TY - JOUR
T1 - Safety and efficacy of propylene glycol-free melphalan as conditioning in patients with AL amyloidosis undergoing stem cell transplantation
AU - Sidiqi, M. Hasib
AU - Aljama, Mohammed A.
AU - Muchtar, Eli
AU - Buadi, Francis K.
AU - Warsame, Rahma
AU - Lacy, Martha Q.
AU - Dispenzieri, Angela
AU - Dingli, David
AU - Leung, Nelson
AU - Gonsalves, Wilson I.
AU - Kapoor, Prashant
AU - Kourelis, Taxiarchis V.
AU - Hogan, William J.
AU - Wolf, Robert C.
AU - Kumar, Shaji K.
AU - Gertz, Morie A.
N1 - Publisher Copyright:
© 2018, Springer Nature Limited.
PY - 2019/7/1
Y1 - 2019/7/1
N2 - High-dose melphalan is the standard conditioning regimen for patients with AL amyloidosis receiving autologous stem cell transplantation. Conventional formulations require propylene glycol (PG) as a co-solvent and melphalan has limited solubility and chemical stability after reconstitution, with potential risks for propylene glycol-related complications. Captisol-stabilized propylene glycol-free (PG-free) melphalan has been developed with improved solubility and chemical stability. We compared a cohort of patients with AL amyloidosis receiving PG melphalan (n = 96) to those receiving PG-free melphalan (n = 48) as conditioning for autologous stem cell transplantation. Median time to neutrophil and platelet engraftment was the same; 14 days PG melphalan vs 14 days PG-free melphalan, p = 0.73 and 16 days PG melphalan vs 16 days PG-free melphalan, p = 0.52, respectively. Hospitalization rate was similar in both cohorts, 68% PG melphalan vs 58% PG-free melphalan, p = 0.27. All-cause mortality at 100 days was not statistically significant, 3% PG melphalan vs 2% PG-free melphalan, p > 0.99. Overall response rate (ORR) and rates of complete response (CR) were similar (ORR 93% PG melphalan vs 94% PG-free melphalan, p > 0.99 and CR 39% PG melphalan vs 32% PG-free melphalan, p = 0.46). PG-free melphalan showed a comparable safety and efficacy profile to PG melphalan in patients with AL amyloidosis receiving stem cell transplantation.
AB - High-dose melphalan is the standard conditioning regimen for patients with AL amyloidosis receiving autologous stem cell transplantation. Conventional formulations require propylene glycol (PG) as a co-solvent and melphalan has limited solubility and chemical stability after reconstitution, with potential risks for propylene glycol-related complications. Captisol-stabilized propylene glycol-free (PG-free) melphalan has been developed with improved solubility and chemical stability. We compared a cohort of patients with AL amyloidosis receiving PG melphalan (n = 96) to those receiving PG-free melphalan (n = 48) as conditioning for autologous stem cell transplantation. Median time to neutrophil and platelet engraftment was the same; 14 days PG melphalan vs 14 days PG-free melphalan, p = 0.73 and 16 days PG melphalan vs 16 days PG-free melphalan, p = 0.52, respectively. Hospitalization rate was similar in both cohorts, 68% PG melphalan vs 58% PG-free melphalan, p = 0.27. All-cause mortality at 100 days was not statistically significant, 3% PG melphalan vs 2% PG-free melphalan, p > 0.99. Overall response rate (ORR) and rates of complete response (CR) were similar (ORR 93% PG melphalan vs 94% PG-free melphalan, p > 0.99 and CR 39% PG melphalan vs 32% PG-free melphalan, p = 0.46). PG-free melphalan showed a comparable safety and efficacy profile to PG melphalan in patients with AL amyloidosis receiving stem cell transplantation.
UR - http://www.scopus.com/inward/record.url?scp=85055983597&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85055983597&partnerID=8YFLogxK
U2 - 10.1038/s41409-018-0388-x
DO - 10.1038/s41409-018-0388-x
M3 - Article
C2 - 30390060
AN - SCOPUS:85055983597
SN - 0268-3369
VL - 54
SP - 1077
EP - 1081
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 7
ER -