C1-inhibitor improves recovery of red blood cell transfusion in patients suffering from autoimmune hemolytic anemia – an open-labeled pilot trial

• Positive (≥1+) monospecific antiglobulin test for C3b and/or C3d with/without positivity for IgM OR strongly positive (≥3+) monospecific antiglobulin test for C3b and/or C3d with positivity for IgG
• Indication for a transfusion with at least 2 red packed cell concentrates based on the clinical assessment by the hematologist in charge
• Hemoglobin value at least < 5 mmol/L (8 g/dl) with/without clinical symptoms
• Clinical signs of hemolysis: not-detectable haptoglobin (mandatory) and increased lactate dehydrogenase (LDH) eventually combined with hyperbilirubinemia (increased direct and/or indirect bilirubin), lactate.
• Age ≥ 18 years
• Written informed consent
• Women of child bearing potential must have had a negative serum pregnancy test 7 days prior to the start of study drug

• History of arterial and/or venous thromboembolic events in the absence of an actual treatment with Vitamin K-antagonists
• Concomitant use of therapeutic doses of heparin
• Female patients who are pregnant or breast feeding or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes. Oral contraceptives only are not acceptable.
• Patients with known HIV seropositivity or chronic active hepatitis
• Patients who have any severe and/or uncontrolled medical condition or other conditions that could affect their participation in the study such as:
– cerebrovascular accidents ≤ 6 months before study drug start
– uncontrolled hypertension