TY - JOUR
T1 - Monitoring Clinical Course and Treatment Response in Chronic Inflammatory Demyelinating Polyneuropathy During Routine Care
T2 - A Review of Clinical and Laboratory Assessment Measures
AU - Allen, Jeffrey A.
AU - Merkies, Ingemar S. J.
AU - Lewis, Richard A.
N1 - Funding Information:
nonfinancial support from Meridian HealthComms during the conduct of the study; personal fees from CSL Behring, Alexion, Biotest, Akcea, and Argenyx outside the submitted work. Dr Merkies reported grants from Talecris Talents Program, GBS CIDP Foundation International, and the FP7 EU programme outside the submitted work. Dr Lewis reported personal fees from CSL Behring, Argenx, Akcea, Alnylam, Annexon, Biotest, Momenta, Pfizer, and Sanofi outside the submitted work. No other disclosures were reported.
Funding Information:
Additional Contributions: Editorial support was provided by Meridian HealthComms Ltd. Funding for editorial support was provided by CSL Behring.
Publisher Copyright:
© 2020 American Medical Association. All rights reserved.
PY - 2020/9
Y1 - 2020/9
N2 - This narrative review provides an overview of specific outcomes that are appropriate for chronic inflammatory demyelinating polyneuropathy monitoring during routine clinical care and highlights the minimum clinically important difference, if known.Importance Identifying clinical change in many neurologic diseases, including chronic inflammatory demyelinating polyneuropathy (CIDP), can be challenging. At the same time, how change is defined heavily influences a patient's diagnostic and treatment pathway. It can be especially problematic when equivocal subjective observations are interpreted as clinically meaningful and then used to make diagnostic and treatment decisions. Change in clinical trials is strictly defined by a preselected metric, but there is a perception that formal outcomes collection during routine clinical care is neither feasible nor necessary. Given the importance placed on how change is interpreted, there is a need to select assessments that can be applied to routine care that are representative of the neurologic disease state. Observations For an outcome measure to be useful during clinical trials, it must have good reliability, validity, be responsive to change, and have clinical meaning. To be useful during routine clinical care, the assessment must additionally be easy to collect without the need for extensive training or equipment and should provide an immediately available result that can be rapidly quantified and interpreted. Chronic inflammatory demyelinating polyneuropathy is clinically heterogeneous and so is best evaluated with a diverse group of assessment tools. Assessing strength impairment, disability, and quality of life is ideally suited for everyday practice when caring for patients with CIDP. While electrophysiologic studies, imaging, cerebrospinal fluid, and nodal/paranodal antibodies can provide diagnostic data, they are less practical and helpful longitudinal assessment tools. Conclusions and Relevance Sound clinimetric outcome measures in CIDP are widely available and have the potential to help clinicians objectify treatment response and disease progression. Such data are critically important when justifying the need for ongoing or periodic immunotherapy, documenting relapse or deterioration, or providing reassurance of disease improvement, stability, or remission.
AB - This narrative review provides an overview of specific outcomes that are appropriate for chronic inflammatory demyelinating polyneuropathy monitoring during routine clinical care and highlights the minimum clinically important difference, if known.Importance Identifying clinical change in many neurologic diseases, including chronic inflammatory demyelinating polyneuropathy (CIDP), can be challenging. At the same time, how change is defined heavily influences a patient's diagnostic and treatment pathway. It can be especially problematic when equivocal subjective observations are interpreted as clinically meaningful and then used to make diagnostic and treatment decisions. Change in clinical trials is strictly defined by a preselected metric, but there is a perception that formal outcomes collection during routine clinical care is neither feasible nor necessary. Given the importance placed on how change is interpreted, there is a need to select assessments that can be applied to routine care that are representative of the neurologic disease state. Observations For an outcome measure to be useful during clinical trials, it must have good reliability, validity, be responsive to change, and have clinical meaning. To be useful during routine clinical care, the assessment must additionally be easy to collect without the need for extensive training or equipment and should provide an immediately available result that can be rapidly quantified and interpreted. Chronic inflammatory demyelinating polyneuropathy is clinically heterogeneous and so is best evaluated with a diverse group of assessment tools. Assessing strength impairment, disability, and quality of life is ideally suited for everyday practice when caring for patients with CIDP. While electrophysiologic studies, imaging, cerebrospinal fluid, and nodal/paranodal antibodies can provide diagnostic data, they are less practical and helpful longitudinal assessment tools. Conclusions and Relevance Sound clinimetric outcome measures in CIDP are widely available and have the potential to help clinicians objectify treatment response and disease progression. Such data are critically important when justifying the need for ongoing or periodic immunotherapy, documenting relapse or deterioration, or providing reassurance of disease improvement, stability, or remission.
KW - QUALITY-OF-LIFE
KW - IMPORTANT DIFFERENCE
KW - NERVE ULTRASOUND
KW - GRIP STRENGTH
KW - INTRAVENOUS IMMUNOGLOBULIN
KW - DISABILITY SCALE
KW - GO TEST
KW - CIDP
KW - NEUROPATHIES
KW - VIGORIMETER
U2 - 10.1001/jamaneurol.2020.0781
DO - 10.1001/jamaneurol.2020.0781
M3 - (Systematic) Review article
C2 - 32338716
SN - 2168-6149
VL - 77
SP - 1159
EP - 1166
JO - JAMA Neurology
JF - JAMA Neurology
IS - 9
ER -