title: Long term follow-up of allogeneic stem cell transplantation in patients with myelodysplastic syndromes using busulfan, cytosine arabinoside, and cyclophosphamide.
abstract: We report here the 10-year follow-up of 86 patients who underwent allogeneic stem cell transplantation (ASCT) for myelodysplastic syndrome (MDS). All patients received the busulfan, cytosine arabinoside, and cyclophosphamide (BAC) preparative regimen which consisted of busulfan 16 mg/kg, cytosine arabinoside 8 g/m(2) IV, and cyclophosphamide 120 mg/kg IV. Fifty-nine patients (69%) had de novo MDS; 26 (30%) had secondary MDS (treatment related), and one had a preceding aplastic anemia which progressed to MDS before transplant. Cytogenetics (80 patients) was classified as good (34%), intermediate (17%), or poor (42%). With a median follow-up for survivors of 124 months, the 10-year Kaplan-Meier estimates for overall survival (OS) was 43% (95% confidence interval [CI]: 31-53%). Cumulative nonrelapse mortality (NRM) and relapse was 43% (95% CI: 32-54%) and 19% (95% CI: 11-27%), respectively. No patient relapsed after 2 years. In patients with RAEB-T/AML, 10-year relapse-free survival (RFS), relapse, and NRM was 36%, 36%, and 27%, respectively. Younger age (P = 0.05), human leukocyte antigen (HLA) match (P = 0.002), good risk cytogenetics (P = 0.008), and having a related donor (P = 0.03) significantly improved overall and RFS in the multivariable analysis. The long-term follow-up of patients receiving the BAC regimen with ASCT in this study indicated durable relapse-free and OS with acceptable toxicity in this group of patients with high-risk features.
authors.affiliation: Medical College of Wisconsin, 9200 W Wisconsin Avenue, Milwuakee, Wisconsin 53226, USA. eatallah@mcw.edu
authors.forename: Ehab
authors.initials: E
authors.lastname: Atallah
authors.forename: Judith
authors.initials: J
authors.lastname: Abrams
authors.forename: Lois
authors.initials: L
authors.lastname: Ayash
authors.forename: Gail
authors.initials: G
authors.lastname: Bentley
authors.forename: Muneer
authors.initials: M
authors.lastname: Abidi
authors.forename: Voravit
authors.initials: V
authors.lastname: Ratanatharathorn
authors.forename: Joseph
authors.initials: J
authors.lastname: Uberti
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002066:Busulfan / Q000008:administration & dosage / Q000627:therapeutic use*; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use*; D003561:Cytarabine / Q000008:administration & dosage / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000401:mortality / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007239:Infections / Q000401:mortality; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000627:therapeutic use*; D009190:Myelodysplastic Syndromes / Q000401:mortality / Q000601:surgery*; D011183:Postoperative Complications / Q000401:mortality; D012008:Recurrence; D012189:Retrospective Studies; D033581:Stem Cell Transplantation; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D019653:Myeloablative Agonists; D003561:Cytarabine; D003520:Cyclophosphamide; D002066:Busulfan