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This combination therapy, developed by Fudan University, is a multi-agent regimen designed for the treatment of intermediate-to-high-risk rhabdomyosarcoma. It integrates the standard-of-care VAC chemotherapy backbone (vincristine, dactinomycin, and cyclophosphamide) with all-trans retinoic acid (ATRA) and the multi-target tyrosine kinase inhibitor anlotinib. ATRA is included to promote the differentiation of myogenic precursor cells, which are often arrested in an undifferentiated state in rhabdomyosarcoma. Anlotinib provides targeted inhibition of angiogenesis and tumor cell signaling by blocking VEGFR, FGFR, PDGFR, and c-Kit. The regimen is currently being evaluated in a Phase II clinical trial to determine if the addition of differentiation therapy and anti-angiogenic agents can improve progression-free and overall survival in patients with advanced rhabdomyosarcoma who have not received prior systemic treatment.
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