Condition

Neural tube defects

Clinical trials and treatment information for Neural tube defects

1.1M
People Affected
80
Active Trials
300K
New Cases/Year
56K
Deaths/Year

Treatment Rankings

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

1
Ventriculoperitoneal (VP) Shunt Placement
90% Effectiveness• 95% Confidence• 55% Safety• 500 trials• 100K participants
HIGH EvidenceExcellent ValueDose: N/A (surgical procedure)
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Comparative Safety Scale(Higher is safer)

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55
DangerousModerateSafe

Time to Effect

Immediate

Duration

Lifetime (requires ongoing management and potential revisions)

Response Rate

95%

Remission Rate

%

Number Needed to Harm (NNH)

5

Common Side Effects:

Shunt malfunction (occlusion, disconnection): 35%
Shunt infection: 10%
Overdrainage (slit ventricle syndrome): 7%
Seizures: 7%

Annual Cost of Care

Drug Cost

$0

Monitoring

$1,500

Side Effects

$5,000

Total Annual

$46,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

12

Cost per Responder

$48,947.37

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+95%
Common Side Effects
Shunt malfunction (occlusion, disconnection)
+35%
Shunt infection
+10%
Overdrainage (slit ventricle syndrome)
+7%

Clinical Trial Phases:

Phase 4
2
Fetal Myelomeningocele Repair (Maternal-Fetal Surgery)
80% Effectiveness• 90% Confidence• 50% Safety• 4 trials• 1K participants
HIGH EvidencePoor ValueDose: N/A (surgical procedure)
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Comparative Safety Scale(Higher is safer)

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50
DangerousModerateSafe

Time to Effect

Immediate surgical correction, long-term neurodevelopmental benefits

Duration

Single surgical intervention (typically 19-26 weeks gestation)

Response Rate

60%

Remission Rate

60%

Number Needed to Treat (NNT)

2.6

Number Needed to Harm (NNH)

21

Common Side Effects:

Preterm birth (before 37 weeks): 46%
Chorioamnionitis: 5%
Uterine dehiscence: 2%
Increased need for shunt revision (postnatal, if initial shunt placed): 10%

Annual Cost of Care

Drug Cost

$0

Monitoring

$10,000

Side Effects

$30,000

Total Annual

$140,000

Cost-Effectiveness

POOR

QALYs Gained

0.96

ICER

$305,055/QALY

Cost per Remission

$233,333.33

Cost per Responder

$233,333.33

Treatment Outcomes
Primary Outcomes
Need for Ventriculoperitoneal (VP) Shunt by 12 Months82% in postnatal repair group
-51.2% (-42 percentage points)
Mental Development Index (MDI) at 30 Months85 points (Bayley-II scale) in postnatal repair group
+5.88% (+5 points)
Psychomotor Development Index (PDI) at 30 Months75 points (Bayley-II scale) in postnatal repair group
+6.67% (+5 points)
Prevalence of Hindbrain Herniation at 6 Months97% in postnatal repair group
-96.9% (-94 percentage points)
Secondary Benefits
Ambulatory Status at 30 Months37% (walking without or with crutches/braces) in postnatal repair group
+59.5% (+22 percentage points)
Tethered Cord Syndrome Requiring Re-operation25% in postnatal repair group
-60% (-15 percentage points)
Incidence of Syringomyelia at 30 Months20% in postnatal repair group
-50% (-10 percentage points)
Common Side Effects
Preterm birth (before 37 weeks)
+46%
Chorioamnionitis
+5%
Uterine dehiscence
+2%

Clinical Trial Phases:

Phase 3Phase 4
3
Postnatal Myelomeningocele Repair
75% Effectiveness• 95% Confidence• 55% Safety• 4 trials• 50K participants
HIGH EvidenceExcellent ValueDose: N/A (surgical procedure)
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Comparative Safety Scale(Higher is safer)

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Meth💀
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Chemo☢️
Alcohol🍺
Morphine💊
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Water💧
55
DangerousModerateSafe

