2025 年 42 巻 5 号 p. 832-835
This review outlines new promising therapies and clinical research in peripheral neuropathies. In 2024, Japanese version of clinical practice guideline for Guillain–Barré syndrome (GBS) was published. The results of a Japanese physician–initiated phase III trial of a complement C5 inhibitor (eculizumab) for the treatment of GBS were also published. Unlike the promising results of the phase II trial, the phase III trial failed to achieve its primary endpoint. Partial results from clinical trials using another complement inhibitor (C1q inhibitor) have shown efficacy. Japanese clinical practice guideline of chronic inflammatory demyelinating peripheral polyneuropathy (CIDP) was also published in 2024. Clinical trials are ongoing for drugs targeting complement for CIDP. The clinical trial for efgartigimod showed very promising results (about 70%–response rate), which is now available for the treatment of CIDP in Japan. For hereditary ATTR amyloidosis (ATTRv), various new therapeutic agents of oligonucleotide therapeutics are emerging, and many clinical trials are underway.
