title: Nonmyeloablative conditioning followed by transplantation of genetically modified HLA-matched peripheral blood progenitor cells for hematologic malignancies in patients with acquired immunodeficiency syndrome.
abstract: To assess the safety and efficacy of nonmyeloablative allogeneic transplantation in patients with HIV infection, a clinical protocol was initiated in patients with refractory hematologic malignancies and concomitant HIV infection. The results from the first 2 patients are reported. The indications for transplantation were treatment-related acute myelogenous leukemia and primary refractory Hodgkin disease in patients 1 and 2, respectively. Only patient 1 received genetically modified cells. Both patients tolerated the procedure well with minimal toxicity, and complete remissions were achieved in both patients, but patient 2 died of relapsed Hodgkin disease 12 months after transplantation. Patient 1 continues in complete remission with undetectable HIV levels and rising CD4 counts, and with both the therapeutic and control gene transfer vectors remaining detectable at low levels more than 2 years after transplantation. These results suggest that nonmyeloablative allogeneic transplantation in the context of highly active antiretroviral therapy is feasible in patients with treatment-sensitive HIV infection.
authors.affiliation: Molecular and Clinical Hematology Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
authors.forename: Elizabeth M
authors.initials: EM
authors.lastname: Kang
authors.forename: Moniek
authors.initials: M
authors.lastname: de Witte
authors.forename: Harry
authors.initials: H
authors.lastname: Malech
authors.forename: Richard A
authors.initials: RA
authors.lastname: Morgan
authors.forename: Sheila
authors.initials: S
authors.lastname: Phang
authors.forename: Charles
authors.initials: C
authors.lastname: Carter
authors.forename: Susan F
authors.initials: SF
authors.lastname: Leitman
authors.forename: Richard
authors.initials: R
authors.lastname: Childs
authors.forename: A John
authors.initials: AJ
authors.lastname: Barrett
authors.forename: Richard
authors.initials: R
authors.lastname: Little
authors.forename: John F
authors.initials: JF
authors.lastname: Tisdale
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000150:complications / Q000188:drug therapy / Q000628:therapy*; D000208:Acute Disease; D000328:Adult; D000900:Anti-Bacterial Agents / Q000627:therapeutic use; D023241:Antiretroviral Therapy, Highly Active; D003520:Cyclophosphamide / Q000494:pharmacology; D016572:Cyclosporine / Q000627:therapeutic use; D005240:Feasibility Studies; D015774:Ganciclovir / Q000627:therapeutic use; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation; D006689:Hodgkin Disease / Q000150:complications / Q000628:therapy*; D006801:Humans; D017053:Infection Control; D007951:Leukemia, Myeloid / Q000150:complications / Q000628:therapy*; D011241:Prednisone / Q000627:therapeutic use; D011292:Premedication; D012008:Recurrence; D012074:Remission Induction; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000494:pharmacology; D019562:Viral Load
chemical_list: D000900:Anti-Bacterial Agents; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine; D015774:Ganciclovir; D011241:Prednisone