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

Bertaina, Alice; Grimm, Paul; Weinberg, Kenneth I.; Parkman, Robertson; Kristovich, Karen; Barbarito, Giulia; Lippner, Elizabeth; Dhamdhere, Girija; Ramachandran, Vasavi; Spatz, Jordan; Fathallah-Shaykh, Sahar; Atkinson, Tracey; Al‐Uzri, Amira; Aubert, Geraldine; Elst, Kim van; Green, Sean G; Agarwal, Rajni; Slepicka, Priscila Ferreira; Shah, Ami J.; Roncarolo, Maria Grazia; Gallo, Amy; Concepcion, Waldo; Lewis, David B.

doi:10.1056/nejmoa2117028

Cited by 17 publications

(12 citation statements)

References 24 publications

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“…The patient in Case 3 patient is planning to undergo peripheral blood stem cell transplantation while kidney function is maintained since bone marrow transplantation may be an effective therapy for SIOD 17 . It has recently been reported that KT followed by HSCT has been successful in three patients 11 . If HSCT in Case 3 successfully maintains kidney and bone marrow functions, HSCT followed by KT may also be a treatment option.…”

Section: Discussionmentioning

confidence: 99%

“…17 It has recently been reported that KT followed by HSCT has been successful in three patients. 11 If HSCT in Case 3 successfully maintains kidney and bone marrow functions, HSCT followed by KT may also be a treatment option. It is equally as important as in the previous report to determine whether HSCT after KT is effective since bone marrow failure may appear after the KT.…”

Section: Discussionmentioning

confidence: 99%

“…To date, only a few cases of SIOD have been reported of having a successful course after KT, resulting in good kidney function, but with other extra‐renal complications. A recent seminal study reported successful KT after bone marrow transplantation 11 . However, since the post‐KT outcome of SIOD remains unelucidated, we report our experience with SIOD patients who underwent KT.…”

Section: Introductionmentioning

confidence: 96%

“…A recent seminal study reported successful KT after bone marrow transplantation. 11 However, since the post-KT outcome of SIOD remains unelucidated, we report our experience with SIOD patients who underwent KT.…”

Section: Introductionmentioning

confidence: 99%

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Clinical course of post‐kidney transplant Schimke immuno‐osseous dysplasia

Woo,

Kim,

Ahn

et al. 2023

Pediatric Transplantation

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BackgroundSchimke immuno‐osseous dysplasia (SIOD) is a rare systemic disease characterized by short stature, proteinuria, and recurrent infections. Patients usually have spondyloepiphyseal dysplasia, and progressive steroid‐resistant nephropathy that leads to kidney failure. However, their clinical course after kidney transplantation (KT) is not yet well known. Here, we present our experience with cases of SIOD treated at our institute.Case PresentationSince 2014, three children have been diagnosed with nephropathy resulting from SIOD. They presented with proteinuria in the nephrotic range at 7, 5, and 3 years of age. Focal segmental glomerulosclerosis was confirmed and progressed to kidney failure approximately 2 years after proteinuria was detected. These patients underwent living‐donor KT from their parents. After KT, Case 1 lost his graft within 7 months due to multi‐organ failure caused by disseminated adenovirus infection and died. Case 2 experienced graft failure 5 years after KT due to acute rejection from poor compliance. In Case 3, the allograft was still functioning 6 years after KT with low‐dose tacrolimus single medication (trough level < 5 ng/mL). Extra‐renal manifestations progressed regardless of KT, namely, right renal vein thrombosis and pulmonary hypertension in Case 1, severe bilateral hip dysplasia and Moyamoya syndrome in Case 2, and neutropenia and thrombocytopenia in Case 3, in addition to recurrent infection.ConclusionIn SIOD patients, KT is complicated with recurrent infections due to their inherent immune dysfunction. Additionally, extra‐renal symptoms may render the patients morbid despite the recovery of kidney function.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 96%

Section: Introductionmentioning

confidence: 99%

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Clinical course of post‐kidney transplant Schimke immuno‐osseous dysplasia

Woo,

Kim,

Ahn

et al. 2023

Pediatric Transplantation

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“…Similarly, the Northwestern group demonstrated complete withdrawal of immunosuppression in 5 out of 10 patients for 16-36 months [22]. These efforts have since expanded to additional patients with slightly modified regimens by these groups and others, including a recent study showcasing efficacy of a modified regimen in patients with Schimke Immuno-osseous Dysplasia (SIOD) [23].…”

Section: Introductionmentioning

confidence: 99%

Antibody-Based Preparative Regimens for Cell, Tissue and Organ Transplantation

Hentenryck,

Li,

Murphy

et al. 2021

OBM Transplant

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The ability to successfully transplant cells and organs from a donor into an immunologically disparate recipient is one of the greatest treatment advances in the history of medicine. Nevertheless, acute and chronic rejection, graft versus host disease, and the inability to identify suitable donors continue to be challenges and limit broader application of cell and organ transplantation to the many patients that could benefit. Immunosuppression before and after allogeneic transplant has been found to dramatically improve allograft survival and, despite side effects, has been a mainstay of patient management. Inducing donor-specific tolerance is the holy grail in allotransplantation and is readily established in experimental animals but has been difficult to achieve in patients in settings apart from hematopoietic cell transplantation. Antibody-based conditioning to prepare the recipient is a promising approach towards achieving transplant tolerance in both hematopoietic and solid organ transplant settings, and multiple targets are currently under consideration including those on circulating lymphocytes and hematopoietic stem cells. Here we review progress in the use of antibodies to support cell and tissue transplantation with a particular focus on induction of donor-specific tolerance for solid organ transplantation.

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