Avapritinib Approved: Gastrointestinal Stromal Tumor With PDGFRA Exon 18 Mutation

The FDA has approved avapritinib (AyvakitTM, Blueprint Medicines) for adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) with platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutations, including D842V mutations, the most common type of exon 18 mutation. Up to 10% of patients with GIST harbor PDGFRA mutations, which have been linked to GIST development. "GIST harboring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST," commented Rich...
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Pexidartinib: First Approval for Tenosynovial Giant Cell Tumor

The FDA has now approved pexidartinib (TuralioTM, Daiichi Sankyo, Inc.) for adults with symptomatic tenosynovial giant cell tumor (TGCT) that does not respond to surgery and is associated with severe morbidity or functional limitations. This is the first FDA approval for TGCT. A locally aggressive neoplasm of the synovium of joints and tendon sheaths, TGCT is a rare disease that causes inflammation, pain, swelling, and the destruction of joints. Symptoms result in part from colony-stimulating fa...
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Soft Tissue Sarcoma In Young Versus Older Adulthood

Soft tissue sarcoma, a rare cancer in tissues that connect, support, and surround body structures, is found in various age groups and typically has a five-year survival rate of 65%. When investigators conducted research comparing the clinical presentation, histological subtype, treatment, and outcome of soft tissue sarcoma in adolescents and young adults versus older adults, they found several differences between the two populations that could aid in future research on this disease. For this stu...
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Immunotherapy Safe for Patients With HIV and Advanced Cancer

Patients infected with HIV are at increased risk of cancer. For example, Kaposi sarcoma is 500 times more likely to occur in patients with HIV than in the general population. Because of their compromised immune systems, patients with HIV are not able to partake in immunotherapy clinical trials. However, researchers at Georgetown University have now found that immunotherapy is quite successful in treating patients with HIV and advanced-stage cancers, producing side effects in only a slight percen...
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Undifferentiated Uterine Sarcoma: Molecular Subtypes Found

Researchers at the Karolinska Institutet in Sweden have identified new molecular subtypes of undifferentiated uterine sarcoma, a rare type of cancer that is highly deadly and has no effective treatment options. "It is too early to propose a new treatment that will be useful for the patients today, but the study opens up new avenues for future research, which will create in time new treatment possibilities for women who suffer from these rare tumors," commented the study's senior author, Joseph C...
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