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Depth of response prior to autologous stem cell transplantation predicts survival in light chain amyloidosis

Bone Marrow Transplantation volume 56, pages 928–935 (2021)Cite this article

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Abstract

The goal of therapy in AL amyloidosis is to inhibit further production of the amyloidogenic light chains, thereby allowing organ recovery and improving survival. We aimed to assess the impact of depth of hematologic response prior to ASCT on survival. We conducted a retrospective study of 128 newly diagnosed AL amyloidosis patients who received induction prior to ASCT between January 2007 and August 2017 at Mayo Clinic. The overall response rate to induction was 86% (CR 18%, VGPR 31% and PR 38%). With a median follow up of 52 months, the median PFS and OS was 48.5 months and not reached, respectively. Response depth to induction therapy was associated with improved PFS and OS. The median PFS was not reached for patients achieving ≥VGPR prior to ASCT and 34.1 months for patients achieving PR or less (P = 0.0009). The median OS was longer in patients with deeper responses (not reached for ≥VGPR vs. 128 months for PR or less (P = 0.02)). On multivariable analysis, independent predictors of OS were melphalan conditioning dose (RR = 0.42; P = 0.036) and depth of response prior to transplant (RR 0.37; P = 0.0295). Hematologic response prior to transplant predicts improved post transplant outcomes in AL amyloidosis.

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Fig. 1: PFS difference between patients achieving CR/VGPR and patients achieving PR or less pre transplant.
Fig. 2: PFS difference between patients achieving CR/VGPR and patients achieving PR or less post transplant.

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Funding

NCI SPORE MM SPORE 5P50 CA186781-04.

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Authors and Affiliations

  1. Division of Hematology, Mayo Clinic, Rochester, MN, USA

    Iuliana Vaxman, M. Hasib Sidiqi, Abdullah S. Al Saleh, Shaji Kumar, Eli Muchtar, Angela Dispenzieri, Francis Buadi, David Dingli, Martha Lacy, Suzanne Hayman, Nelson Leung, Wilson Gonsalves, Taxiarchis Kourelis, Rahma Warsame, William Hogan & Morie Gertz

  2. Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah-Tikva, Israel

    Iuliana Vaxman

  3. Israel Sackler Faculty of Medicine Tel-Aviv University, Tel-Aviv, Israel

    Iuliana Vaxman

  4. Fiona Stanley Hospital, Perth, WA, Australia

    M. Hasib Sidiqi

  5. King Saud bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia

    Abdullah S. Al Saleh

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  1. Iuliana Vaxman
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Contributions

IV designed the study, analyzed the data, wrote the first draft and approved the final version of the manuscript. HS designed the study and critically reviewed the manuscript and approved the final version of the manuscript. ASA analyzed the data and critically reviewed the manuscript and approved the final version of the manuscript. SK, EM, AD, FB, DD, ML, SH, NL, WG, TK, RE, and WH critically revised the manuscript and approved the final version of the manuscript. MAG designed the study, analyzed the data, wrote the first draft and approved the final version of the manuscript.

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Correspondence to Morie Gertz.

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Conflict of interest

Dr. Gertz reports personal fees from Ionis/Akcea, personal fees from Alnylam, personal fees from Prothena, personal fees from Celgene, personal fees from Janssen, grants and personal fees from Spectrum, personal fees from Annexon, personal fees from Appellis, personal fees from Amgen, personal fees from Medscape, personal fees from Physicians Education Resource, personal fees for Data Safety Monitoring board from Abbvie, personal fees from Research to Practice, speaker fees from Teva, Speaker fees from Johnson and Johnson; Speaker fees from Medscape, Speaker fees DAVA oncology; Advisory Board for Pharmacyclics Advisory Board for Proclara outside the submitted work; Development of educational materials for i3Health, Educational Program development i3Health, Royalties from Springer Publishing, Grant Funding Amyloidosis Foundation; International Waldenstrom Foundation. The authors declare that they have no conflict of interest.

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Vaxman, I., Sidiqi, M.H., Al Saleh, A.S. et al. Depth of response prior to autologous stem cell transplantation predicts survival in light chain amyloidosis. Bone Marrow Transplant 56, 928–935 (2021). https://doi.org/10.1038/s41409-020-01136-2

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