title: [Treatment with ranibizumab for choroidal neovascularization secondary to a pseudoxanthoma elasticum: Results of the French observational study PiXEL].
abstract: BACKGROUND
  Pseudoxanthoma elasticum (PXE), a rare hereditary connective tissue disorder, may be complicated by angioid streaks (AS) and choroidal neovascularization (CNV), which may lead to irreversible loss of visual acuity (VA). Here we describe the safety and efficacy of ranibizumab in patients with CNV secondary to PXE.
  
  
  METHODS
  A multicenter (n=23), observational study of a retrospective/prospective cohort, performed under real world conditions in France in all patients with CNV secondary to PXE who received at least one ranibizumab injection as of October 2011. The study objectives were to describe the mean annual number and reason for ranibizumab injections since initiation, evolution of best-corrected visual acuity (BCVA by Early Treatment Diabetic Retinopathy Study [ETDRS] letters), and safety.
  
  
  RESULTS
  Patients (n=72; 98 eyes) had a mean age of 59.6±8.3years and consisted of 54.2% men. The criterion for retreatment was based mainly on loss of VA, progression of CNV and angiographic leakage. CNV was primarily subfoveal or juxtafoveal (73.4%), and the initial mean VA was 64.6 ETDRS letters. On average, visual acuity of all eyes analyzed was relatively stable during the 2-year follow-up (62.3 letters vs 64.6 letters at the first injection), and 88.6% of eyes maintained VA between -15 and +15 letters or gained over 15 letters. No deaths or new intolerances were described.
  
  
  CONCLUSIONS
  These results showed that ranibizumab was able to maintain stable VA in clinical practice for at least 2years in patients with CNV secondary to PXE, and to significantly reduce the frequency of neovascularization relapses, with a limited number of injections. The treatment was well tolerated by the patients.
authors: University Hospital of Angers, 4, rue Larrey, 49100 Angers, France. Electronic address: jean-marc.ebran@hotmail.fr.
authors: J-M
authors: JM
authors: Ebran
authors: École Militaire Ophthalmic Center, 75000 Paris, France.
authors: G
authors: G
authors: Mimoun
authors: Ophthalmic Center of Laser and Medical Imaging, 75000 Paris, France.
authors: S-Y
authors: SY
authors: Cohen
authors: Ophthalmic Center of Laser and Medical Imaging, 75000 Paris, France.
authors: T
authors: T
authors: Grenet
authors: Ophthalmic Center, 2, rue Fréteau-de-Peny, 77011 Melun cedex, France.
authors: A
authors: A
authors: Donati
authors: University Hospital of Sainte-Marguerite, 270, boulevard de Sainte-Marguerite, 13009 Marseille, France.
authors: M-J
authors: MJ
authors: Jean-Pastor
authors: Novartis Pharma France, 2, rue Lionel-Terray, 92500 Rueil-Malmaison, France.
authors: A
authors: A
authors: Ponthieux
authors: Novartis Pharma France, 2, rue Lionel-Terray, 92500 Rueil-Malmaison, France.
authors: C
authors: C
authors: Bouchet
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020256:Choroidal Neovascularization / Q000188:drug therapy* / Q000209:etiology; D015331:Cohort Studies; D005260:Female; D005500:Follow-Up Studies; D005602:France; D006801:Humans; D008297:Male; D008875:Middle Aged; D011561:Pseudoxanthoma Elasticum / Q000150:complications / Q000188:drug therapy*; D000069579:Ranibizumab / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000069579:Ranibizumab
keywords: Best-corrected visual acuity; Choroidal neovascularization; Meilleure acuité visuelle corrigée; Néovascularisation choroïdienne; Pseudoxanthoma elasticum; Pseudoxanthome élastique; Ranibizumab; Stabilisation; Stabilization