Treatment

The treatment of acute leukemia is decided by a hematologist-oncologist specialist depending on the specific subtype and results of molecular testing.

• Pretreatment: All patients should have prechemotherapy screening as part of preparation for cancer treatment.

• Chemotherapy

  • Systemic chemotherapy: The regimen of choice is based on individual patient and disease factors.

  • Intrathecal chemotherapy (commonly used): Consider adding for patients with or at high risk of CNS infiltration (e.g., all patients with ALL).

  • Targeted chemotherapy: Consider adding for leukemias with specific immunophenotype and genetic profiles, e.g., Philadelphia translocation.

• Adjunctive treatment, e.g., radiation therapy, immunotherapy, or stem cell transplantation (SCT): Consider based on individual evaluation.

• Supportive care: Provide holistic care to reduce symptoms and support patients and carers (see “Principles of cancer care”).

• Management of complications: initiate monitoring, prevention, and early aggressive treatment as needed for infection, bleeding, pancytopenia, and oncologic emergencies

Early and aggressive chemotherapy improves the prognosis, e.g., 80–90% of patients with ALL achieve complete remission with chemotherapy.

Regimens vary depending on the subtype of leukemia, the age of the patient, and immunophenotype and genetic study results.

• Induction chemotherapy

  • Average duration for an adult with ALL is 4–8 weeks

  • Reinduction therapy may be required in case of relapse or failure of primary induction

• Consolidation chemotherapy: average duration for an adult with ALL is 4–8 months

• Maintenance chemotherapy: average duration for an adult with ALL is 2–3 years

• Intrathecal chemotherapy: administration of chemotherapeutic agents (e.g., triple therapy with methotrexate, cytarabine, and hydrocortisone) directly into the subarachnoid space via lumbar puncture or using an intraventricular catheter with a reservoir placed under the scalp

  • Indications in ALL: Prevention of leukemic meningitis in all patients at the time of diagnosis

  • Indications in AML

    • Treatment of patients with confirmed CNS infiltration

    • Prevention in patients with high-risk APL

• CNS radiotherapy: Consider directed radiation therapy alongside intrathecal chemotherapy in select patients.

• Targeted therapy: Nonstandard chemotherapy or immunotherapy is indicated if certain mutations or markers are detected.

  • Tyrosine kinase inhibitors (TKIs)

    • Philadelphia chromosome-positive ALL: BCR-ABLTKIs, e.g., imatinib, ponatinib)

    • FLT3-ITD AML: Consider midostaurin

• Autologous or allogeneic stem cell transplantation: Indications include patients with poor prognostic factors (e.g., unfavorable cytogenetics) and those who do not achieve remission with chemotherapy

• Oncologic emergencies

  • Tumor lysis syndrome (TLS)

    • Management: typically includes fluid therapy (oral or IV) and rasburicase

    • Prophylaxis: Consider hydration, and urate-lowering therapy (i.e., allopurinol or rasburicase).

  • Leukostasis (e.g., due to hyperleukocytosis): Consider IV fluid resuscitation and/or cytoreductive therapy.

  • See “Oncologic emergencies” for details.

• Treatment-related complications

  • Anticancer therapy-induced myelosuppression: anemia , thrombocytopenia, and neutropenia

  • Mucositis

• Secondary hyperuricemia

  • Can lead to acute gout, uric acid stones, and urate nephropathy

  • Consider hydration, fluid administration, and urate-lowering therapy (e.g., allopurinol and rasburicase) as prophylaxis prior to chemotherapy.