Objective: To explore the relationship between symptom burden and hematologic responses after treatment with interferon and/or hydroxyurea in patients with polycythemia vera (PV) . Methods: Hematologic responses after continuous treatment with interferon and/or hydroxyurea for six months were evaluated in 190 patients with PV using the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-10 score) . In all patients, the PV diagnosis was based on the 2016 World Health Organization diagnostic definitions. Results: The study cohort comprised 93 (48.9% ) male and 97 (51.1% ) female patients. The median age at the time of MPN-10 assessment was 60 (32-82) years. The median MPN-10 score of the entire cohort was 9 (range, 0-67) . The median MPN-10 score of patients treated with interferon plus hydroxyurea (n=27) was 11 (0-67) , which was significantly higher than those of patients treated with interferon only (n=64) (6[0-56], P=0.019) or hydroxyurea only (n=99) (9[0-64], P=0.047) , whereas the median MPN-10 score was not significantly different between those treated with interferon only and hydroxyurea only (P=0.421) . The rate of severe symptom burden (i.e., any single symptom burden score ≥ 7 and/or total score ≥ 44) was 28.9% (55/190) in the entire cohort, whereas the rate of severe symptom burden was not significantly different among the interferon only (23.4% ) , hydroxyurea only (29.3% ) , and interferon plus hydroxyurea (40.7% ) groups (P>0.05 for all two-group comparisons) . When evaluating MPN-10 score, 37.4% (71/190) of the patients achieved complete hematologic remission (CHR) . Only 28.9% (55/190) patients had adequate disease control, defined as CHR without severe symptom burden. Reasons for inadequate disease control were evaluating blood counts alone, severe symptom burden alone, and evaluating blood counts accompanied with severe symptom burden in 42.1% (80/190) , 8.4% (16/190) , and 20.5% (39/190) of the patients, respectively. Compared to the patients with a platelet count ≤ 400×10(9)/L, those with a platelet count > 400×10(9)/L had a significantly higher rate of severe symptom burden (40.8% [20/49] vs 24.8% [35/141], P=0.044) and a higher median MPN-10 score (14[0-67] vs 7[0-56], P=0.038) . Platelet count > 400×10(9)/L was associated with an increased risk of severe symptom burden (hazard ratio, 2.089; 95% confidence interval, 1.052-4.147, P=0.035) . Conclusions: Symptoms related to disease after treatment with interferon and/or hydroxyurea were rather universal in patients with PV. Some patients still experienced severe symptom burden despite achieving CHR. Platelet count > 400×10(9)/L was associated with an increased risk of severe symptom burden in patients with PV treated with interferon and/or hydroxyurea.
目的： 探讨真性红细胞增多症（PV）患者干扰素和（或）羟基脲治疗后症状负荷改善与血液学疗效之间的关系。 方法： 对190例符合WHO（2016）诊断分型标准、接受干扰素和（或）羟基脲连续治疗≥6个月的PV患者，分别应用外周血细胞计数和骨髓增殖性肿瘤总症状评估量表（MPN-10）评价患者血液学疗效和症状负荷。 结果： 全部190例PV患者中，男93例（48.9%），女97例（51.1%）。进行MPN-10评分时，患者中位年龄为60（32~82）岁。全部患者中位MPN-10总分为9（0~67）分，干扰素+羟基脲组（27例）为11（0~67）分，显著高于干扰素组[64例，6（0~56）分，P＝0.019]和羟基脲组[99例，9（0~64）分，P＝0.047]，而干扰素组与羟基脲组差异无统计学意义（P＝0.421）。28.9%（55/190）的患者存在重度症状（单项症状评分≥7分或总分≥44分），干扰素组、羟基脲组、干扰素+羟基脲组重度症状患者占比分别为23.4%、29.3%、40.7%，组间比较差异均无统计学意义（P>0.05）。全部190例患者中，进行MPN-10评分时71例（37.4%）获得完全血液学缓解（CHR），仅55例（28.9%）获得疾病充分控制（获得CHR且无重度症状）。未获得疾病充分控制的患者包括：血细胞增高80例（42.1%），获得CHR但伴重度症状16例（8.4%），血细胞增高且伴重度症状39例（20.5%）。PLT>400×10(9)/L患者中存在重度症状患者比例、MPN-10总分中位数均高于PLT≤400×10(9)/L患者[40.8%（20/49）对24.8%（35/141），P＝0.044；14（0~67）分对7（0~56）分，P＝0.038]。PLT>400×10(9)/L是存在重度症状的危险因素（HR＝2.089，95%CI 1.052~4.147，P＝0.035）。 结论： 经干扰素和（或）羟基脲治疗后，PV患者症状负荷未获满意控制仍较突出，部分患者虽然获得CHR，仍存在重度症状负荷。PLT>400×10(9)/L是干扰素和羟基脲治疗后患者存在重度症状的危险因素。.
Keywords: Hydroxyurea; Interferon; Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score; Polycythemia vera; Response.