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Parkinson's Disease

Chapter 446 | Part 13: Neurologic Disorders · Part 13 – Neurologic Disorders · Chapter 446


Key Clinical Points

  1. Cardinal motor features of PD are bradykinesia, rest tremor, rigidity, and postural instability.
  2. Pathologic hallmark is the degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNc) and the presence of α-synuclein inclusions (Lewy bodies and Lewy neurites).
  3. α-synuclein pathology may follow a 'Braak hypothesis' progression from the periphery/gut via the vagus nerve to the brainstem and finally to the SNc.
  4. Genetic factors (e.g., SNCA, LRRK2, GBA1) influence age of onset and severity; the 'double-hit' hypothesis suggests an interaction between genetic susceptibility and environmental triggers.
  5. Nonmotor features (anosmia, sleep disorders like RBD, autonomic dysfunction) often precede motor symptoms by years or even decades.
  6. Atypical parkinsonisms (MSA, PSP, CBS) are characterized by early falls, poor levodopa response, and additional neurological signs (e.g., orthostatic hypotension in MSA, slow saccades in PSP).
  7. Secondary parkinsonism results from external factors such as drugs (neuroleptics), toxins (MPTP, manganese), or other conditions like Wilson's disease.
  8. Pimavenserin (34 mg daily) is FDA-approved for psychosis in Parkinson's Disease; clozapine is an alternative.
  9. Orthostatic hypotension is common and requires nonpharmacologic management as a first step.
  10. Levodopa-induced complications include 'wearing off' and dyskinesias, which typically progress over time during chronic treatment.

DEFINITION & OVERVIEW

Definition: Parkinson's disease (PD) is the second most common age-related neurodegenerative disease, exceeded only by Alzheimer's disease (AD). • Cardinal Motor Features: ◦ Bradykinesia (slowing) ◦ Rest tremor ◦ Rigidity (stiffness) ◦ Gait dysfunction with postural instability • Pathologic Hallmarks: ◦ Degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNc) ◦ Reduced striatal dopamine ◦ Intraneuronal proteinaceous inclusions (Lewy bodies and Lewy neurites) that stain for α-synuclein. • Systemic Involvement: ◦ Neuronal degeneration with Lewy pathology can also affect: ◦ Cholinergic neurons of the nucleus basalis of Meynert (NBM) ◦ Norepinephrine neurons of the locus coeruleus (LC) ◦ Serotonin neurons in the raphe nuclei ◦ Olfactory system, cerebral hemispheres, spinal cord, and peripheral autonomic nervous system.


EPIDEMIOLOGY

Global Burden: ◦ Approximately 10.8 million people currently affected. ◦ Expected to double within 20 years due to the aging population. • Demographics: ◦ Mean age of onset: ~60 years. ◦ Lifetime risk: ~3% for men, ~2% for women. ◦ Early-onset cases (even in the 20s) are often associated with pathogenic gene mutations.


ETIOLOGY & PATHOPHYSIOLOGY

Genetic Factors: ◦ ~15% of cases are familial. ◦ Key genes: SNCA, LRRK2, MAPT, GBA1. ◦ 'Double-hit' hypothesis: Interaction between (1) genetic risk factors inducing susceptibility and (2) exposure to toxic environmental factors. Note: No direct evidence currently supports this theory for clinical use. • Environmental Factors: ◦ Potential risks: Pesticides, solvents, rural living, farming, and drinking well water (results are inconsistent). ◦ Protective factors: Caffeine, cigarette smoking, NSAIDs, and calcium channel blockers. • α-Synuclein Pathology: ◦ SNCA gene is the first linked to PD; mutations or duplications/triplications of wild-type SNCA cause PD. ◦ Prion-like model: α-synuclein misfolds → forms toxic oligomers → aggregates into Lewy bodies → spreads to adjacent neurons. ◦ Braak hypothesis: Pathology may begin in the GI tract → travel via vagus nerve → lower brainstem (dorsal motor nucleus of the vagus) → SNc.


