What is the initial management for patients with scleroderma?

Initial Management of Scleroderma (Systemic Sclerosis)

The initial management of scleroderma requires immediate baseline organ screening followed by mycophenolate mofetil as first-line systemic therapy for patients with diffuse cutaneous disease and skin thickening, while simultaneously addressing Raynaud's phenomenon with calcium channel blockers. 1

Immediate Baseline Screening (Before Starting Treatment)

All newly diagnosed scleroderma patients require comprehensive organ assessment to detect subclinical involvement and guide treatment decisions:

• Pulmonary function tests with DLCO to detect interstitial lung disease (ILD), which may be present even without symptoms 1
• High-resolution CT chest as the primary tool to diagnose ILD and establish baseline fibrosis 1
• Echocardiogram to screen for pulmonary arterial hypertension 1
• Blood pressure monitoring to establish baseline for scleroderma renal crisis surveillance 1
• Skin assessment using modified Rodnan skin score (mRSS) to quantify baseline skin thickening 1

First-Line Systemic Therapy

For Diffuse Cutaneous Systemic Sclerosis with Skin Thickening:

Mycophenolate mofetil (MMF) is the preferred first-line treatment, having surpassed cyclophosphamide as the initial drug of choice, with mean mRSS improvement of -4.90 points 1. MMF addresses both skin thickening and potential ILD simultaneously 1.

Alternative first-line option: Methotrexate 25 mg per week if MMF is not tolerated or if musculoskeletal involvement is predominant, with approximately 5-point improvement in mRSS 1. For early diffuse disease specifically, methotrexate at 10-15 mg/week has shown efficacy in reducing skin scores 2.

Critical Caveat - Avoid Corticosteroid Monotherapy:

Corticosteroids should NOT be used as monotherapy because they are associated with substantial long-term morbidity in fibrotic lung disease and significantly increase the risk of scleroderma renal crisis in early diffuse cutaneous disease 1. If steroids are necessary, patients must be carefully monitored for blood pressure and renal function 2.

Organ-Specific Initial Management

Raynaud's Phenomenon (Present in Nearly All Patients):

• Dihydropyridine calcium channel blockers (nifedipine) as first-line therapy to reduce frequency and severity of attacks 2, 1
• Intravenous iloprost or phosphodiesterase-5 inhibitors if calcium channel blockers are insufficient 2, 1

Digital Ulcers:

• Intravenous prostanoids (particularly iloprost) for active digital ulcers 2
• Bosentan should be considered for patients with multiple digital ulcers after failure of calcium antagonists and prostanoid therapy 2

Gastrointestinal Involvement:

• Proton pump inhibitors (PPIs) for prevention of gastro-esophageal reflux, esophageal ulcers, and strictures 2
• Prokinetic drugs for symptomatic motility disturbances (dysphagia, early satiety, bloating) 2

Interstitial Lung Disease (If Detected on Screening):

• Cyclophosphamide should be considered based on two high-quality RCTs showing improvement in lung function tests, dyspnea score, and quality of life, despite known toxicity 2
• MMF is increasingly preferred as first-line for ILD given better tolerability profile 1

Scleroderma Renal Crisis (If Develops):

• ACE inhibitors must be used immediately despite lack of RCTs, based on expert consensus and retrospective data showing mortality benefit 2

Monitoring Strategy on Initial Therapy

• Serial PFTs every 3-6 months to track forced vital capacity (FVC) 1
• mRSS at each visit to quantify skin improvement 1
• Watch for progression indicators: worsening dyspnea, declining FVC >10%, or new HRCT changes 1
• Monitor for MMF or methotrexate side effects: nausea, headache, transient hepatotoxicity 3

Common Pitfalls to Avoid

• Using corticosteroids as monotherapy increases scleroderma renal crisis risk 1
• Inadequate treatment duration - continue therapy for at least 12 months after achieving clinical improvement before tapering 3, 1
• Failing to screen for subclinical organ involvement before symptoms develop 1
• Underestimating the importance of aggressive early treatment of skin disease, which is associated with significant physical and psychological morbidity 3