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[Oral molecular targeting agents in hematological malignancy]
Shihoko Wakabayashi1, Kazuteru Ohashi
1Department of Hematology, Tokyo Metropolitan Infectious Disease Center Komagome Hospital, Tokyo, Japan.
Abstract:
Molecular target therapy is progressive and promising in various hematological malignancies. Imatinib is now the treatment of choice for chronic-phase chronic myeloid leukemia. Eight-year data from the pivotal trial of imatinib, the IRIS trial, showed high long-term response rates and favorable tolerability profile compared with previous therapies. For patients with primary resistance to imatinib, hematologic disease recurrence, or emergent BCR-ABL kinase domain mutations, the potent second generation tyrosine kinase inhibitors dasatinib and nilotinib are now available. Since the introduction of all-trans retinoic acid (ATRA) in the 1980s, the strategy for treating acute promyelocytic leukemia (APL) has shifted from conventional chemotherapy to cell differentiation. The combination of ATRA and anthracycline-based chemotherapy is currently the standard approach to treat newly-diagnosed APL. Multiple myeloma (MM) is also one of the major therapeutic targets in using molecular based technology. The recent availability of clinical data regarding thalidomide and lenalidomide has provided effective treatment options for patients with both newly diagnosed and relapsed/refractory MM. Overall, this paper focuses on a comprehensive review of the current literature and provides data supporting molecular target therapy for patients with CML, APL, or MM.
Insights
Molecular targeted therapies offer promising treatments for blood cancers like chronic myeloid leukemia (CML), acute promyelocytic leukemia (APL), and multiple myeloma (MM). New drugs provide effective options for patients with resistant or relapsed conditions.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Context:
- Molecular targeted therapy represents a significant advancement in treating hematological malignancies.
- Chronic myeloid leukemia (CML) management has been revolutionized by imatinib, with long-term data showing high efficacy and tolerability.
- Alternative therapies like dasatinib and nilotinib are available for imatinib-resistant cases.
Purpose:
- To provide a comprehensive review of current molecular targeted therapies for CML, APL, and MM.
- To present data supporting the efficacy of these targeted treatments.
- To highlight advancements in managing hematological malignancies through molecular approaches.
Summary:
- Imatinib is the standard for chronic-phase CML, with second-generation inhibitors available for resistance.
- Acute promyelocytic leukemia (APL) treatment has shifted to differentiation therapy, combining all-trans retinoic acid (ATRA) with chemotherapy.
- Thalidomide and lenalidomide offer new treatment avenues for multiple myeloma (MM), including relapsed/refractory cases.
Impact:
- Molecular targeted therapies have improved long-term outcomes and quality of life for patients with CML, APL, and MM.
- The development of targeted agents signifies a paradigm shift from conventional chemotherapy to precision medicine in hematologic oncology.
- This review underscores the growing importance and success of molecularly-driven treatment strategies in combating blood cancers.
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