Allergy, Immunology, and Medical Genetics

Medical genetics generates thousands of variants per patient from exome and genome sequencing. Determining which variants cause disease requires integrating population frequency, functional predictions, literature, and clinical phenotype. AI automates ACMG criteria application, achieving 85-95% concordance with expert manual review for clearly pathogenic or benign variants. Facial recognition AI (Face2Gene) shows 90%+ top-10 accuracy for recognizable syndromes. Pharmacogenomics clinical decision support through CPIC guidelines is well-established. But population bias in variant databases, rare disease data scarcity, and polygenic risk score equity challenges remain substantial barriers.

Learning Objectives

After reading this chapter, you will be able to:

  • Evaluate AI systems for genetic variant classification and interpretation
  • Understand AI applications in allergic disease phenotyping
  • Assess immunodeficiency diagnostic support tools
  • Navigate the role of AI in precision medicine and pharmacogenomics
  • Recognize the unique challenges of rare disease AI
  • Apply evidence-based frameworks for genetics and immunology AI

The Clinical Context: These specialties deal with complex pattern recognition (allergic phenotypes, immune dysregulation, genetic variant interpretation) that AI may assist with, but face challenges of rare conditions, limited training data, and evolving knowledge bases.

What Works Well:

Application Evidence Level Key Benefit
Genetic variant classification (ACMG criteria) Strong Consistent application of pathogenicity criteria
Pharmacogenomics CDS (CPIC guidelines) Strong Evidence-based genotype-guided prescribing
Facial dysmorphology AI (Face2Gene) Moderate Rare syndrome recognition support

What’s Emerging:

Application Status Notes
Polygenic risk scores Variable Cardiovascular and breast cancer PRS nearing clinical utility
Allergic phenotyping Research Asthma endotypes, food allergy prediction
Primary immunodeficiency classification Research Pattern recognition from clinical data

Critical Challenges:

  • Population bias: Most variant databases overrepresent European ancestry
  • Novel variants: AI cannot classify variants not in training data
  • Rare diseases: Limited training data by definition
  • Phenotype quality: AI performance depends on accurate clinical description

The Bottom Line: Genetic variant interpretation AI is increasingly mature, with ACMG-based classification tools showing consistent performance. Pharmacogenomics CDS through CPIC guidelines is well-established. Facial recognition for syndromes (Face2Gene) shows 90%+ top-10 accuracy for recognizable conditions. Allergy and immunology AI remains largely research-stage. Polygenic risk scores approach clinical utility for cardiovascular disease and breast cancer but face equity and calibration challenges.


Part 1: Genetic Variant Interpretation AI

The Classification Challenge

Exome and genome sequencing identify thousands of variants per patient. Determining which variant(s) cause disease requires integrating:

  • Population frequency (rare variants more likely pathogenic)
  • Functional impact predictions (protein truncation, missense effects)
  • Published literature and case reports
  • Clinical phenotype match
  • Segregation in family members

The ACMG/AMP guidelines (Richards et al., 2015) standardized variant classification into five categories: pathogenic, likely pathogenic, variant of uncertain significance (VUS), likely benign, and benign. AI automates criteria application and evidence integration.

AI-Assisted Classification Tools

Guidelines-based AI:

Tool Approach Evidence
InterVar Automated ACMG criteria application Widely used in clinical labs
Franklin (Genoox) AI-powered evidence aggregation Commercial, EHR-integrated
Illumina Emedgene ML-enhanced variant prioritization FDA-registered
VarSome Automated ACMG classification Free tier available

Performance: Automated ACMG classification tools show 85-95% concordance with expert manual review for clearly pathogenic or benign variants. Discordance highest for VUS classification (Li et al., 2022).

In silico predictors:

Pathogenicity prediction algorithms (CADD, REVEL, AlphaMissense) provide scores that inform the PP3/BP4 ACMG criteria (computational evidence):

  • AlphaMissense (DeepMind, 2023): Trained on evolutionary conservation and protein structure
  • REVEL: Ensemble of 13 prediction algorithms
  • CADD: Integrates diverse annotations

Limitation: Computational predictors lack clinical validation for standalone classification. They support, not replace, comprehensive variant analysis.

