What question did this study set out to answer?

To evaluate the efficacy of iptacopan combined with cyclophosphamide and prednisone in refractory warm autoimmune hemolytic anemia (wAIHA).

December 8, 2025BloodOpen Access

Severe hemolysis flare of refractory warm hemolytic anemia with positive complement component C3d responsive to iptacopan with cyclophosphamide and prednisone

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Demonstrates improved hematologic responses in patients with refractory warm autoimmune hemolytic anemia, suggesting a new treatment option.

To evaluate the efficacy of iptacopan combined with cyclophosphamide and prednisone in refractory warm autoimmune hemolytic anemia (wAIHA).
Fifteen refractory wAIHA patients were treated with iptacopan, cyclophosphamide, and prednisone during severe hemolytic episodes.
Patients had previously failed at least two treatment lines including glucocorticoid therapies.
The treatment lasted from June 2024 to June 2025, with monitoring of hematologic responses and C3d levels.
Thirteen patients (86.67%) achieved hematologic responses with a hemoglobin increase >20 g/L.
Overall response rate was 73.3% at one month and 80.0% at two months post-treatment.
C3d turned negative or decreased in titer in 86.67% of patients.

Guangsheng He (2025) studied this question.

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Demonstrates improved hematologic responses in patients with refractory warm autoimmune hemolytic anemia, suggesting a new treatment option.