A pilot study of nonmyeloablative allogeneic hematopoietic stem cell transplant for advanced systemic mastocytosis. Bone Marrow Transplant 2006 Feb;37(4):353-8
Date
01/10/2006Pubmed ID
16400343DOI
10.1038/sj.bmt.1705245Scopus ID
2-s2.0-32844466631 (requires institutional sign-in at Scopus site) 67 CitationsAbstract
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.
Author List
Nakamura R, Chakrabarti S, Akin C, Robyn J, Bahceci E, Greene A, Childs R, Dunbar CE, Metcalfe DD, Barrett AJMESH terms used to index this publication - Major topics in bold
AdultDisease Progression
Female
Graft Survival
Graft vs Host Disease
HLA Antigens
Hematopoietic Stem Cell Transplantation
Humans
Male
Mastocytosis, Systemic
Middle Aged
Pilot Projects
Prospective Studies
Recurrence
Siblings
Survival Rate
Transplantation Conditioning
Transplantation, Homologous
Treatment Outcome