Condition

Digestive congenital anomalies

Clinical trials and treatment information for Digestive congenital anomalies

3.2M
People Affected
50
Active Trials
600K
New Cases/Year
48K
Deaths/Year

Treatment Rankings

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

1
Ramstedt Pyloromyotomy for Pyloric Stenosis
98% Effectiveness• 95% Confidence• 55% Safety• 200 trials• 5K participants
HIGH EvidenceExcellent Value
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55
DangerousModerateSafe

Time to Effect

Immediate surgical correction, feeding within 24 hours, full recovery 1 week

Duration

Acute (surgical), 1-3 days hospitalization, minimal long-term follow-up

Response Rate

99%

Remission Rate

99%

Common Side Effects:

Incomplete pyloromyotomy: 1.5%
Duodenal perforation: 0.8%
Wound infection: 3%
Bleeding: 0.2%

Annual Cost of Care

Drug Cost

$300

Monitoring

$20,000

Side Effects

$1,000

Total Annual

$21,300

Cost-Effectiveness

EXCELLENT

QALYs Gained

75

Cost per Remission

$21,515

Cost per Responder

$21,515

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+98%
Response Rate
+99%
Remission Rate
+99%
Common Side Effects
Incomplete pyloromyotomy
+1.5%
Duodenal perforation
+0.8%
Wound infection
+3%
2
Esophageal Atresia Repair (Primary Anastomosis)
90% Effectiveness• 85% Confidence• 45% Safety• 1 trials• 10K participants
MODERATE EvidenceExcellent Value
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45
DangerousModerateSafe

Time to Effect

Immediate surgical correction, functional recovery 2-4 weeks

Duration

Acute (surgical), 2-4 weeks hospitalization, lifelong follow-up

Response Rate

95%

Remission Rate

85%

Common Side Effects:

Anastomotic stricture: 35%
Gastroesophageal reflux (GERD): 50%
Tracheomalacia: 15%
Recurrent tracheoesophageal fistula: 7%
Dysphagia/feeding difficulties: 30%
Infection: 10%

Annual Cost of Care

Drug Cost

$500

Monitoring

$200,000

Side Effects

$20,000

Total Annual

$220,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

65

Cost per Remission

$259,412

Cost per Responder

$232,105

Treatment Outcomes
Primary Outcomes
Oral Caloric Intake0% of total caloric needs (dependent on non-oral feeding)
+100% (Transition from 0% to 100% oral caloric intake)
Frequency of Aspiration-Related Respiratory SymptomsFrequent episodes (e.g., multiple daily coughs, recurrent pneumonia)
-95% (From frequent to rare or absent symptoms)
Weight-for-Age Z-score-2.5 Z-score (indicating moderate-to-severe malnutrition)
+60% (+1.5 Z-score improvement (e.g., from -2.5 to -1.0))
Dependency on Non-Oral Feeding100% dependency on gastrostomy or parenteral nutrition
-100% (Elimination of dependency on non-oral feeding)
Secondary Benefits
Pediatric Quality of Life Inventory (PedsQL) Feeding Impact Module Score40/100 (indicating significant feeding-related distress)
+50% (+20 points on PedsQL score (e.g., from 40 to 60))
Hospitalization Rate for Respiratory/Feeding Complications3-5 hospitalizations per year
-80% (Reduction from multiple to rare/no hospitalizations per year)
Parental Stress Index (PSI) Feeding-related Subscale Score80th percentile (indicating high stress)
-25% (Reduction by 20 percentile points (e.g., from 80th to 60th percentile))
Common Side Effects
Anastomotic stricture
+35%
Gastroesophageal reflux (GERD)
+50%
Tracheomalacia
+15%
3
Intestinal Resection and Anastomosis for Atresia/Stenosis
90% Effectiveness• 90% Confidence• 45% Safety• 200 trials• 5K participants
MODERATE EvidenceExcellent Value
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45
DangerousModerateSafe

Time to Effect

Immediate surgical relief, feeding tolerance over 2-6 weeks

Duration

Acute (surgical), 1-4 weeks hospitalization, long-term nutritional support/follow-up

Response Rate

95%

Remission Rate

85%

Common Side Effects:

Anastomotic leak: 7%
Short Bowel Syndrome (if extensive resection): 10%
Intestinal obstruction due to adhesions: 8%
Infection: 7%

Annual Cost of Care

Drug Cost

$500

Monitoring

$120,000

Side Effects

$15,000

Total Annual

$135,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

70

Cost per Remission

$159,412

Cost per Responder

$142,632

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+95%
Remission Rate
+85%
Common Side Effects
Anastomotic leak
+7%
Short Bowel Syndrome (if extensive resection)
+10%
Intestinal obstruction due to adhesions
+8%
4
Hirschsprung's Disease Pull-through Procedure
90% Effectiveness• 88% Confidence• 48% Safety• 7 trials• 10K participants
MODERATE EvidenceExcellent Value
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48
DangerousModerateSafe

Time to Effect

Immediate surgical relief of obstruction, functional improvement over 3-6 months

Duration

Acute (surgical), 1-2 weeks hospitalization, long-term follow-up (years)

