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This study aims to characterize the clinical features and treatment outcomes of central nervous system involvement in multiple myeloma among a Chinese cohort.

May 30, 2026Open Access

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Clinical Characteristics, Treatment Patterns, and Prognosis of Central Nervous System Involvement in Multiple Myeloma: A Multicenter Retrospective Study From China

WLWenjing LiBeijing Chao-Yang Hospital WTWeiwei TianShanxi Academy of Medical Sciences JMJie MaZhengzhou University

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This study aims to characterize the clinical features and treatment outcomes of central nervous system involvement in multiple myeloma among a Chinese cohort.
Multicenter retrospective study across nine hematology centers in China from July 2017 to June 2024.
Analysis of clinical records from 35 multiple myeloma patients with CNS involvement measuring diagnostic parameters, treatment regimens, and survival outcomes.
Median follow-up duration of 19.1 months, with treatment strategies including chemotherapy, intrathecal therapy, and CAR-T therapy.
CNS involvement occurred at initial diagnosis in 31.4% of cases and at relapse in 68.6%, with a median latency of 22.8 months from diagnosis.
Overall response rate was 62.9%, with 36.4% achieving complete remission, but 56% relapsed within a median of 3.1 months.
Median overall survival was recorded at 13.0 months with a mortality rate of 60% among the population.

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 months (range 1.2-8.4). 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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Clinical Characteristics, Treatment Patterns, and Prognosis of Central Nervous System Involvement in Multiple Myeloma: A Multicenter Retrospective Study From China

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