title: Excellent long-term outcome of renal transplantation in cystinosis patients.
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.
authors.affiliation: Service de Néphrologie-Transplantation, Hôpital Necker, 149 rue de Sèvres, 75015, Paris, France.|Université Sorbonne Paris Cité, Paris, France.
authors.forename: Camille
authors.initials: C
authors.lastname: Cohen
authors.affiliation: Université Sorbonne Paris Cité, Paris, France.|Service de Néphrologie pédiatrique, Hôpital Necker, APHP, Paris, France.
authors.forename: Marina
authors.initials: M
authors.lastname: Charbit
authors.affiliation: Université Sorbonne Paris Cité, Paris, France.|Laboratoire de biochimie B, Hôpital Necker, Paris, France.
authors.forename: Bernadette
authors.initials: B
authors.lastname: Chadefaux-Vekemans
authors.affiliation: Service de Néphrologie-Transplantation, CHU, Nantes, France.
authors.forename: Magali
authors.initials: M
authors.lastname: Giral
authors.affiliation: Service de Néphrologie-Transplantation, CHU, Montpellier, France.
authors.forename: Valérie
authors.initials: V
authors.lastname: Garrigue
authors.affiliation: Service de Néphrologie-Transplantation, CHU, Nancy, France.
authors.forename: Michèle
authors.initials: M
authors.lastname: Kessler
authors.affiliation: Service de Néphrologie-Transplantation, hôpital Saint Louis, APHP, Paris, France.
authors.forename: Corinne
authors.initials: C
authors.lastname: Antoine
authors.affiliation: Service de Néphrologie-Transplantation, Hôpital Necker, 149 rue de Sèvres, 75015, Paris, France.|Université Sorbonne Paris Cité, Paris, France.
authors.forename: Renaud
authors.initials: R
authors.lastname: Snanoudj
authors.affiliation: Université Sorbonne Paris Cité, Paris, France.|Service de Néphrologie pédiatrique, Hôpital Necker, APHP, Paris, France.
authors.forename: Patrick
authors.initials: P
authors.lastname: Niaudet
authors.affiliation: Service de Néphrologie-Transplantation, Hôpital Necker, 149 rue de Sèvres, 75015, Paris, France.|Université Sorbonne Paris Cité, Paris, France.
authors.forename: Henri
authors.initials: H
authors.lastname: Kreis
authors.affiliation: Service de Néphrologie-Transplantation, Hôpital Necker, 149 rue de Sèvres, 75015, Paris, France.|Université Sorbonne Paris Cité, Paris, France.
authors.forename: Christophe
authors.initials: C
authors.lastname: Legendre
authors.affiliation: Service de Néphrologie-Transplantation, Hôpital Necker, 149 rue de Sèvres, 75015, Paris, France. aude.servais@nck.aphp.fr.|Université Sorbonne Paris Cité, Paris, France. aude.servais@nck.aphp.fr.
authors.forename: Aude
authors.initials: A
authors.lastname: Servais
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D003554:Cystinosis / Q000150:complications* / Q000503:physiopathology; D006801:Humans; D007676:Kidney Failure, Chronic / Q000209:etiology / Q000601:surgery*; D016030:Kidney Transplantation; D016896:Treatment Outcome; D055815:Young Adult