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镰状细胞病非清髓性造血细胞移植后的肾功能

Kidney function after nonmyeloablative hematopoietic cell transplant for sickle cell disease.

作者信息

Limerick Emily, Hsieh Matthew M, Barretto Mauricio, Jeffries Neal, Diamantidis Clarissa, Lokhnygina Yuliya, Menon Ritika, Saraf Santosh L, Fitzhugh Courtney D

机构信息

National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.

Department of Medicine, Department of Nephrology, Wake Forest University School of Medicine, Winston-Salem, NC, USA.

出版信息

Bone Marrow Transplant. 2025 May;60(5):690-696. doi: 10.1038/s41409-025-02550-0. Epub 2025 Mar 22.

Abstract

Hematopoietic cell transplantation (HCT) is potentially curative for patients with sickle cell disease (SCD). Both SCD and HCT cause kidney damage. This study analyzed data from 160 patients who received nonmyelablative HCT for SCD. Renal function was assessed at baseline and annually for 3years. The rate of new-onset eGFR <60 ml/min/1.73m was low (2.8%). Rapid kidney function decline in the first year post-HCT was noted in 7.5% of patients but was not associated with subsequently worse renal function. The eGFR decreased post-HCT (1 year: -7.19 p < 0.0001, 2 year: -11.32 p < 0.0001, 3 year: -12.37 ml/min/1.73m p < 0.0001). Mean eGFR remained within normal limits throughout the follow-up period (1 year:119, 2 year:115, 3 year:113 ml/min/1.73m). Hyperfiltration rates decreased with a corresponding increase in patients with normal eGFR post-HCT. Therefore, the decline in eGFR after HCT may represent preservation of renal function. The prevalence of kidney damage increased transiently but, by 3 years post-HCT, was not significantly changed from baseline. Most cases of kidney damage were due to albuminuria. AKI, noted early post-HCT in 39% of patients, was most commonly stage 1 and was associated with decreased survival (p = 0.03). Larger studies with longer follow-up are required to explore the effects of HCT on renal function in patients with SCD.

摘要

造血细胞移植(HCT)对镰状细胞病(SCD)患者有潜在的治愈作用。SCD和HCT都会导致肾脏损伤。本研究分析了160例接受非清髓性HCT治疗SCD患者的数据。在基线时以及之后3年每年评估肾功能。新发估算肾小球滤过率(eGFR)<60ml/min/1.73m²的发生率较低(2.8%)。7.5%的患者在HCT后第一年出现肾功能快速下降,但这与随后较差的肾功能无关。HCT后eGFR下降(1年:-7.19,p<0.0001;2年:-11.32,p<0.0001;3年:-12.37ml/min/1.73m²,p<0.0001)。在整个随访期间,平均eGFR保持在正常范围内(1年:119,2年:115,3年:113ml/min/1.73m²)。HCT后高滤过率降低,同时eGFR正常的患者相应增加。因此,HCT后eGFR的下降可能代表肾功能的保存。肾脏损伤的患病率短暂增加,但在HCT后3年,与基线相比无显著变化。大多数肾脏损伤病例是由于蛋白尿。39%的患者在HCT后早期出现急性肾损伤(AKI),最常见的是1期,且与生存率降低相关(p=0.03)。需要进行更大规模、更长随访时间的研究来探讨HCT对SCD患者肾功能的影响。

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