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

Valerio De Stefano, Elena Rossi, Tommaso Za, E Antonioli, P Guglielmelli, L Pieri, M Finazzi, E Rumi, V Martinelli, N Vianelli, M Randi, I Bertozzi, M Ruggeri, E Elli, R Cacciola, E Cacciola, E Pogliani, F Rodeghiero, M Baccarani, F PassamontiG Finazzi, A Rambaldi, A Bosi, M Cazzola, T Barbui, A. Vannucchi

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78 Citations (Scopus)


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.
Original languageEnglish
Pages (from-to)552-554
Number of pages3
JournalAmerican Journal of Hematology
Publication statusPublished - 2012




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