Eculizumab for atypical hemolytic-uremic syndrome in India: First report from India and the challenges faced

Abstract

Much progress has been made in understanding the pathophysiology and treatment of atypical hemolytic uremic syndrome (aHUS). Plasma therapy is the mainstay of treatment for aHUS. The availability of the first effective anti-complement therapeutic agent, eculizumab, has dramatically changed the outlook of this disease. However, its use in clinical practice raises important questions, such as who should receive the drug, when to start such therapy, and is it safe to stop treatment once the disease is controlled. We describe here for the 1st time in India, use of eculizumab in a 12-year-old boy with aHUS. We also describe in this report challenges faced in procuring the drug, and an ideal, evidence-based method of treating aHUS in children.

Isolated Liver Transplantation: A Worthy Choice for Atypical Hemolytic Syndrome in Resource-Restricted Settings

“…Eculizumab, now considered to be the first-line treatment, seems to be a wonder drug for atypical HUS; however, it is an expensive drug and is not so easy to procure in many countries. There is only 1 report of use of eculizumab from India 5 . There is no data as to when the drug can be stopped or whether the dosing interval can be prolonged.…”

<p>Optimal management of atypical hemolytic uremic disease: challenges and solutions</p>

“…Once labeled as the most expensive drug worldwide by Forbes, the cost of a single 30 mL/300 mg vial of eculizumab is around $6830 or £3150 with an annual cost of treatment being £350,000 or $465,635 approximately 61. The high price and limited availability of the drug limits its use, especially in the developing world 52,62. Reimbursement limitations, even in developed countries like UK, pose challenges to both patients and physicians and restrict the access to this life-saving drug 63.…”

“…61 The high price and limited availability of the drug limits its use, especially in the developing world. 52,62 Reimbursement limitations, even in developed countries like UK, pose challenges to both patients and physicians and restrict the access to this life-saving drug. 63 The biweekly hospitalization for the administration of eculizumab during the maintenance phase of treatment further adds to the rising health care-associated financial burden.…”