title: A pilot study of nonmyeloablative allogeneic hematopoietic stem cell transplant for advanced systemic mastocytosis.
abstract: Systemic mastocytosis (SM) is a disease characterized by tissue infiltration of neoplastic mast cells originating from hematopoietic stem cells. Patients with advanced SM have a poor prognosis, and there is no mast cell ablative therapy available for most patients who carry an activating point mutation in the c-kit gene. We report results of a prospective study evaluating the safety, engraftment, and possibility of inducing a graft-versus-mast cell (GvMC) effect after allogeneic nonmyeloablative hematopoietic cell transplantation (HCT) from an HLA-identical sibling. Three patients with advanced SM were transplanted. All achieved complete donor T cell chimerism followed by clinical evidence for GvMC effect. However, all patients experienced disease progression with the longest response duration of 39 months. The GvMC effect can be observed after nonmyeloablative HCT with limited efficacy. Effective cytoreductive therapy prior to HCT may be required for long-term disease control and cure.
authors.affiliation: Stem Cell Allogeneic Transplant Unit, Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA. rnakamura@coh.org
authors.forename: R
authors.initials: R
authors.lastname: Nakamura
authors.forename: S
authors.initials: S
authors.lastname: Chakrabarti
authors.forename: C
authors.initials: C
authors.lastname: Akin
authors.forename: J
authors.initials: J
authors.lastname: Robyn
authors.forename: E
authors.initials: E
authors.lastname: Bahceci
authors.forename: A
authors.initials: A
authors.lastname: Greene
authors.forename: R
authors.initials: R
authors.lastname: Childs
authors.forename: C E
authors.initials: CE
authors.lastname: Dunbar
authors.forename: D D
authors.initials: DD
authors.lastname: Metcalfe
authors.forename: A J
authors.initials: AJ
authors.lastname: Barrett
mesh_terms: D000328:Adult; D018450:Disease Progression; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology / Q000628:therapy; D006680:HLA Antigens / Q000032:analysis; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006801:Humans; D008297:Male; D034721:Mastocytosis, Systemic / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D012008:Recurrence; D035781:Siblings; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens