Multicenter prospective, randomized, double-masked, placebo-controlled study of Rheopheresis to treat nonexudative age-related macular degeneration: interim analysis.

OBJECTIVE

To evaluate the safety and efficacy of Rheopheresis blood filtration to treat intermediate- to late-stage preangiogenic age-related macular degeneration (AMD) with soft drusen.

DESIGN

Multicenter, prospective, randomized, double-masked, placebo-controlled clinical trial.

PARTICIPANTS

First 43 randomized patients (28 Rheopheresis and 15 placebo-control patients) with available baseline and 3-month postbaseline best corrected visual acuity (BCVA) measurements and intermediate- to late-stage preangiogenic AMD with multiple large soft drusen and elevated serum levels of targeted macromolecules.

INTERVENTION

Patients were randomly assigned to receive eight Rheopheresis or eight placebo procedures over 10 weeks.

MAIN OUTCOME MEASURES

ETDRS BCVA measurements at baseline, 3, 6, 9, and 12 months postbaseline.

RESULTS

In primary eyes, the mean LogMAR line difference between Rheopheresis and placebo-control eyes was 1.6 lines at 12 months postbaseline; the difference was significant throughout the first posttreatment year (P = .0011, repeated measures analysis). Thirteen percent of Rheopheresis compared with 0% of placebo-control eyes had a > or = 3-line improvement in BCVA at 12 months postbaseline. Four percent of Rheopheresis compared with 18% of placebo-control eyes had a > or = 3-line loss in BCVA. The subgroup of patients whose primary eyes had baseline BCVA worse than 20/40 demonstrated a mean LogMAR difference between Rheopheresis and placebo-control eyes equaling 3.0 lines at 12 months postbaseline; the difference was significant throughout the first posttreatment year (P = .0014, repeated measures analysis). Sixteen percent of Rheopheresis compared with 0% of the placebo-control eyes had a > or = 3-line improvement in BCVA at 12 months postbaseline. Five percent of Rheopheresis compared with 29% of placebo-control eyes had a > or = 3-line loss in BCVA. Fifty-eight percent of Rheopheresis eyes improved to 20/40 or better, compared with 14% of placebo-control eyes. No serious treatment-related adverse events were observed.

CONCLUSIONS

Rheopheresis demonstrated statistically significant and clinically relevant effects on BCVA when compared with placebo controls for the 12-month study interval. Untreated patients with BCVA worse than 20/40 with intermediate- to late-stage preangiogenic AMD, soft drusen, and elevated blood factors were at risk for substantial visual loss. A sample size larger than 43 patients is important to provide a basis for widespread adoption of novel therapeutic options for AMD such as Rheopheresis. Therefore, enrollment to 150 patients is continuing.