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Sequential Stem Cell–Kidney Transplantation in Schimke Immuno-osseous Dysplasia

List of authors.
  • Alice Bertaina, M.D., Ph.D.,
  • Paul C. Grimm, M.D.,
  • Kenneth Weinberg, M.D.,
  • Robertson Parkman, M.D.,
  • Karen M. Kristovich, P.N.P.,
  • Giulia Barbarito, M.S.,
  • Elizabeth Lippner, M.D.,
  • Girija Dhamdhere, Ph.D.,
  • Vasavi Ramachandran, M.S.,
  • Jordan M. Spatz, M.D., Ph.D.,
  • Sahar Fathallah-Shaykh, M.D.,
  • T. Prescott Atkinson, M.D., Ph.D.,
  • Amira Al-Uzri, M.D.,
  • Geraldine Aubert, Ph.D.,
  • Kim van der Elst, Ph.D.,
  • Sean G. Green, Pharm.D.,
  • Rajni Agarwal, M.D.,
  • Priscila F. Slepicka, Ph.D.,
  • Ami J. Shah, M.D.,
  • Maria G. Roncarolo, M.D., Ph.D.,
  • Amy Gallo, M.D.,
  • Waldo Concepcion, M.D.,
  • and David B. Lewis, M.D.

Summary

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.)

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Funding and Disclosures

Supported by the Kruzn for a Kure Foundation (to Drs. Bertaina and Lewis).

Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.

This article was updated on June 16, 2022, at NEJM.org.

We thank Dr. Cornelius Boerkoel III (Sanford USD Medical Center and Hospital, Sioux Falls, SD) for useful discussions.

Author Affiliations

From the Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine (A.B., K.W., R.P., K.M.K., G.B., R.A., P.F.S., A.J.S., M.G.R.), the Center for Definitive and Curative Medicine (A.B., K.W., R.P., K.M.K., G.B., R.A., P.F.S., A.J.S., M.G.R.), and the Divisions of Nephrology (P.C.G., W.C.) and Allergy, Immunology, and Rheumatology (E.L., G.D., V.R., J.M.S., D.B.L.), Department of Pediatrics, and the Departments of Surgery (A.G., W.C.) and Pediatrics (W.C.), Stanford University School of Medicine, and Department of Pharmacy (S.G.G.), Stanford Children’s Health — both in Stanford, CA; the Divisions of Pediatric Nephrology (S.F.-S.) and Pediatric Allergy and Immunology (T.P.A.), Department of Pediatrics, University of Alabama, Birmingham; the Division of Nephrology, Department of Pediatrics, Oregon Health Sciences University, Portland (A. A.-U.); the Terry Fox Laboratory, BC Cancer Agency, Vancouver, Canada (G.A.); and the Department of Clinical Pharmacy, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (K.E.).

Dr. Bertaina can be contacted at or at the Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University, 1000 Welch Rd., Suite 301, Stanford, CA 94304.