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ID
Source
Brief title
Health condition
Autoimmune Hemolytic Anemia
Sponsors and support
Intervention
Outcome measures
Primary outcome
• improvement of recovery of RBC transfusion,
• inhibition of complement activation and deposition on RBC via the classical pathway of complement,
• safety
Secondary outcome
• Attenuates the pro-inflammatory response in AIHA
• Affects the response to the basic treatment targeting autoantibody production
Background summary
This is a prospective, multicenter, national open label study to test the efficacy of C1-inh to improve the efficacy of RBC transfusion in patients with AIHA
Study objective
Because C1-inh is an efficient inhibitor of the classical pathway of complement with an excellent safety profile we hypothesized that C1-inh might inhibit autoantibody mediated destruction of donor RBS in order to improve efficacy of RBC transfusion.
Study design
8 timepoints
Intervention
C1 inhibitor (Cinryze)
Inclusion criteria
• 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
Exclusion criteria
• 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
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL8164 |
| Other | METC AMC : METC 2012_266 |