Cornerstone Research Group Inc., Burlington, Ontario, Canada.
Curr Med Res Opin. 2011 Feb;27(2):383-91. doi: 10.1185/03007995.2010.543125. Epub 2010 Dec 23.
Approximately 10-20% of multiple myeloma patients experience dialysis-dependent renal failure. This is principally due to myeloma kidney, a tubulointerstitial injury caused by high circulating concentrations of monoclonal free light chains. Studies have found that between 3% and 37% of patients with myeloma kidney requiring dialysis recover renal function. In-vivo studies indicate that extended haemodialysis using high cut-off dialysers (HCO-HD) can remove significant quantities of free light chains and is associated with a renal recovery rate of 63-74% in these patients.
The objective of this study was to assess the cost-effectiveness of HCO-HD compared to standard HD in the management of myeloma kidney.
The study used a lifetime Excel-based decision tree model that followed all patients from treatment of the initial presentation with myeloma kidney requiring dialysis to death. It was populated with published clinical data, United Kingdom costs and expert opinion, using a National Health Service perspective and 3.5% annual discounting.
HCO-HD was dominant to standard HD, meaning it was both more effective (greater life years and quality adjusted life years) and less costly, due to a greater increase in the proportion of patients recovering renal function. The model projected lifetime costs of £31,345 per patient for patients treated with standard haemodialysis only and £24,845 for the new treatment (discounted). The model predicted an average survival of 19.92 months for patients on standard HD and 33.90 months for the new therapy (discounted).
The analysis found that treatment of myeloma kidney using an extended schedule of HCO-HD may substantially improve renal recovery in multiple myeloma patients compared to standard HD, resulting in greater life expectancy and cost savings due to avoided chronic dialysis. Limitations of the study include those common to rare diseases including small study sizes and limited natural history data.
约 10-20%的多发性骨髓瘤患者出现依赖透析的肾衰竭。这主要是由于骨髓瘤肾，一种由高循环浓度的单克隆游离轻链引起的肾小管间质性损伤。研究发现，3%-37%的需要透析的骨髓瘤肾患者肾功能恢复。体内研究表明，使用高通量透析器（HCO-HD）进行延长血液透析可以去除大量游离轻链，与这些患者 63-74%的肾脏恢复率相关。
本研究旨在评估与标准血液透析相比，HCO-HD 在骨髓瘤肾治疗中的成本效益。
该研究使用了基于 Excel 的终生决策树模型，该模型从所有初始多发性骨髓瘤肾需要透析治疗的患者开始，一直跟踪到死亡。它使用了已发表的临床数据、英国成本和专家意见，采用了国民保健制度的视角和 3.5%的年度贴现率。
HCO-HD 优于标准 HD，这意味着它不仅更有效（增加更多的患者恢复肾功能），而且成本更低，因为恢复肾功能的患者比例更高。该模型预测，仅接受标准血液透析治疗的患者终生费用为 31345 英镑/人，而新治疗方案（折扣后）为 24845 英镑/人。该模型预测，接受标准血液透析的患者平均生存时间为 19.92 个月，而接受新治疗方案的患者为 33.90 个月（折扣后）。
该分析发现，与标准 HD 相比，使用 HCO-HD 延长治疗方案治疗骨髓瘤肾可能会显著提高多发性骨髓瘤患者的肾功能恢复，从而延长预期寿命并节省因避免慢性透析而产生的成本。该研究的局限性包括常见于罕见疾病的局限性，如研究规模小和有限的自然病史数据。
