Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem-cell transplantation.
Long-term survival and outcome were determined for 80 patients with immunoglobulin light chain (AL) amyloidosis treated with high-dose melphalan and stem cell transplantation (HDM/SCT) more than 10 years ago. Seventeen (21%) patients died within the first year of treatment, of treatment-related complications (14%) or progressive disease (8%). Of the 63 surviving evaluable patients at one year, 32 (51%) achieved a complete hematologic response (CR). For all 80 patients, the median survival was 57 months (4.75 yrs). The median survival exceeds 10 years for patients achieving a CR after HDM/SCT, compared with 50 months for those not achieving a CR (P < .001). In conclusion, HDM/SCT leads to durable remissions and prolonged survival, particularly for those patients who achieve a hematologic CR.
Sanchorawala
Vaishali
V
Stem Cell Transplantation Program of the Section of Hematology-Oncology, Boston University School of Medicine, Boston, MA 02118, USA. vaishali.sanchorawala@bmc.org
Skinner
Martha
M
Quillen
Karen
K
Finn
Kathleen T
KT
Doros
Gheorghe
G
Seldin
David C
DC
D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000401:mortality / Q000628:therapy*; D003131:Combined Modality Therapy / Q000401:mortality; D004305:Dose-Response Relationship, Drug; D005500:Follow-Up Studies; D006801:Humans; D006942:Hypergammaglobulinemia / Q000401:mortality / Q000628:therapy*; D007147:Immunoglobulin Light Chains; D053208:Kaplan-Meier Estimate; D008558:Melphalan / Q000008:administration & dosage*; D008875:Middle Aged; D033581:Stem Cell Transplantation; D017741:Survivors; D013997:Time Factors; D016896:Treatment Outcome
D007147:Immunoglobulin Light Chains; D008558:Melphalan