Hematology Unit, AOU Careggi Largo Brambilla 3, Firenze, IT.
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.
羟基脲（羟脲；HU）常用于治疗费城染色体阴性慢性骨髓增生性肿瘤（MPN）患者的长期治疗。正如欧洲白血病网共识会议所述[1]，它被认为是这些疾病的首选治疗药物，尽管在一些国家仅正式批准用于该适应症。据报道，该药物在绝大多数患者中耐受性良好，尽管已报告发生全身和/或局部毒性。已经描述了定义 HU“不耐受”的共识标准；对 HU 不耐受的患者是二线治疗的候选者，并且最近是研究药物的候选者。然而，关于最具临床意义的 HU 相关不良事件发生的流行病学信息尚不可用。在这项研究中，作者报告了一项多中心研究，该研究纳入了 3411 名接受 HU 治疗的患者，其中 184 名（占总数的 5%）出现了严重的药物相关毒性。这些数据提供了对临床显著 HU 相关毒性发生频率的估计，并对其进行了详细描述；这些信息与需要二线治疗的 MPN 患者的管理相关，这些患者在发生 HU 相关不耐受后需要二线治疗。
