title: Autologous transplant for chronic myelogenous leukemia using marrow treated ex vivo with mafosfamide.
abstract: Ten adult patients with Ph-positive chronic myelogenous leukemia (CML) received autologous transplantation using marrow treated ex vivo with mafosfamide. At transplant, 7 patients were in chronic phase (5 in first, 2 in second) and 3 in accelerated phase. The median time to achieve 500 x 10(6)/l neutrophils was 32 days (range 17-72 days). A platelet count of 20 x 10(9)/l was achieved at a median of 40 days (range 24-151 days). After transplant, cytogenetic analysis revealed 100% Ph-negative marrow metaphases in 6 of 9 analyzable patients with a median duration of Ph-negative hematopoiesis of 6.5 months. After a median follow-up of 16 months (range 3-31 months), five patients evolved into blast crisis, two died of non-hematological causes, one is Ph-negative in chronic phase at +4 and one is in chronic phase, but Ph-positive, at +22. In conclusion, this pilot study demonstrates that: (1) engraftment can occur from Ph-negative stem cells selected by mafosfamide, (2) mafosfamide purging may induce a transient period of Ph-negative hematopoiesis, and (3) modifications of the purging procedure and post-transplant manipulations of the immune-hematopoietic system are required to prolong cytogenetic remission.
authors.affiliation: Department of Hematology, Bone Marrow Transplantation Unit, University of Parma, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Carlo-Stella
authors.forename: L
authors.initials: L
authors.lastname: Mangoni
authors.forename: C
authors.initials: C
authors.lastname: Almici
authors.forename: C
authors.initials: C
authors.lastname: Caramatti
authors.forename: L
authors.initials: L
authors.lastname: Cottafavi
authors.forename: G P
authors.initials: GP
authors.lastname: Dotti
authors.forename: V
authors.initials: V
authors.lastname: Rizzoli
mesh_terms: D000328:Adult; D001853:Bone Marrow / Q000187:drug effects* / Q000473:pathology; D016465:Bone Marrow Purging; D016026:Bone Marrow Transplantation; D002908:Chronic Disease; D003520:Cyclophosphamide / Q000031:analogs & derivatives* / Q000494:pharmacology; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000473:pathology; D006801:Humans; D007621:Karyotyping; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D015996:Survival Rate; D014182:Transplantation, Autologous
chemical_list: C048341:mafosfamide; D003520:Cyclophosphamide