Table 4. Studies on thrombotic events in... | American Society of Hematology

Table 4.

Studies on thrombotic events in CD55-deficient patients

First author/reference	Number of patients	Clinical presentation	Thromboembolic events	Rate of TE under complement inhibition by eculizumab	Comment
Matthes³⁰	1	Intravascular hemolytic anemia Abdominal pain	Multiple Splenic infarctions	NA	• Acquired, transient loss of CD55 on RBCs; other peripheral blood cells had weaker than normal CD55 level; CD55 reoccurred during follow-up • Thrombotic complications and hemolysis were absent in other case with transient loss of CD55¹⁵⁷
Kurolap^54,62	6	Chaple No hemolysis, but elevated MAC deposition on white blood cells	Three of 6 with repeated TEs	Only 3 patients treated, but within 18 mo of follow-up, hyper-coagulopathy events did not reoccur after treatment initiation	• Coagulation investigation did not reveal a commonly known cause for prothrombotic disposition • Response to eeculizumab herapy initiated in 3 patients suggests MAC involvement in pathophysiology
Ozen⁶¹	11 (plus 2 deceased historic cases)	Chaple No hemolysis, but elevated MAC deposition on submucosal arterioles	Five of 13 with multiple thrombi at different sites; in total 3 fatalities caused by thrombosis	NA	• For one case resis tance to anticoagulation was mentioned • Biopsy revealed erythrocyte to endothelium interactions
Hagin¹⁵⁸	1	Chaple Evidence of hemolysis	Repeated mesenteric vein thrombosis	NA	Eculizumab herapy improved abdominal symptoms, increased hemoglobin levels; LDH levels did not normalize completely; months after start of Ecu a malignancy was diagnosed and Ecu stopped; patient died of sepsis coinciding with antineoplastic therapy
Ozen⁵⁵	16	Chaple No hemolysis, but elevated MAC deposition on submucosal arterioles	Six of 16 (38%) with thrombotic complications	Four of 16 (25%) with thrombotic complications	Eculizumab herapy • Normalizes plasma C5a and sC5b-9 levels • Substantially reduces pathophysiology including D-dimers and thrombotic events, but not in all patients

First author/reference	Number of patients	Clinical presentation	Thromboembolic events	Rate of TE under complement inhibition by eculizumab	Comment
Matthes³⁰	1	Intravascular hemolytic anemia Abdominal pain	Multiple Splenic infarctions	NA	• Acquired, transient loss of CD55 on RBCs; other peripheral blood cells had weaker than normal CD55 level; CD55 reoccurred during follow-up • Thrombotic complications and hemolysis were absent in other case with transient loss of CD55¹⁵⁷
Kurolap^54,62	6	Chaple No hemolysis, but elevated MAC deposition on white blood cells	Three of 6 with repeated TEs	Only 3 patients treated, but within 18 mo of follow-up, hyper-coagulopathy events did not reoccur after treatment initiation	• Coagulation investigation did not reveal a commonly known cause for prothrombotic disposition • Response to eeculizumab herapy initiated in 3 patients suggests MAC involvement in pathophysiology
Ozen⁶¹	11 (plus 2 deceased historic cases)	Chaple No hemolysis, but elevated MAC deposition on submucosal arterioles	Five of 13 with multiple thrombi at different sites; in total 3 fatalities caused by thrombosis	NA	• For one case resis tance to anticoagulation was mentioned • Biopsy revealed erythrocyte to endothelium interactions
Hagin¹⁵⁸	1	Chaple Evidence of hemolysis	Repeated mesenteric vein thrombosis	NA	Eculizumab herapy improved abdominal symptoms, increased hemoglobin levels; LDH levels did not normalize completely; months after start of Ecu a malignancy was diagnosed and Ecu stopped; patient died of sepsis coinciding with antineoplastic therapy
Ozen⁵⁵	16	Chaple No hemolysis, but elevated MAC deposition on submucosal arterioles	Six of 16 (38%) with thrombotic complications	Four of 16 (25%) with thrombotic complications	Eculizumab herapy • Normalizes plasma C5a and sC5b-9 levels • Substantially reduces pathophysiology including D-dimers and thrombotic events, but not in all patients

Disease: isolated germline encoded homozygous CD55 deficiency (prevalence: too low to calculate). NA, not applicable; TE, thrombotic events.