ACMG/AMP Position on AI

ACMG Guidelines and AI-Assisted Classification

The foundational ACMG/AMP variant classification guidelines (Richards et al., 2015) established standards that AI tools now implement:

Core principles that apply to AI:

  • Classification requires integrating multiple evidence types
  • Population database frequency informs but does not determine pathogenicity
  • Functional studies provide strong evidence when available
  • Clinical correlation remains essential

Implications for AI tools:

  • AI-assisted classification should follow ACMG criteria, not proprietary algorithms
  • Novel variants not in training databases require caution
  • AI output requires review by certified molecular geneticists or pathologists
  • Patient consent should address AI-assisted analysis

2024 Updates: Gene-specific guidelines (e.g., ATM, BRCA1/2) refine criteria application. AI tools must update as guidelines evolve. The UK ACGS released 2024 practice guidelines incorporating AI considerations.

Population Bias in Variant Databases

A critical equity issue: most variant databases overrepresent European ancestry populations.

Database composition (approximate):

Database European Ancestry
ClinVar ~70%
gnomAD ~55% (improving)
UK Biobank ~95%

Clinical consequence: Variants benign in non-European populations may be misclassified as pathogenic when absent from databases. Patients of African, Asian, and Hispanic ancestry face higher VUS rates.

Mitigation: gnomAD v4 expanded global representation. Population-matched databases (e.g., All of Us) are improving equity, but gaps persist.


Part 2: Facial Recognition for Genetic Syndromes

Face2Gene and DeepGestalt

Face2Gene uses the DeepGestalt deep convolutional neural network to identify syndromic patterns from facial photographs. The system compares uploaded photos against ~300 syndrome models and generates a ranked differential diagnosis.

Performance data:

Study Setting Top-10 Accuracy
Gurovich et al., 2019 Multi-center validation 91%
Carrer et al., 2024 Italian pediatric clinic 98% correct in differential
Egyptian study, 2024 Genetic counseling unit 56% top-3 accuracy

Use case: Face2Gene is most useful for: - Generating differential diagnosis for unfamiliar dysmorphic features - Supporting non-geneticists in recognizing syndromic patterns - Ultra-rare conditions that non-specialists may not consider

Current deployment: Face2Gene is used in 2,000+ clinics across 130 countries according to FDNA.

Limitations

Ethnic and Racial Bias in Facial Recognition AI

Facial recognition AI for syndromes faces significant bias concerns:

Training data skew: Most training images are from European populations. Performance may decrease for: - African and African-American patients - East Asian patients - Middle Eastern patients - Mixed-ancestry patients

Clinical implication: Do not rely solely on Face2Gene for patients from underrepresented populations. Clinical gestalt and genetic testing remain essential.

Photo quality dependence: Performance varies with: - Lighting conditions - Image resolution - Patient age (some syndromes more recognizable in childhood) - Presence of glasses, facial hair, or other features

Appropriate Use

Face2Gene should: - Generate hypotheses, not diagnoses - Supplement, not replace, clinical dysmorphology assessment - Be used with awareness of population limitations - Lead to confirmatory genetic testing, not standalone management changes


Part 3: Pharmacogenomics Clinical Decision Support

CPIC Guidelines: The Evidence Base

The Clinical Pharmacogenetics Implementation Consortium (CPIC) provides peer-reviewed, evidence-based guidelines for translating pharmacogenomic test results into prescribing recommendations.

Scope (2024): - 34 genes covered - 164 drugs with recommendations - 28 active guidelines - 85% of published PGx implementation studies reference CPIC

Key gene-drug pairs with strong evidence:

Gene Drugs Recommendation
CYP2C19 Clopidogrel Poor metabolizers: alternative antiplatelet
CYP2D6 Codeine, tramadol Poor/ultrarapid metabolizers: alternative analgesic
HLA-B*57:01 Abacavir Positive: do not prescribe
TPMT/NUDT15 Azathioprine, 6-MP IM/PM: dose reduction
CYP2C9/VKORC1 Warfarin Genotype-based initial dosing

AI-Powered Pharmacogenomics CDS

Electronic health record integration enables automated PGx alerts:

Implementation models:

  1. Pre-emptive testing: Genotype patients before first prescription; CDS fires when relevant drug ordered
  2. Reactive testing: Test when high-risk drug ordered; results guide dosing
  3. Hybrid: Pre-emptive panel for common genes; reactive for specialized drugs

