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Updated: Jun 27, 2026

Determining Immune System Suppression versus CNS Protection for Pharmacological Interventions in Autoimmune Demyelination
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Published on: September 12, 2016

Clinical Course, Risk Factors, and Therapeutic Response in Multiple Myeloma with Central Nervous System Involvement.

Alexander Xiao1, Saurabh S Zanwar2, Yi Lin1

  • 1Mayo Clinic, Rochester, Minnesota, United States.

Blood Advances
|June 25, 2026
PubMed
Summary

Central nervous system multiple myeloma (CNS-MM) is a rare, aggressive complication of extramedullary disease (EMD). Novel therapies like CAR-T cells and bispecific antibodies show promise for improving survival in CNS-MM patients.

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09:41

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Published on: July 15, 2015

Area of Science:

  • Hematology
  • Oncology
  • Neurology

Background:

  • Extramedullary disease (EMD) is increasingly observed in relapsed/refractory multiple myeloma (MM).
  • Central nervous system involvement (CNS-MM) is a rare but aggressive manifestation of EMD with poor outcomes.
  • Limited data exist on CNS-MM incidence and optimal management, especially with new therapies.

Purpose of the Study:

  • To analyze the incidence, clinical features, and outcomes of CNS-MM.
  • To identify predictors of CNS involvement in patients with EMD.
  • To evaluate the efficacy of different treatment modalities, including cellular and bispecific therapies, for CNS-MM.

Main Methods:

  • Retrospective analysis of 304 patients with pathology-confirmed EMD treated between January 2000 and December 2023.
  • CNS-MM defined by parenchymal or leptomeningeal involvement.
  • Analysis of clinical features, cytogenetics, treatments, and overall survival (OS).

Main Results:

  • 20 patients (6.5%) were diagnosed with CNS-MM.
  • CNS-MM was associated with high-risk cytogenetics (OR 3.7) and visceral EMD (OR 4.6).
  • Median OS from CNS-MM diagnosis was 4.2 months; significantly improved to 19.2 months with CAR-T or bispecific therapies versus 1.2 months without.
  • Intrathecal chemotherapy showed limited benefit.

Conclusions:

  • CNS-MM is an aggressive complication of high-risk, multifocal EMD with a poor prognosis.
  • Cellular and bispecific therapies demonstrate promising activity and may improve survival in CNS-MM.
  • Intrathecal therapy has limited efficacy; further prospective studies are needed for optimal treatment strategies.