Scleroderma (Systemic Sclerosis)
BASICS
- Definition: Chronic disease of unknown cause with diffuse fibrosis of skin/viscera and vascular abnormalities.
- Key Feature: Most manifestations have vascular features (Raynaud phenomenon); frank vasculitis is rare.
- Spectrum: Ranges from mild skin involvement to severe systemic disease (can be fatal within months).
- Variants:
- Diffuse: Proximal/distal extremity and truncal skin thickening.
- Limited: Confined to fingers, hands, and face; includes CREST syndrome (Calcinosis, Raynaud, Esophageal dysmotility, Sclerodactyly, Telangiectasia).
- Systems Affected: Skin, renal, cardiovascular, pulmonary, musculoskeletal, GI.
Special Populations
- Geriatrics: Uncommon >75 yrs.
- Pediatrics: Rare.
- Pregnancy: Higher risk of preterm birth; delay pregnancy until disease stable; diffuse form = greater risk of cardiopulmonary/renal complications.
EPIDEMIOLOGY
- Incidence (US): 1–5/100,000/year
- Prevalence (US): 1–25/100,000
- Onset: Peak 30–50 yrs (most in 3rd–5th decades)
- Sex: Female > male (4:1)
ETIOLOGY & PATHOPHYSIOLOGY
- Pathophysiology: Vascular and fibrotic processes; increased TGF-β, PDGF after endothelial activation.
- Trigger: Unknown (possible immune dysregulation, exposure to silica, vinyl chloride, hydrocarbons, bleomycin, rapeseed oil)
- Genetics: Rare familial clustering.
RISK FACTORS
- Unknown (possible environmental/occupational exposures).
DIAGNOSIS
HISTORY
- Raynaud phenomenon—usually first symptom
- Skin changes: Thickening, “puffy hands,” pruritus
- GERD symptoms: Early, often noted
PHYSICAL EXAM
- Skin: Digital ulcers, pitting, tightness, swelling, hyper-/hypopigmentation, narrowed mouth, calcinosis
- Vascular: Telangiectasia
- Musculoskeletal: Flexion contractures, friction rubs, swelling, stiffness, sclerodactyly, proximal weakness
- GI: Dysphagia, reflux, malabsorption, weight loss, xerostomia
- Renal: Hypertension, renal crisis (ARF)
- Pulmonary: Crackles at bases, dyspnea
- Neuro: Peripheral/trigeminal neuropathy
- Cardiac: Conduction defects, cardiomyopathy, pericarditis, cor pulmonale
DIFFERENTIAL DIAGNOSIS
- Mixed connective tissue/overlap syndromes
- Scleredema
- Nephrogenic systemic fibrosis
- Toxic oil syndrome
- Eosinophilia-myalgia syndrome
- Diffuse fasciitis with eosinophilia
DIAGNOSTIC TESTS & INTERPRETATION
Initial Tests
- Nailfold capillaroscopy: Drop-out significant
- Labs: CBC, creatinine, urinalysis, ANA (>90% positive), anti-Scl-70 (diffuse/systemic, risk for ILD), anticentromere (CREST/limited)
- Imaging: Chest X-ray (reticular pattern, pulmonary fibrosis), hand X-ray (soft tissue atrophy, acroosteolysis, calcinosis)
- Pulmonary Function: Reduced capacity, diffusion defect
- Antibodies: Anti-U3-RNP (risk for pulmonary HTN), anti-PM-Scl (myositis), anti-RNA pol III (diffuse/renal crisis)
Further Evaluation
- ECG: Low voltage, arrhythmia, conduction defects
- Echo: Pulmonary hypertension/cardiomyopathy
- Upper GI imaging: Distal esophageal dilatation/dysmotility
- HRCT Chest: Alveolitis, fibrosis (ground glass or lower lobe changes)
- Skin biopsy: Thick collagen, arteriolar fibrosis
- R heart cath: For pulmonary hypertension
TREATMENT
General
- Supportive, symptomatic.
- Esophageal dilation for strictures.
- Lifestyle: Avoid cold, dress warmly, avoid smoking, fingersticks, elevate bed for reflux, use emollients.
- Renal crisis: May require dialysis.
Medications
First Line
- ACE inhibitors: For renal crisis
- Dihydropyridine CCBs: Nifedipine/amlodipine for Raynaud
- Corticosteroids: Low dose only (for myositis, alveolitis); avoid high doses (renal risk)
- NSAIDs: Joint/tendon pain (caution with ACEI)
- Antibiotics: Secondary infection
- Antacids/PPIs: GERD
- Metoclopramide: GI dysmotility
- Hydrophilic ointments: Skin
- Topical abx/PDE5 inhibitors: For cutaneous ulcers
- Immunosuppressants: Cyclophosphamide for ILD/life-threatening disease
Additional Options
- Nitrates, PDE5 antagonists, prostanoids, endothelin-1 antagonists: For Raynaud/pulmonary HTN
- Immunosuppressants/alkylators: For alveolitis/ILD
- Physical therapy, heat therapy for joint function
Surgery/Procedures
- Gastroplasty: GERD correction
- Sympathectomy: Raynaud (limited role)
- Lung transplant: Pulmonary HTN/ILD
- Stem cell transplant: Rapidly progressive SSc
FOLLOW-UP & MONITORING
- Every 3–6 months: Skin, organ involvement, medication review
- Yearly: Echo, PFTs
DIET
- Adequate fluids, adjust diet to GI symptoms
PATIENT EDUCATION
- Stay active, avoid fatigue, report bruising/nonhealing wounds, smoking cessation, Scleroderma Federation: https://scleroderma.org/
PROGNOSIS
- Course: Incurable, variable; improvement possible.
- Poor prognosis: Cardiac, pulmonary, renal involvement early in course.
COMPLICATIONS
- Renal/respiratory failure, contractures, esophageal dysmotility, reflux, arrhythmia, bowel obstruction, cardiomyopathy, pulmonary hypertension, cancer risk, death.
ICD-10
- M34.2 Systemic sclerosis induced by drug/chemical
- M34.9 Systemic sclerosis, unspecified
- M34.0 Progressive systemic sclerosis
CLINICAL PEARLS
- Raynaud phenomenon often initial symptom.
- Skin thickening, puffy hands, and GERD common early findings.
- Proactive monitoring for pulmonary hypertension or ILD is essential.