Abstract
Autologous stem cell transplant (ASCT) has demonstrated to be an effective treatment for patients with light-chain (AL) amyloidosis. However, a high transplant-related mortality (TRM) rate was reported in previous series of patients and questioned the role of transplant in this disease. Recently, experienced groups have shown a significant TRM decrease that has been attributed to an accurate selection of patients. Moreover, application of several supportive measures has decreased toxicity over amyloid-involved organs. We analyzed a series of 66 patients with AL amyloidosis, who underwent ASCT at a single institution and evaluated the impact of these measures beyond patient selection. Four temporary groups were established: group-A (non-selection plus post-transplant G-CSF use) with 29 patients, group-B (selection) with 13, group-C (selection and G-CSF avoidance) with 14, and group-D (selection, G-CSF avoidance and corticosteroid’s prophylaxis) with 10. A decreasing TRM was observed over time from group-A (38%), to group-D (0%); p = 0.02. We also observed a progressive increase of three-year OS from 62% in group-A to 100% in group-D; p = 0.049. On the multivariate analysis, cardiac involvement was the only independent predictor of survival. Therefore, tailored selection policy together with transplant supportive measures have allowed ASCT to be a safe procedure in AL amyloidosis.
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This work has been supported in part by grants PI12/01290 and PI16/0423 from Instituto de Salud Carlos III and Fondo Europeo de Desarrollo Regional (FEDER), and 2017SGR-792 from AGAUR (Generalitat de Catalunya).
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Gonzalo Gutiérrez-García: honoraria from Gilead. María Teresa Cibeira: honoraria from Janssen, Celgene, and Amgen. Carlos Fernández de Larrea: honoraria from Janssen, Celgene, and Amgen; advisory boards: Janssen, Celgene, and Amgen; grants from: Janssen, Celgene, Amgen, and Takeda. Laura Rosiñol: honoraria from Janssen, Celgene, Amgen, and Takeda. The remaining authors declare that they have no conflict of interest.
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Gutiérrez-García, G., Cibeira, M.T., Rovira, M. et al. Improving security of autologous hematopoietic stem cell transplant in patients with light-chain amyloidosis. Bone Marrow Transplant 54, 1295–1303 (2019). https://doi.org/10.1038/s41409-019-0447-y
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DOI: https://doi.org/10.1038/s41409-019-0447-y