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奥地利多发性骨髓瘤三线治疗的真实世界应用:奥地利骨髓瘤登记处(AMR)对下一代骨髓瘤治疗药物使用前治疗格局和临床结果的分析。

Real-World Use of 3rd Line Therapy for Multiple Myeloma in Austria: An Austrian Myeloma Registry (AMR) Analysis of the Therapeutic Landscape and Clinical Outcomes prior to the Use of Next Generation Myeloma Therapeutics.

作者信息

Willenbacher Ella, Weger Roman, Rochau Ursula, Siebert Uwe, Willenbacher Wolfgang

机构信息

Medical University of Innsbruck, Internal Medicine V-Hematology and Oncology, Innsbruck, Austria.

Area 4 Health Technology Assessment and Bioinformatics, ONCOTYROL-Center for Personalized Cancer Medicine, Innsbruck, Austria.

出版信息

PLoS One. 2016 Mar 3;11(3):e0147381. doi: 10.1371/journal.pone.0147381. eCollection 2016.

Abstract

OBJECTIVE

Clinical trials demonstrate improving survival in patients with multiple myeloma (MM) after treatment. However, it is unclear whether increased survival translates to a similar benefit in a real world setting.

METHODS

We analyzed the overall survival of 347 multiple myeloma patients in Austria by means of a national registry (AMR), focused on results from 3rd and later lines of therapy. This benchmark was chosen to define a baseline prior to the broad application of upcoming 2nd generation drugs (carfilzomib, pomalidomide).

RESULTS

Projected 10 years survival for patients with MM in Austria is estimated to be 56% in patients diagnosed in between the years 2011-2014, 21% in patients with a diagnosis made between 2000-2005, and 39% in those with a diagnosis made between 2006-2010). For the same intervals a significant increase in the use of both bortezomib, lenalidomide and thalidomide-so called IMiDs (from 2005 onwards) and their simultaneous use in combination therapies (from 2010 onwards) could be shown. The use of autologous transplantation (ASCT) remained more or less constant at ~ 35% of patients in the 1st line setting over the whole period, comparing well to international practice patterns, while the use of 2nd line ASCT increased from 5.5% to 18.7% of patients. Patients in 3rd or later line treatment (n = 105), showed that even in relapsed and refractory disease median survival was 27 months with a considerable proportion of long-term survivors (~20%).

CONCLUSION & PERSPECTIVE: With the expected emergence of additional active anti-myeloma compounds, we aim to assess survival in patients with relapsed and refractory MM.

摘要

目的

临床试验表明,多发性骨髓瘤(MM)患者经治疗后生存率有所提高。然而,在现实环境中生存率的提高是否能带来类似的益处尚不清楚。

方法

我们通过国家登记处(AMR)分析了奥地利347例多发性骨髓瘤患者的总生存率,重点关注三线及后续治疗的结果。选择这一基准是为了在即将广泛应用第二代药物(卡非佐米、泊马度胺)之前确定一个基线。

结果

预计奥地利MM患者的10年生存率在2011 - 2014年诊断的患者中为56%,在2000 - 2005年诊断的患者中为21%,在2006 - 2010年诊断的患者中为39%。在相同时间段内,可以看出硼替佐米、来那度胺和沙利度胺(即所谓的免疫调节药物,IMiDs,从2005年起)的使用显著增加,并且它们在联合治疗中的同时使用(从2010年起)也有所增加。在整个时期内,一线自体造血干细胞移植(ASCT)的使用率在患者中大致保持在35%左右,与国际实践模式相当,而二线ASCT的使用率从患者的5.5%增加到了18.7%。三线或后续治疗的患者(n = 105)显示,即使在复发和难治性疾病中,中位生存期仍为27个月,有相当比例的长期存活者(约20%)。

结论与展望

随着更多活性抗骨髓瘤化合物的预期出现,我们旨在评估复发和难治性MM患者的生存率。

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