Three months interval therapy of Eculizumab in a patient with atypical hemolytic uremic syndrome with hybrid CFHR1/CFH gene
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Pregnancy-associated atypical hemolytic uremic syndrome (P-aHUS) is a rare condition. It is characterized by very high maternal mortality and morbidity. Most cases of P-aHUS (79%) manifest in the postpartum period; this is probably due to the complement’s involvement in aHUS pathogenesis. Eculizumab is approved for aHUS treatment, but its use is limited due to cost, unknown duration of treatment, and vague dose intervals to keep patients in remission. In this case report, we present a 26-year-old female with P-aHUS with hybrid CFHR1/CFH gene. Eculizumab was initiated after 5 weeks of being on hemodialysis and plasmapheresis sessions. Full remission successfully achieved after 6th dose of Eculizumab, within 13 weeks of onset of aHUS. Due to financial issues and inability to financially cover the cost, Eculizumab was set in hold. Within 6 months, she suffered recurrence of the disease and Eculizumab was re-instated. After re-inducing full remission, the patient was switched to Eculizumab every 3 months instead of the recommended manufacture dose interval of every 2 weeks. We followed this patient for 3 years and she continued to be in remission based on clinical and laboratory data. In conclusion, achievement of successful and maintenance of remission of P-aHUS in this patient who had limited access to Eculizumab raise the attention of the efficacy of Eculizumab at longer time intervals. However, it is time to consider conducting a long-term study to learn about the safety and efficacy of this approach, which may have a major financial advantage for patients.
KeywordsEculizumab aHUS Complement dysregulation nephropathy Pregnancy-aHUS
We would like to thank Prof. Fadi Fakhouri, MD, PhD, Centre Hospitalier, Universitaire de Nantes, Nantes (CHU Nantes), FRANCE for reviewing the manuscript and for his helpful comments and suggestions.
Compliance with ethical standards
Conflict of interest
The authors did not report any potential conflicts of interest.
The present study was performed in accordance with the ethical standards of the institutional research committee and with the Helsinki declaration.
Written consent was obtained from the patient discussed and documentation is available for review upon request.
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