Is Ozurdex (dexamethasone) a viable treatment option for a patient with Coats' Disease and retinal neovascularization who has not responded to previous treatments with Avastin (bevacizumab), Eylea (aflibercept), and Vabysmo (faricimab)?

Ozurdex Authorization for Coats' Disease: Not Recommended

Ozurdex (dexamethasone intravitreal implant) should not be authorized for this patient with Coats' Disease, as it lacks FDA approval for this indication and has no high-quality evidence supporting its use, while carrying significant risks of glaucoma and cataract formation that outweigh potential benefits in this off-label context. 1

FDA Approval Status and Evidence Base

• Ozurdex received FDA approval in 2009 specifically for macular edema secondary to retinal vein occlusion, not for Coats' Disease or retinal neovascularization from other etiologies 1
• The GENEVA study demonstrated that Ozurdex showed visual acuity gains only at 90 days, with complete loss of treatment effect by 6 months in retinal vein occlusion 2, 1
• The therapeutic effect diminishes between 3-6 months, requiring retreatment intervals of at least 4 months 1
• There are no randomized controlled trials evaluating Ozurdex efficacy specifically for Coats' Disease 1

Available Evidence for Coats' Disease

The only published evidence consists of isolated case reports with significant limitations:

• One case report described a 41-year-old female with adult-onset Coats' disease who received Ozurdex followed by laser photocoagulation, showing improvement 3
• Another case series reported two pediatric patients receiving Ozurdex as an adjunct to ranibizumab and laser, noting it required fewer injections than anti-VEGF therapy 4
• These represent anecdotal evidence only, insufficient to establish efficacy or safety 3, 4

Established Treatment Standards for This Patient

For Retinal Neovascularization

• Dense peripheral panretinal photocoagulation (PRP) is the best treatment for retinal neovascularization, as recommended by the American Academy of Ophthalmology 2, 1
• Anti-VEGF agents serve as adjunctive therapy when complete PRP is insufficient to control angiogenesis 2, 1
• This patient has already received multiple anti-VEGF agents (Avastin, Eylea HD, Vabysmo) without adequate response 2

For Coats' Disease Management

• Standard treatment includes laser photocoagulation and cryotherapy to telangiectatic vessels 5, 6
• Anti-VEGF therapy is effective in reducing exudative changes and is commonly combined with laser or cryotherapy 5
• For severe cases with exudative retinal detachment, external drainage with vitrectomy combined with laser/cryotherapy is the surgical approach 5

Significant Safety Concerns

Ocular Complications with Ozurdex

• IOP elevation requiring topical anti-glaucoma therapy occurs in 18-20% of patients treated with Ozurdex 2, 1
• Cataract formation or progression occurs in 10-16% of patients at one year 2, 1
• The COBALT study showed 18% incidence of IOP elevation and 16% cataract incidence at one year with retreatment every 4 months 2
• This patient is pseudophakic in the left eye, eliminating cataract concerns, but glaucoma risk remains substantial 2

Corticosteroid Positioning in Treatment Algorithms

• Intravitreal corticosteroids are considered second-line therapy for macular edema in retinal vein occlusion due to significant ocular side effects 1
• Corticosteroids have a role only when there is failure to respond or inadequate response to anti-VEGF therapy in FDA-approved indications 1
• The American Academy of Ophthalmology explicitly states that anti-VEGF agents are more favorable than steroids as initial therapy due to steroid-associated risks 2

Clinical Context Analysis

Patient's Current Status

• The patient has chronic, diffuse macular leakage with outer retinal disruption and guarded prognosis per the treating physician's assessment
• OCT shows worsening macular edema compared to prior studies despite ongoing Vabysmo treatment
• Visual acuity remains poor at 20/100-125 despite multiple anti-VEGF agents
• The physician notes "no improvement" and is considering PRN treatment with steroids

Critical Treatment Gap

The patient has not received definitive laser photocoagulation or cryotherapy to the areas of central diffuse macular leakage, which represents the evidence-based standard of care for Coats' Disease 5, 6

• The clinical notes mention prior laser treatment in a previous time period for peripheral disease, but no recent laser to address the current central leakage
• Fluorescein angiography is planned to better characterize leakage patterns, which should guide laser treatment 5

Alternative Recommendation

Appropriate Next Steps

1. Complete fluorescein angiography to map areas of telangiectatic vessels and capillary non-perfusion 5, 3
2. Perform targeted laser photocoagulation to areas of central diffuse macular leakage identified on angiography 5, 6
3. Consider cryotherapy for peripheral telangiectatic vessels if not previously adequately treated 5
4. Continue anti-VEGF therapy as adjunctive treatment to reduce exudative changes 5

If Steroid Consideration is Necessary

• If corticosteroid therapy is deemed absolutely necessary after exhausting laser/cryotherapy options, short-term topical or intravitreal triamcinolone would be more appropriate than Ozurdex given the lack of specific evidence for Coats' Disease 2
• However, the risks of glaucoma and the lack of evidence for efficacy in Coats' Disease make any corticosteroid use highly questionable in this context 1

Reimbursement and Coverage Considerations

• The coverage policy correctly identifies Coats' Disease as experimental/investigational for Ozurdex use
• No change in coverage determination is warranted based on available evidence 1
• The absence of FDA approval, lack of controlled trials, and availability of evidence-based alternatives (laser photocoagulation) support denial of authorization