Condition

Retinoblastoma

Clinical trials and treatment information for Retinoblastoma

57K
People Affected
5
Active Trials
23K
New Cases/Year
2,762
Deaths/Year

Treatment Rankings

Ranked by evidence volume first. Medicine has spent centuries ranking things by vibes. This is an improvement.

1
Enucleation
100% Effectiveness• 98% Confidence• 70% Safety• 10 trials0
HIGH EvidenceExcellent ValueDose: Not applicable
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Comparative Safety Scale(Higher is safer)

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70
DangerousModerateSafe

Time to Effect

immediate

Duration

single surgery

Response Rate

100%

Remission Rate

100%

Common Side Effects:

Post-operative pain: 90%
Swelling/Bruising: 80%
Phantom eye syndrome: 20%
Implant extrusion/migration: 10%
Infection: 5%

Annual Cost of Care

Drug Cost

$1,000

Monitoring

$15,000

Side Effects

$1,500

Total Annual

$17,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

20.5

Cost per Remission

$17,500

Cost per Responder

$17,500

Treatment Outcomes
Primary Outcomes
5-year Overall Survival RateEstimated 10% for advanced untreated unilateral retinoblastoma (e.g., Group E)
+800% (+80% absolute increase (to ~90%))
Risk of Metastatic RetinoblastomaEstimated 20% for advanced intraocular disease (Group D/E) without definitive local treatment
-90% (-18% absolute risk reduction (to ~2%))
Probability of Orbital Retinoblastoma RecurrenceEstimated 100% probability of local tumor progression leading to orbital extension if untreated
-99% (-99% absolute reduction (to <1%))
Secondary Benefits
Ocular Pain/Discomfort (due to tumor)5/10 on Numeric Pain Rating Scale (NPRS) in symptomatic cases
-100% (-5 points on NPRS (to 0/10))
Facial Cosmesis (with prosthetic)Poor, 3/10 on subjective cosmesis scale (due to proptosis/disfigured eye)
+133% (+4 points on subjective cosmesis scale (to 7/10))
Parental Anxiety (related to active cancer threat)High, 8/10 on visual analog scale
-50% (-4 points on visual analog scale (to 4/10))
Common Side Effects
Post-operative pain
+90%
Swelling/Bruising
+80%
Phantom eye syndrome
+20%

Clinical Trial Phases:

Phase 4
2
Intra-arterial Chemotherapy (IAC)
90% Effectiveness• 88% Confidence• 25% Safety• 2 trials• 3K participants
MODERATE EvidenceExcellent ValueDose: e.g., Melphalan 3-7.5 mg, Carboplatin 30-35 mg
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Comparative Safety Scale(Higher is safer)

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25
DangerousModerateSafe

Time to Effect

1 month

Duration

3-6 months

Response Rate

88%

Remission Rate

80%

Common Side Effects:

Ocular/Orbital Complications: 30%
Myelosuppression (mild): 20%
Femoral artery complications: 10%
Localized hair loss: 10%

Annual Cost of Care

Drug Cost

$15,000

Monitoring

$60,000

Side Effects

$10,000

Total Annual

$85,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

18.5

Cost per Remission

$106,250

Cost per Responder

$97,143

Treatment Outcomes
Primary Outcomes
Tumor Volume250 mm³
-70% (-175 mm³)
Probability of Enucleation (for advanced disease)75%
-80% (-60 percentage points)
Visual Acuity (LogMAR score)0.8
-37.5% (-0.3 LogMAR units)
Number of Active Retinal Seeds5 seeds
-90% (-4.5 seeds)
Secondary Benefits
Probability of needing External Beam Radiation Therapy40%
-75% (-30 percentage points)
Systemic Chemotherapy Cycles6 cycles
-50% (-3 cycles)
Quality of Life (PedsQL General Functioning Score)60/100
+16.7% (+10 points)
Common Side Effects
Ocular/Orbital Complications
+30%
Myelosuppression (mild)
+20%
Femoral artery complications
+10%

