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Overall survival as a primary end point in multiple myeloma trials

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Median overall survival for patients with newly diagnosed multiple myeloma may surpass ten years. Nonetheless, many patients face considerable treatment-related morbidity and relapsed disease. Owing to this typically long overall survival, most multiple myeloma trials now use progression-free survival as their primary end point. In this Comment, we highlight circumstances in which this end point does not best answer the questions that various trials seek to investigate.

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Change history

  • 31 May 2023

    In the version of this article initially published, the given name and surname for Ghulam Rehman Mohyuddin were presented incorrectly and are now amended in the online version of the article.

References

  1. Mohyuddin, G. R. et al. Use of endpoints in multiple myeloma randomized controlled trials over the last 15 years: A systematic review. Am. J. Hematol. 96, 690–697 (2021).

    Article  PubMed  Google Scholar 

  2. Haslam, A. & Prasad, V. When is crossover desirable in cancer drug trials and when is it problematic? Ann. Oncol. 29, 1079–1081 (2018).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  3. Schjesvold, F. H. et al. Melflufen or pomalidomide plus dexamethasone for patients with multiple myeloma refractory to lenalidomide (OCEAN): a randomised, head-to-head, open-label, phase 3 study. Lancet Haematol. 9, e98–e110 (2022).

    Article  CAS  PubMed  Google Scholar 

  4. Kumar, S. K. et al. Venetoclax or placebo in combination with bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma (BELLINI): a randomised, double-blind, multicentre, phase 3 trial. Lancet Oncol. 21, 1630–1642 (2020).

    Article  CAS  PubMed  Google Scholar 

  5. Rajkumar, S. V. et al. Lenalidomide plus high-dose dexamethasone versus lenalidomide plus low-dose dexamethasone as initial therapy for newly diagnosed multiple myeloma: an open-label randomised controlled trial. Lancet Oncol. 11, 29–37 (2010).

    Article  CAS  PubMed  Google Scholar 

  6. Gill, S. K. et al. Inferior outcomes of patients with quad and penta-refractory multiple myeloma (mm) compared to those of patients who have been quad and penta exposed. Blood 138, 4742–4742 (2021).

    Article  Google Scholar 

  7. Gyawali, B. & Prasad, V. Combining drugs and extending treatment — a PFS end point is not sufficient. Nat. Rev. Clin. Oncol. 14, 521–522 (2017).

    Article  PubMed  Google Scholar 

  8. Mohyuddin, G. R., Koehn, K., Abdallah, A. O., Goodman, A. M. & Prasad, V. Reporting of postprotocol therapies and attrition in multiple myeloma randomized clinical trials: a systematic review. JAMA Netw. Open 4, e218084 (2021).

    Article  PubMed  PubMed Central  Google Scholar 

  9. Munshi, N. C. et al. Association of minimal residual disease with superior survival outcomes in patients with multiple myeloma: a meta-analysis. JAMA Oncol. 3, 28–35 (2017).

    Article  PubMed  PubMed Central  Google Scholar 

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Authors and Affiliations

  1. Program on Regulation, Therapeutics and Law, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA

    Edward R. Scheffer Cliff

  2. Division of Medical Oncology, Dana Farber Cancer Institute, Boston, MA, USA

    Edward R. Scheffer Cliff

  3. Division of Hematology and Hematological Malignancies, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT, USA

    Ghulam Rehman Mohyuddin

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  1. Edward R. Scheffer Cliff
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  2. Ghulam Rehman Mohyuddin
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Correspondence to Edward R. Scheffer Cliff.

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The authors declare no competing interests.

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Cliff, E.R.S., Mohyuddin, G.R. Overall survival as a primary end point in multiple myeloma trials. Nat Rev Clin Oncol 19, 565–566 (2022). https://doi.org/10.1038/s41571-022-00665-7

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