CLINICAL FEATURES

Cardinal Motor Features: ◦ Bradykinesia, rest tremor, rigidity, and postural instability. • Other Motor Features: ◦ Micrographia, masked facies (hypomimia), reduced eye blinking, drooling, soft voice (hypophonia), dysphagia, freezing, and falling. • Nonmotor Features: ◦ Sensory: Anosmia, pain, hyposmia. ◦ Mood/Sleep: Depression, anxiety, apathy; fragmented sleep, REM sleep behavior disorder (RBD). ◦ Autonomic: Orthostatic hypotension, gastrointestinal disturbances, genitourinal/sexual dysfunction. • Pre-motor Phase: ◦ Nonmotor symptoms and specific pathologies (e.g., in the olfactory system or autonomic nervous system) may precede motor features by years.


DIFFERENTIAL DIAGNOSIS

Atypical Parkinsonism: ◦ Multiple-system atrophy (MSA): Characterized by prominent orthostatic hypotension. ◦ Progressive supranuclear palsy (PSP): Characterized by slow saccades with impaired downgaze. ◦ Corticobasal syndrome (CBS). ◦ Features: Early speech/gait impairment, lack of tremor, no motor asymmetry, poor levodopa response. • Secondary Parkinsonism: ◦ Drug-induced (e.g., neuroleptics). ◦ Toxins (e.g., MPTP, manganese, carbon monoxide). ◦ Other: Tumor, infection, vascular, Wilson's disease, NPH. • Clinical Indicators for Differentiation: ◦ Early speech/gait impairment → Atypical parkinsonism ◦ Onset <40 years → Genetic form, Wilson's, or DRD ◦ Hallucinations/dementia preceding motor features → Dementia with Lewy bodies (DLB) ◦ Poor response to levodopa → Atypical or secondary parkinsonism.


INVESTIGATIONS & DIAGNOSIS

  1. Clinical Assessment: ◦ Identify core features: Bradykinesia, rigidity, tremor. ◦ Assess for 'Red Flags': Early falls, lack of asymmetry, and rapid progression.
  2. MDS Clinical Diagnostic Criteria: ◦ Step 1: Confirm motor parkinsonism. ◦ Step 2: Evaluate supporting criteria (e.g., levodopa response, specific nonmotor features). ◦ Step 3: Rule out exclusion criteria (drug-induced, secondary causes). ◦ Step 4: Determine if condition is 'clinically established' or 'clinically probable'.
  3. Imaging & Biomarkers: ◦ [11C]Dihydrotetrabenazine PET: Used as a marker of VMAT2 density to assess presynaptic dopaminergic nerve terminals. ◦ Clinical observation of levodopa response: Key for distinguishing PD from atypical forms.

MANAGEMENT & TREATMENT

  1. Pharmacologic Treatment for Psychosis: ◦ First-line: Pimavenserin (34 mg daily). ◦ Alternative: Clozapine.
  2. Management of Autonomic Dysfunction: ◦ Orthostatic hypotension → prioritize nonpharmacologic measures first.
  3. Cognitive Symptoms in DLB: ◦ Use Cholinesterase inhibitors (rivastigmine, donepezil).
  4. Levodopa Therapy & Monitoring: ◦ Initial: Carbidopa/levodopa (200–1000 mg levodopa/day). ◦ Add-on for 'off' periods: Entacapone (200 mg with each dose), Tolcapone (100–200 mg tid), or Opicapone (50 mg HS). ◦ On-demand therapy: Apomorphine sublingual strip (5–40 mg, up to 5 doses per day).
  5. Supportive Care: ◦ Physical therapy and safety measures for gait/falling.

