title: CY/TBI-800 as a pretransplant regimen for allogeneic bone marrow transplantation for severe aplastic anemia using HLA-haploidentical family donors.
abstract: Allogeneic BMT is the treatment of choice for patients with SAA who have an HLA-identical sibling donor. The results, however, have been relatively poor for transplants from partially matched family donors or unrelated donors because of the high incidence of graft rejection and/or GVHD. Six multiply transfused patients received a novel conditioning regimen of CY 200 mg/kg and TBI 800 cGy prior to receiving marrow from their HLA-haploidentical family donors. Three recipient-donor pairs were mismatched for one HLA locus, one for two loci and two for three loci. A combination of MTX and CsA was used for GVHD prophylaxis. Engraftment was noted in all six patients. Acute GVHD occurred in four patients, two each for grade I and II, respectively. One patient, who was ABO-compatible with her donor had delayed onset of pure red cell aplasia (PRCA) which completely recovered 6 months after additional immunotherapy with prednisolone. There were two deaths; both occurred while patients were on treatment for GVHD. One was from systemic fungemia and the other probably from cytomegalovirus interstitial pneumonitis (CMV-IP). Four patients (66.7%) have been alive and disease-free for more than 8.2, 27.3, 38.4 and 47.2 months after BMT, respectively. The results suggest that CY/TBI-800 may be a simple and effective conditioning regimen for SAA patients receiving BMT from family members other than HLA-identical siblings.
authors.affiliation: Section of Medical Oncology, Department of Medicine, Veterans General Hospital-Taipei, Taiwan, ROC.
authors.forename: C H
authors.initials: CH
authors.lastname: Tzeng
authors.forename: P M
authors.initials: PM
authors.lastname: Chen
authors.forename: S
authors.initials: S
authors.lastname: Fan
authors.forename: J H
authors.initials: JH
authors.lastname: Liu
authors.forename: T J
authors.initials: TJ
authors.lastname: Chiou
authors.forename: R K
authors.initials: RK
authors.lastname: Hsieh
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D006084:Graft Rejection / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D014184:Transplantation, Homologous; D014916:Whole-Body Irradiation
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide