Willenbacher Ella, Weger Roman, Rochau Ursula, Siebert Uwe, Willenbacher Wolfgang
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.
PLoS One. 2016 Mar 3;11(3):e0147381. doi: 10.1371/journal.pone.0147381. eCollection 2016.
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.
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).
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.
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。
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。
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%)。
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。