# Iptacopan for cold agglutinin disease: a case report with literature review

## TL;DR

This case report shows that iptacopan, a complement inhibitor, effectively controlled hemolysis in a patient with cold agglutinin disease who did not respond to other treatments.

## Contribution

The study demonstrates the rapid and sustained efficacy of iptacopan in treating CAD with uncontrolled hemolysis.

## Key findings

- Hemoglobin levels increased from 67 g/L to 101 g/L within 7 weeks of iptacopan treatment.
- The patient achieved transfusion independence and normalized bilirubin levels without adverse events.
- Sustained remission was observed over a 4-month follow-up period.

## Abstract

This study reports a case of cold agglutinin disease (CAD) secondary to lymphoplasmacytic lymphoma in a patient intolerant to rituximab plus bendamustine and with persistent uncontrolled hemolysis following zanubrutinib therapy. The addition of the complement C3 inhibitor iptacopan to a cyclophosphamide and dexamethasone regimen successfully controlled hemolysis and improved hemoglobin levels. Within one week of treatment, the patient achieved transfusion independence, with hemoglobin increasing from 67 g/L to 90 g/L by week 3 and 101 g/L by week 7, alongside normalized bilirubin levels and no adverse events. The follow-up period was 4 months, during which the patient showed sustained remission. These findings suggest that iptacopan can rapidly ameliorate hemolysis in CAD, warranting further investigation into its therapeutic potential.