nintedanib and Scleroderma--Limited

You are consulted to evaluate a 56-year-old woman with known Raynaud's phenomenon, finger swelling of several; months' duration, and new hypertension with a blood pressure of 160/100 mm/Hg. She also reports progressive shortness of breath. Physical examination reveals telangiectasias, sclerodactyly, and proximal skin sclerosis (thick shiny skin on the chest and upper arms), and bibasilar crackles are found on chest examination. Laboratory tests reveal evidence of microangiopathic hemolytic anemia, thrombocytopenia, and elevation of the serum creatinine level (previously normal), and chest computed tomography shows evidence of ground-glass opacification in both lower lung fields.

Topics: Adrenergic alpha-Antagonists; Angiotensin Receptor Antagonists; Angiotensin-Converting Enzyme Inhibitors; Antibodies, Antinuclear; Antibodies, Monoclonal, Humanized; Antihypertensive Agents; Antirheumatic Agents; Calcium Channel Blockers; Complement C3; Complement C4; Complement Inactivating Agents; Cyclophosphamide; Endothelin Receptor Antagonists; Humans; Hypertension; Indoles; Lung Diseases, Interstitial; Plasma Exchange; Protein Kinase Inhibitors; Raynaud Disease; Renal Insufficiency; RNA Polymerase III; Scleroderma, Diffuse; Scleroderma, Limited; Stem Cell Transplantation; Tomography, X-Ray Computed; Transplantation, Autologous; Vasodilator Agents

Systemic sclerosis is a connective tissue disease characterized by progressive skin thickening and a wide spectrum of internal organ involvement. Pathogenesis includes vasculopathy, inflammation, and fibrosis. Although immunosuppressants such as cyclophosphamide and mycophenolate mofetil have shown some benefit in interstitial lung disease management, it is still a major cause of morbi-mortality in these patients. Therefore, there is a current need for new therapies. Here, we report a 65-year-old female patient with limited cutaneous systemic sclerosis, anti-topoisomerase-positive and extensive lung disease. The patient developed progressive lung fibrosis under several immunosuppressants and was started on nintedanib, with clinical and functional stabilization. Nintedanib is a tyrosine-kinase inhibitor that blocks several profibrotic pathways, inhibiting proliferation and migration of fibroblasts and decreasing the synthesis of extracellular matrix proteins. It is approved for idiopathic lung fibrosis and has demonstrated good results in inhibiting migration and proliferation of systemic sclerosis dermal fibroblasts, constituting a promising agent for systemic sclerosis-associated lung fibrosis.

Topics: Aged; Female; Humans; Indoles; Lung Diseases, Interstitial; Protein Kinase Inhibitors; Scleroderma, Limited; Treatment Outcome