We reviewed the evidence for drug treatment other than intravenous immunoglobulin, plasma exchange or corticosteroids for people with Guillain-Barré syndrome (GBS) .
GBS is an acute, paralysing disease caused by inflammation of the nerves. Symptoms are most severe within four weeks of onset. Between 3% and 13% of people with GBS die of its complications. A quarter need a ventilator to help with breathing. Recovery takes several weeks or months and is often incomplete. Plasma exchange (washing harmful substances out of the blood) and intravenous immunoglobulin (infusion of human antibodies harvested from blood donations) can help speed up the recovery of people with GBS. There is no evidence for the efficacy of corticosteroids. Despite these treatments, however, many people who develop GBS experience long-term disability. We need to find what other treatments have been tried as a basis for launching new trials.
We found four trials in the first version of this review (conducted in 2011) and no fresh evidence upon updating in 2013 and 2016. The trials each tested a different treatment. The evidence provided was very low quality. One RCT with only 19 participants compared interferon beta-1a (a drug that is beneficial in multiple sclerosis) with placebo. Another with only 10 participants compared a nerve growth factor which, in theory, should be beneficial in people with GBS, with placebo. A third trial, with 37 participants, compared cerebrospinal fluid filtration (washing the nerve roots around the spinal cord) with plasma exchange. A fourth trial with 43 participants compared the Chinese herbal medicine tripterygium polyglycoside, which is thought to have anti-inflammatory properties, with corticosteroids. The first three trials received commercial company support. Support for the fourth trial is unknown.
Key results and quality of the evidence
None of these trials was large enough to confirm or refute the benefit or harm of any of these drugs in the treatment of people with acute GBS. The only trial which found a difference between treatments was the Chinese herbal medicine trial: participants receiving the herbal medicine were one and a half times more likely to have improved disability after eight weeks than those receiving corticosteroids. However, this estimate was uncertain and the authors did not report other clinical outcomes. Serious adverse events were uncommon with each of the four treatments investigated in the identified studies and rates did not differ significantly to those in the control groups. We identified very little evidence other than that from RCTs.
There is a need to develop and test new treatments for GBS, and to adopt more sensitive outcome measures. Two ongoing trials are testing a complement-inhibiting immunosuppressant drug called eculizumab.
The evidence is up to date to January 2016.
The quality of the evidence was very low. Three small RCTs, comparing interferon beta-1a or brain-derived neurotrophic factor with placebo, and cerebrospinal fluid filtration with plasma exchange, showed no significant benefit or harm for any of the interventions. A fourth small trial showed that the Chinese herbal medicine, tripterygium polyglycoside, hastened recovery in people with GBS to a greater extent than corticosteroids, but this result needs confirmation. We were unable to draw any useful conclusions from the few observational studies we identified.
Plasma exchange and intravenous immunoglobulin, but not corticosteroids, are beneficial in Guillain-Barré syndrome (GBS). The efficacy of other pharmacological agents is unknown. This review was first published in 2011 and updated in 2013 and 2016.
To assess the effects of pharmacological agents other than plasma exchange, intravenous immunoglobulin and corticosteroids for GBS.
On 18 January 2016, we searched the Cochrane Neuromuscular Specialised Register, Cochrane Central Register of Controlled Trials, MEDLINE, and Embase for treatments for GBS. We also searched clinical trials registries.
We included all randomised controlled trials (RCTs) or quasi-RCTs of acute GBS (within four weeks from onset) of all types and degrees of severity, and in individuals of all ages. We discarded trials that investigated only corticosteroids, intravenous immunoglobulin or plasma exchange. We included other pharmacological treatments or combinations of treatments compared with no treatment, placebo or another treatment. We also identified a number of non-randomised studies during the search, the results of which we considered in the Discussion.
We followed standard Cochrane methodology.
We identified no new trials during this update of the review. In previous versions of this review we identified only very low quality evidence for four different interventions published in four studies. Each study had a high risk of bias in at least one respect. One RCT with 19 participants comparing interferon beta-1a and placebo showed no clinically important difference in any outcome between groups. Another with 10 participants comparing brain-derived neurotrophic factor and placebo showed no clinically important difference in any outcome between groups. A third with 37 participants comparing cerebrospinal fluid filtration and plasma exchange also showed no clinically important difference in any outcome between groups. In a fourth with 43 participants, the risk ratio for an improvement by one or more disability grade after eight weeks was greater with the Chinese herbal medicine tripterygium polyglycoside than with corticosteroids (risk ratio 1.47; 95% confidence interval 1.02 to 2.11); other outcomes in this trial showed no difference. Serious adverse events were uncommon with each of these treatments and in the control groups.