TY - JOUR
T1 - The role of autologous stem cell transplantation in amyloidosis
AU - Vaxman, Iuliana
AU - Dispenzieri, Angela
N1 - Funding Information:
advisory board and independent review committee for Janssen and data monitoring safety committees for Oncopeptides and Sorrento and receives research funding from Alynlam, Pfizer, Takeda, and Bristol Myers Squibb.
Funding Information:
function, neurocognitive status, quality of life, and safety. ROADS is a multicenter randomized controlled trial funded by GT Medical Technologies (NCT04365374). The study opened for enrollment in April 2021.
Publisher Copyright:
© 2021 UBM Medica Healthcare Publications. All rights reserved.
PY - 2021
Y1 - 2021
N2 - Autologous stem cell transplantation (ASCT) has been an essential part of the treatment armamentarium in light chain (AL) amyloidosis for several decades. Patients who achieve a complete hematologic response following ASCT have a long overall survival. However, only 1 randomized controlled trial compared ASCT with the standard of care used at the time, which was melphalan and dexamethasone, and the results did not support the use of ASCT in AL amyloidosis. These results are of limited significance due to the unexpected high transplant-related mortality (TRM) (24%). TRM is a major concern in AL amyloidosis, but its incidence can be lessened by better patient selection and by patients receiving ASCT in specialized centers. ASCT in AL amyloidosis is performed only in selected patients; approximately 20% of patients with AL amyloidosis are transplant eligible up front or after bortezomib (Velcade) based conditioning. The introduction of newer agents such as bortezomib and daratumumab (Darzalex), which lead to deep responses and have good safety profiles, encourage revisiting the benefit and timing of ASCT in the modern era. This review provides a comprehensive assessment of eligibility criteria for ASCT in AL amyloidosis, conditioning dosing, efficacy in terms of hematologic and organ response, and future areas of research.
AB - Autologous stem cell transplantation (ASCT) has been an essential part of the treatment armamentarium in light chain (AL) amyloidosis for several decades. Patients who achieve a complete hematologic response following ASCT have a long overall survival. However, only 1 randomized controlled trial compared ASCT with the standard of care used at the time, which was melphalan and dexamethasone, and the results did not support the use of ASCT in AL amyloidosis. These results are of limited significance due to the unexpected high transplant-related mortality (TRM) (24%). TRM is a major concern in AL amyloidosis, but its incidence can be lessened by better patient selection and by patients receiving ASCT in specialized centers. ASCT in AL amyloidosis is performed only in selected patients; approximately 20% of patients with AL amyloidosis are transplant eligible up front or after bortezomib (Velcade) based conditioning. The introduction of newer agents such as bortezomib and daratumumab (Darzalex), which lead to deep responses and have good safety profiles, encourage revisiting the benefit and timing of ASCT in the modern era. This review provides a comprehensive assessment of eligibility criteria for ASCT in AL amyloidosis, conditioning dosing, efficacy in terms of hematologic and organ response, and future areas of research.
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U2 - 10.46883/ONC.2021.3508.0471
DO - 10.46883/ONC.2021.3508.0471
M3 - Review article
C2 - 34398591
AN - SCOPUS:85114165441
SN - 0890-9091
VL - 35
SP - 471
EP - 478
JO - ONCOLOGY
JF - ONCOLOGY
IS - 8
ER -