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Effect of granulocyte macrophage colony‐stimulating factor in a patient with benign systemic mastocytosis
Author(s) -
Zuberbier T.,
Welker P.,
Grabbe J.,
Henz B.M.
Publication year - 2001
Publication title -
british journal of dermatology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.304
H-Index - 179
eISSN - 1365-2133
pISSN - 0007-0963
DOI - 10.1046/j.1365-2133.2001.04435.x
Subject(s) - tryptase , systemic mastocytosis , mast cell , cutaneous mastocytosis , granulocyte macrophage colony stimulating factor , medicine , bone marrow , immunology , granulocyte , immunoglobulin e , pathology , cytokine , antibody
We report the in vitro and in vivo effects of granulocyte macrophage colony stimulating factor (GM‐CSF), a known inhibitor of in vitro mast cell differentiation, in a patient with benign, adult‐onset systemic mastocytosis. In vitro effects of GM‐CSF on bone marrow cultures before the start of treatment showed a marked inhibition of mast cell marker expression [tryptase, Kit, and high‐affinity IgE receptor (FcεRIα)] at both protein and mRNA levels. Therefore, the patient was treated with daily injections of GM‐CSF for 10 weeks. After an initial improvement, increasing worsening of clinical symptoms was noted, and the patient refused further treatment. Lesional skin biopsies showed an increase of toluidine blue‐positive mast cells, compared with uninvolved skin, with further significant increase after treatment. Similar results were obtained on staining for mast cell‐specific tryptase and Kit, as well as for CD1a and FcεRIα. These findings show that GM‐CSF inhibits human bone marrow mast cell differentiation in vitro , and also in mastocytosis. However, GM‐CSF apparently enhances recruitment of mast cell as well as dendritic cell precursors into the tissue during systemic treatment. These findings and the observed adverse clinical effects in the present patient make it unlikely that GM‐CSF monotherapy will be beneficial for the treatment of mastocytosis.