Understanding how to keep leukemia in remission for longer in older patients remains a significant challenge in cancer treatment. While achieving complete remission through initial treatment is a major milestone, the disease often returns within months, making it essential to find better ways to maintain remission. A new study has now provided key insights into how continued treatment with azacitidine can help extend remission periods in elderly patients with acute myeloid leukemia, a type of blood cancer that affects the bone marrow and blood cells.
A team of researchers led by Dr. Esther Natalie Oliva from Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Italy, alongside scientists from institutions across Europe and Japan, conducted an in-depth investigation into the effects of azacitidine as a follow-up treatment. Their work, published in the International Journal of Molecular Sciences, analyzes how the body responds to azacitidine at a cellular level. Using advanced genetic sequencing, a technique used to analyze the DNA of cells to identify mutations or changes in genes, the team examined changes in leukemia-related genes before and after treatment to better understand how azacitidine helps prevent relapse.
Dr. Oliva and her team’s study observed elderly leukemia patients who had achieved remission after intensive chemotherapy, a treatment that uses powerful drugs to kill cancer cells or stop them from growing. These individuals were then assigned to receive either subcutaneous azacitidine or supportive medical care without additional treatment. The results showed that patients who received azacitidine stayed in remission for nearly twice as long as those who did not. This benefit remained even after several years, reinforcing the potential of azacitidine in helping patients stay free of disease.
One of the most notable findings was that specific genetic traits, such as mutations in the FANCA gene, were linked to a higher risk of relapse. The study found that patients with these genetic changes were significantly more likely to see their leukemia return. “Our findings help clarify how genetic changes after remission affect the likelihood of relapse and highlight why some patients may need additional treatment strategies,” explained Dr. Oliva. This insight could pave the way for more tailored treatment plans based on a patient’s genetic profile, a unique set of genetic characteristics that can influence how a person responds to treatment.
The researchers also emphasized that while subcutaneous azacitidine shows promise in extending remission, it has not yet been linked to an overall survival benefit. In contrast, oral azacitidine has demonstrated an improvement in overall survival, which is why oral azacitidine is currently commercialized whereas subcutaneous azacitidine is not.
Importantly, they reported that subcutaneous azacitidine was generally well tolerated, with the most common side effect being a low white blood cell count, a measure of immune system health that can affect the body’s ability to fight infections. No patients died before relapse, suggesting that azacitidine is a safe option for elderly individuals who have already undergone intensive treatment. These findings support previous research showing that azacitidine provides a safer alternative to more aggressive chemotherapy in older patients.
Looking ahead, the team plans to further explore how other genetic factors influence relapse in acute myeloid leukemia. The use of high-tech genetic screening, a test used to identify genetic mutations that may affect disease risk or treatment response, to monitor patients after remission could provide valuable insights into predicting treatment responses and identifying which patients would benefit most from azacitidine therapy. “These results suggest that understanding genetic factors can help refine treatment plans after remission, offering new hope for older leukemia patients,” Dr. Oliva noted.
Dr. Oliva and colleagues’ study marks an important step forward in the treatment of acute myeloid leukemia, highlighting the potential of azacitidine as a valuable follow-up therapy for elderly patients. By combining genetic analysis with clinical data, scientists continue to advance leukemia treatment, moving closer to a more personalized approach, a method of tailoring medical treatment to an individual’s specific characteristics, including genetics and overall health, to cancer care.
Journal Reference
Oliva, E.N., Cuzzola, M., Porta, M.D., Candoni, A., Salutari, P., Palumbo, G.A., Reda, G., Iannì, G., Zampini, M., D’Amico, S., et al. “Translational Research on Azacitidine Post-Remission Therapy of Acute Myeloid Leukemia in Elderly Patients (QOL-ONE Trans-2).” International Journal of Molecular Sciences, 2024. DOI: https://doi.org/10.3390/ijms252111646
About the Author
Esther Natalie Oliva earned degrees in Medicine and Surgery in 1990 and a degree in General Haematology in 1994 from the University of Messina, Italy, From 1991 to 1997, she earned a Mario Negri Institute research grant to conduct research at the Nephrology Unit of the Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy.
Since 1998 she has been a practicing haematologist at the Haematology Unit of the same hospital in Reggio Calabria, in charge of the Myelodysplastic Syndromes, Aplastic Anemia, Anemia and Benign Haematology sections. Since 2018 she has a national scientific qualification for Associate Professor of Blood Diseases, Oncology and Rheumatology. She is junior editor of the American Journal of Blood Research, associate editor of Hemasphere and of the Hematological Malignancies specialty section of Frontiers in Oncology, guest editör of Journal of Clinical Medicine specialty section “Myelodysplastic Syndrome: Recent Advances and Future Directions”, of Cancers, specialty section “Chemotherapy for Hematologic Malignancies and special issue “Symptom Burden in Cancer: Assessment and Management”.
Dr. Oliva has developed a specific instrument for the evaluation of quality of life in myelodysplastic syndromes (QOL-E) and an instrument for the measurement of patient- reported outcomes in patients with haematological malignancies (HM-PRO).
She has also developed and coordinated clinical trials in pharmacological research and in quality of life. She is a founder member and since 2019 Vice President of Associazione QOL- ONE, a non-profit association for scientific research.
Since 2017 she is an examiner for the European Hematology Association (EHA) European Passport of Hematology. She is the Co-Chair of the EHA Scientific Working Group Quality of Life and Symptoms and is a member of the EHA Specialized Working Group Committee. She is regional representative (Calabria – Basilicata) of the Italian Society of Experimental Hematology (SIES) and she is an active member of the American Society of Hematology and the Italian Society of Hematology.
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