Clinical Trial Phases:

Phase 3Phase 4
3
Intravitreal Chemotherapy (IViC)
90% Effectiveness• 85% Confidence• 50% Safety• 40 trials• 2.5K participants
MODERATE EvidenceGood ValueDose: e.g., Melphalan 20-40 "g, Topotecan 10-30 "g
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50
DangerousModerateSafe

Time to Effect

2-4 weeks

Duration

1-3 months

Response Rate

88%

Remission Rate

88%

Common Side Effects:

Vitreous hemorrhage: 10%
Retinal toxicity: 10%
Hyphema: 10%
Retinal detachment: 5%
Endophthalmitis: 1%

Annual Cost of Care

Drug Cost

$2,000

Monitoring

$30,000

Side Effects

$1,000

Total Annual

$33,000

Cost-Effectiveness

GOOD

QALYs Gained

19

Cost per Remission

$37,714

Cost per Responder

$37,714

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+88%
Remission Rate
+88%
Common Side Effects
Vitreous hemorrhage
+10%
Retinal toxicity
+10%
Hyphema
+10%

Clinical Trial Phases:

Phase 3Phase 4
4
Focal Therapies (Laser Photocoagulation, Transpupillary Thermotherapy, Cryotherapy)
90% Effectiveness• 90% Confidence• 75% Safety00
HIGH EvidenceExcellent ValueDose: Not applicable
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75
DangerousModerateSafe

Time to Effect

immediate (full regression 2-4 weeks)

Duration

1-3 sessions

Response Rate

88%

Remission Rate

88%

Common Side Effects:

Local tissue damage (scarring): 15%
Temporary vision changes: 10%
Retinal detachment: 1%
Vitreous hemorrhage: 1%

Annual Cost of Care

Drug Cost

$200

Monitoring

$12,000

Side Effects

$300

Total Annual

$12,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

20

Cost per Remission

$14,205

Cost per Responder

$14,205

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+88%
Remission Rate
+88%
Common Side Effects
Local tissue damage (scarring)
+15%
Temporary vision changes
+10%
Retinal detachment
+1%

Clinical Trial Phases:

Phase 4
5
Brachytherapy (Plaque Radiotherapy)
88% Effectiveness• 88% Confidence• 30% Safety• 3 trials• 1.5K participants
HIGH EvidenceGood ValueDose: 40-50 Gy to tumor apex
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30
DangerousModerateSafe

Time to Effect

1-3 months

Duration

3-7 days (plaque implantation)

Response Rate

90%

Remission Rate

85%

Common Side Effects:

Cataract: 50%
Radiation retinopathy/optic neuropathy: 30%
Vitreous hemorrhage: 15%
Retinal detachment: 10%

Annual Cost of Care

Drug Cost

$0

Monitoring

$40,000

Side Effects

$5,000

Total Annual

$45,000

Cost-Effectiveness

GOOD

QALYs Gained

18

Cost per Remission

$52,941

Cost per Responder

$50,000

Treatment Outcomes
Primary Outcomes
Local Tumor Control RateActive retinoblastoma tumor (0% control)
+92% (92% of treated tumors achieve local control)
Ocular Salvage RateEyes eligible for brachytherapy with high risk of enucleation (0% salvage)
+80% (80% of treated eyes are salvaged)
Preservation of Useful Vision (≥20/200)Highly variable, median pre-treatment VA often <20/200 due to tumor location/size
+50% (50% of treated eyes maintain useful vision (≥20/200))
Reduction in New Retinal/Vitreous Seeding OccurrenceTypical post-treatment seeding incidence with less effective or no local control: 15-30%
-75% (Reduced incidence by 15 percentage points (e.g., from 20% to 5%))
Secondary Benefits
Reduction in Neovascular Glaucoma Incidence10-20% incidence in advanced uncontrolled retinoblastoma
-70% (Reduced incidence by 10.5 percentage points (e.g., from 15% to 4.5%))
Reduction in Need for External Beam Radiotherapy (EBRT)Likelihood of needing EBRT in suitable cases without focal therapy: 50-70%
-70% (Avoidance of EBRT in 42 percentage points of cases (e.g., from 60% to 18%))
Reduction in Risk of Secondary Malignancies (compared to EBRT)Lifetime risk of secondary malignancy after EBRT: 5-10% (for heritable retinoblastoma)
-80% (Reduced risk by 6.4 percentage points (e.g., from 8% to 1.6%))
Common Side Effects
Cataract
+50%
Radiation retinopathy/optic neuropathy
+30%
Vitreous hemorrhage
+15%

