Stable renal function in children and adolescents with sickle cell disease after nonmyeloablative hematopoietic stem cell transplantation

• Published in: Pediatric blood & cancer Vol. 67; no. 9; pp. e28568 - n/a
• Main Authors: Pedersen, Sasia J. V., Monagel, Dania A., Mammen, Cherry, Lewis, Victor A., Guilcher, Gregory M. T., Bruce, Aisha A.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.09.2020
• Subjects: Adolescent; Anemia, Sickle Cell - pathology; Anemia, Sickle Cell - therapy; Child; Child, Preschool; Children; Epidermal growth factor receptors; Female; Follow-Up Studies; Glomerular Filtration Rate; Graft vs Host Disease - etiology; Graft vs Host Disease - pathology; Hematology; hematopoietic stem cell therapy; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic stem cells; Humans; Hypertension; Hypertension - etiology; Hypertension - pathology; Infant; Infant, Newborn; Kidney diseases; Kidney transplantation; Male; nonmyeloablative; Oncology; Patients; pediatric; Pediatrics; Prognosis; Proteinuria; Proteinuria - etiology; Proteinuria - pathology; Renal function; Renal Insufficiency, Chronic - etiology; Renal Insufficiency, Chronic - pathology; Retrospective Studies; Sickle cell disease; Stem cell transplantation; Stem cells; Transplantation Conditioning; Transplantation, Homologous; Transplants & implants; nonmyeloablative; hematopoietic stem cell therapy; pediatric; sickle cell disease
• ISSN: 1545-5009; 1545-5017
• DOI: 10.1002/pbc.28568

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.