Long-term use of anagrelide in young patients with essential thrombocythemia

E. C. Storen, Ayalew Tefferi

Research output: Contribution to journalArticle

154 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)863-866
Number of pages4
JournalBlood
Volume97
Issue number4
DOIs
StatePublished - Feb 15 2001

Fingerprint

Essential Thrombocythemia
Platelets
Platelet Count
Drug dosage
Anemia
Therapeutics
Toxicity
Drug-Related Side Effects and Adverse Reactions
Hemoglobins
anagrelide
Blood Platelets
Maintenance
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Hematology

Cite this

Long-term use of anagrelide in young patients with essential thrombocythemia. / Storen, E. C.; Tefferi, Ayalew.

In: Blood, Vol. 97, No. 4, 15.02.2001, p. 863-866.

Research output: Contribution to journalArticle

@article{d940ad729fba458aaaecb1c69caabc0a,
title = "Long-term use of anagrelide in young patients with essential thrombocythemia",
abstract = "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.",
author = "Storen, {E. C.} and Ayalew Tefferi",
year = "2001",
month = "2",
day = "15",
doi = "10.1182/blood.V97.4.863",
language = "English (US)",
volume = "97",
pages = "863--866",
journal = "Blood",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "4",

}

TY - JOUR

T1 - Long-term use of anagrelide in young patients with essential thrombocythemia

AU - Storen, E. C.

AU - Tefferi, Ayalew

PY - 2001/2/15

Y1 - 2001/2/15

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=0035865604&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0035865604&partnerID=8YFLogxK

U2 - 10.1182/blood.V97.4.863

DO - 10.1182/blood.V97.4.863

M3 - Article

C2 - 11159509

AN - SCOPUS:0035865604

VL - 97

SP - 863

EP - 866

JO - Blood

JF - Blood

SN - 0006-4971

IS - 4

ER -