Condition

Hepatoblastoma

Clinical trials and treatment information for Hepatoblastoma

34K
People Affected
5
Active Trials
17K
New Cases/Year
2,416
Deaths/Year

Treatment Rankings

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

1
Surgical Resection (Partial Hepatectomy)
90% Effectiveness• 95% Confidence• 55% Safety• 500 trials• 25K participants
HIGH EvidenceExcellent Value
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Comparative Safety Scale(Higher is safer)

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55
DangerousModerateSafe

Time to Effect

immediate

Duration

Acute event, weeks to months recovery

Response Rate

85%

Remission Rate

80%

Common Side Effects:

Post-operative pain: 100%
Infection: 10%
Bleeding (requiring transfusion): 8%
Bile leak: 7%
Liver insufficiency: 3%

Annual Cost of Care

Drug Cost

$500

Monitoring

$15,000

Side Effects

$5,000

Total Annual

$120,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

20

Cost per Remission

$150,625

Cost per Responder

$141,764.7

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+85%
Remission Rate
+80%
Common Side Effects
Post-operative pain
+100%
Infection
+10%
Bleeding (requiring transfusion)
+8%

Clinical Trial Phases:

Phase 4
2
Cisplatin-based Chemotherapy
85% Effectiveness• 90% Confidence• 25% Safety• 1 trials• 15K participants
HIGH EvidenceGood ValueDose: Various regimens, e.g., Cisplatin 80-100 mg/m2 per cycle
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Comparative Safety Scale(Higher is safer)

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25
DangerousModerateSafe

Time to Effect

2-4 weeks

Duration

4-6 months

Response Rate

70%

Remission Rate

70%

Common Side Effects:

Nausea and vomiting: 85%
Myelosuppression: 80%
Ototoxicity: 40%
Nephrotoxicity: 35%
Fatigue: 80%

Annual Cost of Care

Drug Cost

$5,000

Monitoring

$40,000

Side Effects

$50,000

Total Annual

$95,000

Cost-Effectiveness

GOOD

QALYs Gained

18

Cost per Remission

$135,714.28

Cost per Responder

$135,714.28

Treatment Outcomes
Primary Outcomes
Serum Alpha-fetoprotein (AFP)50,000 ng/mL
-95% (-47,500 ng/mL)
Tumor Diameter (Largest Dimension)8 cm
-50% (-4 cm)
Viable Tumor Percentage in Resected Specimen100% (before chemotherapy)
-85% (-85 percentage points)
Secondary Benefits
Post-operative Complications (Grade III-V)15% (for primary complex liver resections)
-33% (-5 percentage points)
PedsQL Cancer Module Score (Age 8-12)60/100 (reflecting impact of disease and initial treatment)
+20% (+12 points)
Length of Hospital Stay (Post-Resection)14 days (for complex liver resection)
-20% (-3 days)
Common Side Effects
Nausea and vomiting
+85%
Myelosuppression
+80%
Ototoxicity
+40%

Clinical Trial Phases:

Phase 3Phase 4
3
Liver Transplantation
80% Effectiveness• 90% Confidence• 25% Safety• 5 trials• 8K participants
HIGH EvidenceModerate Value
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Comparative Safety Scale(Higher is safer)

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25
DangerousModerateSafe

Time to Effect

immediate (tumor removal), long recovery

Duration

Acute event, lifelong immunosuppression

Response Rate

80%

Remission Rate

75%

Common Side Effects:

Organ rejection: 20%
Infection: 50%
Biliary complications: 15%
Kidney dysfunction: 30%
Post-transplant lymphoproliferative disorder (PTLD): 5%

Annual Cost of Care

Drug Cost

$30,000

Monitoring

$30,000

Side Effects

$20,000

Total Annual

$580,000

Cost-Effectiveness

MODERATE

QALYs Gained

20

ICER

$110,000/QALY

Cost per Remission

$773,333.33

Cost per Responder

$725,000

Treatment Outcomes
Primary Outcomes
5-Year Overall SurvivalExpected 5-year survival <10% (for advanced hepatoblastoma requiring LT)
+650% (+65 percentage points (e.g., from 10% to 75%))
5-Year Event-Free SurvivalExpected 5-year EFS <5% (for advanced hepatoblastoma requiring LT)
+1200% (+60 percentage points (e.g., from 5% to 65%))
Tumor Recurrence RateExpected 5-year recurrence rate >90% (without definitive resection/LT)
-70% (-63 percentage points (e.g., from 90% to 27%))
Alpha-fetoprotein (AFP) LevelMedian 50,000 ng/mL (pre-treatment, often >1000 ng/mL)
-99.9% (-49,980 ng/mL (e.g., from 50,000 ng/mL to 20 ng/mL))
Secondary Benefits
Pediatric Quality of Life Inventory (PedsQL) Total ScorePedsQL score: 50/100 (for children with severe chronic illness)
+40% (+20 points (e.g., from 50 to 70))
PedsQL Physical Functioning ScorePedsQL Physical Functioning: 45/100
+55.56% (+25 points (e.g., from 45 to 70))
School Participation Days50% of school days attended (due to illness/hospitalization)
+70% (+35 percentage points (e.g., from 50% to 85%))
Common Side Effects
Organ rejection
+20%
Infection
+50%
Biliary complications
+15%

