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Treatment of acute lymphoblastic leukaemia

Authoring team

The backbone of therapy is multi-agent chemotherapy with vincristine, corticosteroids and an anthracycline with allogeneic stem cell transplantation for eligible candidates (1):

  • chemotherapy consists of
    • induction
    • consolidation
    • long-term maintenance
      • maintenance therapy utilizes oral 6-mercaptopurine or methotrexate delivered once weekly or once monthly (2)
  • with CNS prophylaxis given at intervals throughout therapy
  • elderly patients are often unable to tolerate such regimens and carry a particularly poor prognosis

If the patient has Ph-chromosome positive ALL, the current treatment includes the use of tyrosine kinase inhibitors like imatinib, nilotinib, dasatinib, or ponatinib (2,3)

  • have been associated with improved 4-year survival rates of 85% to 90% in Philadelphia chromosome–positive ALL

Children respond well to treatment:

  • 95% achieve complete remission
  • 50-60% of whom are cured with postremission chemotherapy

Fewer advances have been achieved in adults:

  • 80% achieve complete remission
  • about 35% achieve long-term disease-free survival

Notes:

  • allogenic transplantation has shown clear benefits in the following patients who are at high risk of ALL:
    • individuals with Philadelphia chromosome-positive disease
    • poor initial response to treatment (4)
    • it is also useful in improving the outcome of adults with the t(4;11) subtype of ALL (but the benefits in infants with this genotype is controversial) (4)

References:

  1. Terwilliger T, Abdul-Hay M. Acute lymphoblastic leukemia: a comprehensive review and 2017 update. Blood Cancer J. 2017 Jun 30;7(6):e577.
  2. Puckett Y, Chan O. Acute Lymphocytic Leukemia. [Updated 2023 Aug 26]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan.
  3. Kantarjian H, Aldoss I, Jabbour E. Management of Adult Acute Lymphoblastic Leukemia: A Review. JAMA Oncol. Published online May 01, 2025.
  4. Pui CH, Robison LL, Look AT. Acute lymphoblastic leukaemia. Lancet. 2008;371(9617):1030-43.

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