What is the treatment for Behcet's disease?

Treatment of Behçet's Disease

Treatment of Behçet's disease must be stratified by organ involvement: mucocutaneous and joint disease requires symptom-based therapy with colchicine as first-line, while sight-threatening eye disease, vascular, neurological, or gastrointestinal involvement demands immediate aggressive immunosuppression with high-dose corticosteroids plus azathioprine to prevent irreversible organ damage and death. 1, 2

Treatment Algorithm by Organ System

Mucocutaneous and Joint Disease

For oral and genital ulcers:

• Start with topical corticosteroids for symptomatic relief 1
• Initiate colchicine 1-2 mg/day as first-line systemic therapy, which is particularly effective for erythema nodosum and genital ulcers, especially in women 1, 2
• Colchicine has proven efficacy in two randomized controlled trials specifically for arthritis and erythema nodosum 2
• For acute monoarticular arthritis, use intra-articular corticosteroids 1
• For refractory oral ulcers, apremilast is FDA-approved and represents a safe and effective option 3

These manifestations impair quality of life but do not cause permanent organ damage, so treatment can be tailored according to quality of life impact versus medication risks 1, 4

Ocular Involvement (Sight-Threatening)

Any patient with posterior segment eye disease requires mandatory aggressive treatment:

• Immediately start high-dose glucocorticoids combined with azathioprine as mandatory first-line therapy 1, 2
• For acute sight-threatening exacerbations or severe disease, add either infliximab or interferon-alpha to the azathioprine-corticosteroid combination 1, 2
• Cyclosporine A can be added to azathioprine-corticosteroid combination for severe eye disease, but only if there is no history of neurological involvement 2

The goal is to prevent blindness, which historically occurred in 75% of affected eyes but has been reduced to 20% with proper aggressive management 5

Neurological Involvement (Parenchymal Disease)

For acute parenchymal Neurobehçet's disease:

• Initiate high-dose intravenous methylprednisolone followed by oral prednisolone 2
• Combine with azathioprine 2.5 mg/kg/day as the preferred steroid-sparing agent from the outset 1, 2
• For severe or refractory disease failing conventional therapy, escalate to TNF-alpha inhibitors, particularly infliximab 1, 2

Critical pitfall: Never use cyclosporine A in patients with any history of CNS involvement, even if inactive, due to significant neurotoxicity risk 1, 2

Vascular Involvement

For acute deep vein thrombosis:

• Use mandatory treatment with glucocorticoids combined with immunosuppressives (azathioprine, cyclophosphamide, or cyclosporine A) 1, 2
• Do NOT use anticoagulation, antiplatelet agents, or antifibrinolytic agents for venous thrombosis in Behçet's disease 2
• For refractory venous thrombosis, escalate to monoclonal anti-TNF antibodies 1

For pulmonary and peripheral arterial aneurysms:

• Use cyclophosphamide combined with corticosteroids 2
• Do NOT anticoagulate pulmonary artery aneurysms due to bleeding risk 1

Gastrointestinal Involvement

For acute exacerbations:

• Use glucocorticoids for rapid ulcer healing 1
• Add disease-modifying agents such as 5-ASA or azathioprine 1
• Before considering surgery (except emergencies), trial sulfasalazine, corticosteroids, azathioprine, TNF-alpha antagonists, or thalidomide sequentially 2
• For severe or refractory disease, use monoclonal anti-TNF antibodies and/or thalidomide 1

High-Risk Populations Requiring Aggressive Treatment

Young males with early disease onset require more aggressive immunosuppression and closer monitoring due to higher risk of severe disease and major organ involvement 2, 4

Refractory Disease Management

For patients failing conventional immunosuppression across any organ system:

• TNF-alpha inhibitors (infliximab or adalimumab) represent the next therapeutic step 2, 6
• Infliximab combined with azathioprine shows particular promise for severe or refractory cardiac involvement 2
• Emerging options for refractory disease include IL-1 inhibitors, ustekinumab, secukinumab, and tocilizumab 3

Long-Term Management and Tapering

Disease manifestations typically ameliorate over time, allowing for treatment tapering and potential discontinuation in stable patients 1, 2, 4

Regular monitoring of inflammatory markers and organ-specific assessments remains essential even during remission 2

Critical Pitfalls to Avoid

• Never delay immunosuppression in major organ involvement, as this results in permanent damage 1
• Never use cyclosporine A in patients with any neurological history 1, 2
• Never anticoagulate pulmonary artery aneurysms or use anticoagulation for venous thrombosis in Behçet's disease 1, 2
• Never undertreat chronic scarring ulcers, as this causes irreversible anatomical damage including oropharyngeal narrowing and obliterative genital scarring 1