AOP 2022
Program

Disclosures

Did you receive any funding to support your research for this TOPIC?
No

Were you provided with any honoraria, payment or other compensation for your work on this study?
No

Do you have any financial relationship with any entity which may closely compete with the medications, materials or instruments covered by your study?
No

Do you own or have you applied for any patents in conjunction with the instruments, medications or materials discussed in your study?
No

This work was not supported by any direct or non direct funding. It is under the author's own responsability

Objectives: To summarize the current research in geographic atrophy (GA) due to non-exudative age-related macular degeneration.

Introduction: To date, there is no FDA-approved treatment that has been shown to effectively prevent, slow, or stop GA progression. While the exact pathophysiology is not clear, Complement has been most implicated in the disease process. Natural history studies are available and they either measure global GA growth (i.e., disregarding the directionality of GA growth) or those that correlate measures of local GA growth (i.e., considering the directionality of GA growth). Pilot therapies include pharmacotherapy, laser photostimulation, cell-based regeneration and gene therapy.

Materials / method: Literature review

Conclusion: Ongoing research aims to elucidate pathophysiology of GA to help with development of treatment targets. Clinical trials are investigating various approach to treatment including intravitreal pharmacotherapy, cellular regenerative and gene therapy.

Disclosures

Did you receive any funding to support your research for this TOPIC?
No

Were you provided with any honoraria, payment or other compensation for your work on this study?
No

Do you have any financial relationship with any entity which may closely compete with the medications, materials or instruments covered by your study?
No

Do you own or have you applied for any patents in conjunction with the instruments, medications or materials discussed in your study?
No

This work was not supported by any direct or non direct funding. It is under the author's own responsability

Objectives: to report Arabian Gulf area experience with Voretigene neparvovec (VN) for RPE65-related retinal dystrophy (RD65).

Introduction: Voretigene neparvovec has been approved for the treatment of RPE65-related retinal dystrophy. The results of the pivotal phase 3 clinical trial documented the positive efficacy of subretinal delivery on functional vision. The outcomes were maintained over 4 years following therapy which was generally safe and well tolerated.

Materials / method: Consecutive case series (6 Emirati RD65 patients, 12 eyes). Five were treated at Cleveland Clinic Abu Dhabi and 1 abroad despite local availability. Main outcome measures included real world evidence of improved function; full-field stimulus testing (FST) and Snellen visual acuity (VA) measurements; and adverse events.

Results: We treated 5 patients (10 eyes) to date. All had real world evidence of improved function. VA (HM-20/200) was stable in 4 patients and improved in 1 and FST threshold improved in 2 patients (-4 and -7 dB at baseline to -16 and -17 dB both eyes at last test). Over 1-14months of follow up, 1 eye developed medically controllable transient intraocular pressure elevation. The 6th patient, treated abroad bilaterally, presented after having noted loss of peripheral field. She had monocular advanced glaucoma refractory to medical therapy, necessitating glaucoma surgery in that eye.

Conclusion: Our experience confirms VN’s efficacy in the Emirati population and highlights the importance of proper follow-up.

Disclosures

Did you receive any funding to support your research for this TOPIC?
No

Were you provided with any honoraria, payment or other compensation for your work on this study?
No

Do you have any financial relationship with any entity which may closely compete with the medications, materials or instruments covered by your study?
No

Do you own or have you applied for any patents in conjunction with the instruments, medications or materials discussed in your study?
No

This work was not supported by any direct or non direct funding. It is under the author's own responsability