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  5. /Autoimmune Pathophysiology: Molecular
CNPLE·Canada·Advanced practice
GeneralNPCanada exam scope

Canadian NP licensure (CNPLE track)

Autoimmune Pathophysiology: Molecular

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Pathophysiology

Clinical meaning

Autoimmunity results from the failure of self-tolerance — the immune system's ability to distinguish self from non-self. Central tolerance eliminates autoreactive T and B cells during development (negative selection in the thymus for T cells, bone marrow for B cells), but some autoreactive cells escape. Peripheral tolerance mechanisms (regulatory T cells [Tregs], anergy, deletion, immune checkpoint molecules) normally suppress these escapees. Autoimmune disease occurs when genetic susceptibility + environmental triggers overwhelm tolerance mechanisms. Molecular mechanisms: (1) Molecular mimicry — pathogen antigens share structural similarity with self-antigens, causing cross-reactive immune responses (Group A Strep M protein mimics cardiac myosin → rheumatic heart disease; Campylobacter lipooligosaccharides mimic gangliosides → Guillain-Barré syndrome). (2) Epitope spreading — initial immune response against one antigen damages tissue, exposing previously sequestered self-antigens to the immune system, broadening the autoimmune attack (occurs in MS, SLE). (3) Bystander activation — non-specific inflammation at a tissue site activates autoreactive T cells that happen to be present (viral infections causing type 1 diabetes). (4) Defective Tregs — FOXP3+ regulatory T cells normally suppress autoreactive effectors; loss of Treg function (IPEX syndrome = FOXP3 mutation) causes multi-organ autoimmunity. (5) Immune checkpoint failure — CTLA-4 and PD-1 are inhibitory receptors that prevent excessive immune activation; polymorphisms increase autoimmune risk; therapeutic checkpoint inhibitors (pembrolizumab, nivolumab) can trigger autoimmune adverse effects (thyroiditis, colitis, hepatitis). HLA associations: HLA-B27 → ankylosing spondylitis, reactive arthritis; HLA-DR4 → rheumatoid arthritis; HLA-DQ2/DQ8 → celiac disease; HLA-DR3/DR4 → type 1 DM. Pathogenic autoantibodies: anti-dsDNA (SLE nephritis), anti-Smith (SLE — highly specific), anti-CCP (RA — more specific than RF), anti-SSA/SSB (Sjögren), anti-centromere (limited scleroderma/CREST), anti-Scl-70 (diffuse scleroderma), anti-Jo-1 (polymyositis/dermatomyositis with ILD).

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Diagnosis & workup

Diagnostics & workup: - ANA (antinuclear antibody): screening test — positive in SLE (>95%), drug-induced lupus, Sjögren, scleroderma; but also positive in 5-15% of healthy individuals (low specificity alone) - ANA pattern on IF: homogeneous (anti-dsDNA, drug-induced lupus), speckled (anti-Smith, anti-RNP, anti-SSA/SSB), nucleolar (scleroderma), centromere (limited scleroderma/CREST) - Anti-dsDNA: highly specific for SLE; correlates with disease activity and nephritis - Anti-CCP (cyclic citrullinated peptide): highly specific for RA (>95%); more specific than RF; positive early in disease - Complement levels (C3, C4): LOW in active SLE (consumed by immune complex formation); normal in RA, scleroderma - ESR and CRP: nonspecific inflammatory markers; CRP typically NOT elevated in SLE flares (important distinction from infection)

Risk factors: - Female sex (autoimmunity is 2-10× more common in women — estrogen enhances immune responses, testosterone is immunosuppressive) - HLA susceptibility genes (strongest genetic associations: HLA-B27, HLA-DR4, HLA-DQ2/DQ8) - Environmental triggers: infections (molecular mimicry), UV exposure (SLE flares), smoking (RA, increased anti-CCP), medications (drug-induced lupus: hydralazine, procainamide, isoniazid) - Family history of autoimmune disease (clustering — patients often have multiple autoimmune conditions) - Vitamin D deficiency (vitamin D has immunomodulatory effects; deficiency associated with MS, T1DM, RA) - Checkpoint inhibitor therapy (pembrolizumab, nivolumab, ipilimumab — releases immune tolerance, causing autoimmune adverse effects)

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