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Background <p>The prognostic significance of minimal residual disease (MRD) negativity in multiple myeloma (MM) is well-established, and MRD negativity serves as a surrogate marker for treatment outcomes. However, in various clinical trials, achieving MRD negativity often leads to treatment continuation until disease progression. In real-world clinical practice, discontinuing treatment could lower healthcare costs and reduce adverse events.</p> Methods <p>We retrospectively analyzed patients who reached MRD negativity and discontinued treatment.</p> Results <p>A total of 39 multiple MM cases were included (17 eligible and 22 ineligible for autologous stem cell transplantation). The median time to next treatment was 42.4&#xa0;months. Ten patients (25%) required additional treatment due to paraproteins or clinical relapse. Cumulative incidence of relapse at 12 and 48&#xa0;months was 11.7% (95% confidence interval [CI], 4.5–28.2%) and 26.4% (95% CI, 12.8–49.6%), respectively. Multivariate analysis revealed that elevated lactate dehydrogenase (LDH) at first visit and t(4;14) were the only baseline factors significantly associated with worse outcomes. Eight patients (20%) with International Staging System (ISS) = I and with no risk factors (history of extramural disease, elevated LDH, high-risk cytogenetics) had no recurrence.</p> Conclusion <p>Although treatment discontinuation in high-risk cases is potentially unsafe, low-risk cases demonstrate potential for treatment-free remission.</p>

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Retrospective analysis of treatment discontinuation in minimal residual disease negative multiple myeloma

  • 期刊论文

  • 发表日期：

    2025年3月9日

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  • Shuku Sato,
  • Emi Sawazaki,
  • Shun Tsunoda,
  • Wataru Kamata,
  • Tomiteru Togano,
  • Yotaro Tamai

摘要

Background

The prognostic significance of minimal residual disease (MRD) negativity in multiple myeloma (MM) is well-established, and MRD negativity serves as a surrogate marker for treatment outcomes. However, in various clinical trials, achieving MRD negativity often leads to treatment continuation until disease progression. In real-world clinical practice, discontinuing treatment could lower healthcare costs and reduce adverse events.

Methods

We retrospectively analyzed patients who reached MRD negativity and discontinued treatment.

Results

A total of 39 multiple MM cases were included (17 eligible and 22 ineligible for autologous stem cell transplantation). The median time to next treatment was 42.4 months. Ten patients (25%) required additional treatment due to paraproteins or clinical relapse. Cumulative incidence of relapse at 12 and 48 months was 11.7% (95% confidence interval [CI], 4.5–28.2%) and 26.4% (95% CI, 12.8–49.6%), respectively. Multivariate analysis revealed that elevated lactate dehydrogenase (LDH) at first visit and t(4;14) were the only baseline factors significantly associated with worse outcomes. Eight patients (20%) with International Staging System (ISS) = I and with no risk factors (history of extramural disease, elevated LDH, high-risk cytogenetics) had no recurrence.

Conclusion

Although treatment discontinuation in high-risk cases is potentially unsafe, low-risk cases demonstrate potential for treatment-free remission.