Treatment of Chronic Myeloid Leukemia: Frontline Therapy

Chemotherapy with hydroxyurea and busulfan were the main options for many years, and although these agents can control hematologic manifestations of CML, they rarely if ever induce the cytogenetic responses required to change the natural history of the disease. [Interferon] then became the standard therapy for most patients because of its ability to induce cytogenetic responses in 40% to 60% of patients, including 5% to 30% with a complete cytogenetic response.

Treatment with [interferon] was associated with significant toxicity, but many responses were durable, and some patients have been cured with [interferon] alone.[Kantarjian 2003a] Allogeneic stem cell transplantation can induce long-term remissions in many patients eligible for this procedure, although with risks including death and chronic complications such as chronic graft-vs-host disease.

The reported 20-year event-free survival for patients transplanted in chronic phase was 40% to 45%.[Robin 2005] Stem cell transplantation is generally now considered a second- or third-line option. The current standard first-line therapy for CML in the chronic phase is with a tyrosine kinase inhibitor.

Current guidelines recommend imatinib, dasatinib, or nilotinib as the choice of first-line therapy for newly diagnosed chronic phase CML (Management Guidelines).[NCCN] However, the guidelines state that preliminary data suggest patients with an intermediate- or high-risk score may benefit from treatment with dasatinib or nilotinib vs imatinib.

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