II ongoing trial (NCT02115295) enrolled 95 patients with a median age of 49 years old who were newly diagnosed with:

•  Acute myeloid leukemia (90%)
• Mixed phenotype acute leukemia (3%)
• High-risk myelodysplastic syndrome (7%)

We had the opportunity to interview the lead investigator of this study, Dr. Tapan Kadia, who shared insights into the study results. 

How Effective Was the Combination?

• Patients received one to six cycles, with a median of one cycle required to achieve remission 
• 93% of patients achieved a complete response 
• Overall response rate of 95% 
• 82% of AML patients who achieved a composite complete response were also  MRD negative
• For AML, the relapse-free survival was 78% at one year, and for MDS, it was 86% 
• The 2-year probability of survival was estimated at 73% for AML and 100% for high-risk MDS patients

What Were the Most Common Side Effects?

All patients experienced low blood counts during treatment, which is expected with intensive chemotherapy. This led to:

• Neutropenic fever (high temperature due to very low white blood cell counts) in about 8 out of 10 patients
• A recovery period of approximately 4 weeks for blood counts to return to safer levels
• Need for close monitoring and supportive care during this time

Other significant side effects included:

• Infections: About 3 in 10 patients developed bloodstream infections, and 2 in 10 developed pneumonia
• Gastrointestinal: About 1 in 7 patients experienced inflammation of the bowel (colitis)
• Liver: Some patients showed temporary changes in liver function tests

Importantly, despite these side effects, the treatment was generally manageable with proper medical care, with very low treatment-related mortality (2% within the first two months). Patients received preventive medications and growth factors to help manage these side effects.

The Importance of New Treatment Combinations

Using new treatment combinations can increase the number of patients with different diagnoses who can receive a treatment that quickly and effectively controls their cancer cells.  

The combination of venetoclax with CLIA chemotherapy demonstrated high effectiveness in younger patients with newly diagnosed acute myeloid leukemia and high-risk myelodysplastic syndrome. It offered participants the chance to achieve a deep response and proceed to allogeneic stem cell transplantation. This combination also could offer a long-term durability of response, with an estimated four-year overall survival rate of 71% in patients with newly diagnosed acute myeloid leukemia. 

These findings highlight the potential of CLIA-venetoclax as a powerful induction therapy that offers deep and sustained remissions in high-risk patients.

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Source:

• A Phase II Trial of Venetoclax in Combination with Cladribine, Idarubicin, and Cytarabine in Newly Diagnosed Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome