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Adverse effects and benefits of two years of anagrelide treatment for thrombocythemia in chronic myeloproliferative disorders

Birgegard, G; Bjorkholm, M; Kutti, J; Larfars, G; Lofvenberg, E; Markevarn, B; Merup, M; Palmblad, J; Mauritzson, Nils and Westin, Jan LU , et al. (2004) In Haematologica 89(5). p.520-527
Abstract
Background and Objectives. Although anagrelide is widely used in the treatment of throm-bocythemia 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. A multicenter, open, phase II study of anagrelide treatment was performed by the Swedish Myeloproliferative Disorder Study Group. The study included 60 patients with thrombocythemia due to myeloproliferative disease, 42 with essential thrombocythemia (ET), 17 with polycythemia vera (PV) and one with myelofibrosis (MF). Results. Complete response (CR), defined as a platelet count... (More)
Background and Objectives. Although anagrelide is widely used in the treatment of throm-bocythemia 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. A multicenter, open, phase II study of anagrelide treatment was performed by the Swedish Myeloproliferative Disorder Study Group. The study included 60 patients with thrombocythemia due to myeloproliferative disease, 42 with essential thrombocythemia (ET), 17 with polycythemia vera (PV) and one with myelofibrosis (MF). 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. (Less)
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Contribution to journal
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published
subject
keywords
thrombocythemia, anagrelide, myeloproliferative disease
in
Haematologica
volume
89
issue
5
pages
520 - 527
publisher
Ferrata Storti Foundation
external identifiers
  • pmid:15136214
  • wos:000221292400003
  • scopus:2542419052
ISSN
1592-8721
language
English
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yes
id
3157fc21-328c-44f7-9ea9-a283aff653a1 (old id 279424)
date added to LUP
2007-08-02 10:48:21
date last changed
2017-11-26 03:48:22
@article{3157fc21-328c-44f7-9ea9-a283aff653a1,
  abstract     = {Background and Objectives. Although anagrelide is widely used in the treatment of throm-bocythemia 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. A multicenter, open, phase II study of anagrelide treatment was performed by the Swedish Myeloproliferative Disorder Study Group. The study included 60 patients with thrombocythemia due to myeloproliferative disease, 42 with essential thrombocythemia (ET), 17 with polycythemia vera (PV) and one with myelofibrosis (MF). Results. Complete response (CR), defined as a platelet count &lt;400x10(9)/L in symptomatic patients and &lt; 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 &gt;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.},
  author       = {Birgegard, G and Bjorkholm, M and Kutti, J and Larfars, G and Lofvenberg, E and Markevarn, B and Merup, M and Palmblad, J and Mauritzson, Nils and Westin, Jan and Samuelsson, J},
  issn         = {1592-8721},
  keyword      = {thrombocythemia,anagrelide,myeloproliferative disease},
  language     = {eng},
  number       = {5},
  pages        = {520--527},
  publisher    = {Ferrata Storti Foundation},
  series       = {Haematologica},
  title        = {Adverse effects and benefits of two years of anagrelide treatment for thrombocythemia in chronic myeloproliferative disorders},
  volume       = {89},
  year         = {2004},
}