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Basic Neuroscience · Neuropathology

Autoantibodies in Neurology

Diagnostic autoantibodies in neurological disease, grouped by category. Each lists the associated disorder(s) and the target antigen with key clinical pearls.

AntibodyDiseaseTarget / Description
Demyelinating CNS
AQP4 AbNeuromyelitis optica (NMO)Aquaporin-4.
MOG AbChildhood MS, ADEM, AQP4-negative NMO, AQP4-negative optic neuritisMyelin oligodendrocyte glycoprotein.
Neuromuscular junction & muscle
AChR AbMyasthenia gravisAcetylcholine-receptor antibody. Positive in 85% of myasthenia patients.
MuSK AbMyasthenia gravisMuscle-specific kinase antibody → inhibits AChR clustering at the motor end plate. Positive in ~50% of AChR-negative patients. More common in women and African Americans; no eye involvement, more neck and bulbar involvement; less responsive to anticholinesterase medications or thymectomy.
LRP4 AbMyasthenia gravisLDL receptor-related protein 4 — a receptor for neural agrin that activates MuSK. Positive in ~9% of double-seronegative patients (AChR- and MuSK-negative).
Striational Ab (RyR / Titin Ab)Myasthenia gravisAgainst striated-muscle proteins (titin and ryanodine receptor). Present only in AChR-positive myasthenia, usually elderly (>60) and patients with thymoma. Sensitive but not specific for thymoma (50% of positives have thymoma; 95% of thymoma patients have titin Ab). Associated with a more severe course; respond to calcineurin inhibitors (tacrolimus, cyclosporine). Anti-RyR can react against both skeletal RyR1 and cardiac RyR2 receptors.
VGCC AbLambert-Eaton myasthenic syndrome (LEMS)Positive in 90% of LEMS. Associated with SCLC — screen with CXR every 6 months for lung cancer.
GADStiff-person syndrome; SPS-plus (PERM)Glutamic acid decarboxylase.
Glycine receptor AbStiff-person syndrome-plus / "PERM" (progressive encephalomyelitis with rigidity and myoclonus)Against the glycine receptor.
Channels / surface antigens
VGKC (CASPR2)Isaacs syndrome (neuromyotonia); Morvan syndrome (neuromyotonia, encephalopathy, hyperhidrosis); limbic encephalitisContactin-associated protein-2.
VGKC (LGI-1)Limbic encephalitisLeucine-rich, glioma-inactivated protein 1. Brief faciobrachial dystonic seizures, memory loss, disorientation, hyponatremia in 60%. CSF lymphocytosis and oligoclonal bands in 50%.
NMDANMDA-receptor encephalitisPsychiatric features, cognitive dysfunction, seizures. May be associated with ovarian teratoma — obtain pelvic MRI.
DPPXDPPX-associated encephalitisDipeptidyl-peptidase-like protein 6, a peptide related to VGKC responsible for attenuation of back-propagation of action potentials. Starts with diarrhea and weight loss, followed by CNS hyperexcitability (hyperekplexia, myoclonus, seizures) over a few months.
Onconeuronal (paraneoplastic) antibodies
AmphiphysinStiff-person syndrome (paraneoplastic)SCLC & breast cancer. Protein present on the cytoplasmic surface of synaptic vesicles.
Hu (ANNA-1)Encephalomyelitis (limbic, brainstem, cortical, myelitis); sensory peripheral neuronopathy; cerebellar degenerationSCLC & neuroblastoma. Directed against a neuronal nuclear protein (present in all neurons).
YoCerebellar degenerationOvarian, endometrial & breast cancer.
Ri (ANNA-2)Cerebellar degeneration; opsoclonusOvarian, endometrial & breast cancer. Directed against the NOVA protein. Most common cause of opsoclonus in adults: anti-Hu, Ri, Yo (SCLC & breast). In children: neuroblastoma, usually with negative anti-Hu/Ri/Yo.
Ma2Cerebellar degeneration; limbic encephalitis; stiff-person syndromeTesticular tumors.
CV2 (CRMP5)Cerebellar degeneration; limbic encephalitis; peripheral neuropathySCLC, thymoma & uterine sarcoma.
Glycoproteins / gangliosides
GQ1bMiller-Fisher syndrome; GBS with ophthalmoplegia; Bickerstaff encephalitis; pharyngo-cervical-brachial GBSPositive in 90% of patients with Miller-Fisher syndrome.
GM1AMAN; AMSAN; multifocal motor neuropathy (MMN)Associated with axonal variants of GBS.
GD1bPure sensory variant of GBSAgainst gangliosides on sensory neurons in the dorsal root ganglia.
MAGAnti-MAG neuropathy (chronic sensorimotor demyelinating neuropathy); multiple sclerosis; SLE; MGUS / WaldenströmMyelin-associated glycoprotein (present in peripheral and central myelin). Present in 50% of patients with monoclonal gammopathy (MGUS or Waldenström) and peripheral neuropathy → test for MAG in MGUS/Waldenström patients with neuropathy.
Other
Endothelial cell AbSusac syndromeTriad of encephalopathy, branch retinal artery occlusion, and hearing loss.
SSA (Ro) / SSB (La)Sjögren syndromeAxonal neuropathy (pure sensory or sensorimotor), sensory ganglionopathy, small-fiber neuropathy, autonomic neuropathy. SSA/SSB are positive in only 40% of Sjögren patients presenting with neurological disease.

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