GENETIC SUBTYPES

Dominantly Inherited PD: ◦ SNCA: Median AAO 46y (19–77); features include early-onset, severe parkinsonism with cognitive dysfunction. ◦ LRRK2: Median AAO 56y (20–95); clinically typical PD with slightly slower progression. ◦ VPS35: Median AAO 52y (26–75); very rare form of PD. ◦ CHCHD2: Likely clinically typical PD; predominantly found in Asia. ◦ RAB32: Likely clinically typical, possibly more frequent dementia. ◦ GBA1: Clinically overall typical; however, faster progression and greater risk of cognitive impairment. • Recessively Inherited PD: ◦ PRKN: Median AAO 31y (3–81); often presents with dystonia in a leg. ◦ PINK1: Median AAO 32y (9–67); prominent psychiatric features reported. ◦ PARK7: Median AAO 27y (15–40); clinically similar to PRKN and PINK1.


KEY PEARLS & CLINICAL TRAPS

Diagnostic Clues: ◦ Lack of tremor + early falls → suspect Atypical Parkinsonism. ◦ Presence of orthostatic hypotension → suggests MSA. ◦ Slow saccades/impaired downgaze → suggest PSP. ◦ REM sleep behavior disorder (RBD) → prodromal marker for synucleinopathies. • Treatment Pitfalls: ◦ Avoid neuroleptics in patients with parkinsonism to prevent worsening of motor symptoms. ◦ Recognize that 'wearing off' and dyskinesias are expected complications of long-term levodopa use.


Reference Tables

TABLE 446-1 Clinical Features of Parkinson’s Disease

Harrison's 22e, p.3494

CARDINAL MOTOR
FEATURES
OTHER MOTOR
FEATURES
NONMOTOR FEATURES
Bradykinesia
Rest tremor
Rigidity
Postural instability
Micrographia
Masked facies
(hypomimia)
Reduced eye blinking
Drooling
Soft voice (hypophonia)
Dysphagia
Freezing
Falling
Anosmia
Sensory disturbances
(e.g., pain, hyposmia)
Mood disorders
(e.g., depression, anxiety, apathy)
Sleep disturbances
(e.g., fragmented sleep, RBD)
Autonomic disturbances
Orthostatic hypotension
Gastrointestinal disturbances
Genitourinal disturbances
Sexual dysfunction
Cognitive impairment/dementia

TABLE 446-2 Differential Diagnosis of Parkinsonism Parkinson’s disease

Harrison's 22e, p.3495

Parkinson’s disease
Sporadic
Genetic
PD with dementia/dementia
with Lewy bodies
Atypical parkinsonism
Multiple-system atrophy (MSA)
Cerebellar type (MSA-c)
Parkinson type (MSA-p)
Progressive supranuclear palsy
Parkinsonian variant
Richardson variant
Corticobasal syndrome
Secondary parkinsonism
Drug-induced
Tumor
Infection
Vascular
Normal-pressure hydrocephalus
Trauma
Liver failure
Toxins (e.g., carbon monoxide, manganese,
MPTP, cyanide, hexane, methanol, carbon
disulfide)
Other neurodegenerative disorders associated with
parkinsonism
Wilson’s disease
Huntington’s disease
Neurodegeneration with brain iron accumulation
SCA 3 (spinocerebellar ataxia)
Fragile X–associated ataxia-tremor-parkinsonism
Prion diseases
X-linked dystonia-parkinsonism
Alzheimer’s disease with parkinsonism
Dopa-responsive dystonia

TABLE 446-3 Features Suggesting an Atypical or Secondary Cause of Parkinsonism SYMPTOMS/SIGNS History Early speech and…

Harrison's 22e, p.3497

SYMPTOMS/SIGNS ALTERNATIVE DIAGNOSIS TO
CONSIDER
History
Early speech and gait impairment (lack
of tremor, lack of motor asymmetry,
early falls)
Atypical parkinsonism
Onset prior to age 40 years Genetic form of PD, Wilson’s disease,
DRD
Hallucinations and dementia which
precede the development of PD features
Dementia with Lewy bodies
Poor or no response to an adequate trial
of levodopa
Atypical or secondary parkinsonism
Physical Examination
Prominent orthostatic hypotension MSA
Slow saccades with impaired downgaze PSP

TABLE 446-4 Confirmed Genetic Causes of Parkinson’s Disease (PD) with a Clinical Presentation Similar to Idiopathic PD…

