hus – Page 16 – eFoodAlert

In one of the first rays of hope for sufferers of hemolytic uremic syndrome (HUS), FDA announced today its approval of Soliris “… to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.”

According to FDA, atypical HUS accounts for 5-10% of all cases of hemolytic uremic syndrome and affects children disproportionately. Soliris is the first drug to be approved in the USA for treating atypical HUS.

Soliris (eculizumab) is a humanized monoclonal antibody that was first approved by FDA in 2007 to treat a rare type of blood disorder (paroxysmal nocturnal hemoglobinuria), and is classified as an orphan drug. It is marketed in the USA by Alexion Pharmaceuticals.

While today’s FDA approval of Soliris is limited to atypical HUS, the agency’s action represents the first hint that a specific treatment for more common forms of hemolytic uremic syndrome may be in the offing. HUS is a life-threatening syndrome that affects a significant percentage of people – especially children – who are infected with E. coli O157:H7 and other shiga-toxin producing strains of E. coli.

More than 900 victims of this summer’s E. coli O104:H4 outbreak developed HUS, and 34 of those people died of the disease. The death toll might have been even higher, had doctors not administered eculizumab (Soliris) to 3 children with severe HUS and central nervous system complications. The children were at risk of permanent neurologic damage and not responding to conventional treatment.

All three children responded to the Soliris treatment, and eventually were discharged from hospital with apparently normal neurological status and little or no residual effects of their illness. Six months later, all three children were “in full remission,” according to a report published in New England Journal of Medicine.

Doctors who want to use Soliris to treat HUS must jump through a few extra hoops, due to the limited nature of its clinical trial. Soliris is available only through a restricted program, according to FDA, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.

Nevertheless, today’s approval is major news for HUS sufferers, so many of whom are children. Parents whose children are suffering from HUS – and physicians who treat HUS patients – should educate themselves about this new treatment possibility.

The official numbers reported this morning by Germany’s Robert Koch Institut are worthy of a Hollywood thriller. Nine people dead, and 470 notified cases of hemolytic uremic syndrome (HUS) – a life-threatening complication of enterohemorrhagic E. coli infections.

The German outbreak data reported by BBC and others are even worse. Sixteen deaths, including one in Sweden; 1,534 infected with the outbreak strain known as E. coli O104:H4 – 470 of them suffering from HUS.

The outbreak began in Northern Germany; the heaviest concentration of confirmed cases – and of HUS – can be found in the states of Hamburg and Bremen. As of May 26th, thirteen German states had reported 214 cases of HUS – Hamburg (59), Bremen (11), Schleswig-Holstein (21), Mecklenburg-Vorpommern (10), Hesse (31), Saarland (5), Lower Saxony (28), North Rhine-Westphalia (31), Berlin (3), Baden-Württemberg (8), Bavaria (5), Thuringia (1) and Rhineland-Palatinate (1). These numbers already are far out of date, as the total number of cases has more than doubled since that report was issued.

Thanks to the ease of international travel – especially within the European Union, where most borders are invisible and undefended – the outbreak has not been confined to Germany. Eight other countries have reported confirmed or suspect cases of enterohemorrhagic E. coli in German nationals visiting their country or in individuals who reported having traveled to Germany:

Austria: 2 cases
Denmark: 14 cases, 6 with HUS
France: 6 cases
Netherlands: 7 cases, 3 with HUS
Spain: 1 case
Sweden: 41 cases, 15 with HUS and one death
Switzerland: 2 cases
United Kingdom: 3 cases, 2 with HUS

Despite the best efforts of Germany’s epidemiologists, the source of this outbreak is unknown – except that it appears to be associated with consumption of raw tomatoes, cucumber and leaf salad.

For a time, suspicion fell on raw cucumbers imported from Spain – especially after lab analysis of two cucumber samples yielded enterohemorrhagic E. coli. But the cucumber contaminant did not match the outbreak strain.

The most frightening aspect of this outbreak, other than the lack of success in finding its cause, is the unusually high percentage of victims who have developed HUS – more than 30% of victims, as compared to the 5-10% HUS rate experienced in most other outbreaks.

HUS is a life-threatening complication, usually beginning with kidney failure and often progressing to failure of other organs and systems. Some 5-10% of HUS patients die, and most others suffer life-long consequences of their fight for survival – even with treatment. Conventional treatment consists of dialysis and, where necessary, plasma exchange.

The massive and deadly Canadian E. coli O157:H7 outbreak that was centered in Walkerton, Ontario in 2000 spurred Canadian researchers to study ways of preventing HUS – by preventing the bacterial toxins from binding to its target cells in the body. Researchers at the University of Maryland and in Argentina independently explored similar approaches – using monoclonal antibodies against the toxin in order to block its activity.

Just a few days ago, researchers in Canada, France and Germany reported that they used yet another antibody to treat successfully three 3-year-old children who were suffering from severe cases of HUS. But this new development might be too late to help many of the German outbreak victims.

The German outbreak has many unknowns: