The PATH Study

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment With Subcutaneous Immunoglobulin (IgPro20)

The PATH Study was a trial that investigated immunoglobulin treatment infused with a pump into the tissue under the skin (subcutaneously). There are several advantage of subcutaneous over intravenous treatment, e.g. patients or their caregivers can treat themselves at home, there are less severe side effects and patients might have a better disease control avoiding the ups and downs of intravenous (into the vein) treatment. The study recruited 172 patients all around the world within 3 and a half years. The results included only patients who were successfully treated with IVIG (intravenous immunoglobulin) before. Patients went through a “drug-holiday” to check whether they still needed immunoglobulin therapy. After that, everybody was treated with the same regime of the study’s IVIG brand (Privigen) before they were evenly distributed to 2 doses of the subcutaneous drug (Hizentra) or placebo. The results showed that Hizentra effectively prevented worsening of CIDP symptoms, there were few side effects and many more patients preferred s.c. over i.v treatment. The treatment could usually be given in one or 2 days per week. The company has applied for a marketing authorization in the US, Europe and plans to apply in Japan and other countries throughout the world with first approvals and availablity in 2018 / 2019. Hizentra is already approved for other diseases such as immune deficiences.

The reference of the study is:

O. Mielke

Japanese Ecuilizumab Trial for GBS

A new treatment with compliment inhibition improves motor function in Guillain- Barré syndrome: Japanese eculizumab trial for Guillain- Barré syndrome (JET-GBS)

Recent basic studies on the pathophysiology of Guillain- Barré syndrome (GBS) have shown that complement activation has a major role on peripheral nerve damage in GBS. Eculizumab (Alexion Pharmaceuticals Inc) is a humanized monoclonal antibody against complement C5, and is approved to treat complement-mediated blood diseases. At the Peripheral Nerve Society Congress (8-12 July 2017, Sitges, Spain), Japanese GBS study group presented results of a randomized placebo-controlled phase 2 trial with eculizumab in patients with severe GBS (unable to walk). At 24 weeks, marked improvement in motor function was observed in patients treated with eculizumab; 74% of patients in the eculizumab group (n=23) regained ability to run, compared with 15% in the placebo group (N=11).

Satoshi Kuwabara
Department of Neurology, Chiba University Hospital, Chiba, Japan