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Results of delayed or salvage autologous hematopoietic stem cell transplantation for multiple myeloma

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Abstract

Autologous hematopoietic cell transplantation (autoHCT) remains a therapeutic option for multiple myeloma (MM) at relapse. We retrospectively analyzed 650 patients who underwent delayed (n = 335) or salvage (n = 315) autoHCT at a single center from 2006–2023. Median age was 61.4 years; 22% were Black, and 21% had high-risk cytogenetics. Forty-nine percent received >3 prior therapy lines, and 33% were lenalidomide-refractory. Non-relapse mortality was 3% at day 100 and 4% at 1 year. Median progression-free survival (mPFS) was 17.5 months and median overall survival (mOS) 47.3 months, with no significant difference between delayed and salvage autoHCT (mPFS 16.3 vs. 19.1 months; mOS 43.2 vs. 50.8 months). In salvage autoHCT, transplant ≥24 months after first autoHCT was associated with superior outcomes (mPFS 20.6 vs. 8.4 months; mOS 54.6 vs. 12.5 months; p < 0.001). Multivariable analysis identified adverse factors for PFS and OS including high-risk cytogenetics, R-ISS stage II–III, lenalidomide- or carfilzomib-refractory disease, anti-CD38 antibody non-exposure, and >3 prior therapy lines; achieving CR post-transplant and receiving maintenance predicted improved outcomes. This largest single-center cohort demonstrates delayed or salvage autoHCT is feasible and effective, particularly for patients with prolonged first remissions, and provides a benchmark for emerging therapies in relapsed/refractory MM.

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Fig. 1: Disease response rates before transplant, at day 100 post-transplant, and best response achieved post-transplant.
Fig. 2: Survival outcomes of the study cohort based on transplant type.

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Data availability

The data that support the findings of this study are available on request from the corresponding author.

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Acknowledgements

This work was supported in part by the Cancer Center Support Grant (NCI Grant P30 CA016672). RZO, the Florence Maude Thomas Cancer Research Professor, would like to acknowledge support from the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, the Riney Family Multiple Myeloma Research Fund at MD Anderson from the Paula and Rodger Riney Foundation, and the MD Anderson Cancer Center High Risk Multiple Myeloma Moon Shot.

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Authors

Contributions

OP and MHQ conceived and designed the study; OP collected and assembled the data; OP, CM, and DRM analyzed and verified the data; OP, CM, and MHQ wrote the manuscript. OP, CM, DRM, AAH, MRT, QB, SS, NS, PS, PL, JR, YN, AHM, URS, AJ, GT, YA, HCL, KKP, PK, SKT, RZO, REC, EJS, and MHQ interpreted the data, wrote and approved the article, and are accountable for the publication.

Corresponding author

Correspondence to Muzaffar H. Qazilbash.

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Competing interests

HCL has received consulting fees from Bristol Myers Squibb, Alexion Pharmaceuticals, Janssen, Regeneron, GlaxoSmithKline, Sanofi, Takeda Pharmaceuticals, Allogene Therapeutics, Pfizer, and Menarini. HCL has also received research funding from Amgen, Bristol Myers Squibb, Janssen, GlaxoSmithKline, Regeneron, Takeda Pharmaceuticals, and Alexion Pharmaceuticals. All other authors report no conflicts of interest.

Ethics approval and consent to participate

This study was approved by the University of Texas MD Anderson Institutional Review Board (IRB) under protocol number PA17-0450. Approval was obtained from the IRB to waive informed consent for this retrospective chart review. The study was conducted in accordance with the Declaration of Helsinki and the 1996 Health Insurance Portability and Accountability Act.

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Pasvolsky, O., Marcoux, C., Milton, D.R. et al. Results of delayed or salvage autologous hematopoietic stem cell transplantation for multiple myeloma. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02771-3

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