Chronic Myeloid Leukemia Coverage from Every Angle

Self-Initiated Discontinuation of Nilotinib in CML: Case Report

By: Julia Fiederlein
Posted: Thursday, June 25, 2020

A patient profile published in Case Reports in Hematology describes a 55-year-old man who self-initiated the discontinuation of tyrosine kinase inhibitor (TKI) therapy for chronic myeloid leukemia (CML), despite having suboptimal molecular remission. Although the patient experienced treatment-free remission, Stephen E. Langabeer, PhD FRCPath, of Saint James Hospital, Dublin, and colleagues advised against this approach.

“A major advance in CML management has been the ability to discontinue TKI therapy achieving a treatment-free remission, yet this option is only available to eligible patients who present with low-risk disease and who subsequently attain deep and sustained molecular responses,” the authors commented. Treatment discontinuation by ineligible patients may result in ineffective treatment, molecular relapse, and increased patient anxiety.

In this case report, the patient presented with fatigue, headache, left upper quadrant abdominal discomfort, and palpable splenomegaly. Molecular analysis confirmed high levels of e14a2 BCR-ABL1 transcripts. He was subsequently diagnosed with chronic phase CML with a low-risk Sokal score and was enrolled in a phase II clinical trial to evaluate the efficacy of a TKI.

The patient received 300 mg of nilotinib twice daily. The patient reported fatigue, headaches, and an overall poor quality of life. He did not achieve a deep and prolonged molecular response and therefore was not considered a candidate for treatment-free remission.

After approximately 10 years of treatment, the patient reported self-initiated discontinuation of the nilotinib treatment. He did not experience any symptoms consistent with TKI withdrawal syndrome and continued to receive monthly BCR-ABL1 monitoring. At 18 months after treatment cessation, the patient was in good health and maintained stable BCR-ABL1 levels.

In closing, the investigators still suggest following the existing guidelines regarding treatment-free remission eligibility. However, they noted, the guidelines may change as more TKI discontinuation trials are conducted.

Disclosure: The study authors reported no conflicts of interest.

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