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Interferon alfa for the treatment of chronic myeloid leukemia

Robert S Negrin, MD
Charles A Schiffer, MD
Section Editor
Richard A Larson, MD
Deputy Editor
Alan G Rosmarin, MD


Interferon alfa (IFNa) has been extensively studied as treatment for patients with chronic myeloid leukemia (CML) since 1981, initially using partially purified IFNa [1], followed by recombinant IFNa-2a [2]. Prior to the development of the tyrosine kinase inhibitor (TKI) imatinib mesylate, interferon was the non-transplant treatment of choice for most patients with CML. However, with the development of TKIs and the increased toxicity of interferon compared with TKIs, this latter drug has been used much less commonly for the treatment of CML.

The use of interferon alfa in the treatment of CML will be discussed here. Other treatment options are discussed separately. (See "Overview of the treatment of chronic myeloid leukemia" and "Initial treatment of chronic myeloid leukemia in chronic phase" and "Treatment of chronic myeloid leukemia in accelerated phase" and "Treatment of chronic myeloid leukemia in blast crisis" and "Clinical use of tyrosine kinase inhibitors for chronic myeloid leukemia" and "Hematopoietic cell transplantation in chronic myeloid leukemia".)


Hematologic remissions have been noted in the majority of patients using IFNa-2a as a single agent and complete cytogenetic remissions (ie, absence of any Ph+ metaphase by conventional cytogenetics) were noted in a minority of patients, ranging from 13 to 27 percent [3,4].

The rate of cytogenetic response was higher in younger patients and in those with lower Sokal risk categories [5,6]. Although the degree of cytogenetic response can improve over time, it is uncommon to observe cytogenetic responses with further treatment if no reductions in the percent of Philadelphia chromosome metaphases have been seen during the first year of treatment.

In occasional patients, cytogenetic remissions were sustained for long periods of time, and in those patients with major cytogenetic responses, survival appeared to be improved [7]. Because of the considerable side effects from interferon, many clinicians chose to stop interferon therapy if cytogenetic improvement had not been seen after approximately one year of treatment. In two studies, 10-year survival ranged from 72 to 78 percent in patients achieving complete cytogenetic response to IFNa [3,4]. The majority of patients who achieve complete cytogenetic remission following treatment with IFNa still have molecular evidence of disease when assessed by the more sensitive polymerase chain reaction (PCR) [4,8-10], although there have been occasional patients in long term cytogenetic response who have not relapsed after cessation of the interferon. In part because of this observation, there are ongoing or planned clinical trials that administer IFNa late in the treatment course as a means of increasing the depth of response so as to permit discontinuation of tyrosine kinase inhibitors in a higher fraction of patients.

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Literature review current through: Sep 2017. | This topic last updated: Jan 13, 2017.
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