What are the treatment options for leukemia?

Treatment Options for Leukemia

The standard treatment for leukemia consists of induction chemotherapy with an anthracycline and cytarabine, followed by consolidation therapy, with treatment approaches varying based on leukemia subtype and patient risk factors. 1

Types of Leukemia and Risk Assessment

• Leukemia is broadly classified into acute and chronic forms, with acute forms further divided into Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL), each requiring different treatment approaches 1
• Risk assessment includes patient's age, initial leukocyte count, leukemia subtype, karyotype data, and medical history 2
• Favorable prognostic factors in AML include chromosomal translocations t(15;17) (acute promyelocytic leukemia), t(8;21), and t(16;16) 2
• Poor prognostic factors include advanced age (>60 years), antecedent myelodysplastic syndrome, and complex aberrant karyotypes 2

Treatment of Acute Myeloid Leukemia (AML)

Induction Therapy

• Standard induction regimen includes 3 days of an anthracycline (daunorubicin 60 mg/m² or higher, idarubicin 10-12 mg/m², or mitoxantrone 10-12 mg/m²) and 7 days of cytarabine (100-200 mg/m² continuous IV) 2
• For Acute Promyelocytic Leukemia (APL), induction should include an anthracycline plus all-trans retinoic acid (ATRA) 2
• Patients with excessive leukocytosis may require emergency leukapheresis before starting induction chemotherapy 2

Consolidation Therapy

• Patients achieving complete remission should receive one or more cycles of post-remission therapy 2
• Good-risk patients should receive chemotherapy only, preferably including high-dose cytarabine 2
• Intermediate-risk patients aged <40 years and high-risk patients aged <55 years with an HLA-identical sibling are candidates for allogeneic stem cell transplantation in first remission 2
• For APL, consolidation should include ATRA 2

Maintenance Therapy

• Maintenance chemotherapy and ATRA are beneficial in APL 2
• Maintenance is generally not routinely administered for non-APL AML outside of clinical trials 2

Treatment of Acute Lymphoblastic Leukemia (ALL)

Induction Therapy

• For adults <65 years with Ph-negative ALL, multiagent regimens based on vincristine, anthracyclines, corticosteroids, and L-asparaginase are recommended 3
• For pediatric patients, standard-risk cases receive 3-drug induction without anthracyclines, while high-risk cases receive 4-drug induction including anthracyclines 3

Consolidation and Maintenance

• Consolidation typically includes high-dose methotrexate, cytarabine, and other agents 3
• CNS prophylaxis with intrathecal chemotherapy is essential in all ALL treatment regimens 3
• Standard maintenance regimens include daily mercaptopurine, weekly methotrexate, monthly vincristine, and pulse dexamethasone 3

Treatment of Chronic Myeloid Leukemia (CML)

• Tyrosine kinase inhibitors (TKIs) targeting BCR::ABL1 are the standard of care, with six FDA-approved options 4
• First-line options include imatinib, dasatinib, bosutinib, nilotinib, and asciminib 4
• TKIs have improved CML-related mortality from 10-20% per year to 1-2% per year 4
• Allogeneic stem cell transplantation is reserved for patients who don't respond to second-generation TKIs, those with intolerance to multiple TKIs, or those with accelerated-phase or blast-phase CML 4

Treatment of Relapsed or Refractory Disease

• Patients in second or subsequent remission may qualify for allogeneic transplantation with an unrelated HLA-matched donor 2
• In relapsed APL, arsenic trioxide can induce remission even in patients who have become refractory to ATRA 2
• For CML, patients who fail first-line TKI therapy can be switched to alternative TKIs (dasatinib, bosutinib, nilotinib, ponatinib, or asciminib) 4

Response Evaluation and Monitoring

• Response to induction is monitored through clinical examination, serial peripheral blood counts, and bone marrow aspirates 2
• Complete remission requires normal cellularity of bone marrow, blast levels <5%, and recovery of peripheral blood counts 2
• For CML, molecular monitoring of BCR::ABL1 levels is essential to assess treatment response 4

Special Considerations

• Treatment should be conducted in centers with multidisciplinary expertise and adequate infrastructure 2
• Supportive care including transfusion support, infection prophylaxis, and management of complications is critical 2
• Medication adherence is particularly important for CML patients on TKI therapy to maintain treatment responsiveness 4
• TKI selection for CML should consider agent-specific adverse effects such as pleural effusion (dasatinib), arterio-occlusive events (nilotinib, ponatinib), or gastrointestinal disturbances (bosutinib) 4