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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.

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Acknowledgements

The authors would like to acknowledge our transplant coordinators and transplant nurses for their dedicated care of our patients, and all the members of the Bone Marrow Transplant Team for their constant support of the program. We also thank the Warren Grant Magnusson Clinical Center Intensive Care Unit for their exemplary patient care.

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Correspondence to R Nakamura.

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Nakamura, R., Chakrabarti, S., Akin, C. et al. A pilot study of nonmyeloablative allogeneic hematopoietic stem cell transplant for advanced systemic mastocytosis. Bone Marrow Transplant 37, 353–358 (2006). https://doi.org/10.1038/sj.bmt.1705245

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