Excellent Long-term Outcome of Renal Transplantation in Cystinosis Patients

Overview

Journal Orphanet J Rare Dis

Publisher Biomed Central

Specialty General Medicine

Date 2015 Jul 26

PMID 26208493

Citations 13

Authors

Camille Cohen

Marina Charbit

Bernadette Chadefaux-Vekemans

Magali Giral

Valerie Garrigue

Michele Kessler

Corinne Antoine

Renaud Snanoudj

Patrick Niaudet

Henri Kreis

Christophe Legendre

Aude Servais

Affiliations

Soon will be listed here.

Abstract

Background: Cystinosis is a rare lysosomal disorder leading to end stage renal disease in more than 90 % of patients before 20 years of age. Data about safety and efficiency of renal transplantation in patients with cystinosis is scarce. We evaluated long-term outcomes of renal transplantation in adult patients with cystinosis.

Methods: Data of renal transplantation (n = 31) in 30 adult patients with cystinosis in 5 French university transplant centers between 1980 and 2013 were retrospectively analyzed. A control cohort of 93 patients was matched for age, graft date, living/deceased donor status and transplant center.

Results: Median age at transplantation was 20.4 years (7-36.5). At transplantation, all patients with cystinosis had corneal cystine deposits, 3 had diabetes and 7 had hypothyroidism. Graft survival was better in patients with cystinosis than in control patients (p = 0.013). Multivariate analysis confirmed that cystinosis was an independent protective factor for graft survival (Hazard Ratio (HR) 0.11; CI95 [0.02-0.61]). Specific complications of cystinosis occurred during follow up: diabetes mellitus (n = 4), hypothyroidism (n = 1), liver involvement (n = 1), neurologic involvement (n = 2). Proportion of post-transplant diabetes mellitus (PTDM) was not statistically different in cystinosis group compared to control group: 4 (13.0 %) compared to 5 (5.0 %), respectively (p = 0.25), with no differences regarding calcineurin inhibitors and steroids treatments during follow-up.

Conclusions: Renal transplantation appears to be safe with excellent long-term outcomes in patients with cystinosis. These patients may receive standard immunosuppressive regimens with steroids and calcineurin inhibitors.

Citing Articles

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Monogenic Kidney Diseases in Kidney Transplantation.

Gillion V, Devresse A, Olinger E, Dahlqvist G, Demoulin N, Godefroid N Kidney Int Rep. 2024; 9(3):549-568.

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Newborn Screening: Review of its Impact for Cystinosis.

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Cystinosis beyond kidneys: gastrointestinal system and muscle involvement.

Topaloglu R, Gultekingil A, Gulhan B, Ozaltin F, Demir H, Ciftci T BMC Gastroenterol. 2020; 20(1):242.

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