What is Goodpasture syndrome (Anti-GBM disease)?

From the Guidelines

Goodpasture syndrome, also known as Anti-GBM disease, is a rare autoimmune disorder that requires immediate treatment with a combination of plasmapheresis, glucocorticoids, and immunosuppressive medications, such as cyclophosphamide, to prevent permanent kidney and lung damage. This condition occurs when the immune system mistakenly produces antibodies against type IV collagen, which is found in the basement membranes of the lungs and kidneys 1. The symptoms of Goodpasture syndrome include coughing up blood (hemoptysis), difficulty breathing, fatigue, and signs of kidney dysfunction like blood in urine and decreased urine output.

Key Characteristics of Goodpasture Syndrome

• Rare autoimmune disorder
• Antibodies attack the basement membrane of lungs and kidneys
• Requires prompt recognition and early treatment to prevent kidney failure and high mortality rate 1
• Treatment typically involves plasmapheresis, glucocorticoids, and immunosuppressive medications, such as cyclophosphamide 1

Treatment Approach

• Initiate immunosuppression with cyclophosphamide and glucocorticoids plus plasmapheresis in all patients with anti-GBM GN, except those who are treated with dialysis at presentation, have 100% crescents or > 50% global glomerulosclerosis in an adequate biopsy sample, and do not have pulmonary hemorrhage 1
• Plasmapheresis removes harmful antibodies from the blood
• Glucocorticoids, such as prednisone, reduce inflammation
• Immunossuppressants, such as cyclophosphamide, prevent further antibody production

Importance of Early Diagnosis and Treatment

• Early diagnosis and treatment are crucial to prevent permanent kidney and lung damage
• Without prompt treatment, Goodpasture syndrome can rapidly progress to kidney failure and severe respiratory distress, potentially becoming life-threatening 1

From the Research

Definition and Characteristics

• Goodpasture syndrome, also known as Anti-GBM disease, is a rare clinical entity characterized by rapidly progressive glomerulonephritis, diffuse pulmonary hemorrhage, and the presence of circulating autoantibodies to the glomerular basement membrane (GBM) 2.
• Autoantibodies bind to reactive epitopes of the noncollagenous domain of the collagen type IV alpha-3 chain in glomerular and alveolar basement membranes, activating the complement cascade and resulting in tissue injury by the type II hypersensitivity reaction 2, 3.

Clinical Presentation and Prognosis

• The syndrome typically presents with renopulmonary symptoms, including renal failure, hematuria, proteinuria, and hemoptysis 2, 4.
• Prognostic factors include the renal excretory function and the degree of renal and lung damage at the time of presentation 2.
• Patients with severe renal failure are less likely to recover renal function, and those requiring immediate dialysis have a poorer prognosis 4.
• Prompt diagnosis and early, aggressive medical treatment are vital for patients with Goodpasture syndrome 2, 4, 3.

Treatment and Outcomes

• Treatment typically involves a combination of glucocorticoids, cyclophosphamide, and plasma exchange therapy 2, 4, 5.
• Patient and renal survival rates vary depending on the severity of renal failure at presentation, with better outcomes observed in patients with less severe renal impairment 4.
• Some patients may experience antibody-negative relapse, emphasizing the importance of clinical vigilance and prompt treatment 6, 5.

Atypical Presentations

• Rarely, Goodpasture syndrome may present with negative anti-GBM antibody testing, making diagnosis challenging 6, 5.
• Clinicians should have a high suspicion for Goodpasture syndrome in cases of unexplained severe pulmonary or renal disease, even in the absence of positive antibody testing 6, 5.