Sequential Stem Cell–Kidney Transplantation in Schimke Immuno-osseous Dysplasia
Alice Bertaina, Paul C. Grimm, Kenneth I. Weinberg, Robertson Parkman, Karen Kristovich, Giulia Barbarito, Elizabeth Lippner, Girija Dhamdhere, Vasavi Ramachandran, Jordan Spatz, Sahar Fathallah-Shaykh, Tracey Atkinson, Amira Al‐Uzri, Geraldine Aubert, Kim van Elst, Sean G. Green, Rajni Agarwal, Priscila Ferreira Slepicka, Ami J. Shah, Maria Grazia Roncarolo, Amy Gallo, Waldo Concepcion, David B. Lewis
Abstract
Lifelong immunosuppression is required for allograft survival after kidney transplantation but may not ultimately prevent allograft loss resulting from chronic rejection. We developed an approach that attempts to abrogate immune rejection and the need for post-transplantation immunosuppression in three patients with Schimke immuno-osseous dysplasia who had both T-cell immunodeficiency and renal failure. Each patient received sequential transplants of αβ T-cell-depleted and CD19 B-cell-depleted haploidentical hematopoietic stem cells and a kidney from the same donor. Full donor hematopoietic chimerism and functional ex vivo T-cell tolerance was achieved, and the patients continued to have normal renal function without immunosuppression at 22 to 34 months after kidney transplantation. (Funded by the Kruzn for a Kure Foundation.).