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Blood, 15 February 2001, Vol. 97, No. 4, pp. 863-866
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Long-term use of anagrelide in young patients with essential
thrombocythemia
Elizabeth C. Storen and
Ayalew Tefferi
From the Division of Hematology and Internal Medicine,
Mayo Clinic, Rochester, MN.
Anagrelide is a novel platelet-lowering agent that has recently
been approved for use in essential thrombocythemia (ET) and related
disorders. Short-term drug efficacy and toxicity data have previously
been presented. The purpose of this study was to obtain additional
information regarding long-term anagrelide use. This is a retrospective
series of 35 young patients (17 to 48 years) with ET who received
anagrelide treatment before 1992. Initial drug dosage ranged between 1 and 10 mg/d, and the median maintenance dosage was 2.5 mg/d. The
overall initial response rate of 94% included 74% complete remissions
and 20% partial remissions. Of the 33 responding patients, 27 (82%)
remained on anagrelide therapy for a median of 10.8 years (range, 7 to
15.5). Of these, 66% maintained a complete and 34% a partial
remission over the study period. In general, the reporting of somatic
side effects decreased over time, and anemia was the only new side
effect that emerged after long-term therapy. Eight patients (24%)
experienced a more than 3 g/dL decrease in hemoglobin level. Despite
active therapy, 20% of the patients experienced a total of 10 thrombotic episodes, and a similar proportion experienced major
hemorrhagic events. All thrombohemorrhagic complications occurred at a
platelet count of more than 400 × 109/L. It is concluded
that long-term treatment of ET with anagrelide is associated with
decreased reporting of initial side effects and the development of
mild-to-moderate anemia. Complete normalization of platelet counts may
be needed to minimize residual thrombohemorrhagic risk during therapy.
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