Department of Internal Medicine, University Hospitals of Leuven, Leuven, Belgium.
Department of Hematology, University Hospitals of Leuven, Herestraat 49, 3000, Leuven, Belgium.
Ann Hematol. 2019 Jun;98(6):1421-1426. doi: 10.1007/s00277-019-03654-6. Epub 2019 Mar 27.
Hydroxyurea (HU) resistance or intolerance occurs in 15 to 24% of patients with polycythemia vera (PV). Resistance to HU is associated with a shortened life expectancy, intolerance has no prognostic value. We assessed the occurrence of HU resistance or intolerance comparing the original (ELNo) versus the modified European Leukemia Net (ELNm) criteria as applied in recent large clinical trials including PV patients. We retrospectively analyzed 106 patients with PV treated with HU at the University Hospitals of Leuven between 1990 and 2016 for occurrence of HU resistance/intolerance when using both ELNo as ELNm. After a mean duration of treatment of 5.1 years, when applying the ELNo 20.7% of patients had shown resistance or intolerance to HU in comparison to 39.6% when using the ELNm. When using the ELNo 4.7% of patients were resistant to HU versus 23.6% when applying the ELNm. In total, 16.0% of patients were HU intolerant. This rate was identical when using both ELNo and ELNm. 20.7% of PV patients were considered as HU-resistant or intolerant when using the original ELN criteria. However, when applying the modified ELN criteria 39.6% of PV patients were resistant or intolerant to HU. In our hands, no patient received a minimum dose of 2 g HU a day, as such the ELNm seem better adapted for daily clinical use. However, the prognostic value of HU-resistance in PV, when defined by the ELNm, still needs to be confirmed.
羟基脲 (HU) 耐药或不耐受发生于 15%至 24%的真性红细胞增多症 (PV) 患者中。HU 耐药与预期寿命缩短有关，不耐受则无预后价值。我们评估了原始 (ELNo) 与改良欧洲白血病网 (ELNm) 标准在近期大型临床试验中应用于评估 HU 耐药或不耐受的发生率，这些试验包括 PV 患者。我们回顾性分析了 1990 年至 2016 年在鲁汶大学医院接受 HU 治疗的 106 例 PV 患者，应用 ELNo 和 ELNm 评估 HU 耐药/不耐受的发生情况。在平均 5.1 年的治疗后，应用 ELNo 时 20.7%的患者对 HU 耐药或不耐受，而应用 ELNm 时则为 39.6%。应用 ELNo 时 4.7%的患者对 HU 耐药，而应用 ELNm 时则为 23.6%。总共有 16.0%的患者 HU 不耐受。应用 ELNo 和 ELNm 时这一比例相同。应用原始 ELN 标准时，20.7%的 PV 患者被认为 HU 耐药或不耐受。然而，应用改良 ELN 标准时，39.6%的 PV 患者对 HU 耐药或不耐受。在我们的研究中，没有患者接受每天 2g HU 的最低剂量，因此 ELNm 似乎更适合于日常临床应用。然而，ELNm 定义的 HU 耐药在 PV 中的预后价值仍有待确认。
