Cladribine therapy for systemic mastocytosis
| Autor: | Rob Fijnheer, O Aart van Dobbenburgh, Jasper J. van Doormaal, Jan W Van 't Wout, Gregor Verhoef, J Marja Oldhoff, Hanneke C. Kluin-Nelemans, W. B. J. Gerrits, Suzanne G.M.A. Pasmans |
|---|---|
| Přispěvatelé: | Damage and Repair in Cancer Development and Cancer Treatment (DARE), Stem Cell Aging Leukemia and Lymphoma (SALL) |
| Jazyk: | angličtina |
| Rok vydání: | 2003 |
| Předmět: |
Adult
Male medicine.medical_specialty medicine.drug_class Antimetabolites DISORDERS medicine.medical_treatment Immunology Biochemistry Gastroenterology Antimetabolite PATIENT Mastocytosis Systemic Internal medicine Leukemia Myelogenous Chronic BCR-ABL Positive HISTAMINE Medicine Humans MAST-CELL DISEASE Systemic mastocytosis Cladribine MUTATION Aged Chemotherapy business.industry URTICARIA Cell Biology Hematology Middle Aged Mast cell leukemia medicine.disease Mast cell Surgery C-KIT Leukemia medicine.anatomical_structure Treatment Outcome Bone marrow suppression Myelodysplastic Syndromes Female Drug Eruptions INTERFERON-ALPHA business Biomarkers medicine.drug |
| Zdroj: | Blood, 102(13), 4270-4276. AMER SOC HEMATOLOGY |
| ISSN: | 0006-4971 |
| Popis: | 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. (C) 2003 by The American Society of Hematology. |
| Databáze: | OpenAIRE |
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