Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma

Meletios Dimopoulos; Pieter Sonneveld; Salomon Manier; Annette Lam; Tito Roccia; Jordan M. Schecter; Patricia Cost; Lida Pacaud; Abbey Poirier; Gabriel Tremblay; Tommy Lan; Satish Valluri; Shaji Kumar

doi:10.1186/s12885-024-12263-0

Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma

Meletios Dimopoulos, Pieter Sonneveld, Salomon Manier, Annette Lam, Tito Roccia, Jordan M. Schecter, Patricia Cost, Lida Pacaud, Abbey Poirier, Gabriel Tremblay, Tommy Lan, Satish Valluri, Shaji Kumar^*

^*Corresponding author for this work

Hematology

Research output: Contribution to journal › Article › Academic › peer-review

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Abstract

Objectives:

The goal of the research was to assess the quantitative relationship between median progression-free survival (PFS) and median overall survival (OS) specifically among patients with relapsed/refractory multiple myeloma (RRMM) based on published randomized controlled trials (RCTs).

Methods:

Two bibliographic databases (PubMed and Embase, 1970–2017) were systematically searched for RCTs in RRMM that reported OS and PFS, followed by an updated search of studies published between 2010 and 2022 in 3 databases (Embase, MEDLINE, and EBM Reviews, 2010–2022). The association between median PFS and median OS was assessed using the nonparametric Spearman rank and parametric Pearson correlation coefficients. Subsequently, the quantitative relationship between PFS and OS was assessed using weighted least-squares regression adjusted for covariates including age, sex, and publication year. Study arms were weighted by the number of patients in each arm.

Results:

A total of 31 RCTs (56 treatment arms, 10,450 patients with RRMM) were included in the analysis. The average median PFS and median OS were 7.1 months (SD 5.5) and 28.1 months (SD 11.8), respectively. The Spearman and Pearson correlation coefficients between median PFS and median OS were 0.80 (P < 0.0001) and 0.79 (P < 0.0001), respectively. In individual treatment arms of RRMM trials, each 1-month increase in median PFS was associated with a 1.72-month (95% CI 1.26–2.17) increase in median OS.

Conclusion:

Analysis of the relationship between PFS and OS incorporating more recent studies in RRMM further substantiates the use of PFS to predict OS in RRMM.

Original language	English
Article number	541
Journal	BMC Cancer
Volume	24
Issue number	1
DOIs	https://doi.org/10.1186/s12885-024-12263-0
Publication status	Published - 29 Apr 2024

Bibliographical note

Publisher Copyright:
© The Author(s) 2024.

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This output contributes to the following UN Sustainable Development Goals (SDGs)

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Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myelomaFinal published version, 1.04 MBLicence: CC BY

Cite this

Dimopoulos, M., Sonneveld, P., Manier, S., Lam, A., Roccia, T., Schecter, J. M., Cost, P., Pacaud, L., Poirier, A., Tremblay, G., Lan, T., Valluri, S., & Kumar, S. (2024). Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma. BMC Cancer, 24(1), Article 541. https://doi.org/10.1186/s12885-024-12263-0

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title = "Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma",

abstract = "Objectives: The goal of the research was to assess the quantitative relationship between median progression-free survival (PFS) and median overall survival (OS) specifically among patients with relapsed/refractory multiple myeloma (RRMM) based on published randomized controlled trials (RCTs). Methods: Two bibliographic databases (PubMed and Embase, 1970–2017) were systematically searched for RCTs in RRMM that reported OS and PFS, followed by an updated search of studies published between 2010 and 2022 in 3 databases (Embase, MEDLINE, and EBM Reviews, 2010–2022). The association between median PFS and median OS was assessed using the nonparametric Spearman rank and parametric Pearson correlation coefficients. Subsequently, the quantitative relationship between PFS and OS was assessed using weighted least-squares regression adjusted for covariates including age, sex, and publication year. Study arms were weighted by the number of patients in each arm. Results: A total of 31 RCTs (56 treatment arms, 10,450 patients with RRMM) were included in the analysis. The average median PFS and median OS were 7.1 months (SD 5.5) and 28.1 months (SD 11.8), respectively. The Spearman and Pearson correlation coefficients between median PFS and median OS were 0.80 (P < 0.0001) and 0.79 (P < 0.0001), respectively. In individual treatment arms of RRMM trials, each 1-month increase in median PFS was associated with a 1.72-month (95% CI 1.26–2.17) increase in median OS. Conclusion: Analysis of the relationship between PFS and OS incorporating more recent studies in RRMM further substantiates the use of PFS to predict OS in RRMM.",

author = "Meletios Dimopoulos and Pieter Sonneveld and Salomon Manier and Annette Lam and Tito Roccia and Schecter, {Jordan M.} and Patricia Cost and Lida Pacaud and Abbey Poirier and Gabriel Tremblay and Tommy Lan and Satish Valluri and Shaji Kumar",

