Overview

Definition:
-Retinoblastoma is the most common primary intraocular malignancy of childhood, arising from photoreceptor precursor cells
-Red reflex screening is a simple, non-invasive method to detect potential abnormalities in the ocular media, particularly leukocoria (white pupillary reflex), which is a classic sign of retinoblastoma.
Epidemiology:
-Incidence is approximately 1 in 15,000 to 20,000 live births worldwide
-It accounts for about 3-5% of all childhood cancers
-Bilateral cases are more common in heritable forms (germline mutation), while unilateral cases are more frequent in sporadic forms
-Peak age of diagnosis is around 18-24 months.
Clinical Significance:
-Early detection of retinoblastoma through red reflex screening is crucial for successful treatment and preservation of vision
-Delayed diagnosis significantly worsens the prognosis, increasing the risk of enucleation, metastasis, and mortality
-This screening is a fundamental skill for pediatricians and ophthalmologists.

Clinical Presentation

Symptoms:
-The most common presenting sign is leukocoria (a white or yellowish reflex in the pupil)
-Other symptoms may include strabismus (crossed or wandering eyes), decreased visual acuity, eye pain, redness, or a visible mass
-In advanced stages, exophthalmos may be present.
Signs:
-A normal red reflex appears as a symmetric, symmetrical reddish-orange glow in both pupils when illuminated by an ophthalmoscope or penlight
-An abnormal reflex can be white, yellow, or absent
-Other signs include visible tumors within the eye, vitreous seeds, or retinal detachment
-Strabismus can be a primary observation.
Diagnostic Criteria:
-Diagnosis is primarily clinical and confirmed by ocular imaging
-Definitive diagnosis relies on ophthalmoscopic visualization of a retinoblastoma tumor
-Histopathological confirmation after enucleation or biopsy (less common) is the gold standard, but usually not required for initial diagnosis when typical findings are present.

Diagnostic Approach

History Taking:
-Inquire about any noticed abnormalities in the child's eyes, especially a white or unusual reflex
-Ask about family history of retinoblastoma or other cancers
-Note the age of onset of symptoms and any associated visual complaints or neurological symptoms.
Physical Examination:
-Perform a thorough ophthalmic examination including visual acuity assessment (age-appropriately)
-Assess for strabismus
-Crucially, perform the red reflex test using an ophthalmoscope or penlight from a distance of about 30 cm, observing the pupillary reflex in each eye
-Follow with a funduscopic examination to visualize the retina and any intraocular lesions.
Investigations:
-Ocular ultrasound (B-scan) is vital for assessing tumor size, calcification, and calcification extent
-MRI of the orbits and brain is essential for staging, assessing optic nerve and orbital involvement, and ruling out intracranial extension
-CT scans can also be used, but MRI is preferred due to better soft tissue contrast and avoidance of radiation
-Genetic testing may be indicated for suspected hereditary retinoblastoma.
Differential Diagnosis:
-Conditions mimicking leukocoria include persistent hyperplastic primary vitreous (PHPV), Coats disease, toxocariasis, retinopathy of prematurity (ROP), congenital cataracts, vitreous hemorrhage, and ocular inflammatory conditions
-Strabismus can be due to refractive errors, accommodative excess, or other cranial nerve palsies.

Management

Initial Management:
-Management is multidisciplinary, involving ophthalmologists, pediatric oncologists, and radiation oncologists
-The primary goal is to preserve the eye and vision, while eradicating the tumor and preventing metastasis
-Staging of the tumor is critical for treatment planning.
Medical Management:
-Systemic chemotherapy is the mainstay for many cases, particularly for bilateral disease or larger tumors
-Common agents include carboplatin, etoposide, and vincristine
-Localized chemotherapy delivery via intra-arterial infusion (IA chemotherapy) offers a targeted approach with reduced systemic toxicity
-Intravitreal chemotherapy may be used for vitreous seeding.
Surgical Management:
-Enucleation (surgical removal of the eye) is indicated for eyes with advanced tumors, extensive optic nerve involvement, or when other therapeutic modalities have failed
-It is the definitive treatment to prevent spread
-Cryotherapy and thermotherapy are used for focal, peripheral tumors, often in conjunction with chemotherapy.
Supportive Care:
-Supportive care includes managing treatment-related side effects like myelosuppression, nausea, and alopecia
-Psychological support for the child and family is paramount
-Prosthetic eyes are fitted after enucleation to maintain cosmetic appearance.

Complications

Early Complications:
-Chemotherapy side effects (neutropenia, anemia, thrombocytopenia, nausea, vomiting, alopecia)
-Surgical complications of enucleation include infection, hemorrhage, and anesthetic risks
-Ocular complications from local treatment include inflammation, cataract formation, and retinal detachment.
Late Complications:
-Secondary malignancies (in heritable retinoblastoma), impaired vision or blindness in the affected eye, cosmetic disfigurement from enucleation, recurrence of tumor, and psychological distress
-Long-term effects of chemotherapy can include infertility and cardiac issues.
Prevention Strategies:
-Regular and thorough red reflex screening in infancy and early childhood
-Prompt referral for any suspicious findings
-Accurate staging and appropriate treatment selection based on consensus guidelines (e.g., International Classification of Retinoblastoma)
-Close follow-up to monitor for recurrence or complications.

Prognosis

Factors Affecting Prognosis:
-Tumor size and location, presence of optic nerve or uveal involvement, vitreous seeding, bilaterality, and the specific treatment received
-Early diagnosis and treatment significantly improve outcomes.
Outcomes:
-With modern multimodal therapy, the cure rate for retinoblastoma is over 90%
-Vision can often be preserved in many cases, especially with early detection and focal treatments
-However, enucleation may be necessary for advanced disease
-Metastasis and death are rare with timely and appropriate care.
Follow Up:
-Lifelong surveillance is recommended for survivors, particularly those with heritable retinoblastoma, due to the increased risk of secondary malignancies
-Regular ophthalmic examinations are required to monitor for tumor recurrence, assess vision, and detect any complications
-Screening for other cancers may also be advised.

Key Points

Exam Focus:
-Leukocoria is the cardinal sign of retinoblastoma
-Differentiate retinoblastoma from other causes of leukocoria
-Understand the role of red reflex screening in early detection
-Know the main chemotherapeutic agents and local treatment modalities
-Recognize the importance of genetic counseling in heritable forms.
Clinical Pearls:
-Never assume leukocoria is benign
-Always perform a thorough ophthalmic examination and consider retinoblastoma in any child with leukocoria or strabismus
-Remember that a normal red reflex is bright red and symmetrical
-Early referral to a pediatric ophthalmologist is paramount.
Common Mistakes:
-Failing to perform red reflex screening in all infants and young children
-Delaying referral for suspected leukocoria or strabismus
-Misinterpreting benign conditions as retinoblastoma or vice versa
-Inadequate staging leading to suboptimal treatment
-Not considering the possibility of hereditary retinoblastoma.