Palandri Francesca, Rossi Elena, Auteri Giuseppe, Breccia Massimo, Paglia Simona, Benevolo Giulia, Elli Elena M, Cavazzini Francesco, Binotto Gianni, Tieghi Alessia, Tiribelli Mario, Heidel Florian H, Bonifacio Massimiliano, Pugliese Novella, Caocci Giovanni, Crugnola Monica, Mendicino Francesco, D'Addio Alessandra, Tomassetti Simona, Martino Bruno, Polverelli Nicola, Ceglie Sara, Mazzoni Camilla, Mullai Rikard, Ripamonti Alessia, Garibaldi Bruno, Pane Fabrizio, Cuneo Antonio, Krampera Mauro, Semenzato Gianpietro, Lemoli Roberto M, Vianelli Nicola, Palumbo Giuseppe A, Andriani Alessandro, Cavo Michele, Latagliata Roberto, De Stefano Valerio
Istituto di Ematologia "Seràgnoli", IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40138 Bologna, Italy.
Section of Hematology, Department of Radiological and Hematological Sciences, Catholic University School of Medicine, 00168 Rome, Italy.
Cancers (Basel). 2023 Jul 21;15(14):3706. doi: 10.3390/cancers15143706.
In polycythemia vera (PV), the prognostic relevance of an ELN-defined complete response (CR) to hydroxyurea (HU), the predictors of response, and patients' triggers for switching to ruxolitinib are uncertain. In a real-world analysis, we evaluated the predictors of response, their impact on the clinical outcomes of CR to HU, and the correlations between partial or no response (PR/NR) and a patient switching to ruxolitinib. Among 563 PV patients receiving HU for ≥12 months, 166 (29.5%) achieved CR, 264 achieved PR, and 133 achieved NR. In a multivariate analysis, the absence of splenomegaly ( = 0.03), pruritus ( = 0.002), and a median HU dose of ≥1 g/day ( < 0.001) remained associated with CR. Adverse events were more frequent with a median HU dose of ≥1 g/day. Overall, 283 PR/NR patients (71.3%) continued HU, and 114 switched to ruxolitinib. In the 449 patients receiving only HU, rates of thrombosis, hemorrhages, progression, and overall survival were comparable among the CR, PR, and NR groups. Many PV patients received underdosed HU, leading to lower CR and toxicity rates. In addition, many patients continued HU despite a PR/NR; however, splenomegaly and other symptoms were the main drivers of an early switch. Better HU management, standardization of the criteria for and timing of responses to HU, and adequate intervention in poor responders should be advised.
在真性红细胞增多症（PV）中，欧洲白血病网络（ELN）定义的对羟基脲（HU）的完全缓解（CR）的预后相关性、缓解的预测因素以及患者改用鲁索替尼的触发因素尚不确定。在一项真实世界分析中，我们评估了缓解的预测因素、它们对HU治疗CR的临床结局的影响以及部分缓解或无缓解（PR/NR）与患者改用鲁索替尼之间的相关性。在563例接受HU治疗≥12个月的PV患者中，166例（29.5%）达到CR，264例达到PR，133例达到NR。在多变量分析中，无脾肿大（ = 0.03）、瘙痒（ = 0.002）以及HU中位剂量≥1 g/天（ < 0.001）仍与CR相关。HU中位剂量≥1 g/天时不良事件更常见。总体而言，283例PR/NR患者（71.3%）继续使用HU，114例改用鲁索替尼。在仅接受HU治疗的449例患者中，CR、PR和NR组之间的血栓形成、出血、病情进展和总生存率相当。许多PV患者接受的HU剂量不足，导致CR率和毒性率较低。此外，许多患者尽管处于PR/NR状态仍继续使用HU；然而，脾肿大和其他症状是早期换药的主要驱动因素。应建议更好地管理HU、规范HU治疗反应的标准和时机以及对反应不佳者进行充分干预。
