title: Verteporfin photodynamic therapy for neovascular age-related macular degeneration: cohort study for the UK.
abstract: OBJECTIVE
  The verteporfin photodynamic therapy (VPDT) cohort study aimed to answer five questions: (a) is VPDT in the NHS provided as in randomised trials?; (b) is 'outcome' the same in the nhs as in randomised trials?; (c) is 'outcome' the same for patients ineligible for randomised trials?; (d) is VPDT safe when provided in the NHS?; and (e) how effective and cost-effective is VPDT?
  
  
  METHODS
  Treatment register.
  
  
  METHODS
  All hospitals providing VPDT in the NHS.
  
  
  METHODS
  All patients attending VPDT clinics.
  
  
  METHODS
  Infusion of verteporfin followed by infrared laser exposure is called VPDT, and is used to treat neovascular age-related macular degeneration (nAMD). The VPDT cohort study advised clinicians to follow patients every 3 months during treatment or active observation, retreating based on criteria used in the previous commercial 'TAP' (Treatment of Age-related macular degeneration with Photodynamic therapy) trials of VPDT.
  
  
  METHODS
  The primary outcome was logarithm of the minimum angle of resolution monocular best-corrected distance visual acuity (BCVA). Secondary outcomes were adverse reactions and events; morphological changes in treated nAMD (wet) lesions; and for a subset of patients, 6-monthly contrast sensitivity, generic and visual health-related quality of life (HRQoL) and resource use. Treated eyes were classified as eligible for the TAP trials (EFT), ineligible (IFT) or unclassifiable (UNC).
  
  
  RESULTS
  Forty-seven hospitals submitted data for 8323 treated eyes in 7748 patients; 4919 eyes in 4566 patients were treated more than 1 year before the last data submission or had completed treatment. Of 4043 eyes with nAMD in 4043 patients, 1227 were classified as EFT, 1187 as IFT and 1629 as UNC. HRQoL and resource use data were available for about 2000 patients. The mean number of treatments in years 1 and 2 was 2.3 and 0.4 respectively. About 50% of eyes completed treatment within 1 year. BCVA deterioration in year 1 did not differ between eligibility groups. EFT eyes lost 11.6 letters (95% confidence interval 10.1 to 13.0 letters) compared with 9.9 letters in VPDT-treated eyes in the TAP trials. EFT eyes had poorer BCVA at baseline than IFT and UNC eyes. Adverse reactions and events were reported for 1.4% of first visits - less frequently than those reported in the TAP trials. Associations between BCVA in the best-seeing eye with HRQoL and community health and social care resource use showed that the 11-letter difference in BCVA between VPDT and sham treatment in the TAP trials corresponded to differences in utility of 0.012 and health and social service costs of £60 and £92 in years 1 and 2, respectively. VPDT provided an incremental cost per quality-adjusted life-year (QALY) of £170,000 over 2 years.
  
  
  CONCLUSIONS
  VPDT was administered less frequently than in the TAP trials, with less than half of those treated followed up for > 1 year in routine clinical practice. Deterioration in BCVA over time in EFT eyes was similar to that in the TAP trials. The similar falls in BCVA after VPDT across the pre-defined TAP eligibility groups do not mean that the treatment is equally effective in these groups because deterioration in BCVA can be influenced by the parameters that determined group membership. Safety was no worse than in the TAP trials. The estimated cost per QALY was similar to the highest previous estimate. Although VPDT is no longer in use as monotherapy for neovascular AMD, its role as adjunctive treatment has not been fully explored. VPDT also has potential as monotherapy in the management of vascular malformations of the retina and choroid and with trials underway in neovascularisation due to myopia and polypoidal choroidopathy.
  
  
  BACKGROUND
  The National Institute for Health Research Health Technology Assessment programme.
authors.affiliation: London School of Hygiene and Tropical Medicine, London, UK.
authors.forename: B C
authors.initials: BC
authors.lastname: Reeves
authors.forename: S P
authors.initials: SP
authors.lastname: Harding
authors.forename: J
authors.initials: J
authors.lastname: Langham
authors.forename: R
authors.initials: R
authors.lastname: Grieve
authors.forename: K
authors.initials: K
authors.lastname: Tomlin
authors.forename: J
authors.initials: J
authors.lastname: Walker
authors.forename: C
authors.initials: C
authors.lastname: Guerriero
authors.forename: J
authors.initials: J
authors.lastname: Carpenter
authors.forename: W P
authors.initials: WP
authors.lastname: Patton
authors.forename: K A
authors.initials: KA
authors.lastname: Muldrew
authors.forename: T
authors.initials: T
authors.lastname: Peto
authors.forename: U
authors.initials: U
authors.lastname: Chakravarthy
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D015331:Cohort Studies; D003362:Cost-Benefit Analysis; D005260:Female; D006801:Humans; D008268:Macular Degeneration / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care; D017319:Photosensitizing Agents / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D011166:Porphyrins / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic; D012042:Registries; D015861:Retinal Neovascularization / Q000188:drug therapy; D013222:State Medicine; D006113:United Kingdom; D000077362:Verteporfin
chemical_list: D017319:Photosensitizing Agents; D011166:Porphyrins; D000077362:Verteporfin