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Blood, 15 December 2003, Vol. 102, No. 13, pp. 4270-4276.
Prepublished online as a Blood First Edition Paper on August 21, 2003; DOI 10.1182/blood-2003-05-1699.


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CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS

Cladribine therapy for systemic mastocytosis

Hanneke C. Kluin-Nelemans, J. Marja Oldhoff, Jasper J. van Doormaal, Jan W. van 't Wout, Gregor Verhoef, Wim B. J. Gerrits, O. Aart van Dobbenburgh, Suzanne G. Pasmans, and Rob Fijnheer

From the Department of Hematology, University Hospital Groningen, the Netherlands; the Department of Allergology, University Hospital Groningen, the Netherlands; the Department of Dermatology, University Medical Center Utrecht, the Netherlands; the Department of Hematology, University Medical Center Utrecht, the Netherlands; the Department of Internal Medicine, Bronovo Hospital, The Hague, the Netherlands; the Department of Hematology, Leyenburg Hospital, The Hague, the Netherlands; the Department of Internal Medicine, Gelre Hospital, Zutphen, the Netherlands; and the Department of Hematology, University Hospital, Leuven, Belgium.

Patients with systemic mastocytosis (SM) can suffer from disabling symptoms related to mast cell mediator release or mast cell infiltration, requiring mast cell eradication. In the present absence of any curative therapy, a recent case report describing the efficacy of cladribine showed promising results. In a pilot study, the efficacy of cladribine (0.10-0.13 mg/kg in a 2-hour infusion, days 1-5; repeated at 4-8 weeks until 6 cycles) was studied. Ten patients with SM with severe symptoms were treated. Four patients were classified as having indolent or smoldering mastocytosis, 3 as having aggressive systemic mastocytosis, and 3 as having SM with an accompanying hematologic malignancy. Nine patients received 6 courses, 1 patient stopped because of toxicodermia. All responded concerning signs, symptoms, and mast cell parameters (serum tryptase and urinary histamine metabolite excretion), although none achieved a complete remission. Prolonged follow-up is required, as response is ongoing in most cases. One patient relapsed within 11 months and showed a second response. Side effects were mainly related to bone marrow suppression. Single-agent cladribine is an effective and relatively safe treatment for severe systemic mastocytosis. The optimal dose and schedule need to be explored. (Blood. 2003;102:4270-4276)


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