Sequential Stem Cell-Kidney Transplantation in Schimke Immuno-osseous Dysplasia
Bertaina, Alice; Grimm, Paul C; Weinberg, Kenneth; Parkman, Robertson; Kristovich, Karen M; Barbarito, Giulia; Lippner, Elizabeth; Dhamdhere, Girija; Ramachandran, Vasavi; Spatz, Jordan M; Fathallah-Shaykh, Sahar; Atkinson, T Prescott; Al-Uzri, Amira; Aubert, Geraldine; van der Elst, Kim; Green, Sean G; Agarwal, Rajni; Slepicka, Priscila F; Shah, Ami J; Roncarolo, Maria G; Gallo, Amy; Concepcion, Waldo; Lewis, David B
(2022) The New England journal of medicine, volume 386, issue 24, pp. 2295 - 2302
(Article)
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
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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.
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Keywords: Arteriosclerosis/genetics, Graft Rejection/prevention & control, Hematopoietic Stem Cell Transplantation, Humans, Immunologic Deficiency Syndromes/therapy, Kidney Transplantation/adverse effects, Kidney/physiology, Nephrotic Syndrome/genetics, Osteochondrodysplasias/genetics, Primary Immunodeficiency Diseases/genetics, Pulmonary Embolism/genetics, Transplantation Conditioning/methods, Journal Article
ISSN: 0028-4793
Publisher: Massachussetts Medical Society
Note: Funding Information: Supported by the Kruzn for a Kure Foundation (to Drs. Ber-taina and Lewis). Disclosure forms provided by the authors are available with the full text of this article at NEJM.org. We thank Dr. Cornelius Boerkoel III (Sanford USD Medical Center and Hospital, Sioux Falls, SD) for useful discussions. Publisher Copyright: Copyright © 2022 Massachusetts Medical Society.
(Peer reviewed)