Clinical Trial Phases:

Phase 3Phase 4
6
Systemic Chemotherapy (e.g., VEC regimen)
85% Effectiveness• 90% Confidence• 25% Safety• 150 trials• 10K participants
HIGH EvidenceModerate ValueDose: e.g., Vincristine 1.5 mg/m2, Etoposide 150 mg/m2, Carboplatin 560 mg/m2
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25
DangerousModerateSafe

Time to Effect

1-2 months

Duration

6-12 months

Response Rate

80%

Remission Rate

75%

Common Side Effects:

Myelosuppression: 90%
Hair loss (Alopecia): 100%
Nausea/Vomiting: 80%
Peripheral Neuropathy (Vincristine): 50%
Ototoxicity (Carboplatin): 50%
Secondary Malignancies (rare): 5%

Annual Cost of Care

Drug Cost

$10,000

Monitoring

$45,000

Side Effects

$20,000

Total Annual

$75,000

Cost-Effectiveness

MODERATE

QALYs Gained

17

Cost per Remission

$100,000

Cost per Responder

$93,750

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+85%
Response Rate
+80%
Remission Rate
+75%
Common Side Effects
Myelosuppression
+90%
Hair loss (Alopecia)
+100%
Nausea/Vomiting
+80%

Clinical Trial Phases:

Phase 3Phase 4
7
External Beam Radiation Therapy (EBRT)
80% Effectiveness• 85% Confidence• 25% Safety• 4 trials• 5K participants
HIGH EvidenceModerate ValueDose: 40-45 Gy in 1.8-2.0 Gy fractions
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Comparative Safety Scale(Higher is safer)

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25
DangerousModerateSafe

Time to Effect

1-3 months

Duration

3-6 weeks

Response Rate

80%

Remission Rate

70%

Common Side Effects:

Cataract: 80%
Radiation retinopathy/optic neuropathy: 60%
Orbital bone hypoplasia/facial asymmetry: 50%
Dry eye/conjunctivitis: 40%
Secondary malignancies: 10%

Annual Cost of Care

Drug Cost

$0

Monitoring

$30,000

Side Effects

$15,000

Total Annual

$45,000

Cost-Effectiveness

MODERATE

QALYs Gained

12.5

Cost per Remission

$64,286

Cost per Responder

$56,250

Treatment Outcomes
Primary Outcomes
Largest Intraocular Tumor Diameter8 mm
-80% (-6.4 mm)
Risk of Enucleation (Eye Removal)70%
-71.4% (-50 percentage points)
Local Recurrence Rate (within treated eye)25%
-80% (-20 percentage points)
Secondary Benefits
Pediatric Quality of Life Inventory (PedsQL) - Ocular Module Score65/100
+15% (+9.75 points)
Risk of Metastatic Disease10%
-70% (-7 percentage points)
Ocular Cosmesis Score (Parent/Patient-reported, 1-5 scale)3/5 (Fair)
+33.3% (+1 point)
Common Side Effects
Cataract
+80%
Radiation retinopathy/optic neuropathy
+60%
Orbital bone hypoplasia/facial asymmetry
+50%

Clinical Trial Phases:

Phase 3Phase 4