What are the treatment options for Atypical Hemolytic Uremic Syndrome (aHUS)?

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From the FDA Drug Label

1.2 Atypical Hemolytic Uremic Syndrome (aHUS) SOLIRIS is indicated for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

2.3 Recommended Dosage Regimen – aHUS For patients 18 years of age and older, SOLIRIS therapy consists of: 900 mg weekly for the first 4 weeks, followed by 1200 mg for the fifth dose 1 week later, then 1200 mg every 2 weeks thereafter

The treatment option for Atypical Hemolytic Uremic Syndrome (aHUS) is eculizumab (IV), which is administered as follows:

  • For patients 18 years of age and older:
    • 900 mg weekly for the first 4 weeks
    • 1200 mg for the fifth dose 1 week later
    • 1200 mg every 2 weeks thereafter 1

From the Research

The primary treatment for atypical hemolytic uremic syndrome (aHUS) is eculizumab, a terminal complement inhibitor that blocks the formation of the membrane attack complex, with treatment typically beginning with induction dosing of 900 mg weekly for four weeks, followed by 1,200 mg at week 5 and then every two weeks thereafter as maintenance therapy, as supported by the most recent study 2.

Treatment Overview

The treatment of aHUS involves the use of complement inhibitors, such as eculizumab, to block the uncontrolled complement activation that drives the microvascular thrombosis and organ damage characteristic of aHUS.

  • Eculizumab is administered intravenously, with a dosing regimen that typically includes induction dosing of 900 mg weekly for four weeks, followed by 1,200 mg at week 5 and then every two weeks thereafter as maintenance therapy.
  • For pediatric patients, dosing is weight-based, and before starting eculizumab, patients must receive meningococcal vaccination at least two weeks prior to treatment due to increased risk of meningococcal infections, as noted in 3.
  • Alternative complement inhibitors, such as ravulizumab, may also be used, with a longer half-life allowing for less frequent dosing, as seen in 4.

Supportive Care

In addition to complement inhibitors, supportive care is essential in the management of aHUS, including:

  • Blood pressure control
  • Dialysis for kidney failure
  • Management of anemia
  • Plasma exchange or plasma infusion may be used as a bridge therapy until complement inhibitors can be initiated, particularly in critically ill patients, as discussed in 5 and 6.

Monitoring and Adjustment

The effectiveness of eculizumab treatment can be monitored by measuring complement activity, allowing for safe tailoring of the frequency of eculizumab administration, thus avoiding excessive drug exposure while keeping the disease in remission, as demonstrated in 2.

  • Treatment is typically long-term, though some patients may eventually discontinue therapy under close monitoring, with the goal of minimizing morbidity, mortality, and improving quality of life.

References

Research

Safety and effectiveness of restrictive eculizumab treatment in atypical haemolytic uremic syndrome.

Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 2018

Research

An effective treatment of atypical hemolytic uremic syndrome with plasma exchange and eculizumab: A case report.

Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis, 2015

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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