Time is a key factor: a spotlight on people affected by Guillain-Barré Syndrome

At Hansa Biopharma we are committed to help raise awareness for those living with rare immunologic diseases. Today we want to raise the attention around Guillain-Barré syndrome (GBS), a rare autoimmune disease characterized by severe pain, and that can lead to a severe paresis of the arms and legs (leaving 20% of patients unable to walk after six months). With current standard of care, GBS is fatal in 3-7% of cases.

We have reached out to Professor Shahram Attarian, Head of Department of Neuromuscular Diseases and ALS, Hopitaux Universitaires de Marseille (APHM). Prof Attarian dedicates his life to treat patients affected by GBS and other sever neuromuscular diseases and can help us understand the difficulties that GBS patient face and the challenges that healthcare professional have in diagnosing and treating this painful rare condition.

Prof Attarian, you are an expert in treatment of Guillain-Barré syndrome. Could you briefly describe this syndrome?

Guillain-Barré syndrome, or GBS, is a rare, paralyzing, inflammatory disorder of the peripheral nervous system that induces an autoimmune response targeting peripheral nerves and their spinal roots. The disease is acute, meaning that it comes on suddenly and requires urgent medical attention. Although it is classed as a rare disease, approximately 100,000 people each year contract this severe and debilitating condition worldwide.

?In most patients, the acute onset of neurological symptoms is preceded by an infectious disease, which is followed by progressive weakening of the limbs that can last up to four weeks. Usually, after this initial acute phase the disease progression slows, and begins to plateau. Several infections are known to be associated with GBS, with infections from the bacterium Campylobacter jejuni (a common cause of food poisoning) being the most common and widely reported.

Given that GBS is usually preceded by an infection, how difficult is it to do a successful early diagnosis of this condition?

The classic presentation of the syndrome is usually not particularly challenging to diagnose. However, there are atypical variants that can go undetected when ignored. The recognition of GBS variants is clinically important to avoid treatment delays. In my career, I have seen cases where patients had to make three visits to the emergency room before the diagnosis of GBS was made. This complicates things, as a timely diagnosis and treatment is crucial to reduce the severity of the symptoms and minimize long term damage.

Many GBS patients experience prolonged pain. How severe is this pain?

The neuropathic pain can be very severe, often localized in the neck and back. Recovery is a long and difficult process, and in some instances, the disease causes permanent damage or even death. Two-thirds of patients with GBS develop such pain in the acute phase. To counteract the worsening of neuropathic pain, it is important to recognize it quickly and start specific treatment on time, with adequate doses, for a prolonged period of time.

What is the most difficult challenge in treating people diagnosed with GBS?

In the treatment of GBS, time is a key factor. In the acute phase, usually within the first two weeks of disease onset, patients are at risk of developing significant complications and nerve damage. Ventilator support is required in 20% of patients with GBS. For those patients at risk of potential respiratory and autonomic failure (unconscious nerve control of bodily processes such as heartbeat and blood vessel expansion and contraction), admission to an intensive care unit is recommended for close monitoring of disease progression. Immunotherapy should be initiated as soon as patients show signs of disability.

Why is it important to raise awareness around this condition and the thousands of people affected by it?

The rarity of the disease, especially in its initial stages, could be responsible for the wandering diagnosis and delay in treatment. This is a particularly serious problem for a disease where acting fast and starting treatment as early as possible has a crucial impact on the success of the recovery.

Although current standard of care can accelerate recovery in GBS, even when treated with standard immunotherapies, approximately 5% of people die, and up to 20% cannot walk independently at 1 year from disease onset.