Monthly Lucentis - Safe and Effective for PXE?

NOVEMBER 18, 2011
by Christine Vocke, Director of Education & Information, PXE International

This article first appeared in the Fall 2011 PXE MemberGram

Monthly Ranibizumab for Choroidal Neovascularizations Secondary to Angioid Streaks in Pseudoxanthoma Elasticum: A One-Year Prospective Study, Finger RP, Charbel Issa P, Hendig D, Scholl HP, Holz FG., Am J Ophthalmol. 2011 Oct;152(4):695-703.

In October 2011 the American Journal of Ophthalmology published the results of the first prospective study of the use of Ranibizumab (Lucentis®) in the treatment of CNV (choroidal neovascularization) in pseudoxanthoma elasticum (PXE). A team of researchers from Germany designed the 12-month study to evaluate the efficacy and safety of monthly injections of Lucentis, specifically in patients with active CNV due to PXE.

Prior to this study, several retrospective case studies reported on the use of bevacizumab (Avastin®) to treat CNV in patients with pseudoxanthoma elasticum. These studies found that Avastin was effective in maintaining visual function, and that long-term use of up to two years had favorable outcomes. See:

The prospective study reported here was a 12-month, open-label, uncontrolled, non- randomized study of the use of Lucentis in patients with PXE and active CNV. The researchers followed seven patients with PXE, and each patient received 12 injections of Lucentis, one per month. The researchers measured a number of visual functions at each monthly visit - distance and reading visual acuity (VA), reading speed, angiographic findings, and central retinal thickness (CRT). In addition, central retinal light increment sensitivity (LIS) was measured at baseline, at six months, and three to four months after the last visit.

The results showed that patients’ best corrected VA increased significantly from baseline to the final visit (20/62 to 20/32 or 61 to 72 ETDRS letters) and that the effect was maintained three months later. Reading acuity and speed was maintained throughout the study. Central LIS improved, leakage from active CNVs subsided, and intraretinal and subretinal fluid largely disappeared. Only small areas of atrophy of the retinal pigment epithelium (RPE appeared during the follow-up period. CRT decreased from the baseline to month 12 and 15, and no serious adverse events occurred. In particular, there were no thromboembolic or cardiovascular adverse events during the study and follow-up period.

This is the first study to investigate reading ability in patients with pseudoxanthoma elasticum treated with Lucentis or Avastin. The authors suggest that because reading ability is an important part of quality of life in individuals with macular disease, therapeutic interventions in PXE should aim to maintain quality of life, including preserving reading ability.

The authors also suggest that because PXEers have an increased cardiovascular risk profile, the possible complications of treatments with anti-VEGF agents, such as thromboembolic events, need to be considered and discussed. None of these events occurred in this study, but the authors suggest that treatment on an as-needed (PRN) basis with close followup may be a reasonable alternative to monthly anti-VEGF injections.

This study concludes that Lucentis appears to be a safe and effective treatment for people with pseudoxanthoma elastsicum and active CNV, and has a beneficial therapeutic effect on central visual functions.

Anti-VEGF (vascular endothelial growth factor) agent – A chemical or drug designed to inhibit a growth factor that operates on blood vessels.

Non-randomized – Relating to a clinical trial in which the subjects are not randomly distributed into groups which are either subjected to the experimental procedure (as use of a drug) or which serve as controls.

Open label – Relating to a clinical trial in which both the researchers and the patients know who receives the drug and who receives a placebo.

Prospective study – A study that starts with the present condition of a population of individuals and follows them into the future.

Retrospective study – A study that starts with the present condition of a population of individuals and collects data about their past history to explain their present condition.

Thromboembolic event – An event, such as a stroke, associated with the blocking of a blood vessel by a particle that has broken away from a blood clot at its site of formation.

Uncontrolled – Relating to a clinical trial in which NO subjects serve as controls – in other words, all subjects receive the treatment.