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

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

Research output: Contribution to journalArticlepeer-review

13 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)2295-2302
Number of pages8
JournalNew England Journal of Medicine
Issue number24
StatePublished - Jun 16 2022

ASJC Scopus subject areas

  • Medicine(all)


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