Time to Effect

Immediate surgical correction, long-term care

Duration

Single surgical intervention (typically within 72 hours of birth)

Response Rate

100%

Remission Rate

%

Number Needed to Harm (NNH)

10

Common Side Effects:

Surgical site infection: 7%
Hydrocephalus requiring ventriculoperitoneal shunt: 80%
CSF leak: 10%
Meningitis: 3%

Annual Cost of Care

Drug Cost

$0

Monitoring

$5,000

Side Effects

$20,000

Total Annual

$90,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

12

Cost per Responder

$90,000

Treatment Outcomes
Primary Outcomes
Incidence of Ventriculoperitoneal (VP) Shunt for Hydrocephalus by 12 months75% (estimated risk without early surgical closure)
-13.33% (-10% absolute reduction in incidence)
Incidence of Meningitis within 30 days of birth50% (estimated risk with open neural defect)
-94% (-47% absolute reduction in incidence)
Proportion of Children Achieving Independent Ambulation (K level 3 or higher) by 5 years20% (estimated for severe lesions without optimized care)
+75% (+15% increase in rate of independent ambulation)
Secondary Benefits
Incidence of Pressure Ulcers at 1 year (among mobile patients)30% (estimated for patients with severe sensory deficits without prophylactic care/repair)
-66.67% (-20% absolute reduction in incidence)
Bayley Scales of Infant Development (BSID-III) Cognitive Composite Score at 12 months85/100 (score indicative of mild developmental delay often associated with MMC)
+5.88% (+5 points)
Bowel Continence (Social continence with management by 7 years)15% (estimated for individuals with neurogenic bowel without optimized care)
+166.67% (+25% increase in rate of social continence)
Common Side Effects
Surgical site infection
+7%
Hydrocephalus requiring ventriculoperitoneal shunt
+80%
CSF leak
+10%

Clinical Trial Phases:

Phase 4
4
Folic Acid Supplementation (Prevention)
70% Effectiveness• 95% Confidence• 90% Safety• 1000 trials• 1M participants
HIGH EvidenceExcellent ValueDose: 0.4mg-4mg daily
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Comparative Safety Scale(Higher is safer)

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90
DangerousModerateSafe

Time to Effect

Ongoing during periconceptional period

Duration

Periconceptional period (at least 1 month before conception through first trimester), or for all women of childbearing age

Response Rate

70%

Remission Rate

%

Number Needed to Treat (NNT)

150

Common Side Effects:

Nausea: 1%
Bloating: 1%
Loss of appetite: 1%

Annual Cost of Care

Drug Cost

$15

Monitoring

$1,000

Side Effects

$0

Total Annual

$1,015

Cost-Effectiveness

EXCELLENT

QALYs Gained

25

Cost per Responder

$21.43

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+70%
Response Rate
+70%
Common Side Effects
Nausea
+1%
Bloating
+1%
Loss of appetite
+1%

Clinical Trial Phases:

Phase 4
5
Posterior Fossa Decompression (for symptomatic Chiari II)
70% Effectiveness• 80% Confidence• 55% Safety• 50 trials• 5K participants
MODERATE EvidenceModerate ValueDose: N/A (surgical procedure)
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
55
DangerousModerateSafe

Time to Effect

Days to weeks

Duration

Single surgical intervention

Response Rate

75%

Remission Rate

%

Number Needed to Treat (NNT)

4

Number Needed to Harm (NNH)

15

Common Side Effects:

CSF leak: 7%
Surgical site infection: 4%
Persistent symptoms: 25%
Meningitis: 2%

Annual Cost of Care

Drug Cost

$0

Monitoring

$750

Side Effects

$3,000

Total Annual

$53,750

Cost-Effectiveness

MODERATE

QALYs Gained

0.7

Cost per Responder

$71,666.67

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+70%
Response Rate
+75%
Common Side Effects
CSF leak
+7%
Surgical site infection
+4%
Persistent symptoms
+25%

Clinical Trial Phases:

Phase 4