Hydroxyurea-related toxicity in 3,411 patients with Ph'-negative MPN

Am J Hematol. 2012 May;87(5):552-4. doi: 10.1002/ajh.23160. Epub 2012 Apr 4.


Hydroxyurea (Hydroxycarbamide; HU) is commonly used for the long-term treatment of patients with Philadelphia-chromosome negative chronic myeloproliferative neoplasms (MPNs). It is considered a first-choice agent for the treatment of these disorders as underlined by the European Leukemia Net Consensus Conference [1], although it is formally approved for this indication in some countries only. The drug is reportedly well tolerated in the large majority of subjects, although systemic and/or localized toxicities have been reported. Consensus criteria for definition of "intolerance" to HU have been described;patients who develop intolerance are candidate for second-line therapy and, more recently, for investigational drugs. However, no epidemiologic information about the occurrence of the most clinically significant HU-associated adverse events is yet available. In this study, the authors report on a multicenter series of 3,411 patients who were treated with HU among which 184, accounting for 5% of total, developed significant drug-related toxicities. These data provide an estimate of the frequency and a detailed characterization of clinically significant HU-related toxicities; these information have relevance for the management of MPN patients who require second-line therapy after developing HU-related intolerance.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antimetabolites / adverse effects*
  • Antimetabolites / therapeutic use
  • Carcinoma, Squamous Cell / chemically induced
  • Carcinoma, Squamous Cell / epidemiology
  • Drug Eruptions / epidemiology
  • Drug Eruptions / etiology*
  • Female
  • Fever / chemically induced*
  • Fever / epidemiology
  • Humans
  • Hydroxyurea / adverse effects*
  • Hydroxyurea / therapeutic use
  • Keratosis, Actinic / chemically induced
  • Keratosis, Actinic / epidemiology
  • Male
  • Middle Aged
  • Multicenter Studies as Topic / statistics & numerical data
  • Myeloproliferative Disorders / complications
  • Myeloproliferative Disorders / drug therapy*
  • Myeloproliferative Disorders / genetics
  • Pneumonia / epidemiology
  • Pneumonia / etiology
  • Retrospective Studies
  • Skin Neoplasms / chemically induced
  • Skin Neoplasms / epidemiology
  • Skin Ulcer / chemically induced
  • Skin Ulcer / epidemiology
  • Young Adult


  • Antimetabolites
  • Hydroxyurea