What is the use of Nilotinib (Tasigna)?

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Last updated: March 28, 2025 • View editorial policy

From the Guidelines

Nilotinib is a targeted cancer medication primarily used to treat Philadelphia chromosome-positive chronic myeloid leukemia (CML) in adults, with a recommended dose of 300 mg twice daily, as indicated by the most recent guidelines 1. The medication works by blocking the BCR-ABL protein that drives the abnormal growth of leukemia cells in CML. Some key points to consider when using nilotinib include:

  • Taking the medication on an empty stomach to avoid excess drug exposure with fat-containing food 1
  • Monitoring for potential side effects such as rash, headache, fatigue, nausea, and elevated liver enzymes
  • Regular ECG monitoring to detect potential QT interval prolongation, which can cause serious heart rhythm problems 2, 1
  • Avoiding interactions with medications that affect CYP3A4 enzymes and certain foods like grapefruit
  • Regular blood tests to monitor response to therapy and detect side effects Nilotinib has been shown to be effective in treating CML, with studies demonstrating high rates of hematologic and cytogenetic responses 2, 1. However, it is essential to carefully manage side effects and drug interactions to ensure safe and effective use. In terms of specific patient populations, nilotinib may be prescribed with caution in patients with risk factors such as diabetes or coronary, cerebrovascular, or peripheral arterial disease 1. Overall, nilotinib is a valuable treatment option for patients with CML, and its use should be guided by the most recent clinical guidelines and patient-specific factors.

From the FDA Drug Label

Nilotinib is an inhibitor of the BCR-ABL kinase. Nilotinib binds to and stabilizes the inactive conformation of the kinase domain of ABL protein. In vitro, nilotinib inhibited BCR-ABL mediated proliferation of murine leukemic cell lines and human cell lines derived from patients with Ph+ CML

  • The primary use of Nilotinib (Tasigna) is to inhibit the BCR-ABL kinase, which is involved in the proliferation of leukemic cells in patients with Ph+ CML.
  • Nilotinib is used to treat Philadelphia chromosome-positive (Ph+) Chronic Myeloid Leukemia (CML).
  • The mechanism of action of Nilotinib involves binding to and stabilizing the inactive conformation of the kinase domain of ABL protein, thereby inhibiting BCR-ABL mediated proliferation of leukemic cells 3

From the Research

Use of Nilotinib (Tasigna)

  • Nilotinib is used in the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (CML) in chronic phase 4, 5, 6, 7, 8.
  • It is a second-generation tyrosine kinase inhibitor that works by inhibiting the tyrosine kinase activity of BCR-ABL, thereby decreasing ABL-associated cell proliferation and kinase autophosphorylation 4, 7, 8.
  • Nilotinib is effective as a first-line treatment for newly diagnosed CML in chronic phase, with studies showing higher rates of major molecular responses and complete cytogenetic responses compared to imatinib 4, 6, 8.
  • It is also used in the treatment of CML in patients who are resistant or intolerant to imatinib, with studies showing significant responses and improved survival rates 5, 7.
  • The recommended dosage of nilotinib is 400 mg orally twice daily, and it is generally well-tolerated with common side effects including rash, pruritus, nausea, fatigue, and headache 7.

Efficacy of Nilotinib

  • Studies have shown that nilotinib is effective in achieving major molecular responses, complete cytogenetic responses, and complete hematologic responses in patients with CML in chronic phase 4, 5, 6.
  • Nilotinib has also been shown to improve overall survival and progression-free survival rates in patients with CML in chronic phase 5, 6.
  • The efficacy of nilotinib has been demonstrated in both newly diagnosed patients and those who are resistant or intolerant to imatinib 4, 5, 6, 7, 8.

Safety and Tolerability of Nilotinib

  • Nilotinib is generally well-tolerated, with common side effects including rash, pruritus, nausea, fatigue, and headache 7.
  • Serious side effects include thrombocytopenia, neutropenia, elevated lipase, hyperglycemia, and hypophosphatemia 7.
  • Nilotinib has also been associated with a prolonged QT interval and sudden death, and concurrent use of CYP3A4 inhibitors should be avoided 7.

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.