ABSTRACT
BACKGROUND: Chronic inflammatory demyelinating polyneuropathy (CIDP) is an inflammatory disease affecting the peripheral nerves and the most frequent autoimmune polyneuropathy. Given the lack of established biomarkers or risk factors for the development of CIDP and patients' treatment response, this research effort seeks to identify potential clinical factors that may influence disease progression and overall treatment efficacy. METHODS: In this multicenter, retrospective analysis, we have screened 197 CIDP patients who presented to the University Hospitals in Düsseldorf, Berlin, Cologne, Essen, Magdeburg and Munich between 2018 and 2022. We utilized the respective hospital information system and examined baseline data with clinical examination, medical letters, laboratory results, antibody status, nerve conduction studies, imaging and biopsy findings. Aside from clinical baseline data, we analyzed treatment outcomes using the Standard of Care (SOC) definition, as well as a comparison of an early (within the first 12 months after manifestation) versus late (more than 12 months after manifestation) onset of therapy. RESULTS: In terms of treatment, most patients received intravenous immunoglobulin (56%) or prednisolone (39%) as their first therapy. Patients who started their initial treatment later experienced a worsening disease course, as reflected by a significant deterioration in their Inflammatory Neuropathy Cause and Treatment (INCAT) leg disability score. SOC-refractory patients had worse clinical outcomes than SOC-responders. Associated factors for SOC-refractory status included the presence of fatigue as a symptom and alcohol dependence. CONCLUSION: Timely diagnosis, prompt initiation of treatment and careful monitoring of treatment response are essential for the prevention of long-term disability in CIDP and suggest a "hit hard and early" treatment paradigm.
ABSTRACT
INTRODUCTION: Chronic inflammatory demyelinating polyneuropathy (CIDP) is one of the most common immune neuropathies leading to severe impairments in daily life. Current treatment options include intravenous immunoglobulins (IVIG), which are administered at intervals of 4-12 weeks. Determination of individual treatment intervals is challenging since existing clinical scores lack sensitivity to objectify small, partially fluctuating deficits in patients. End-of-dose phenomena described by patients, manifested by increased fatigue and worsening of (motor) symptoms, are currently difficult to detect. From a medical and socio-economic point of view, it is necessary to identify and validate new, more sensitive outcome measures for accurate mapping of disease progression and, thus, for interval finding. Digital health technologies such as wearables may be particularly useful for this purpose, as they record real-life data and consequently, in contrast to classic clinical 'snapshots', can continuously depict the disease course. METHODS: In this prospective, observational, non-interventional, single-center, investigator-initiated study, CIDP patients treated with IVIG will be continuously monitored over a period of 6 months. Clinical scores and blood analyses will be assessed and collected during three visits (V1, V2, V3). Additionally, activity, sleep, and cardiac parameters will be recorded over the entire period using a wearable device. Further, patients' subjective disease development and quality of life will be recorded at various visits (read-outs). The usability of the smartwatch will be assessed at the end of the study. PERSPECTIVE: The study aims to evaluate different digital measurements obtained with the smartwatch and blood-based analyses for monitoring disease activity and progress in CIDP patients. In conjunction, both means of monitoring might offer detailed insights into behavioral and biological patterns associated with treatment-related fluctuations such as end-of-dose phenomena. TRIAL REGISTRATION: The study protocol was registered at ClinicalTrials.gov. Identifier: NCT05723848. Initially, the protocol was submitted prospectively on January 10, 2023. The trial was publicly released after formal improvements on February 13, 2023, after first patients were included according to the original protocol.
