Real-World Clinical Characterisation of Polycythaemia Vera Patients from a Prospective Registry in Portugal: Is Resistance to Hydroxyurea a Reality?

IF 1.1 Q4 HEMATOLOGY Hematology Reports Pub Date : 2023-09-13 DOI:10.3390/hematolrep15030056
Maria Sarmento, Marta Duarte, Sandra Ponte, Juan Sanchez, Diana Roriz, Laura Fernandes, Maria José Monteiro Silva, Judite Pacheco, Gisela Ferreira, Jorge Freitas, Inês Costa, Daniel Brás
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Abstract

Patients with polycythaemia vera (PV) are at increased risk of thrombosis and haemorrhages. Although hydroxyurea (HU) has been the frontline therapy for patients at high risk of vascular complications, about 25% of patients develop resistance/intolerance to this therapy. The aim of this non-interventional, multicentre cohort study was to understand the clinical characteristics and HU treatment response of Portuguese PV patients. HU resistance/intolerance was defined according to adjusted European LeukemiaNet (ELN) criteria. In total, 134 PV patients with a mean (SD) disease duration of 4.8 (5.0) years were included and followed up for 2 years. At baseline, most patients were ≥60 years old (83.2%), at high risk for thrombotic events (87.2%), and receiving HU therapy (79.1%). A total of 10 thrombotic events and 8 haemorrhagic events were reported, resulting in a 5-year probability of thrombo-haemorrhagic events of 17.2%. Haematocrit (p = 0.007), haemoglobin (p = 0.012) and MPN10 symptom score (12.0 (11.6) vs. 10.3 (9.1); p = 0.041) decreased significantly at the 24-month visit compared to baseline. Overall, 75.9% of patients met at least one of the adjusted ELN criteria for HU resistance, and 14.4% of patients remained on HU throughout the study. The results from this real-world study may help identify the subset of patients at higher risk for disease sequelae who may benefit from earlier second-line treatment.

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葡萄牙前瞻性登记的维拉性红细胞增多症患者的真实世界临床特征:羟基脲耐药性是现实吗?
真性红细胞增多症(PV)患者发生血栓和出血的风险增加。尽管羟基脲(HU)一直是血管并发症高危患者的一线治疗方法,但约25%的患者对该疗法产生耐药性/不耐受。这项非介入性、多中心队列研究的目的是了解葡萄牙PV患者的临床特征和HU治疗反应。HU抵抗/不耐受根据调整后的欧洲白血病网(ELN)标准进行定义。总共纳入134名PV患者,其平均(SD)疾病持续时间为4.8(5.0)年,并随访2年。基线时,大多数患者年龄≥60岁(83.2%),有血栓事件的高风险(87.2%),并接受HU治疗(79.1%)。共报告了10例血栓事件和8例出血事件,导致血栓出血事件的5年概率为17.2%。血细胞压积(p=0.007)、血红蛋白(p=0.012)和MPN10症状评分(12.0(11.6)对10.3(9.1);p=0.041)在24个月访视时与基线相比显著降低。总体而言,75.9%的患者至少符合一项调整后的ELN HU耐药性标准,14.4%的患者在整个研究期间仍使用HU。这项现实世界研究的结果可能有助于确定可能受益于早期二线治疗的疾病后遗症风险较高的患者子集。
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来源期刊
Hematology Reports
Hematology Reports HEMATOLOGY-
CiteScore
0.90
自引率
0.00%
发文量
47
审稿时长
10 weeks
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