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First line treatment of adult patients with primary immune thrombocytopenia: a real-world study.

成人原发性免疫性血小板减少症患者的一线治疗: 一项真实世界的研究。

  • 影响因子:2.36
  • DOI:10.1080/09537104.2019.1572875
  • 作者列表:"Wang L","Xu L","Hao H","Jansen AJG","Liu G","Li H","Liu X","Zhao Y","Peng J","Hou M
  • 发表时间:2020-01-01
Abstract

:Immune thrombocytopenia (ITP) is an autoimmune disease with a mild to severe risk of bleeding complications. First line treatment includes corticosteroids, immunoglobulins, or other. In this large cohort study, first-line strategies for treatment-naive adult primary ITP was studied in a real-world setting. Records from all adult ITP patients who received first-line treatment between January 2010 and December 2017 at Qilu Hospital were reviewed retrospectively (n = 699). During the study period, 271 patients were treated with high-dose dexamethasone (HDD) and 289 patients were treated with conventional prednisone (alone or in combination with other drugs). Initial responses were similar for the two groups (88.56% vs. 86.51%, P = 0.462), but patients in the HDD group responded earlier than the prednisone group (3 days vs. 5 days, P < 0.001). The sustained response (SR) at 6 months was lower in the HDD group than in the prednisone group (35.4% vs. 47.1%, P = 0.040). However, the SR at 12 months and at the end of our follow-up were not significantly different between the groups. Overall duration of response (DOR) in the prednisone group was longer than in the HDD group throughout the follow-up period (P = 0.007). However, the incidence of SR and overall DOR were not significantly different between the HDD group and the prednisone 3 months group (prednisone terminated within 3 months). The presence of anti-GPIb-IX autoantibodies was a predictive factor for a poor initial response to corticosteroids therapy (P < 0.05). However, neither of the two antiplatelet autoantibodies were correlated with the opportunity to achieve SR and overall DOR in both groups throughout the follow-up period (P > 0.05). Adverse events were more frequent and long-lasting in the prednisone group. Our study showed that HDD provided an effective and more rapid response as initial treatment of ITP, with comparable long-term prognosis and better tolerance when compared with conventional PDN (less than 3 months) in the real-world setting.

摘要

: 免疫性血小板减少症 (ITP) 是一种自身免疫性疾病,有轻度至重度出血并发症的风险。一线治疗包括皮质类固醇、免疫球蛋白或其他。在这项大型队列研究中,在真实世界环境中研究了初治成人原发性ITP的一线策略。回顾性分析 2010 年 1 月至 2017 年 12 月期间在齐鲁医院接受一线治疗的所有成人ITP患者的记录 (n = 699)。在研究期间,271 例患者接受大剂量地塞米松 (HDD) 治疗,289 例患者接受常规泼尼松 (单独或与其他药物联合) 治疗。两组的初始反应相似 (88。5 6% vs. 86.5 1%,P = 0.462),但HDD组患者反应早于泼尼松组 (3 天vs. 5 天,P <0.001)。HDD组 6 个月时的持续反应 (SR) 低于泼尼松组 (35.4% vs. 47.1%,P = 0.040)。然而,12 个月和随访结束时的SR在两组之间没有显著差异。在整个随访期间,泼尼松组的总体缓解持续时间 (DOR) 长于HDD组 (P = 0.007)。然而,HDD组和泼尼松 3 个月组 (泼尼松在 3 个月内终止) 的SR和总体DOR发生率无显著差异。抗GPIb-IX自身抗体的存在是皮质类固醇治疗初始反应不佳的预测因素 (P <0.05)。然而,在整个随访期间,两种抗血小板自身抗体均与两组获得SR和总体DOR的机会无关 (P> 0.05)。泼尼松组不良事件发生率更高,且持续时间更长。我们的研究表明,与传统PDN (小于 3 个月) 相比,HDD作为ITP的初始治疗提供了有效和更快速的反应,具有相当的长期预后和更好的耐受性在现实世界的设置。

