title: High-dose melphalan with or without stem cell support before myeloablative allo-SCT for remission induction in patients with advanced relapsed or refractory AML.
abstract: Treatment strategies for relapsed/refractory AML are limited and disappointing. Recently, high-dose melphalan (HDM) chemotherapy and autologous hematopoietic SCT (HSCT) has been proposed for AML re-induction. We investigated the impact of HDM remission induction in highly advanced relapsed/refractory AML patients planned for allogeneic HSCT. A total of 23 patients with relapsed/refractory AML were prospectively scheduled for HDM with or without stem cell support followed by myeloablative allogeneic HSCT. Patients included nine individuals with a history of previous HSCT (seven allogeneic, two autologous). A total of 18 patients (78%) achieved a leukemia-free state and an additional four had substantial reduction of the initial leukemia burden warranting treatment continuation. There were no differences between patients with or without immediate stem cell support regarding mucositis or other organ toxicity. A total of 20 patients proceeded to myeloablative allogeneic HSCT. Outcome of allogeneic HSCT was poor: 11 patients (55%) relapsed, 7 patients (35%) died from TRM and only 2 patients (10%) were alive at the last follow-up. Our study shows that HDM is effective in inducing a leukemia-free state in patients with highly advanced relapsed/refractory AML. Leukemia burden reduction with HDM, however, did not translate into improved OS.
authors.affiliation: Division of Hematology, Department of Internal Medicine, University Hospital of Basel, Basel, Switzerland. AOmeara@uhbs.ch
authors.forename: A
authors.initials: A
authors.lastname: O'Meara
authors.forename: T
authors.initials: T
authors.lastname: Pabst
authors.forename: D
authors.initials: D
authors.lastname: Heim
authors.forename: S
authors.initials: S
authors.lastname: Gerull
authors.forename: C
authors.initials: C
authors.lastname: Bucher
authors.forename: J
authors.initials: J
authors.lastname: Halter
authors.forename: C
authors.initials: C
authors.lastname: Arber
authors.forename: A
authors.initials: A
authors.lastname: Rovò
authors.forename: A
authors.initials: A
authors.lastname: Tichelli
authors.forename: A
authors.initials: A
authors.lastname: Gratwohl
authors.forename: M
authors.initials: M
authors.lastname: Stern
mesh_terms: D000328:Adult; D000368:Aged; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006412:Hematopoietic Stem Cells / Q000187:drug effects; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000627:therapeutic use; D012008:Recurrence; D012074:Remission Induction / Q000379:methods; D019172:Transplantation Conditioning / Q000379:methods*; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D019653:Myeloablative Agonists; D008558:Melphalan