Title |
Excellent long-term outcome of renal transplantation in cystinosis patients
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Published in |
Orphanet Journal of Rare Diseases, July 2015
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DOI | 10.1186/s13023-015-0307-9 |
Pubmed ID | |
Authors |
Camille Cohen, Marina Charbit, Bernadette Chadefaux-Vekemans, Magali Giral, Valérie Garrigue, Michèle Kessler, Corinne Antoine, Renaud Snanoudj, Patrick Niaudet, Henri Kreis, Christophe Legendre, Aude Servais |
Abstract |
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. 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. 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. 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. |
X Demographics
Geographical breakdown
Country | Count | As % |
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United States | 1 | 33% |
Italy | 1 | 33% |
Unknown | 1 | 33% |
Demographic breakdown
Type | Count | As % |
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Scientists | 2 | 67% |
Members of the public | 1 | 33% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 58 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 11 | 19% |
Researcher | 10 | 17% |
Other | 9 | 16% |
Student > Bachelor | 4 | 7% |
Professor > Associate Professor | 4 | 7% |
Other | 12 | 21% |
Unknown | 8 | 14% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 28 | 48% |
Biochemistry, Genetics and Molecular Biology | 5 | 9% |
Pharmacology, Toxicology and Pharmaceutical Science | 4 | 7% |
Psychology | 4 | 7% |
Agricultural and Biological Sciences | 3 | 5% |
Other | 5 | 9% |
Unknown | 9 | 16% |