September 30, 2025Open Access

Clinical characteristics and prognosis of central nervous system involvement in multiple myeloma: a multicenter retrospective analysis from China

WLWenjing LiLoughborough University WTWei-Wei TianShanxi Medical University JMJie MaFirst Affiliated Hospital of Zhengzhou University

Key Points

CNS involvement significantly reduces overall survival, with a median of 13.0 months observed in patients with multiple myeloma.
Among 35 cases studied, 68.6% experienced CNS symptoms at relapse, indicating high aggression of the disease after initial diagnosis.
The analysis involved a multicenter retrospective review across nine hematology centers in China, providing extensive clinical insights.
Despite multimodal treatment approaches, including chemotherapy and CAR-T therapy, 56% of patients relapsed quickly with a median of only 3.1 months.

Abstract

Abstract Background Central nervous system (CNS) involvement is a rare and aggressive complication of multiple myeloma (MM), with limited data on its clinical characteristics and prognosis. This multicenter study characterizes the largest Chinese cohort of MM patients with CNS involvement (MM-CNS) to date. Methods We conducted a multicenter retrospective study across nine hematology centers in China (July 2017-June 2024), analyzing clinical records of MM-CNS patients. Data collection encompassed diagnostic parameters, treatment regimens, and survival outcomes. Results Among 35 identified MM-CNS cases (median age 55 years, range 40–83), CNS manifestations presented at initial MM diagnosis in 31.4% (11/35) versus relapse in 68.6% (24/35), with median latency of 22.8 months from MM diagnosis. Predominant features included IgG subtype (51.4%), λ light chain restriction (54.3%), and high-risk cytogenetics: 1q21 amplification (48.3%), t(4;14) translocation (13.8%), and complex karyotypes of chromosome (35.7%). Diagnostic confirmation combined CSF analysis (protein elevation, plasma cell detection) with neuroimaging (meningeal/parenchymal lesions). Treatments included systemic chemotherapy (88.6%), intrathecal therapy (51.4%), radiotherapy (17.1%), chimeric antigen receptor T-cell (CAR-T) therapy (11.4%), and autologous stem cell transplantation (ASCT) (14.3%). Despite 62.9% overall response rate (36.4% complete remission), 56% relapsed within median 3.1 (range 1.2–8.4) months. The median follow-up duration was 19.1 months (range: 0.1–46.8). Median overall survival (OS) at 13.0 months (range 0.1–43.1) with 60% mortality (21/35). Conclusions CNS involvement in MM is associated with aggressive disease and poor prognosis. Despite transient responses to multimodal therapies, rapid progression and high mortality persist. Novel strategies are urgently needed to improve outcomes in this high-risk population.

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