Performance: - CPIC API supports 80,000+ monthly queries - Epic’s foundational genomics module integrates CPIC content - Studies show improved adherence to PGx-guided prescribing with CDS

AAAAI Framework for Augmented Intelligence

AAAAI Health Informatics, Technology, and Education Committee Framework

The American Academy of Allergy, Asthma and Immunology published a framework for AI in the specialty (Shaker et al., 2022):

Key recommendations:

  • Allergist-immunologists should be involved in design, validation, and implementation of AI tools for the specialty
  • Data science and bioinformatics training should be incorporated into fellowship programs
  • AI applications should be evaluated for bias before clinical deployment
  • Electronic health records provide rich data sources for AI in allergy

Identified applications:

  • Allergen component-resolved diagnostics
  • Asthma phenotype classification
  • Drug allergy de-labeling decision support
  • Immunodeficiency pattern recognition

Current status: Most applications remain research-stage. The AAAAI continues to develop educational resources on AI for members.


Part 4: Allergic Disease AI Applications

Asthma Phenotyping

AI enables classification of asthma into clinically relevant subtypes:

T2-high vs. T2-low asthma: - T2-high: Eosinophilic, responds to biologics (omalizumab, mepolizumab, dupilumab) - T2-low: Neutrophilic or paucigranulocytic, limited biologic options

AI approaches: - Cluster analysis of clinical features, biomarkers, lung function - Treatment response prediction based on phenotype - Biomarker integration (FeNO, blood eosinophils, IgE)

Clinical status: Research-stage. Phenotyping tools not yet integrated into routine practice.

Food Allergy Prediction

AI models attempt to predict: - Which sensitized patients will react on oral food challenge - Severity of reactions - Development of tolerance

Inputs: - Skin prick test wheal size - Specific IgE levels and component testing - Clinical history - Age and other demographics

Performance: Research models show AUC 0.75-0.85 for predicting challenge outcomes. Not yet validated for clinical use.

Drug Allergy Assessment

AI tools for: - Penicillin allergy de-labeling risk stratification - Cross-reactivity prediction (beta-lactam, sulfonamide families) - Distinguishing IgE-mediated from non-immune reactions

Clinical relevance: 90% of patients labeled penicillin-allergic are not truly allergic. AI could identify low-risk candidates for direct challenge without testing.


Part 5: Primary Immunodeficiency AI

The Diagnostic Challenge

Primary immunodeficiencies (PIDs) are underdiagnosed: - >450 distinct genetic conditions - Many present with common infections in early stages - Delayed diagnosis: median 6+ years from symptom onset

AI Approaches

Pattern recognition from clinical data: - Infection types, frequency, pathogens - Autoimmune features - Family history patterns - Growth and development

Genetic prioritization: - Phenotype-driven gene panel selection - Variant interpretation in immune-related genes - Immunologic phenotype-genotype correlation

Jeffrey Modell Foundation Warning Signs

AI could operationalize PID warning signs: - 4+ ear infections in one year - 2+ serious sinus infections in one year - 2+ months on antibiotics with little effect - 2+ pneumonias within one year - Failure to thrive - Recurrent deep skin or organ abscesses - Persistent thrush after age 1 - Need for IV antibiotics to clear infections - 2+ deep-seated infections - Family history of PID

Status: Research-stage. Pattern recognition tools for PID not validated for clinical deployment.


Part 6: Polygenic Risk Scores

Current Evidence

Polygenic risk scores (PRS) aggregate the effects of thousands of genetic variants to estimate disease risk. The 2024 evidence base:

Cardiovascular disease: - PRS validated in multiple large cohorts - HEART study (2024) showed feasibility of clinical integration - PRS combined with traditional risk factors may improve prediction - Several commercial labs offer CAD-PRS

Breast cancer: - PRS could stratify screening intensity - Prospective trials needed before screening guideline changes - Debate continues on PRS for enhancing vs. de-intensifying screening

Implementation status: The eMERGE Network selected 10 conditions for PRS implementation based on performance, actionability, and evidence in diverse populations (Lennon et al., 2024).