Response Rate

90%

Remission Rate

85%

Common Side Effects:

Hirschsprung-associated enterocolitis (HAEC): 20%
Anastomotic stricture: 10%
Fecal incontinence/soiling: 20%
Constipation: 15%
Anastomotic leak: 3%

Annual Cost of Care

Drug Cost

$500

Monitoring

$80,000

Side Effects

$10,000

Total Annual

$90,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

68

Cost per Remission

$106,471

Cost per Responder

$100,556

Treatment Outcomes
Primary Outcomes
Bowel Movement Frequency1-2 movements/week (often requiring intervention)
+300% (+4.5 movements/week)
Annual Hirschsprung-Associated Enterocolitis (HAEC) Episodes2-3 episodes/year
-85% (-2 episodes/year)
Weight Percentile for Age5th percentile (failure to thrive)
+400% (+20 percentile points)
Frequency of Significant Abdominal DistensionDaily severe episodes
-80% (Reduced from daily to 1-2 times/week)
Secondary Benefits
PedsQL Generic Core Scales Score (Child/Parent Report)50/100 (significant impairment)
+25% (+12.5 points)
Dependence on Daily Rectal Irrigation/EnemasDaily use
-90% (Reduced from daily to <1 time/week)
Proportion of Calories from Oral Feeding20% (remaining from tube feeds)
+400% (+80 percentage points (from 20% to 100%))
Common Side Effects
Hirschsprung-associated enterocolitis (HAEC)
+20%
Anastomotic stricture
+10%
Fecal incontinence/soiling
+20%
5
Posterior Sagittal Anorectoplasty (PSARP) for Imperforate Anus
88% Effectiveness• 85% Confidence• 48% Safety• 1 trials• 8K participants
MODERATE EvidenceExcellent Value
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48
DangerousModerateSafe

Time to Effect

Immediate anatomical correction, functional bowel control develops over 1-3 years

Duration

Acute (staged surgical), 1-2 weeks hospitalization, long-term follow-up and bowel management (years)

Response Rate

90%

Remission Rate

80%

Common Side Effects:

Fecal incontinence/soiling: 35%
Constipation: 45%
Anal stricture: 15%
Wound infection: 7%

Annual Cost of Care

Drug Cost

$500

Monitoring

$70,000

Side Effects

$10,000

Total Annual

$80,500

Cost-Effectiveness

EXCELLENT

QALYs Gained

65

Cost per Remission

$100,625

Cost per Responder

$89,444

Treatment Outcomes
Primary Outcomes
Frequency of Fecal SoilingDaily (7 episodes/week)
-70% (-5 episodes/week)
Need for Bowel Management Aids4-5 times/week (e.g., laxatives, enemas)
-60% (-3 times/week)
Frequency of Severe Constipation3-4 episodes/month (impacting daily activity)
-75% (-2 to -3 episodes/month)
Daily Time Spent on Bowel Care60-90 minutes/day
-50% (-30-45 minutes/day)
Secondary Benefits
Disease-Specific Quality of Life Score50/100 (significant impairment)
+30% (+15 points)
Perianal Skin Irritation Severity (Visual Analogue Scale)7/10 (moderate to severe)
-50% (-3.5 points)
Participation in Social Activities (Parent-reported scale)2/5 (limited participation)
+75% (+1.5 points)
Common Side Effects
Fecal incontinence/soiling
+35%
Constipation
+45%
Anal stricture
+15%
6
Kasai Portoenterostomy for Biliary Atresia
70% Effectiveness• 90% Confidence• 40% Safety• 20 trials• 5K participants
MODERATE EvidenceExcellent Value
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40
DangerousModerateSafe

Time to Effect

Bile flow improvement over 1-3 months

Duration

Acute (surgical), 1-3 weeks hospitalization, lifelong follow-up and potential transplant

Response Rate

70%

Remission Rate

50%

Common Side Effects:

Cholangitis (bacterial infection of bile ducts): 45%
Portal hypertension: 30%
Ascites: 15%
Failure to establish bile flow: 35%

Annual Cost of Care

Drug Cost

$1,000

Monitoring

$100,000

Side Effects

$20,000

Total Annual

$121,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

55

Cost per Remission

$242,000

Cost per Responder

$172,857

Treatment Outcomes
Primary Outcomes
Total Bilirubin12.0 mg/dL
-83.3% (-10.0 mg/dL)
Conjugated Bilirubin10.0 mg/dL
-90% (-9.0 mg/dL)
Serum Gamma-Glutamyl Transferase (GGT)800 U/L
-75% (-600 U/L)
Serum Bile Acids180 µmol/L
-77.8% (-140 µmol/L)
Secondary Benefits
Pruritus Severity Score7/10 (severe pruritus)
-57.1% (-4 points)
Serum Albumin3.2 g/dL
+12.5% (+0.4 g/dL)
International Normalized Ratio (INR)1.5
-26.7% (-0.4)
Common Side Effects
Cholangitis (bacterial infection of bile ducts)
+45%
Portal hypertension
+30%
Ascites
+15%