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    Subjects/Ophthalmology/Retinoblastoma
    Retinoblastoma
    medium
    eye Ophthalmology

    A 3-year-old girl with a family history of retinoblastoma is diagnosed with unilateral intraocular retinoblastoma on imaging. Regarding management and prognosis, all of the following statements are correct EXCEPT:

    A. Enucleation is indicated if the tumor is large (>8 mm in thickness or >15 mm in base diameter) with poor vision
    B. Systemic chemotherapy followed by focal therapy is the standard approach for globe-salvaging in bilateral disease
    C. Intra-arterial chemotherapy (IAC) is contraindicated in all cases of retinoblastoma due to high systemic toxicity
    D. The International Classification of Retinoblastoma (Murphree classification) is used to stratify risk and guide treatment intensity

    Explanation

    ## Retinoblastoma: Treatment Strategies and Classification ### Treatment Paradigm Shift **Key Point:** Modern retinoblastoma management prioritizes **globe salvage** over enucleation whenever possible, especially in unilateral disease with good visual potential and in all cases of bilateral disease. **High-Yield:** The treatment approach depends on: 1. **Unilateral vs. bilateral disease** 2. **Tumor size and location** 3. **Visual prognosis** 4. **Risk stratification using International Classification** ### Enucleation Indications **Key Point:** Enucleation is reserved for: - **Large tumors** (>8 mm in thickness OR >15 mm in base diameter) - **Poor visual prognosis** (vision <20/200 or rubeotic glaucoma) - **Neovascular glaucoma** - **Phthisical eye** - **Failed globe-salvaging therapy** - **Extraocular extension** **Clinical Pearl:** Even in unilateral disease, globe salvage is attempted if there is any potential for useful vision. ### Chemotherapy Approaches #### Systemic Chemotherapy - **Induction chemotherapy** (vincristine, etoposide, carboplatin) followed by focal therapy (laser, cryotherapy, brachytherapy) - Standard for bilateral disease and high-risk unilateral disease - Goal: reduce tumor burden and allow focal consolidation #### Intra-Arterial Chemotherapy (IAC) **High-Yield:** Intra-arterial chemotherapy is **NOT contraindicated** — it is an **established, effective globe-salvaging modality**. **Key Point:** IAC is indicated for: - **Chemotherapy-resistant tumors** - **Vitreous seeding** - **Recurrent or refractory disease** - **Unilateral tumors with good visual potential** **Clinical Pearl:** IAC delivers high drug concentration directly to the ophthalmic artery, minimizing systemic exposure compared to systemic IV chemotherapy. It has become a cornerstone of globe-salvaging therapy in specialized centers. ### International Classification of Retinoblastoma (Murphree) **Key Point:** The Murphree classification stratifies intraocular disease into five groups (A–E) based on tumor size, location, vitreous involvement, and other factors. | Group | Risk Profile | Treatment | | --- | --- | --- | | A | Smallest, best prognosis | Observation, focal therapy | | B | Small to medium | Systemic chemo + focal therapy | | C | Medium, vitreous seeding | Systemic chemo + focal therapy ± IAC | | D | Large, extensive seeding | Systemic chemo + IAC ± external beam | | E | Phthisical, pain, NVG | Enucleation | **High-Yield:** This classification guides treatment intensity and helps predict globe salvage rates and overall survival. ### Treatment Algorithm ```mermaid flowchart TD A[Retinoblastoma diagnosed]:::outcome --> B{Unilateral or Bilateral?}:::decision B -->|Unilateral| C{Group A-B or C-E?}:::decision B -->|Bilateral| D[Systemic chemotherapy induction]:::action C -->|A-B| E[Focal therapy ± systemic chemo]:::action C -->|C-E| F[Systemic chemotherapy]:::action D --> G[Assess response]:::decision F --> G G -->|Good response| H[Focal therapy: laser, cryo, brachytherapy]:::action G -->|Resistant| I[IAC or external beam]:::action H --> J{Globe salvaged?}:::decision I --> J J -->|Yes| K[Long-term surveillance]:::outcome J -->|No| L[Enucleation]:::action L --> M[Adjuvant therapy if high-risk pathology]:::action ``` [cite:Boyd 5e Ch 17]

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