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Stable renal function in children and adolescents with sickle cell disease after nonmyeloablative hematopoietic stem
Sasia J V Pedersen1, Dania A Monagel2, Cherry Mammen3
1Department of Pediatrics, Division of Immunology, Hematology/Oncology and Palliative Care, Stollery Children's Hospital, Edmonton, AB, Canada.
Insights
Pediatric patients with sickle cell disease (SCD) showed stable kidney function after nonmyeloablative hematopoietic stem cell transplant (NMA-HSCT). This treatment may preserve renal function, reducing the risk of chronic kidney disease in children.
Area of Science:
- Nephrology
- Pediatric Hematology
- Transplantation Immunology
Background:
- Sickle cell disease (SCD) frequently causes renal complications from infancy.
- Allogeneic hematopoietic stem cell transplant (HSCT) with nonmyeloablative (NMA) conditioning is promising for pediatric SCD.
- Renal outcomes post-NMA-HSCT in pediatric SCD patients remain undescribed.
Purpose of the Study:
- To evaluate baseline renal parameters in pediatric SCD patients.
- To assess short- and long-term renal outcomes following NMA-HSCT.
- To determine the impact of NMA-HSCT on kidney function in pediatric SCD.
Main Methods:
- Retrospective chart review of pediatric SCD patients undergoing NMA-HSCT in Alberta, Canada.
- Short-term renal outcomes: acute kidney injury (AKI), fluid overload (FO), and hypertension.
- Long-term renal outcomes: estimated glomerular filtration rate (eGFR), hyperfiltration, proteinuria, and hypertension.
Main Results:
- No post-transplant AKI or FO observed; eGFR remained >90 mL/min/1.73 m².
- Hyperfiltration decreased significantly post-HSCT (44.4% pre- vs. 22.2% post-HSCT, P < 0.0001).
- Hypertension was present in 33.3% of patients long-term post-HSCT.
Conclusions:
- NMA-HSCT demonstrates stable kidney function in pediatric SCD patients without AKI or FO.
- Reduced hyperfiltration post-HSCT suggests potential for preserved long-term renal function.
- NMA-HSCT may be a protective strategy against progressive chronic kidney disease in pediatric SCD.
Background:
Sickle cell disease (SCD) is associated with renal complications starting as early as infancy. Allogeneic hematopoietic stem cell transplant (HSCT) treatments using newer nonmyeloablative (NMA) conditioning regimens show promising results in treating SCD in the pediatric population, but renal outcome parameters after transplantation have not been described.
Aim:
To describe baseline renal parameters as well as short- and long-term renal outcomes in pediatric patients with SCD who underwent NMA-HSCT.
Methods:
A retrospective chart review of pediatric patients who received NMA-HSCT in Alberta, Canada. Short-term renal outcomes evaluated were: (1) acute kidney injury (AKI), (2) fluid overload (FO), and (3) hypertension. Long-term outcomes evaluated were: (1) estimated glomerular filtration rate (eGFR) development and at last follow-up with hyperfiltration defined as eGFR ≥ 150 mL/min/1.73 m2 , (2) proteinuria, and (3) hypertension.
Results:
The mean follow-up time was 128.6 weeks (standard deviations, 69.3). No posttransplant AKI events or FO were observed. eGFR remained > 90 mL/min/1.73 m2 at last follow-up in all patients, whereas hyperfiltration was present in eight (44.4%) and four (22.2%) patients pre- and post-HSCT, respectively, which are significantly different (P < 0.0001). Consequently, median GFR was significantly higher pre-HSCT compared with 24 months HSCT (P < 0.009). Long-term hypertension post-HSCT was present in six patients (33.3%).
Conclusion:
This study describes stable kidney function in children with SCD after NMA-HSCT without evidence of AKI or FO episodes. Rates of hyperfiltration decreased post-HSCT, which signifies that NMA-HSCT could potentially preserve long-term renal function in this population at risk of progressive chronic kidney disease. Further prospective studies are needed to confirm these novel findings.
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