Critical Limitations

Polygenic Risk Score Equity Concerns

Population transferability: - Most PRS developed in European populations - Predictive accuracy drops 30-50% when applied to African ancestry populations - Calibration (absolute risk estimation) often poor outside development populations

Clinical utility uncertainty: - Does knowing PRS change patient behavior or outcomes? - How should PRS integrate with established risk factors? - Threshold for “high risk” classification varies by study

Current guidance: - PRS should not be used as standalone risk assessment - Population-matched validation required before clinical use - Genetic counseling should accompany PRS results - Equity review essential: PRS may widen rather than narrow health disparities


Part 7: Rare Disease Diagnosis

The Diagnostic Odyssey

  • 7,000+ rare diseases
  • Average 4-7 years from symptom onset to diagnosis
  • Many conditions seen by non-specialists unfamiliar with presentations

AI Solutions

Phenotype-driven prioritization: - HPO (Human Phenotype Ontology) standardizes phenotype description - Exomiser, Phenomizer: Match patient phenotypes to disease databases - LIRICAL: Likelihood ratio-based phenotype matching

Facial analysis: - Face2Gene (discussed above) - GestaltMatcher: Open-source alternative

Multi-omic integration: - AI combines genomic, transcriptomic, methylation data - Helps identify variants when WES/WGS inconclusive

Undiagnosed Diseases Programs

The NIH Undiagnosed Diseases Program and Network use systematic approaches including AI-assisted analysis. Success rates for diagnosis: 25-35% of referred cases.

AI contribution: Prioritizes variants, identifies candidate genes, suggests additional testing.


Part 8: Professional Society Positions

ACMG Position

The American College of Medical Genetics emphasizes: - AI-assisted variant classification should follow established ACMG/AMP criteria - Certified clinical laboratory geneticists/pathologists must review AI output - Population diversity in training data is essential - Patient consent should address AI-assisted analysis

AAAAI Position

The American Academy of Allergy, Asthma and Immunology: - Published framework for augmented intelligence in the specialty (2022) - Emphasizes allergist involvement in AI tool development - Supports training fellows in data science fundamentals - Issued 2024 position statement on EHR allergy documentation terminology

CPIC Implementation

CPIC provides implementation resources: - Machine-readable guidelines (JSON format) - EHR integration support - Standardized terminology for CDS - Regular guideline updates


Clinical Scenarios

Case: A 3-year-old with developmental delay undergoes exome sequencing. A variant in a developmental disorder gene is classified as VUS by the AI-assisted analysis. Parents ask if this explains their child’s condition.

Question: How should the geneticist counsel about this VUS?

Discussion

Understanding VUS classification:

  • VUS means insufficient evidence to classify as pathogenic or benign
  • It does NOT mean “probably causes disease”
  • Many VUS are eventually reclassified as benign

Counseling approach:

  1. Explain that VUS cannot be used for clinical decision-making
  2. Describe factors that could lead to reclassification (more affected individuals, functional studies)
  3. Offer periodic reanalysis (1-2 years) as databases grow
  4. Consider additional family testing if feasible (segregation)
  5. Do not change management based on VUS

AI limitation: AI classified this as VUS because evidence is genuinely insufficient. The appropriate classification is uncertain, not “probably pathogenic.”

Teaching point: VUS is the most common challenging classification. AI cannot resolve genuine uncertainty. Time and additional evidence, not AI refinement, will reclassify most VUS.

Case: A pediatrician sees a 2-year-old with dysmorphic features and developmental delay. They upload a photo to Face2Gene, which suggests Cornelia de Lange syndrome in the top 3 matches. The pediatrician has never seen this syndrome.

Question: What should the pediatrician do with this suggestion?

Discussion

Appropriate next steps:

  1. Review the syndrome features: Does the patient have cardinal features (synophrys, limb anomalies, growth restriction)?
  2. Refer to genetics: Face2Gene suggestions require expert evaluation
  3. Do not diagnose based on Face2Gene alone: The tool generates hypotheses, not diagnoses
  4. Genetic testing: If features are consistent, targeted gene panel or exome sequencing can confirm

Face2Gene value here:

  • Helped a non-specialist consider a rare diagnosis
  • Generated a hypothesis that would otherwise require dysmorphology expertise
  • Appropriate use: hypothesis generation leading to referral

What not to do:

  • Tell parents “the computer says your child has Cornelia de Lange syndrome”
  • Change management without genetic confirmation
  • Rely on Face2Gene for populations underrepresented in training data
Teaching point: Face2Gene is most valuable when it prompts consideration of diagnoses the clinician would not have considered. It should accelerate the path to genetics referral, not replace it.

