自体造血干细胞移植治疗轻链沉积症后的长期疗效和肾脏反应:代表欧洲血液和骨髓移植学会慢性恶性肿瘤工作组进行的一项回顾性研究。

IF 8.2 1区 医学 Q1 HEMATOLOGY
Laurent Garderet, Luuk Gras, Linda Koster, Liesbeth De Wreede, Rovira Montserrat, Laure Vincent, Roland Fenk, Kamaraj Karunanithi, Dries Deeren, Martin Kaufmann, Jürgen Kuball, Hakan Ozdogu, Maria Jesus Pascual Cascon, Jakob Passweg, Adam Rye, Urpu Salmenniemi, John Snowden, Charlotte Toftmann Hansen, Xavier Leleu, Lauris Gastaud, Joanna Drozd Sokolowska, Kavita Raj, Meral Beksac, Stefan Schönland, Patrick Hayden, Donal McLornan
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引用次数: 0

摘要

关于自体造血干细胞移植(ASCT)对轻链沉积症(LCDD)疗效的长期结果数据很少。我们在EBMT登记处找到了51名LCDD患者,他们在1995年至2021年间接受了前期ASCT。血清肌酐中位数为 280 μmol/L,45% 的患者在移植时需要接受肾脏替代治疗 (RRT)。23%的患者美法仑剂量为100毫克/平方米,55%为140毫克/平方米,21%为200毫克/平方米。非常好的部分反应或更好的部分反应率从移植前的41%提高到移植后第100天的66%。在不依赖 RRT 的患者中,头 3 个月的肾功能略有改善;eGFR 中位数从 44 ml/min/1.73 m2 增加到 51 ml/min/1.73 m2。ASCT 术后 3 个月至 12 个月期间没有进一步变化。ASCT 时不依赖 RRT 的患者在 ASCT 后第 + 100 天时均不再依赖 RRT。ASCT 后的中位随访时间为 84 个月(IQR:46-122)。ASCT后6年的总生存率(OS)为88%(95% CI:78-98%),PFS为44%(95% CI:28-60%)。复发和非复发死亡率(NRM)的2年累计发生率分别为17%(95% CI:6-27%)和2%(95% CI:0-6%)。ASCT后4年的肾移植累计发生率为27%(95% CI:13-41),肾移植时间为ASCT后6.3至52.9个月(中位24.7个月)。ASCT是LCDD患者的可行选择,即使患者在移植时依赖RRT。结果良好,NRM 低,长期 OS 良好。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Long-term outcomes and renal responses following autologous hematopoietic stem cell transplantation for light chain deposition disease: a retrospective study on behalf of the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation.

There is little long-term outcome data on the efficacy of autologous hematopoietic stem cell transplantation (ASCT) in light chain deposition disease (LCDD). We identified 51 LCDD patients in the European Society for Blood and Bone Marrow transplantation registry who had undergone upfront ASCT between 1995 and 2021. The median serum creatinine was 280 μmol/L and 45% required renal replacement therapy (RRT) at time of transplant. The melphalan dose was 100 mg/m2 in 23%, 140 mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pretransplant to 66% at day +100 post- ASCT. In RRT-independent patients, there was a modest improvement in renal function within the first 3 months; the median estimated glomerular filtration rate increased from 44 to 51 mL/min/1.73 m2. There was no further change between 3 and 12 months post-ASCT. No patient who was RRT-independent at ASCT became RRT dependent by day + 100 post-ASCT. Median follow- up post-ASCT was 84 months (interquartile range [IQR]: 46-122). At 6-years post ASCT, overall survival was 88% (95% confidence interval [CI]: 78-98) and PFS was 44% (95% CI: 28-60). The 2-year cumulative incidence of relapse and non-relapse mortality was 17% (95% CI: 6-27) and 2% (95% CI: 0-6), respectively. The cumulative incidence of renal transplantation at 4 years after ASCT was 27% (95% CI: 13-41) with renal transplantation performed between 6.3 and 52.9 months post-ASCT (median 24.7 months). ASCT represents a feasible option for LCDD patients even if RRT dependent at time of transplant. Outcomes are favorable with low non-relapse mortality and good long-term overall survival.

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来源期刊
Haematologica
Haematologica 医学-血液学
CiteScore
14.10
自引率
2.00%
发文量
349
审稿时长
3-6 weeks
期刊介绍: Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research. Scope: The scope of the journal includes reporting novel research results that: Have a significant impact on understanding normal hematology or the development of hematological diseases. Are likely to bring important changes to the diagnosis or treatment of hematological diseases.
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