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影响因子:3.70
发表时间:2020-01-01
DOI:10.1093/ndt/gfy373
作者列表:["Caluwé R","Verbeke F","De Vriese AS"]

METHODS::The cardinal biological role of vitamin K is to act as cofactor for the carboxylation of a number of vitamin K-dependent proteins, some of which are essential for coagulation, bone formation and prevention of vascular calcification. Functional vitamin K deficiency is common and severe among dialysis patients and has garnered attention as a modifiable risk factor in this population. However, no single biochemical parameter can adequately assess vitamin K status. For each biological function of vitamin K, the degree of carboxylation of the relevant vitamin K-dependent protein most accurately reflects vitamin K status. Dephosphorylated uncarboxylated matrix Gla protein (dp-ucMGP) is the best biomarker for vascular vitamin K status when cardiovascular endpoints are studied. Dp-ucMGP levels are severely elevated in haemodialysis patients and correlate with markers of vascular calcification and mortality in some but not all studies. The aetiology of vitamin K deficiency in haemodialysis is multifactorial, including deficient intake, uraemic inhibition of the vitamin K cycle and possibly interference of vitamin K absorption by phosphate binders. The optimal vitamin K species, dose and duration of supplementation to correct vitamin K status in dialysis patients are unknown. Dp-ucMGP levels dose-proportionally decrease with supraphysiological vitamin K2 supplementation, but do not normalize even with the highest doses. In the general population, long-term vitamin K1 or K2 supplementation has beneficial effects on cardiovascular disease, bone density and fracture risk, and insulin resistance, although some studies reported negative results. In haemodialysis patients, several trials on the effects of vitamin K on surrogate markers of vascular calcification are currently ongoing.

翻译标题与摘要 下载文献
影响因子:2.36
发表时间:2020-01-01
来源期刊:Platelets
DOI:10.1080/09537104.2019.1572875
作者列表:["Wang L","Xu L","Hao H","Jansen AJG","Liu G","Li H","Liu X","Zhao Y","Peng J","Hou M"]

METHODS::Immune thrombocytopenia (ITP) is an autoimmune disease with a mild to severe risk of bleeding complications. First line treatment includes corticosteroids, immunoglobulins, or other. In this large cohort study, first-line strategies for treatment-naive adult primary ITP was studied in a real-world setting. Records from all adult ITP patients who received first-line treatment between January 2010 and December 2017 at Qilu Hospital were reviewed retrospectively (n = 699). During the study period, 271 patients were treated with high-dose dexamethasone (HDD) and 289 patients were treated with conventional prednisone (alone or in combination with other drugs). Initial responses were similar for the two groups (88.56% vs. 86.51%, P = 0.462), but patients in the HDD group responded earlier than the prednisone group (3 days vs. 5 days, P < 0.001). The sustained response (SR) at 6 months was lower in the HDD group than in the prednisone group (35.4% vs. 47.1%, P = 0.040). However, the SR at 12 months and at the end of our follow-up were not significantly different between the groups. Overall duration of response (DOR) in the prednisone group was longer than in the HDD group throughout the follow-up period (P = 0.007). However, the incidence of SR and overall DOR were not significantly different between the HDD group and the prednisone 3 months group (prednisone terminated within 3 months). The presence of anti-GPIb-IX autoantibodies was a predictive factor for a poor initial response to corticosteroids therapy (P < 0.05). However, neither of the two antiplatelet autoantibodies were correlated with the opportunity to achieve SR and overall DOR in both groups throughout the follow-up period (P > 0.05). Adverse events were more frequent and long-lasting in the prednisone group. Our study showed that HDD provided an effective and more rapid response as initial treatment of ITP, with comparable long-term prognosis and better tolerance when compared with conventional PDN (less than 3 months) in the real-world setting.

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