Treatment of Atypical HUS

Treatment Options for Atypical HUS

There is no cure for Atypical HUS. In fact, there is not a standard treatment, as each case is different. (Note : With the advent of Soliris, this may change over time). The short term strategy is to be supportive and reactive to whatever is taking place. Atypical HUS patients are much more likely to develop complications. These complications may be:

  • Recurrence
  • Kidney failure
  • Seizures/neurological problems or other multi-organ involvement.
  • No response to supportive treatments
  • Return of atypical HUS even after a kidney transplant occurs
  • Severe hypertension (high blood pressure)

The Short-Term Treatment

Involves dealing with the individual symptoms on a case-by-case basis. Usually the first line of defense is to use plasma exchange (pheresis, or infusions) to help combat the effect of the defective protein (caused by the mutated gene). Then, the other symptoms are dealt with on an individual basis. These may include:

  • Decreased urine output (rising BUN, increased creatinine)
  • Infections/flu like symptoms
  • Seizures/neurological problems or other multi-organ involvement.
  • Elevated blood pressure
  • Return of atypical HUS even after a kidney transplant occurs
  • Severe hypertension (high blood pressure)

The Long-Term Treatment

Involves finding out the genetic root cause of the disease, and then determining the appropriate long-term path. At least one of the genes, MCP, will respond well to a kidney transplant if that treatment proves to be necessary. MCP is a protective coating on the kidneys, and a transplanted kidney will contain that protective coating, so no additional steps are needed. MCP is the root cause of estimated 5% of patients. To this end, the MCP gene diagnosis is a totally separate category from most of the other gene mutations. Most of the other non-MCP genetic mutations do not respond well to a transplant. The good news is that many of the genes involved in the disease have been identified.

Atypical HUS Treatment Information

The Identified Genes of Atypical HUS are:

  • Factor H
  • Factor H Related 1-3 (Deletions –FHR1)
  • Factor H Related 5
  • Factor I
  • Factor B
  • C3
  • Thrombomodulin
  • MCP

Treatment Breakthroughs

Recently, several patients have used the Drug Eculizimab or Soliris after transplantation with success. Right now, Soliris has not been used in clinical trials for post-transplant evaluation, but it has undergone clinical trials on a pre-transplant basis. Read more about the results of the Soliris clinical trials. To date, this drug is fast becoming the “Gold Standard” for treatment.

The long-term treatment options currently available are:

  1. Do nothing but treat the symptoms are they appear.
  2. Perform a dual Liver/Kidney, as many of the genetic mutations occurred as a result of proteins that are synthesized in the kidneys.
  3. Use of Soliris (Eculizimab) in a proactive, preventable fashion more...