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Cord blood-derived mesenchymal stromal cells in children with steroid-dependent nephrotic syndrome: a prospective
William Morello1, Elisa Montelatici2, Cristiana Lavazza2
1Pediatric Nephrology, Dialysis and Transplant Unit, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milano, Italy.
Insights
Cord-blood-derived mesenchymal stromal cells (CB-MSCs) were tested in children with steroid-dependent nephrotic syndrome (SDNS). This novel therapy was safe but did not improve relapse-free survival after immunosuppressive treatment withdrawal.
Area of Science:
- Pediatric Nephrology
- Immunology
- Regenerative Medicine
Background:
- Steroid-dependent nephrotic syndrome (SDNS) in children necessitates long-term immunosuppressive (IS) therapy, impacting quality of life.
- Cord-blood-derived mesenchymal stromal cells (CB-MSCs) possess immunomodulatory properties and were investigated as a potential alternative treatment.
Purpose of the Study:
- To evaluate the efficacy of CB-MSCs in achieving sustained remission in children with SDNS after IS withdrawal.
- To assess the safety and relapse-free survival rates associated with CB-MSC therapy.
Main Methods:
- An adaptive, open-label, single-arm, phase II trial was conducted in children (3-18 years) with SDNS.
- Part 1 involved three CB-MSC infusions with IS tapering; Part 2 modified IS withdrawal timing and increased CB-MSC dose/infusions.
- A historical cohort served as a post-hoc control group.
Main Results:
- Part 1 failed to meet the primary endpoint, with 7/9 patients relapsing.
- Part 2 showed initial remission in 9/11 patients, but 6/11 relapsed within 6 months.
- No significant difference in relapse-free survival was observed between Part 1 and Part 2 (P = .25), and historical controls had better outcomes (P = .0007).
Conclusions:
- CB-MSC therapy is safe in children with SDNS.
- The investigated CB-MSC regimen did not improve relapse-free survival at 6 months post-IS withdrawal.
- Further research may be needed to explore alternative dosing or patient selection for CB-MSC therapy in SDNS.
Background:
Children with steroid-dependent nephrotic syndrome (SDNS) require long-term immunosuppressive (IS) treatment, significantly impacting their quality of life. Cord-blood-derived mesenchymal stromal cells (CB-MSCs) are multipotent stem cells with proven immunomodulatory properties.
Methods:
We conducted an adaptive, open-label, single-arm, phase II trial with an adaptive second part to evaluate the efficacy of CB-MSCs in children aged 3-18 years with SDNS in remission for ≥6 months on IS therapy. In Part 1, patients received three intravenous infusions of CB-MSCs (1.5 × 10⁶/kg) at 1-2-week intervals, with rapid IS tapering and discontinuation after the third infusion. The primary endpoint was relapse-free survival at 6 months post-IS withdrawal, with a threshold of <4/11 relapsing patients. In the adaptive Part 2, IS was discontinued before the first infusion to enhance CB-MSCs activity, and the dose was increased to 2 × 10⁶/kg, with a booster fourth infusion. A historical cohort of SDNS children tapering IS under standard care served as a post-hoc control.
Results:
In Part 1, 9 patients were enrolled, and 7/9 (78%) relapsed, failing to meet the prespecified efficacy threshold. In Part 2, 11 patients were enrolled; 9 (82%) were in remission at 2 weeks post-third infusion and received the boosted dose. However, 6/11 (55%) relapsed within 6 months, with no significant difference in relapse-free survival compared to Part 1 (P = .25). Historical controls had significantly better relapse-free survival (P = .0007).
Conclusion:
In children with SDNS, this novel therapy with CB-MSCs was safe but failed to improve relapse-free survival 6 months after IS withdrawal.
