Anagrelide in Essential Thrombocythemia (ET): Results from 150 patients over 25 years by the "Ph1-negative Myeloproliferative Neoplasms Latium Group"

Eur J Haematol. 2020 Sep;105(3):335-343. doi: 10.1111/ejh.13454. Epub 2020 Jun 16.

Abstract

Background and aims: Anagrelide is a drug effective in reducing platelet counts in essential thrombocythemia (ET) and Ph1-negative myeloproliferative neoplasms. The aim of this study was to evaluate the real-life use of anagrelide in patients with ET followed over 25 years at the Haematological Institutes belonging to "Ph1-negative Myeloproliferative Neoplasms Latium Group."

Patients and methods: Eligibility criteria were diagnosis of ET and treatment with anagrelide. Data were collected through an ad hoc case report form.

Results: One hundred and fifty patients received anagrelide for a median time of 7.4 years (0.1-23.2). Anagrelide was administered as first-line therapy in 34.7% of patients, as second-line in 52% and as third-line in 13.3%: 85.4% responded to therapy. Sixty-eight/136 evaluable patients reported side effects: palpitations, peripheral vasodilation, anaemia, diarrhoea and gastric distress. Fourteen thrombotic (arterial 10, venous 4) and 51 bleeding events (minor 48, major 3) occurred. Sixteen/150 (10.6%) patients developed secondary myelofibrosis and 3/150 (2%) an acute myeloid leukaemia.

Conclusions: In our experience, anagrelide is an effective drug in reducing platelet levels in a high percentage of patients with ET. It is especially addressed to younger people. A careful assessment of the thrombotic risk and monitoring of cardiac function, at diagnosis and during follow-up, is mandatory.

Keywords: adverse events; anagrelide; essential thrombocythemia; haemorrhage; thrombosis.

Publication types

  • Multicenter Study

MeSH terms

  • Adult
  • Age Factors
  • Aged
  • Aged, 80 and over
  • Anemia / diagnosis
  • Anemia / etiology
  • Disease Management
  • Disease Susceptibility
  • Drug Substitution
  • Female
  • Follow-Up Studies
  • Health Care Surveys
  • Humans
  • Italy / epidemiology
  • Male
  • Middle Aged
  • Pregnancy
  • Pregnancy Complications, Hematologic
  • Prognosis
  • Quinazolines / administration & dosage
  • Quinazolines / adverse effects
  • Quinazolines / therapeutic use*
  • Retreatment
  • Retrospective Studies
  • Thrombocythemia, Essential / diagnosis
  • Thrombocythemia, Essential / drug therapy*
  • Thrombocythemia, Essential / epidemiology*
  • Thrombocythemia, Essential / etiology
  • Thrombosis
  • Treatment Outcome
  • Young Adult

Substances

  • Quinazolines
  • anagrelide