Major Advances in Targeted Therapies Transform Treatment for Blood Cancers

There have been major advances in targeted therapies for blood cancers, especially chronic lymphocytic leukemia (CLL), large B-cell lymphoma (LBCL), and myelofibrosis (MF).

In CLL, treatment has largely shifted from chemotherapy to BTK inhibitors. Newer drugs like acalabrutinib (Calquence) and zanubrutinib (Brukinsa) provide strong, lasting responses with fewer side effects than older drugs such as ibrutinib, making chemotherapy-free regimens the standard of care.

For LBCL, CAR T-cell therapy has become a highly effective treatment option. Real-world data confirm that outcomes in clinical practice closely match those seen in trials, validating its effectiveness.

In myelofibrosis and polycythemia vera, four JAK inhibitors are now FDA-approved, each suited to different patient needs. Pacritinib (Vonjo) is helpful for patients with low blood counts, momelotinib (Ojjaara) benefits anemic patients, and fedratinib (Inrebic) is effective after ruxolitinib. Research is ongoing to develop more precise JAK inhibitors, including drugs targeting specific mutations or inactive forms of JAK2.

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