Approaches to the Treatment of Patients with Myelofibrosis and Polycythemia Vera with Constitutional Symptoms in Real-World Clinical Practice in the Russian Federation: Intermediate Results of a Multi-Center Observational Prospective Clinical Study

Q4 Medicine
Анаит Левоновна Меликян, И. Н. Суборцева, С. М. Куликов, Ю. А. Чабаева, Е. А. Гилязитдинова, К. П. Новоселов, Е. А. Князева, А. С. Егорова, И. С. Степочкин, Е. В. Королева, Т. М. Сычева, В. П. Бельгесова, А. Ю. Путинцева, О. М. Сендерова, И. В. Васильева, Е. Ю. Комарцева, А. А. Каплина, В. И. Бахтина, М. А. Михалев, Ю. Б. Черных, Е. Н. Паровичникова
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 Materials & Methods. The analysis focused on the data of 1229 patients. In 629 (51.18 %) patients, PV was diagnosed, MF was identified in 521 (42.39 %) patients. The diagnosis of 79 (6.43 %) patients was not reported. Early stage of primary MF (PMF) was detected in 182 (34.93 %) patients, PMF fibrosis stage was identified in 251 (48.18 %) patients, post-polycythemic MF was registered in 61 (11.71 %) patients, and 13 (2.5 %) patients showed post-thrombocythemic MF. In 14 (2.69 %) patients, MF type was not reported. By the time of diagnosis, the median age of PV patients was 56 years (range 17–86 years), and that of MF patients was 55 years (range 16–83 years) (p = 0.022). The proportion of women among PV patients was 57 %, among MF patients it was 65 % (p = 0.0065).
 Results. The assessment of thrombotic complication risk in PV showed that 51.01 % (n = 302) of patients belong to the low-risk, 39.86 % (n = 236) belong to the intermediate-risk, and only 9.12 % (n = 54) of patients belong to the high-risk groups. Distribution of MF patients between risk groups demonstrates favorable prognosis for most patients. The group of low and intermediate-1 risks includes 56.43 % (n = 294) patients according to the prognostic scoring system IPSS and 68.52 % (n = 357) according to the prognostic scoring system DIPSS. In the vast majority of cases, patients received hydroxycarbamide therapy: 81.81 % (n = 832) in the total cohort, 83.33 % (n = 465) in the PV group, and 79.96 % (n = 367) in the MF group. Interferon-α was administered to 19.71 % (n = 110) of PV patients and 29.85 % (n = 137) of MF patients. Ruxolitinib was assigned to 3.14 % (n = 19) of PV patients and 21.35 % (n = 98) of MF patients.
 Conclusion. Regular monitoring of the PV and MF course and treatment efficacy can provide recommendations for adequate change of therapy in case of the failure of previous treatment. It should be emphasized that the timely switch to the second-line therapy results in reduced disability and mortality among PV and MF patients with myeloproliferative neoplasms.","PeriodicalId":36905,"journal":{"name":"Klinicheskaya Onkogematologiya/Clinical Oncohematology","volume":"659 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2023-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Klinicheskaya Onkogematologiya/Clinical Oncohematology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.21320/2500-2139-2023-16-2-146-153","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"Medicine","Score":null,"Total":0}
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Abstract

Aim. To describe the methods of drug therapy implemented for the disease control in patients with polycythemia vera (PV) and myelofibrosis (MF) as well as to analyze manifestations and severity of the disease symptoms in real-world clinical practice. Materials & Methods. The analysis focused on the data of 1229 patients. In 629 (51.18 %) patients, PV was diagnosed, MF was identified in 521 (42.39 %) patients. The diagnosis of 79 (6.43 %) patients was not reported. Early stage of primary MF (PMF) was detected in 182 (34.93 %) patients, PMF fibrosis stage was identified in 251 (48.18 %) patients, post-polycythemic MF was registered in 61 (11.71 %) patients, and 13 (2.5 %) patients showed post-thrombocythemic MF. In 14 (2.69 %) patients, MF type was not reported. By the time of diagnosis, the median age of PV patients was 56 years (range 17–86 years), and that of MF patients was 55 years (range 16–83 years) (p = 0.022). The proportion of women among PV patients was 57 %, among MF patients it was 65 % (p = 0.0065). Results. The assessment of thrombotic complication risk in PV showed that 51.01 % (n = 302) of patients belong to the low-risk, 39.86 % (n = 236) belong to the intermediate-risk, and only 9.12 % (n = 54) of patients belong to the high-risk groups. Distribution of MF patients between risk groups demonstrates favorable prognosis for most patients. The group of low and intermediate-1 risks includes 56.43 % (n = 294) patients according to the prognostic scoring system IPSS and 68.52 % (n = 357) according to the prognostic scoring system DIPSS. In the vast majority of cases, patients received hydroxycarbamide therapy: 81.81 % (n = 832) in the total cohort, 83.33 % (n = 465) in the PV group, and 79.96 % (n = 367) in the MF group. Interferon-α was administered to 19.71 % (n = 110) of PV patients and 29.85 % (n = 137) of MF patients. Ruxolitinib was assigned to 3.14 % (n = 19) of PV patients and 21.35 % (n = 98) of MF patients. Conclusion. Regular monitoring of the PV and MF course and treatment efficacy can provide recommendations for adequate change of therapy in case of the failure of previous treatment. It should be emphasized that the timely switch to the second-line therapy results in reduced disability and mortality among PV and MF patients with myeloproliferative neoplasms.
俄罗斯联邦现实世界临床实践中具有体质症状的骨髓纤维化和真性红细胞增多症患者的治疗方法:一项多中心观察性前瞻性临床研究的中间结果
的目标。描述真性红细胞增多症(PV)和骨髓纤维化(MF)患者疾病控制的药物治疗方法,并分析现实临床实践中疾病症状的表现和严重程度。 材料,方法。分析的重点是1229名患者的数据。629例(51.18%)患者确诊PV, 521例(42.39%)患者确诊MF。79例(6.43%)患者未报告诊断。182例(34.93%)患者出现早期原发性MF (PMF), 251例(48.18%)患者出现PMF纤维化阶段,61例(11.71%)患者出现红细胞增多性MF, 13例(2.5%)患者出现血小板增多性MF。14例(2.69%)患者未报告MF型。确诊时PV患者的中位年龄为56岁(范围17-86岁),MF患者的中位年龄为55岁(范围16-83岁)(p = 0.022)。PV患者中女性比例为57%,MF患者中为65% (p = 0.0065)。 结果。对PV患者血栓形成并发症风险的评估显示,51.01% (n = 302)的患者属于低危组,39.86% (n = 236)的患者属于中危组,仅有9.12% (n = 54)的患者属于高危组。MF患者在危险组之间的分布表明大多数患者预后良好。根据预后评分系统IPSS,低、中危组患者占56.43% (n = 294);根据预后评分系统DIPSS,低、中危组患者占68.52% (n = 357)。在绝大多数病例中,患者接受了羟基脲治疗:总队列中81.81% (n = 832), PV组为83.33% (n = 465), MF组为79.96% (n = 367)。19.71%的PV患者(n = 110)和29.85%的MF患者(n = 137)给予干扰素α治疗。Ruxolitinib分配给3.14% (n = 19)的PV患者和21.35% (n = 98)的MF患者。结论。定期监测PV和MF病程和治疗效果,可以在先前治疗失败的情况下提供适当改变治疗的建议。应该强调的是,及时转向二线治疗可降低PV和MF合并骨髓增生性肿瘤患者的致残率和死亡率。
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来源期刊
CiteScore
0.80
自引率
0.00%
发文量
20
审稿时长
12 weeks
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