Case: A cardiologist orders clopidogrel for a 65-year-old post-PCI. The EHR displays a pharmacogenomics alert: “Patient is CYP2C19 poor metabolizer. Per CPIC guidelines, consider alternative antiplatelet therapy.”

Question: How should the cardiologist respond?

Discussion

Clinical context:

  • CYP2C19 poor metabolizers have reduced conversion of clopidogrel to active metabolite
  • Higher rates of cardiovascular events on clopidogrel vs. normal metabolizers
  • CPIC recommends alternative antiplatelet (prasugrel or ticagrelor) for poor metabolizers

Appropriate response:

  1. Review the genotype result in the EHR
  2. Confirm patient has no contraindications to alternatives (bleeding risk, prior ICH)
  3. Switch to ticagrelor or prasugrel per CPIC recommendation
  4. Document rationale for genotype-guided prescribing

If cardiologist disagrees:

  • Document clinical reasoning for overriding alert
  • Consider that poor metabolizer status may have contributed to events in patients labeled “clopidogrel non-responders”

AI/CDS value:

  • Alert appeared at point of prescribing
  • Linked to evidence-based CPIC guideline
  • Actionable recommendation provided
Teaching point: Pharmacogenomics CDS is among the most mature AI applications in genetics. CPIC alerts are evidence-based and should generally be followed unless patient-specific factors warrant override.

Case: A 45-year-old woman asks about a direct-to-consumer (DTC) genetic test that reported her “coronary artery disease polygenic risk score is in the 90th percentile.” She is concerned about her heart disease risk.

Question: How should the physician counsel this patient?

Discussion

Understanding PRS results:

  • 90th percentile means her PRS is higher than 90% of the reference population
  • This does NOT mean 90% chance of heart disease
  • Relative risk increase is typically 1.5-2x compared to average, not absolute risk

Counseling points:

  1. Contextualize: Ask about the reference population. If patient is non-European and score was developed in Europeans, interpretation is limited.

  2. Integrate with traditional factors: What is her BP, lipids, smoking status, family history? These matter more than PRS.

  3. Actionability: What would she do differently? Standard cardiovascular prevention applies regardless of PRS.

  4. Limitations of DTC testing:

    • Validation varies by company
    • Quality of genetic counseling often inadequate
    • May not use most validated PRS algorithms

Recommended response:

  • Perform comprehensive cardiovascular risk assessment
  • Do not order additional testing based on PRS alone
  • Recommend standard primary prevention based on established guidelines
  • Note that elevated PRS may provide additional motivation for lifestyle modification
Teaching point: PRS from DTC testing should not drive clinical decisions. Standard risk assessment and prevention guidelines apply. PRS may add modest predictive value but does not change management in most cases.

Key Takeaways

Clinical Bottom Line

Genetic variant interpretation:

  • AI tools consistently apply ACMG criteria; 85-95% concordance with experts for clear cases
  • VUS classification remains challenging; AI cannot resolve genuine uncertainty
  • Population bias in databases affects patients of non-European ancestry
  • All AI classifications require certified geneticist review

Facial recognition for syndromes:

  • Face2Gene achieves 90%+ top-10 accuracy for recognizable syndromes
  • Useful for hypothesis generation, not diagnosis
  • Performance varies across ethnic groups
  • Most valuable for non-geneticists considering rare diagnoses

Pharmacogenomics CDS:

  • CPIC guidelines are the evidence-based standard
  • EHR-integrated CDS improves PGx-guided prescribing
  • Pre-emptive genotyping enables point-of-care alerts
  • 34 genes and 164 drugs have CPIC recommendations

Allergy and immunology AI:

  • Most applications remain research-stage
  • Asthma phenotyping and food allergy prediction are active research areas
  • AAAAI framework supports allergist involvement in AI development

Polygenic risk scores:

  • Cardiovascular and breast cancer PRS approaching clinical utility
  • Population transferability and equity are critical concerns
  • PRS should supplement, not replace, standard risk assessment

Rare disease diagnosis:

  • AI accelerates phenotype-to-gene matching
  • Facial analysis and HPO-based tools support diagnosis
  • Definitive diagnosis still requires genetic confirmation

References