TY - JOUR
T1 - Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014
T2 - Cracking the glass ceiling of early death
AU - Muchtar, Eli
AU - Gertz, Morie A.
AU - Kumar, Shaji K.
AU - Lacy, Martha Q.
AU - Dingli, David
AU - Buadi, Francis K.
AU - Grogan, Martha
AU - Hayman, Suzanne R.
AU - Kapoor, Prashant
AU - Leung, Nelson
AU - Fonder, Amie
AU - Hobbs, Miriam
AU - Hwa, Yi Lisa
AU - Gonsalves, Wilson
AU - Warsame, Rahma
AU - Kourelis, Taxiarchis V.
AU - Russell, Stephen
AU - Lust, John A.
AU - Lin, Yi
AU - Go, Ronald S.
AU - Zeldenrust, Steven
AU - Kyle, Robert A.
AU - Rajkumar, S. Vincent
AU - Dispenzieri, Angela
N1 - Publisher Copyright:
© 2017 by The American Society of Hematology.
PY - 2017/4/13
Y1 - 2017/4/13
N2 - In light of major advances in immunoglobulin light chain (AL) amyloidosis, we evaluated the trends in presentation, management, and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000 to 2014. As compared with the 2 intervals 2000-2004 and 2005-2009, patients diagnosed in 2010-2014 were less likely to have >2 involved organs. Utilization of autologous stemcell transplant (ASCT)wassimilar across all periods, about one-third of patients, but there was an increase in the use of pre- ASCT bortezomib induction and of unattenuated melphalan conditioning in 2010-2014 compared with earlier periods. Non-ASCT first-line regimen changed with 65% of patients in 2010-2014 received bortezomib-based therapy, 79% of patients in 2005-2009 received melphalan-dexamethasone, and 64% of patients in 2000-2004 received melphalan-prednisone. The rate of better than very good partial response (VGPR) was higher inmore recent periods (66% vs 58% vs 51%;P5.001), a change largely driven by improved VGPR rates in the non-ASCT population. Overall survival (OS) has improved, with inflection points for improvement differing for the ASCT and non-ASCT groups. In the ASCT population, the greatest gainswere after 2010 (4-year OS, 91%comparedwith 73% and 65%). In the non-ASCT group, greatest gains were after 2005 (4-year OS, 38%, 32%, and 16%). Fewer patients died within 6 months of diagnosis in the 2 later periods (24% vs25%vs 37%;P<.001). Overall, outcomes among patients with AL amyloidosis have improved with earlier diagnosis, higher rates of VGPR, lower early mortality, and improved OS.
AB - In light of major advances in immunoglobulin light chain (AL) amyloidosis, we evaluated the trends in presentation, management, and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000 to 2014. As compared with the 2 intervals 2000-2004 and 2005-2009, patients diagnosed in 2010-2014 were less likely to have >2 involved organs. Utilization of autologous stemcell transplant (ASCT)wassimilar across all periods, about one-third of patients, but there was an increase in the use of pre- ASCT bortezomib induction and of unattenuated melphalan conditioning in 2010-2014 compared with earlier periods. Non-ASCT first-line regimen changed with 65% of patients in 2010-2014 received bortezomib-based therapy, 79% of patients in 2005-2009 received melphalan-dexamethasone, and 64% of patients in 2000-2004 received melphalan-prednisone. The rate of better than very good partial response (VGPR) was higher inmore recent periods (66% vs 58% vs 51%;P5.001), a change largely driven by improved VGPR rates in the non-ASCT population. Overall survival (OS) has improved, with inflection points for improvement differing for the ASCT and non-ASCT groups. In the ASCT population, the greatest gainswere after 2010 (4-year OS, 91%comparedwith 73% and 65%). In the non-ASCT group, greatest gains were after 2005 (4-year OS, 38%, 32%, and 16%). Fewer patients died within 6 months of diagnosis in the 2 later periods (24% vs25%vs 37%;P<.001). Overall, outcomes among patients with AL amyloidosis have improved with earlier diagnosis, higher rates of VGPR, lower early mortality, and improved OS.
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U2 - 10.1182/blood-2016-11-751628
DO - 10.1182/blood-2016-11-751628
M3 - Article
C2 - 28126928
AN - SCOPUS:85018894692
SN - 0006-4971
VL - 129
SP - 2111
EP - 2119
JO - Blood
JF - Blood
IS - 15
ER -