Acute myeloid leukemia (AML) treatment

Acute myeloid leukemia (AML) is a rapidly growing blood and bone marrow cancer with the excessive growth of defective white blood cells. The abnormal cells build up and disrupt the formation of healthy blood cells, causing potentially fatal conditions like infection, anemia, and bleeding. AML is the most prevalent acute leukemia in adults and has a high tendency to worsen rapidly if not treated.

Early diagnosis and appropriate treatment strategy are critical to better outcomes. With the progress of medical science, such as chemotherapy, stem cell transplantation, targeted therapy, and newer alternatives like CAR T-cell therapy, the treatment scenario of AML is changing worldwide.

What is the Treatment/Procedure?

Treatment for AML involves several strategies that may be used alone or in combination, depending on the patient’s age, overall health, genetic mutations, and subtype of AML. Common treatments include:

• Chemotherapy: The primary treatment for most AML patients.

• Targeted Therapy: Medications that specifically attack leukemia cells with genetic mutations.

• Stem Cell Transplantation: Also known as a bone marrow transplant, it replaces diseased bone marrow with healthy cells.

• Radiation Therapy: Less common, used to treat specific areas or before a stem cell transplant.

• Immunotherapy and CAR T-Cell Therapy: Still under clinical evaluation but promising for certain relapsed or refractory cases.

Indications

Treatment is initiated based on several factors, including:

• Confirmed diagnosis of AML through bone marrow biopsy and genetic tests.

• Presence of symptoms like fatigue, bleeding, fever, or infections.

• White blood cell count and cytogenetic findings.

• Patient’s age and comorbid conditions.

• Whether the leukemia is de novo or therapy-related (secondary AML).

Procedure Details

1. Induction Therapy: The first phase aims to induce remission by killing leukemia cells in the blood and bone marrow. The standard chemotherapy regimen involves a combination of cytarabine and an anthracycline (often called “7+3”).

2. Consolidation Therapy: After remission, high-dose chemotherapy or a stem cell transplant is used to destroy any remaining leukemia cells and reduce relapse risk.

3. Stem Cell Transplant: In patients with high relapse risk, allogeneic hematopoietic stem cell transplantation (HSCT) is recommended. The process involves high-dose chemotherapy followed by infusion of donor stem cells to regenerate healthy bone marrow.

4. Targeted Therapy: Drugs such as midostaurin, gilteritinib, and enasidenib are used for patients with specific gene mutations like FLT3, IDH1, or IDH2.

5. CAR T-Cell Therapy: Though still emerging in AML, China’s biotechnology sector is actively exploring CAR T-cell therapy for refractory or relapsed AML. In this method, a patient’s own T cells are modified in a lab to target leukemia cells and then reinfused. While currently more established in treating ALL, ongoing clinical trials in China show potential in AML.

Effectiveness

The effectiveness of AML treatment varies widely based on patient-specific factors. Overall:

• Complete remission can be achieved in 60-80% of younger adults and about 40-60% of older adults.

• Long-term survival is possible, especially in patients who undergo stem cell transplants.

• Targeted therapies have significantly improved outcomes in patients with actionable mutations.

• CAR T-cell therapy and novel immunotherapies are offering hope for patients with resistant AML, although they are still in early stages for this disease.

Risks and Side Effects

AML treatments, especially chemotherapy and HSCT, come with significant risks:

• Short-term effects: Fatigue, infections, nausea, vomiting, mucositis, hair loss, bleeding, and organ toxicity.

• Long-term effects: Fertility issues, cardiac complications, secondary cancers, and chronic graft-versus-host disease (in transplant cases).

• CAR T-cell therapy risks: Cytokine release syndrome (CRS) and neurotoxicity, requiring specialized care.

Close monitoring and supportive care are vital throughout the treatment process.

Recovery and Aftercare

Recovery from AML treatment can be physically and emotionally demanding:

• Hospital stays may be prolonged during induction and transplant phases.

• Regular follow-ups are essential to monitor for relapse, side effects, and secondary complications.

• Blood counts are frequently monitored post-treatment.

• Supportive care includes transfusions, antibiotics, antifungals, and nutritional support.

• Psychosocial support: Counseling and support groups help patients and caregivers cope with the journey.

Post-remission maintenance therapy and survivorship plans are becoming more standardized, especially in developed countries.

Cost and Availability

AML treatment cost varies depending on the therapy used, hospital facilities, duration of treatment, and country. While chemotherapy is relatively more accessible, stem cell transplants and CAR T-cell therapy are expensive and require specialized centers.

Globally, leading cancer hospitals offer comprehensive AML treatment, but patients may consider medical travel for affordability, availability of clinical trials, or shorter wait times.

Patient Experiences

Many AML survivors describe their journey as physically exhausting but life-transforming. Common themes include:

• The shock of diagnosis, followed by rapid initiation of treatment.

• Emotional and financial challenges, especially during long hospitalizations.

• Resilience, with strong support from family and oncology teams.

• Post-treatment fears, such as the risk of relapse and late side effects.

Access to personalized treatment, advanced diagnostics, and supportive care significantly improves the patient experience.

Cost in Countries

FAQ

Is AML curable?
Yes, especially in younger patients and those who achieve complete remission and undergo successful stem cell transplantation.

How long does AML treatment last?
Initial chemotherapy can take 4-6 weeks. Full treatment, including consolidation or transplant, may span several months.

What is the survival rate of AML?
Overall 5-year survival is about 30%, but it depends on age, mutations, and response to treatment.

Can AML relapse after treatment?
Yes, relapse is common. Patients may need salvage chemotherapy, a second transplant, or experimental therapies.

Is CAR T-cell therapy available for AML?
It is not yet standard but under active research, especially in China and the USA, for relapsed/refractory cases.

Can patients travel abroad for AML treatment?
Yes. Many patients from developing nations travel to countries like India, China, or Turkey for affordable, high-quality care.

What are the latest developments in AML treatment?
Advances include targeted drugs (FLT3, IDH1/2 inhibitors), monoclonal antibodies, and immunotherapies like CAR T and bispecific T-cell engagers.

Conclusion

Acute myeloid leukemia is a serious and aggressive disease, but with advancements in diagnostics, personalized medicine, and global access to high-quality care, the prognosis continues to improve for many patients. Chemotherapy remains the backbone of treatment, complemented by targeted therapies and transplants for eligible patients.

Emerging technologies like CAR T-cell therapy offer new hope, especially for those who do not respond to standard treatments. China’s leadership in biotech and cell therapy, including CAR T trials for AML, could redefine global care standards in the coming years.

Navigating AML treatment requires a multidisciplinary approach, strong patient support, and timely intervention. With the right guidance and access, remission—and even cure—is increasingly within reach for many.