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year = "2024",

month = apr,

day = "29",

doi = "10.1186/s12885-024-12263-0",

language = "English",

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T1 - Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma

AU - Dimopoulos, Meletios

AU - Sonneveld, Pieter

AU - Manier, Salomon

AU - Lam, Annette

AU - Roccia, Tito

AU - Schecter, Jordan M.

AU - Cost, Patricia

AU - Pacaud, Lida

AU - Poirier, Abbey

AU - Tremblay, Gabriel

AU - Lan, Tommy

AU - Valluri, Satish

AU - Kumar, Shaji

PY - 2024/4/29

Y1 - 2024/4/29

N2 - Objectives: The goal of the research was to assess the quantitative relationship between median progression-free survival (PFS) and median overall survival (OS) specifically among patients with relapsed/refractory multiple myeloma (RRMM) based on published randomized controlled trials (RCTs). Methods: Two bibliographic databases (PubMed and Embase, 1970–2017) were systematically searched for RCTs in RRMM that reported OS and PFS, followed by an updated search of studies published between 2010 and 2022 in 3 databases (Embase, MEDLINE, and EBM Reviews, 2010–2022). The association between median PFS and median OS was assessed using the nonparametric Spearman rank and parametric Pearson correlation coefficients. Subsequently, the quantitative relationship between PFS and OS was assessed using weighted least-squares regression adjusted for covariates including age, sex, and publication year. Study arms were weighted by the number of patients in each arm. Results: A total of 31 RCTs (56 treatment arms, 10,450 patients with RRMM) were included in the analysis. The average median PFS and median OS were 7.1 months (SD 5.5) and 28.1 months (SD 11.8), respectively. The Spearman and Pearson correlation coefficients between median PFS and median OS were 0.80 (P < 0.0001) and 0.79 (P < 0.0001), respectively. In individual treatment arms of RRMM trials, each 1-month increase in median PFS was associated with a 1.72-month (95% CI 1.26–2.17) increase in median OS. Conclusion: Analysis of the relationship between PFS and OS incorporating more recent studies in RRMM further substantiates the use of PFS to predict OS in RRMM.

AB - Objectives: The goal of the research was to assess the quantitative relationship between median progression-free survival (PFS) and median overall survival (OS) specifically among patients with relapsed/refractory multiple myeloma (RRMM) based on published randomized controlled trials (RCTs). Methods: Two bibliographic databases (PubMed and Embase, 1970–2017) were systematically searched for RCTs in RRMM that reported OS and PFS, followed by an updated search of studies published between 2010 and 2022 in 3 databases (Embase, MEDLINE, and EBM Reviews, 2010–2022). The association between median PFS and median OS was assessed using the nonparametric Spearman rank and parametric Pearson correlation coefficients. Subsequently, the quantitative relationship between PFS and OS was assessed using weighted least-squares regression adjusted for covariates including age, sex, and publication year. Study arms were weighted by the number of patients in each arm. Results: A total of 31 RCTs (56 treatment arms, 10,450 patients with RRMM) were included in the analysis. The average median PFS and median OS were 7.1 months (SD 5.5) and 28.1 months (SD 11.8), respectively. The Spearman and Pearson correlation coefficients between median PFS and median OS were 0.80 (P < 0.0001) and 0.79 (P < 0.0001), respectively. In individual treatment arms of RRMM trials, each 1-month increase in median PFS was associated with a 1.72-month (95% CI 1.26–2.17) increase in median OS. Conclusion: Analysis of the relationship between PFS and OS incorporating more recent studies in RRMM further substantiates the use of PFS to predict OS in RRMM.

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DO - 10.1186/s12885-024-12263-0

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AN - SCOPUS:85191830909

SN - 1471-2407

VL - 24

JO - BMC Cancer

JF - BMC Cancer

IS - 1

M1 - 541

ER -

Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma

Abstract

Bibliographical note

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