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Symptom and Treatment Satisfaction in Members of the US and Canadian GBS/CIDP Foundations with a Diagnosis of Chronic Inflammatory Demyelinating Polyneuropathy

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Abstract

Introduction

Current guidelines for defining good outcomes in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) are predominately defined by experts. At present, we do not have a patient-anchored definition of what constitutes a “good” outcome. Our study aimed to assess the symptom burden of people living with CIDP, as well as satisfaction with treatments and clinical outcomes.

Methods

We conducted an online-survey in CIDP patients registered with the US and Canadian GBS/CIDP foundations. Respondents answered general demographic and clinical questions, as well as satisfaction with current symptom burden and treatments, plus validated outcome measures.

Results

A total of 318 individuals with self-reported CIDP completed the online survey, of whom 128 (40%) considered their current disease burden as satisfactory while 190 (60%) did not. Of 305 patients who answered the treatment satisfaction question, 222(74%) were satisfied with their treatments. Patients who were satisfied with their current symptoms had, on average, better scores in quality of life and disease severity scales, although regression modeling showed that only ability to walk, stable symptoms, and health utility scores were associated with symptom satisfaction. Treatment satisfaction was associated with stable symptoms, use of IVIG, and use of one versus no medication.

Conclusions

A high proportion of members of the US and Canadian GBS/CIDP Foundations reporting a diagnosis of CIDP were unsatisfied with current symptoms, despite a high level of overall satisfaction with treatments. There is an unmet need for improving long-term outcomes in people with a diagnosis of CIDP, and for studying patient-centered long-term treatment goals.

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Data Availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on request.

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Acknowledgements

We would like to thank the members of the GBS/CIDP Foundation in the US and Canada for their participation in this survey.

Medical Writing/Editorial Assistance

The authors did not use medical writing or editorial assistance for this article.

Funding

This study received funding from Grifols (Grant CLinician Initiated Research), through an investigator-initiated grant. The grant includes the journal’s Rapid Service Fee. The sponsor had no role in data collection, analyses or manuscript generation.

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Authors and Affiliations

  1. Toronto General Hospital, Ellen and Martin Prosserman Centre for Neuromuscular Disease, 200 Elizabeth Street 5ECW-334, Toronto, ON, M5G2C4, Canada

    Meg Mendoza, Vera Bril, Hans D. Katzberg & Carolina Barnett-Tapia

  2. Division of Neurology, University Health Network and University of Toronto, Toronto, ON, Canada

    Christopher Tran, Vera Bril, Hans D. Katzberg & Carolina Barnett-Tapia

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  1. Meg Mendoza
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Contributions

Meg Mendoza: contributed to study design, data collection, data analysis, and manuscript preparation. Christopher Tran: contributed to study design and data collection. Hans D. Katzberg: contributed to patient recruitment for the pilot survey and critical review of manuscript. Vera Bril: contributed to patient recruitment for the pilot survey and critical review of manuscript. Carolina Barnett-Tapia: contributed to study design, data collection, data analysis, and manuscript preparation. Statistical analysis completed by Dr. Carolina Barnett-Tapia and Meg Mendoza.

Corresponding author

Correspondence to Carolina Barnett-Tapia.

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Conflict of Interest

Meg Mendoza reports no disclosures. Christopher Tran reports no disclosures. Hans D. Katzberg has been a consultant to Grifols, CSL Behring, UCB, Takeda (Shire), Alnylam, Octapharma, Akcea, Alexion, Pfizer, Biogen and Terumo: he has received research support from Takeda (Shire) and CSL Behring. Vera Bril has been a consultant to Grifols, CSL Behring, UCB, Argenx, Takeda (Shire), Alnylam, Octapharma, Powell Mansfield Inc., Akcea, Immunovant and Alexion; she has received research support from Grifols, CSL Behring, UCB, Argenx, Takeda (Shire), Octapharma, Baxalta and Biogen. Carolina Barnett-Tapia has been consultant to Alexion, Argenx, UCB and Sanofi; she has received grant support from Grifols and Octapharma. She is the developer of the MGII and may receive royalties.

Ethical Approval

This study was reviewed and approved by the University Health Network Research Ethics Board (Study number: 18-5514) and conforms to the World Medical Association Declaration of Helsinki. Participants of the pilot test provided written consent and answering the anonymous electronic survey was considered as implicit consent. We obtained written permission from the developers of the INCAT, RODS and CAPPRI to use the measures in this study. We obtained a license for the use of the SF-12 from QualityMetric, and we registered our study to use the EQ-5D-5L with EuroQoL (non-commercial, fast-track digital, no license needed).

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Mendoza, M., Tran, C., Bril, V. et al. Symptom and Treatment Satisfaction in Members of the US and Canadian GBS/CIDP Foundations with a Diagnosis of Chronic Inflammatory Demyelinating Polyneuropathy. Adv Ther 40, 5188–5203 (2023). https://doi.org/10.1007/s12325-023-02661-4

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