Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Overall survival as a primary end point in multiple myeloma trials

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.

This is a preview of subscription content, access via your institution

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

All prices are NET prices.

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  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  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  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  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  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  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  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  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Edward R. Scheffer Cliff.

Ethics declarations

Competing interests

The authors declare no competing interests.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Cliff, E.R.S., Rehman Mohyuddin, G. 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

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41571-022-00665-7

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing