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Outcomes with early vs. deferred stem cell transplantation in light chain amyloidosis

Abstract

In the presence of effective treatment options for systemic light chain (AL) amyloidosis, autologous stem cell transplantation (ASCT) is sometimes deferred after stem cell collection. We designed this retrospective study to compare overall survival (OS) between patients who proceed directly to ASCT after stem cell collection and those who defer ASCT. We included patients with AL amyloidosis who had stem cell collection at Mayo Clinic, Minnesota, from 2004 to 2018. ASCT was considered “early” if performed within 90 days of collection, and “deferred” if performed after 90 days, or not done by last follow up. We included 651 patients; 527 underwent early ASCT and 124 deferred ASCT. There was no difference in OS with early vs. deferred ASCT (median OS: 13.0 vs. 11.4 years, respectively, P = 0.28). There was no difference in OS between the 2 groups among patients with early or advanced Mayo Stage. Among patients who achieved ≥very good partial response at the time of collection, OS in the early and deferred groups was 14.2 and 13.4 years, respectively (P = 0.06). Survival outcomes are similar with early and deferred ASCT. Further studies are needed to identify patients who would benefit from each approach.

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Fig. 1: Study population.
Fig. 2: Reasons for deferring transplant.
Fig. 3: Overall survival.
Fig. 4: Overall survival in patients who received treatment prior to collection.
Fig. 5: Overall survival in patients with treatment response.

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Correspondence to Shaji Kumar.

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Conflict of interest

S.S. served as a consultant for Janssen. A.D. received research funding from Celgene, Millennium Pharmaceuticals, Pfizer, and Janssen, and received a travel grant from Pfizer. M.G. served as a consultant for Millennium Pharmaceuticals and received honoraria from Celgene, Millennium Pharmaceuticals, Onyx Pharmaceuticals, Novartis, GlaxoSmithKline, Prothena, Ionis Pharmaceuticals, and Amgen. M.L. received research funding from Celgene. N.L. serves on an advisory board for Takeda Pharmaceuticals. S.K. served as a consultant for Celgene, Millennium Pharmaceuticals, Onyx Pharmaceuticals, Janssen, and Bristol-Myers Squibb, and received research funding from Celgene, Millennium Pharmaceuticals, Novartis, Onyx Pharmaceuticals, AbbVie, Janssen, and Bristol-Myers Squibb. The remaining authors declare no competing interests.

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Abdallah, N., Sidana, S., Dispenzieri, A. et al. Outcomes with early vs. deferred stem cell transplantation in light chain amyloidosis. Bone Marrow Transplant 55, 1297–1304 (2020). https://doi.org/10.1038/s41409-020-0964-8

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