What is the initial management for a patient with scleroderma?

Initial Management of Scleroderma (Systemic Sclerosis)

Mycophenolate mofetil (MMF) is the first-line treatment for systemic sclerosis with skin involvement, regardless of antibody status, to address both skin thickening and potential interstitial lung disease. 1

Immediate Baseline Screening Required

Before initiating treatment, complete the following essential evaluations:

• Pulmonary function tests (PFTs) with DLCO to detect subclinical interstitial lung disease, which is present in many patients at diagnosis 1
• High-resolution CT (HRCT) of the chest as the primary tool to diagnose ILD 1
• Echocardiogram to screen for pulmonary arterial hypertension 1
• Blood pressure monitoring to establish baseline for scleroderma renal crisis surveillance 1
• Modified Rodnan skin score (mRSS) assessment at 17 anatomical sites (fingers, hands, forearms, arms, chest, abdomen, thighs, legs, feet) with scores 0-3 at each site for a maximum of 51 points 2

First-Line Systemic Treatment Algorithm

Primary Option: Mycophenolate Mofetil

• MMF has surpassed cyclophosphamide as the initial treatment of choice and is the most commonly prescribed first-line drug for diffuse cutaneous SSc, with a mean mRSS improvement of -4.90 points 1
• MMF addresses both skin thickening and potential ILD progression simultaneously 1

Alternative Option: Methotrexate

• Methotrexate 25 mg per week is an alternative first-line treatment if MMF is not tolerated or if musculoskeletal involvement is predominant, with approximately 5-point improvement in mRSS 1
• For early diffuse SSc specifically, methotrexate may be considered for skin manifestations 3

Critical Treatment Caveat

Avoid corticosteroids as monotherapy - they are associated with substantial long-term morbidity in fibrotic lung disease and increase risk of scleroderma renal crisis in early diffuse cutaneous SSc 1. If steroids are used, patients must be carefully monitored for blood pressure and renal function 3.

Adjunctive Management for Raynaud Phenomenon

Since Raynaud phenomenon is often the earliest manifestation:

• Dihydropyridine calcium channel blockers (especially nifedipine) as first-line for Raynaud 1
• Phosphodiesterase 5 inhibitors or intravenous iloprost if calcium channel blockers are insufficient 1

Organ-Specific Initial Management

Gastrointestinal Involvement

• Proton pump inhibitors (PPI) should be used for prevention of gastro-oesophageal reflux, oesophageal ulcers and strictures 3
• Prokinetic drugs for symptomatic motility disturbances (dysphagia, GORD, early satiety, bloating, pseudo-obstruction) 3

Scleroderma Renal Crisis Prevention

• ACE inhibitors should be used in treatment of scleroderma renal crisis if it develops 3
• Monitor blood pressure closely, especially in patients on steroids 3

Monitoring Strategy on Initial Treatment

Serial Assessments Every 3-6 Months:

• PFTs to track forced vital capacity (FVC) 1
• mRSS at each visit to quantify skin improvement (minimal clinically important difference is 3.5-5.3 points) 2
• Repeat HRCT at defined intervals to assess fibrosis progression 1

Watch for Progression Indicators:

• Worsening dyspnea 1
• Declining FVC >10% 1
• New HRCT changes 1
• Rapid skin progression (rapid increase in mRSS) is a risk factor for scleroderma renal crisis 2

Second-Line Options if Disease Progresses

If disease progresses on MMF:

• Consider rituximab (anti-CD20) or tocilizumab (anti-IL-6) as second-line options 1
• If ILD is fibrotic and progressing despite immunosuppression, add nintedanib as antifibrotic therapy 1
• For refractory cases with advanced skin fibrosis, intravenous immunoglobulins (IVIG) have demonstrated potential benefit 4

High-Risk Features Requiring Aggressive Management

A high mRSS (>24) at baseline is associated with increased risk of mortality and lower rates of survival without progression 2. High skin scores or moderate scores with progressive ILD are criteria for considering autologous hematopoietic stem cell transplantation in patients with early diffuse cutaneous SSc 2.

Common Pitfalls to Avoid

• Do not delay treatment initiation - time to treatment affects response 5
• Do not use steroids as monotherapy - increases scleroderma renal crisis risk 1
• Do not underestimate skin disease - it is associated with significant physical and psychological morbidity 6
• Do not stop treatment prematurely - inadequate treatment duration leads to relapse 6