Conflict of interest: Nothing to report
Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine†
Article first published online: 9 OCT 2009
Copyright © 2009 Wiley-Liss, Inc.
American Journal of Hematology
Volume 84, Issue 12, pages 790–794, December 2009
How to Cite
Lim, K. H., Pardanani, A., Butterfield, J. H., Li, C.-Y. and Tefferi, A. (2009), Cytoreductive therapy in 108 adults with systemic mastocytosis: Outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am. J. Hematol., 84: 790–794. doi: 10.1002/ajh.21561
- Issue published online: 23 NOV 2009
- Article first published online: 9 OCT 2009
- Accepted manuscript online: 9 OCT 2009 12:00AM EST
- Manuscript Accepted: 5 OCT 2009
- Manuscript Revised: 2 OCT 2009
- Manuscript Received: 20 AUG 2009
Cytoreductive therapy in systemic mastocytosis (SM) includes several drugs whose individual merit has not been well characterized. We retrospectively studied 108 Mayo Clinic patients who met the 2008 WHO diagnostic criteria for SM and received at least one cytoreductive drug. The numbers of patients who were evaluable for response to treatment with interferon-alpha with or without prednisone (IFN-α), hydroxyurea (HU), imatinib mesylate (IM) or 2-chlorodeoxyadenosine (2-CdA) were 40, 26, 22, and 22, respectively. The corresponding overall (major) response rates, according to recently published consensus criteria, were 53% (18%), 19% (0%), 18% (9%), and 55% (37%). The respective overall response rates in indolent SM, aggressive SM and SM associated with another clonal hematological nonmast cell lineage disease (SM-AHNMD) were 60%, 60%, 45% for IFN-α, 0, 0, 21% for HU, 14%, 50%, 9% for IM and 56%, 50%, 55% for 2-CdA. The absence of mast cell mediator release symptoms in IFN-α-treated patients and presence of circulating immature myeloid cells in 2-CdA-treated patients predicted inferior response. TET2 mutational status did not influence treatment response. Although the major response rates with these four cytoreductive agents were still suboptimal and HU was mainly used in patients with SM-AHNMD, the current study favors 2-CdA or IFN-α as first-line current therapy in SM and identifies patients who are likely to respond to such therapy. Am. J. Hematol., 2009. © 2009 Wiley-Liss, Inc.