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Abstract: SA-PO706

A Prospective Study to Assess Safety and Efficacy of Bone-Marrow Derived Mesenchymal Stromal Cells for Severe Frequently Relapsing or Steroid-Dependent Idiopathic Nephrotic Syndrome: The MESNEPH Study

Session Information

Category: Glomerular Diseases

  • 1303 Glomerular Diseases: Clinical‚ Outcomes‚ and Trials

Authors

  • Vivarelli, Marina, Division of Nephrology, Bambino Gesù Pediatric Hospital IRCCS, Rome, RM, Italy
  • Colucci, Manuela, Division of Nephrology, Bambino Gesù Pediatric Hospital IRCCS, Rome, RM, Italy
  • Ruggenenti, Piero Luigi, Istituto di Ricerche Farmacologiche "Mario Negri" IRCCS, Bergamo, BG, Italy
  • Casiraghi, Federica, Istituto di Ricerche Farmacologiche "Mario Negri" IRCCS, Bergamo, BG, Italy
  • Zotta, Federica, Division of Nephrology, Bambino Gesù Pediatric Hospital IRCCS, Rome, RM, Italy
  • Emma, Francesco, Division of Nephrology, Bambino Gesù Pediatric Hospital IRCCS, Rome, RM, Italy
  • Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche "Mario Negri" IRCCS, Bergamo, BG, Italy
Background

An immune etiology of idiopathic nephrotic syndrome (INS) has been suggested by the efficacy of steroids and steroid-sparing agents, which present side effects.

Methods

This phase I study aimed to assess feasibility, safety and efficacy of autologous bone marrow-derived mesenchymal stromal cells (BM-MSC) in children and young adults (5-40 age years old) with steroid-dependent or multirelapsing INS. Following BM-MSC infusions, oral immunosuppression (IS) was gradually tapered. Safety, efficacy and immunomodulatory effects on different B and T cell subsets in vivo were monitored for 12 months.

Results

A total of 16 patients (10 children, 6 adults) were enrolled. A sufficient amount of autologous BM-MSC was obtained for all. Infusions were well tolerated. Adverse events were limited and not reconducible to BM-MSC infusion. At baseline, all patients were on steroids and most were on 1-2 steroid-sparing agents. At the end of the study, the number of required IS was significantly reduced in children (p=0.022) but not in adults. Whereas all patients relapsed during the 12-month follow-up, with a mean time of 8,5 months, the median number of relapses was significantly reduced in children (1 [IQR 1-1] vs [2 [IQR 2-3.25] compared to the previous 12 months, (p=<0.001) but not in adults.
Among B and T cell subsets, a significant but transient reduction of total CD19+, mature and switched memory B cells and an increase of transitional B cells and regulatory T cells (p<0.05) was observed in vivo between 1 and 3 months following BM-MSC infusion, despite the tapering of immunosuppression, especially in children.

Conclusion

Infusion of autologous BM-MSC was safe. A significant reduction of number of IS drugs and of relapses was found in children but not in young adults. The immunomodulatory effect of a cycle of autologous BM-MSC infusions on specific B and T cell subsets was transient.

Funding

  • Government Support – Non-U.S.