Eculizumab Therapy Leads to Rapid Resolution of Thrombocytopenia in Atypical Hemolytic Uremic Syndrome

Table 1

Characteristics of the patients.

Case	Age, y	Gender	Comorbidity	Presenting features	Pathology	Molecular defects
1	61	F	None	Abdominal pain, diarrhea, and confusion	None available	None detected
2	57	F	3 months after auto-HCT for advanced multiple myeloma; MM in remission	Abdominal pain, diarrhea, vomiting, and confusion	None available	Anti-CFH, CFHR1-CFHR3 +/−, and CFB c.1697A>C, p.Glu566Ala*
3	50	M	None	Abdominal pain, nausea, vomiting, headache, gross hematuria, confusion, and hypertension (highest blood pressures 220/146 mmHg)	Kidney biopsy: TMA	None detected
4-1	22	F	Gestation at 22 weeks	Anorexia, anasarca, scotoma, and hypertension (highest blood pressures 205/134 mmHg)	Kidney biopsy: TMA	CFH c.3607C>T, p.Arg1203Trp**
4-2			3 months after discontinuing eculizumab	Nausea, vomiting, dyspnea, and hypertension (highest blood pressures 221/159 mmHg)
5	66	F	Gemcitabine/carboplatin and cholangiocarcinoma	Fatigue, somnolence, and confusion	Kidney biopsy: TMA	None detected

An uncommon variant that is found in chimpanzee’s CFB, which is 99% identical to human CFB.
**A rare variant (prevalence of CT genotype, 0.001) in the CFH SCR-20 domain of uncertain consequence.
Abbreviations: CFH: complement factor H; HCT: hematopoietic stem cell therapy; MM: multiple myeloma; TMA: thrombotic microangiopathy.