title: Autologous bone marrow transplantation with marrow purged by mafosfamide in seven patients with myelodysplastic syndromes in transformation (AML-MDS): a pilot study.
abstract: Seven patients with acute myeloblastic leukemia (AML) occurring on myelodysplastic syndromes (MDS) were consolidated while in complete remission (CR) by autologous bone marrow transplantation (ABMT) with a marrow purged in vitro by mafosfamide. The median age of population was 44 years (range 39-55). MDS FAB diagnosis was established before progression to AML in five patients: refractory anaemia with excess of blast (RAEB) in three patients, RAEB in transformation (RAEB-t) in one patient, and chronic myelomonocytic leukemia (CMML) in one patient. In the remaining two patients, the diagnosis of MDS (as a secondary malignancy in one) was made retrospectively at time of overt AML. Three out the seven patients had karyotypic abnormalities. The median interval between the obtention of CR and ABMT was 7 months (range 6-18). One patient died from transplant related toxicity. Engraftment occurred at a median of 41 days (range 27-60), for white blood cells (> 10(9)/l) and 120 days (range 60-180) for platelets (> 50 x 10(9)/l). Four patients relapsed at 2.5, 6.8, and 25 months post-ABMT. Two patients are alive and well at 10 and 28 months, respectively. ABMT with marrow purged by mafosfamide is feasible in patients with AML following MDS with a prospect of cure. However, further studies are needed to assess the real value of this approach.
authors.affiliation: Bone Marrow Transplant Unit, Service des Maladies du Sang and Formation Associée Claude-Bernard, Paris, France.
authors.forename: J P
authors.initials: JP
authors.lastname: Laporte
authors.forename: F
authors.initials: F
authors.lastname: Isnard
authors.forename: S
authors.initials: S
authors.lastname: Lesage
authors.forename: P
authors.initials: P
authors.lastname: Fenaux
authors.forename: L
authors.initials: L
authors.lastname: Douay
authors.forename: M
authors.initials: M
authors.lastname: Lopez
authors.forename: J
authors.initials: J
authors.lastname: Stachowiak
authors.forename: A
authors.initials: A
authors.lastname: Najman
authors.forename: N C
authors.initials: NC
authors.lastname: Gorin
mesh_terms: D000328:Adult; D000970:Antineoplastic Agents; D016465:Bone Marrow Purging; D016026:Bone Marrow Transplantation; D003520:Cyclophosphamide / Q000031:analogs & derivatives*; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D010865:Pilot Projects; D012074:Remission Induction; D014182:Transplantation, Autologous
chemical_list: D000970:Antineoplastic Agents; C048341:mafosfamide; D003520:Cyclophosphamide