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Kidney function after nonmyeloablative hematopoietic cell transplant for sickle cell disease

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/1.73m2 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/1.73m2 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/1.73m2). 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.

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Fig. 1: Schematic of patients included in baseline and 1, 2, and 3-year post-HCT analyses.
Fig. 2: Slope of eGFR decline post-transplant.
Fig. 3: Trend of eGFR post-HCT shown by mean values with 95% confidence intervals.
Fig. 4
Fig. 5: The proportion of patients at each time point with kidney damage defined by UACR ≥ 30 mg/dL and/or eGFR <60 mL/min/1.73 m2.
Fig. 6: A lower baseline eGFR is seen in patients who developed AKI and remains lower throughout the follow-up period.
Fig. 7: Kaplan Meier curve demonstrating differences in survival up to 10 years after HCT between patients with and without AKI.

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Data availability

The datasets analysed in this study are available from the corresponding author on reasonable request.

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Acknowledgements

Drs. Delbert Wigfall and Annabelle Chua of Duke Medical Center for contributing pediatric nephrology expertise.

Funding

This research was funded by the Cooperative Assessment of Late Effects for SCD Curative Therapies (COALESCE, 1U01HL156620-01, National Heart, Lung, and Blood Institute (NHLBI)) and was supported by the intramural research program of the NHLBI, National Institutes of Health.

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Contributions

EL, CD, YL, SS, and CF conceived and designed the analyses; EL, MH, and SS collected data; MB and RM contributed data; EL and NJ performed the analyses, EL and CF drafted the manuscript. All authors reviewed and approved the final manuscript.

Corresponding authors

Correspondence to Santosh L. Saraf or Courtney D. Fitzhugh.

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All methods were performed in accordance with the relevant guidelines and regulations. Approval has been obtained from the National Institutes of Health and University of Illinois Chicago institutional review boards. Informed consent was obtained from all participants.

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Limerick, E., Hsieh, M.M., Barretto, M. et al. Kidney function after nonmyeloablative hematopoietic cell transplant for sickle cell disease. Bone Marrow Transplant 60, 690–696 (2025). https://doi.org/10.1038/s41409-025-02550-0

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