Targeting complement dysregulation: eculizumab in scleroderma renal crisis management-a case-based review

Area of Science:

• Nephrology
• Rheumatology
• Immunology

Background:

• Systemic sclerosis (SSc) presents management challenges, particularly with scleroderma renal crisis (SRC), a severe complication.
• Thrombotic microangiopathy (TMA) can co-occur with SRC, complicating treatment strategies.

Observation:

• A 41-year-old female SSc patient experienced SRC with concurrent TMA, unresponsive to plasma exchange and RAAS blockade.
• Treatment with eculizumab, a C5 complement inhibitor, was initiated for refractory disease.

Findings:

• Eculizumab therapy rapidly improved platelet counts and reduced lactate dehydrogenase levels.
• The patient achieved complete renal function recovery following eculizumab treatment.
• Genetic analysis identified a variant of unknown significance in the thrombomodulin (THBD) gene, potentially linked to complement pathways.

Implications:

• This case underscores the critical role of complement dysregulation in SRC and co-occurring TMA.
• Eculizumab demonstrates potential as a therapeutic option for refractory SRC and associated TMA.
• Further research into complement pathways and eculizumab efficacy in SRC is warranted to optimize patient outcomes.