蛋白酶体抑制剂
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从不可治愈到长期生存,多发性骨髓瘤创新疗法带来“功能性治愈”新希望
Yang Zi Wan Bao Wang· 2025-09-05 10:41
Core Viewpoint - Multiple myeloma, the second most common hematological malignancy, poses significant treatment challenges due to its incurable nature and high relapse rates, but advancements in medical technology have led to innovative therapies that offer hope for "functional cure" and improved quality of life for patients [1][8]. Group 1: Disease Overview - Multiple myeloma is characterized by malignant proliferation of plasma cells, which are crucial for immune function, leading to the production of ineffective immunoglobulins that harm normal bodily functions [3][4]. - The disease's early symptoms are often non-specific, leading to misdiagnosis, with common symptoms summarized by the "CRAB" acronym: hypercalcemia, renal impairment, anemia, and bone lesions [3][4]. Group 2: Diagnosis and Monitoring - Patients often seek treatment for symptoms like renal issues or bone pain, mistakenly attributing them to other conditions, which can delay diagnosis and worsen outcomes [4][5]. - Monitoring serum protein levels is critical, with levels exceeding 50 g/L warranting further investigation for multiple myeloma [4]. Group 3: Treatment Approaches - Treatment for multiple myeloma is individualized, with options including traditional chemotherapy, targeted therapy, immunotherapy, and stem cell transplantation, depending on patient-specific factors [7][9]. - Recent advancements have introduced bispecific antibodies as a standard treatment for relapsed/refractory multiple myeloma, showing efficacy rates over 60% and complete response rates around 35% [9]. Group 4: Prognosis and Future Directions - The five-year survival rate for multiple myeloma patients has improved significantly, with many now living for over a decade, thanks to new therapies [8][9]. - The concept of "functional cure" is emerging, where patients may have residual tumor cells that are undetectable and coexist with the immune system, allowing for a normal quality of life [9].