Department of Pediatrics, Division of Immunology, Hematology/Oncology and Palliative Care, Stollery Children's Hospital, Edmonton, AB, Canada.
Departments of Oncology and Pediatrics, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada.
Pediatr Blood Cancer. 2020 Sep;67(9):e28568. doi: 10.1002/pbc.28568. Epub 2020 Jul 16.
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.
To describe baseline renal parameters as well as short- and long-term renal outcomes in pediatric patients with SCD who underwent NMA-HSCT.
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 m , (2) proteinuria, and (3) hypertension.
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 m 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%).
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.
镰状细胞病（SCD）从婴儿期开始就与肾脏并发症有关。使用新型非清髓性（NMA）预处理方案的同种异体造血干细胞移植（HSCT）治疗在儿科人群治疗 SCD 方面显示出良好的效果，但移植后的肾脏预后参数尚未描述。
描述接受 NMA-HSCT 的 SCD 儿科患者的基线肾脏参数以及短期和长期肾脏结果。
对加拿大艾伯塔省接受 NMA-HSCT 的儿科患者进行回顾性图表审查。评估的短期肾脏结果包括：（1）急性肾损伤（AKI），（2）液体超负荷（FO）和（3）高血压。长期结果评估包括：（1）估算肾小球滤过率（eGFR）的发展和最后一次随访时的高滤过定义为 eGFR≥150mL/min/1.73m，（2）蛋白尿和（3）高血压。
平均随访时间为 128.6 周（标准差为 69.3）。未观察到移植后 AKI 事件或 FO。所有患者的最后一次随访时 eGFR 仍>90mL/min/1.73m，而分别有 8 名（44.4%）和 4 名（22.2%）患者在 HSCT 前后存在高滤过，差异具有统计学意义（P<0.0001）。因此，HSCT 前的中位 GFR 明显高于 HSCT（P<0.009）。HSCT 后有 6 名患者（33.3%）出现长期高血压。
本研究描述了 NMA-HSCT 后 SCD 儿童稳定的肾功能，无 AKI 或 FO 发作的证据。HSCT 后高滤过率降低，这表明 NMA-HSCT 可能在该高危人群中潜在地保护长期肾功能免受进行性慢性肾脏病的影响。需要进一步的前瞻性研究来证实这些新发现。
