Abstract | BACKGROUND AND OBJECTIVES: Although anagrelide is widely used in the treatment of thrombocythemia in myeloproliferative diseases, there is currently limited information on the efficacy and toxicity of its long-term use. This prospective study investigated clinical toxicity and efficacy of anagrelide during two years of treatment. DESIGN AND METHODS: RESULTS: Complete response (CR), defined as a platelet count <400x10(9)/L in symptomatic patients and < 600x10(9)/L in asymptomatic patients was achieved in 67% of the patients and partial response (PR) in 6%. The response rate was higher in patients with ET than in those with PV (p = 0.05). Primary treatment failure occurred in 27% due to lack of efficacy at a tolerable dose (n=13) or insufficient platelet response without side effects (n=3). In addition, another 14 patients withdrew from treatment before the end of the two-year period due to side effects. Side effects included palpitations (70%), headache (52%), nausea (35%), diarrhea or flatulence (33%), edema (22%) and fatigue (23%). Patients and doctors rated their satisfaction with the anagrelide treatment on a 10-grade scale from 7.6 at 3 months to >9 at 24 months. After two years, 50% ( n=30) of the patients continued anagrelide treatment. INTERPRETATION AND CONCLUSIONS: Side effects and toxic discontinuation rates were higher than in previous studies, probably because this is the first long-term prospective study of the feasibility and toxicity of anagrelide treatment. Nevertheless, anagrelide is a valuable alternative for treatment of thrombocythemia in myeloproliferative disorders for patients who tolerate the drug well.
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Authors | Gunnar Birgegård, Magnus Björkholm, Jack Kutti, Gerd Lärfars, Eva Löfvenberg, Berit Markevärn, Mats Merup, Jan Palmblad, Nils Mauritzson, Jan Westin, Jan Samuelsson |
Journal | Haematologica
(Haematologica)
Vol. 89
Issue 5
Pg. 520-7
(May 2004)
ISSN: 1592-8721 [Electronic] Italy |
PMID | 15136214
(Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- Platelet Aggregation Inhibitors
- Quinazolines
- anagrelide
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Topics |
- Adult
- Aged
- Chronic Disease
- Diarrhea
(chemically induced)
- Dose-Response Relationship, Drug
- Female
- Headache
(chemically induced)
- Heart Rate
(drug effects)
- Humans
- Male
- Middle Aged
- Myeloproliferative Disorders
(complications)
- Nausea
(chemically induced)
- Patient Compliance
- Patient Dropouts
- Platelet Aggregation Inhibitors
(adverse effects, therapeutic use)
- Prospective Studies
- Quinazolines
(adverse effects, therapeutic use)
- Thrombocytosis
(drug therapy, etiology)
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