title: Treatment of leukemia relapse with recombinant granulocyte-macrophage colony stimulating factor (rhGM-CSF) following unrelated umbilical cord blood transplant: Induction of graft-vs.-leukemia.
abstract: An infant with congenital leukemia in complete remission (CR1) received an unrelated donor umbilical cord blood cell transplant from a one-HLA disparate donor. The conditioning regimen consisted of thiotepa, busulfan and cyclophosphamide. GVHD prophylaxis consisted of tacrolimus and mini-methotrexate. Engraftment occurred and a bone marrow aspirate obtained on day 28 showed 100% donor cells. The post-transplant course was complicated by skin and liver GVHD, grade III, that responded to therapy with methylprednisolone, anti-thymocyte globulin and daclizumab (Zenapax), in addition to tacrolimus. A bone marrow aspirate obtained on day 187 showed relapse, with 17% blasts. The patient was then treated for 30 days with recombinant human granulocyte-macrophage colony-stimulating factor treatment (rhGM-CSF). A bone marrow aspirate obtained 17 days after the initiation of rhGM-CSF treatment showed 2% blasts. Ascites was the predominant side-effect of the rhGM-CSF treatment. The patient remains in complete remission 24 months after relapse and 30 months after transplantation. This case documents that rhGM-CSF and withdrawal of immunosuppression can induce a durable complete remission after relapse following an unrelated donor cord blood transplant.
authors.affiliation: Department of Pediatrics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Box 87, Houston, TX 77030, USA. lworth@mdanderson.org
authors.forename: Laura L
authors.initials: LL
authors.lastname: Worth
authors.forename: Craig A
authors.initials: CA
authors.lastname: Mullen
authors.forename: Mary
authors.initials: M
authors.lastname: Choroszy
authors.forename: Susannah
authors.initials: S
authors.lastname: Koontz
authors.forename: KaWah
authors.initials: K
authors.lastname: Chan
mesh_terms: D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D020544:Graft vs Leukemia Effect / Q000187:drug effects* / Q000276:immunology*; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000151:congenital / Q000188:drug therapy*; D011994:Recombinant Proteins; D012008:Recurrence
chemical_list: D007166:Immunosuppressive Agents; D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor