title: Tandem cycles of high-dose melphalan and autologous stem cell transplantation increases the response rate in AL amyloidosis.
abstract: Clinical outcomes of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation (HDM/SCT) are tightly linked to the achievement of a hematologic complete response (HCR). We conducted a prospective trial to determine whether a second cycle of HDM/SCT could induce HCR in patients in whom the plasma cell dyscrasia persisted following initial treatment with HDM/SCT. Sixty-two patients were enrolled. Nine patients (15%) were removed from the protocol. Of the 53 patients continuing in this study, four died within 100 days of treatment (8%), and 27 (55%) achieved an HCR at 6 months after the first cycle of HDM/SCT. Of the 22 patients who did not achieve an HCR after initial treatment, 17 received a second HDM/SCT, 1 died within 100 days of treatment (6%), while 5 (31%) achieved an HCR. Thus, the HCR rate was 67% (32/48) for surviving patients on study, 60% (32/53) for all patients who received initial cycle of HDM/SCT, and 56% (35/62) by intention-to-treat. The median survival for all patients enrolled on the trial has not yet been reached. Thus, tandem cycles of HDM/SCT can increase the proportion of patients who achieve an HCR.
authors.affiliation: Department of Medicine [corrected] Stem Cell Transplantation Program, Section of Hematology-Oncology, Boston University School of Medicine [corrected] Boston, MA, USA. Vaishali.Sanchorawala@bmc.org
authors.forename: V
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: D G
authors.initials: DG
authors.lastname: Wright
authors.forename: K
authors.initials: K
authors.lastname: Quillen
authors.forename: K T
authors.initials: KT
authors.lastname: Finn
authors.forename: L M
authors.initials: LM
authors.lastname: Dember
authors.forename: J L
authors.initials: JL
authors.lastname: Berk
authors.forename: G
authors.initials: G
authors.lastname: Doros
authors.forename: C
authors.initials: C
authors.lastname: Fisher
authors.forename: M
authors.initials: M
authors.lastname: Skinner
authors.forename: D C
authors.initials: DC
authors.lastname: Seldin
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage* / Q000009:adverse effects; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000401:mortality; D006801:Humans; D007147:Immunoglobulin Light Chains; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D010352:Patient Dropouts; D011446:Prospective Studies; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D007147:Immunoglobulin Light Chains; D008558:Melphalan