Clinical Trial Phases:

Phase 3Phase 4
4
Doxorubicin (as part of multi-agent chemotherapy)
75% Effectiveness• 85% Confidence• 25% Safety• 200 trials• 10K participants
HIGH EvidenceGood ValueDose: Various regimens, e.g., 30-60 mg/m2 per cycle
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25
DangerousModerateSafe

Time to Effect

weeks

Duration

Part of a 4-6 month regimen

Response Rate

70%

Remission Rate

70%

Common Side Effects:

Myelosuppression: 85%
Nausea and vomiting: 80%
Hair loss: 95%
Cardiotoxicity (cumulative dose): 20%
Mucositis: 50%

Annual Cost of Care

Drug Cost

$2,000

Monitoring

$5,000

Side Effects

$10,000

Total Annual

$17,000

Cost-Effectiveness

GOOD

QALYs Gained

15

Cost per Remission

$24,285.71

Cost per Responder

$24,285.71

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+75%
Response Rate
+70%
Remission Rate
+70%
Common Side Effects
Myelosuppression
+85%
Nausea and vomiting
+80%
Hair loss
+95%

Clinical Trial Phases:

Phase 3Phase 4
5
Liposomal Doxorubicin
70% Effectiveness• 75% Confidence• 28% Safety• 6 trials• 2K participants
MODERATE EvidenceModerate ValueDose: Various regimens, e.g., 20-30 mg/m2 per cycle
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Comparative Safety Scale(Higher is safer)

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28
DangerousModerateSafe

Time to Effect

weeks

Duration

Part of a 4-6 month regimen

Response Rate

70%

Remission Rate

70%

Common Side Effects:

Myelosuppression: 75%
Hand-foot syndrome: 35%
Nausea and vomiting: 70%
Mucositis: 45%
Cardiotoxicity (reduced): 10%

Annual Cost of Care

Drug Cost

$15,000

Monitoring

$5,000

Side Effects

$10,000

Total Annual

$30,000

Cost-Effectiveness

MODERATE

QALYs Gained

15.5

Cost per Remission

$42,857.14

Cost per Responder

$42,857.14

Treatment Outcomes
Primary Outcomes
Alpha-fetoprotein (AFP) Levels150,000 ng/mL
-90% (-135,000 ng/mL)
Tumor Size (Largest Diameter)8 cm
-50% (-4 cm)
Number of Hepatic Tumor Nodules3 nodules
-66.7% (-2 nodules)
Secondary Benefits
Pediatric Quality of Life (PedsQL) ScorePedsQL score: 60/100
+10% (+6 points)
Left Ventricular Ejection Fraction (LVEF)65%
-5% (-3.25%)
WHO Oral Mucositis Scale ScoreWHO Oral Mucositis Scale: 2/4
-50% (-1 point)
Common Side Effects
Myelosuppression
+75%
Hand-foot syndrome
+35%
Nausea and vomiting
+70%

Clinical Trial Phases:

Phase 2Phase 3
6
Vincristine + 5-Fluorouracil (5-FU)
60% Effectiveness• 70% Confidence• 30% Safety• 5 trials• 3K participants
MODERATE EvidenceModerate ValueDose: Vincristine 1.5-2 mg/m2; 5-FU 500 mg/m2
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30
DangerousModerateSafe

Time to Effect

weeks

Duration

Variable, often in specific protocols

Response Rate

40%

Remission Rate

40%

Common Side Effects:

Peripheral neuropathy (Vincristine): 60%
Myelosuppression (5-FU): 60%
Mucositis (5-FU): 45%
Constipation (Vincristine): 40%
Diarrhea (5-FU): 40%

Annual Cost of Care

Drug Cost

$1,000

Monitoring

$5,000

Side Effects

$7,000

Total Annual

$13,000

Cost-Effectiveness

MODERATE

QALYs Gained

8

Cost per Remission

$32,500

Cost per Responder

$32,500

Treatment Outcomes
Primary Outcomes
Alpha-Fetoprotein (AFP) Levels1,000,000 ng/mL
-95% (-950,000 ng/mL)
Tumor Volume Reduction (based on imaging)200 cm³
-50% (-100 cm³)
Pathologic Necrosis in Resected Tumor10% necrotic tissue
+700% (+70% (from 10% to 80%))
Secondary Benefits
Pain Score (Wong-Baker FACES Scale)6/10
-50% (-3 points (from 6 to 3))
Serum Bilirubin2.5 mg/dL
-60% (-1.5 mg/dL)
Body Weight15 kg
+10% (+1.5 kg)
Common Side Effects
Peripheral neuropathy (Vincristine)
+60%
Myelosuppression (5-FU)
+60%
Mucositis (5-FU)
+45%

Clinical Trial Phases:

Phase 2Phase 3