Harrison's 22e, p.3498

DESIGNATIONa
AND REFERENCE
GENEREVIEWS AND OMIM REFERENCE CLINICAL CLUESB COMMENTS
Dominantly Inherited PD
PARK-SNCA GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1223/
OMIM 168601
Median AAO: 46 years (range 19–77 years); 25th/75th
percentile: 36/54 years. Gene duplications cause
classical PD. Most missense mutations and triplications
cause early-onset, severe parkinsonism with prominent
cognitive dysfunction
Very rare form of PD, α-synuclein
protein main component of Lewy
bodies, the pathological hallmark
of PD
GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1208/
OMIM 607060
Median AAO: 56 years (range 20–95 years); 25th/75th
percentile: 47/64 years. Clinically typical PD with slightly
slower progression
PARK-VPS35 GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1223/
OMIM 616710
Median AAO: 52 years (range 26–75 years); 25th/75th
percentile: 45/61 years. Clinically typical PD
Very rare form of PD
GeneReviews
N/A
OMIM 614203
Likely clinically typical PD. Systematic MDSGene review
not yet available
PARK-RAB32 GeneReviews
N/A
OMIM 612906 (disease link not yet included)
Likely clinically typical PD, possibly more frequent
dementia. Systematic MDSGene review not yet available
Most recently found form of PD. All
currently identified patients and
families carry the same founder
pathogenic variant
GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1223/
OMIM 168600/606463
Clinically overall typical PD; however, faster progression
and greater risk of cognitive impairment. Systematic
MDSGene review not yet available
Recessively Inherited PD
PARK-PRKN GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1155/
OMIM 600116
Median AAO: 31 years (range 3–81 years); 25th/75th
percentile: 23/38 years. Often presents with dystonia,
typically in a leg
Most common early-onset form of
genetic PD. Protein name: Parkin
GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1223/
OMIM 605909
Median AAO: 32 years (range 9–67 years); 25th/75th
percentile: 24/40 years. Prominent psychiatric features
have been described in several families
PARK-PARK7 GeneReviews
http://www.ncbi.nlm.nih.gov/books/NBK1223/
OMIM 606324
Median AAO: 27 years (range 15–40 years); 25th/75th
percentile: 22/34
Clinically very similar to PARK-PRKN
and PARK-PINK1, but rarest of all
forms. Protein name: DJ-1

TABLE 446-5 Drugs Commonly Used for Treatment of Parkinson’s Disease a AGENT Levodopa a

Harrison's 22e, p.3504

AGENT AVAILABLE DOSAGES TYPICAL DOSING
Levodopaa
Carbidopa/levodopa 10/100, 25/100, 25/250 mg 200–1000 mg
levodopa/day
Benserazide/levodopa 25/100, 50/200 mg
Carbidopa/levodopa CR 25/100, 50/200 mg
Benserazide/levodopa
MDS
25/200, 25/250 mg
Parcopa 10/100, 25/100, 25/250 mg
Rytary (carbidopa/
levodopa)
Carbidopa/levodopa/
entacapone
23.75/95, 36.25/145,
48.75/195, 61.25/245
12.5/50/200, 18.75/75/200,
25/100/200, 31.25/125/200,
37.5/150/200, 50/200/200 mg
See conversion
tables
0.125, 0.25, 0.5, 1.0, 1.5 mg
0.375, 0.75, 1.5. 3.0, 4.5 mg
0.25, 0.5, 1.0, 3.0 mg
2, 4, 6, 8 mg
2-, 4-, 6-, 8-mg patches
2–8 mg
COMT inhibitors
Entacapone 200 mg 200 mg with each
levodopa dose
Tolcapone
Opicapone
100, 200 mg
50 mg
100–200 mg tid
50 mg HS
5 mg
0.5, 1.0 mg
100 mg
On-demand therapy for off
periods
Inhaled levodopa
Apomorphine sublingual
strip
5–40 mg Up to 5 doses per day
Up to 5 doses per day
20, 40 mg
100–400 mg