Characteristics, ImmunoglobulinTreatment Patterns, and Effectiveness in CIDP, Heredofamilial Amyloidosis, and Guillain-Barré Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 170
- 试验地点
- 1
- 主要终点
- Overall neuropathy limitations scale
研究概览
简要总结
To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR) adult patients at a single U.K. centre.
详细描述
CIDP (chronic inflammatory demyelinating polyneuropathy) is an immune-mediated peripheral nerve disorder, that can be progressive or relapsing, with sensory or motor symptoms. It is typically diagnosed by a combination of progressive history, ruling out other neuropathic conditions, or by being refractive to immunoglobulin or corticosteroid treatment. Recently published guidelines introduced a pathway to diagnosis (Van den Burgh, 2021). Challenges with CIDP can result in misdiagnoses of hereditary amyloidosis (hATTR) and Guillain-Barré syndrome (GBS).
Studies in France, Italy, and Japan indicate that 15-53% of patients with hereditary amyloidosis (hATTR) have previously been diagnosed with CIDP (Russo 2020; Cortese 2017; Plante-Bordeneuve 2007; Koike 2011).
A study in the Netherlands highlighted the diagnostic challenges in diagnosis of acute onset CIDP versus (GBS) during the early acute phase onset (Ruts 2010). This diagnostic challenge may be driven by the lack of diagnostic tests or clinical features that can distinguish GBS acute onset CIDP (Van den Burgh, 2021).
A Hospital, Episode Statistics (HES) analysis of previous and subsequent primary diagnosis codes for CIDP patients highlighted hATTR and GBS, as well as unspecified [inflammatory] neuropathy codes, as three conditions with diagnostic challenges that might lead to reclassification of patients.
This current study is designed to describe the real-world demographics, clinical characteristics, and treatment pathways of patients diagnosed with CIDP, GBS and hATTR. In addition, this study will describe the real-world effectiveness and safety of immunoglobulin therapies in the treatment of patients with CIDP or GBS.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed or treated at the study centre with a diagnosis of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre syndrome (GBS), or heredofamilial amyloidosis (hATTR).
- •Patients aged 18 years and over.
- •Patients who did not "opt out" of their health data being used for research.
排除标准
- •Patients who "opted out" of their health data being used for research.
结局指标
主要结局
Overall neuropathy limitations scale
时间窗: At diagnosis or pre-Ig treatment initiation.
The ONLS allows semi-objective measurement of function, which is useful to detect changes with therapy, and relatively earlier changes may be detected than on the standard five-point Medical Research Council scale used in routine examination of muscle power. The ONLS is graded separately for upper and lower limbs as follows: Arm scale score (0 to 5) + Leg scale score (0 to 7) for the total out of 12.
Berg balance test
时间窗: At diagnosis or pre-Ig treatment initiation.
The Berg balance uses a stopwatch, a ruler or a measuring tape, a chair, a step, and an object that can be picked up. It takes \~15 min to complete and includes 14 tasks scored 0-4 and scores are added for a total out of 56. Berg balance test scoring system: 0 to 20: Score - need the assistance of a wheelchair to move around safely 21 to 40: Score - need some type of walking assistance, cane or a walker 41 to 56: Score - considered independent and able to move around safely without assistance
Patient baseline characteristics
时间窗: At diagnosis
Patient demographics, clinical characteristics, weight (kg), and medical history.
9-hole peg test
时间窗: At diagnosis or pre-Ig treatment initiation.
Average of four measurements (two per hand) in seconds, measured using stopwatch.
10 meter walk test
时间窗: At diagnosis or pre-Ig treatment initiation.
Average speed in meters/second over 10 meters, measured using stopwatch.
Grip strength test
时间窗: At diagnosis or pre-Ig treatment initiation.
Average measurement of grip strength using dynamometer over 3 trials per hand, measured in kilograms of force.
Medical research council muscle strength score
时间窗: At diagnosis or pre-Ig treatment initiation.
The Medical Research Council (MRC) Scale for Muscle Strength is a commonly used scale for assessing muscle strength from Grade 5 (normal) to Grade 0 (no visible contraction) The Criteria requires that each of the six muscle groups listed below are examined bilaterally, each with a score from 0 to 5 according to the scale: Shoulder abductors, Elbow flexors, Wrist extensors, Hip flexors, Knee extensors, Foot dorsiflexors.
Inflammatory neuropathy cause and treatment (INCAT) sensory sum (ISS) score
时间窗: At diagnosis or pre-Ig treatment initiation up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.
The ISS score is inversely related to function, with 0 representing no functional impairment and 10 representing inability to make any purposeful movement with either arms or legs.
次要结局
- Time to treatment initiation(From diagnosis to day 1 of treatment)
- Treatment outcomes(Treatment outcomes to be measured at 6 week intervals after the date of first immunoglobulin up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.)
- Treatment patterns(From the date of first immunoglobulin up to December 31, 2022 or death or lost-to-follow-up, whichever comes first.)
- Relapse(From 6 weeks after the date of first immunoglobulin treatment to the date of first documented relapse up to a maximum of 173 months of follow-up)