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Survey Highlights Treatment Needs, Perspectives of Patients With Chronic Inflammatory Demyelinating Polyneuropathy

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In total, 82% of patients were interested in more convenient treatments, emphasizing the importance of shared decision-making and open discussions with physicians in rare disease management.

Jeffrey Allen, MD, associate professor of neurology at the University of Minnesota Medical School

Jeffrey Allen, MD

Recently announced results from a survey study led by CSL Behring showed that a majority of patients with chronic inflammatory demyelinating polyneuropathy (CIDP) are interested in exploring more convenient treatment options; however, less than half of this cohort claimed they discussed potential new treatment options or switching treatments with a physician.1

The study was conducted in 100 patients with CIDP, aged at least 18 years old, and 100 neurologists/neuromuscular specialists who see such patients. Coming into the study, only 2 of these patients were not currently on treatment for CIDP. Following the survey, which took place between February 27, 2023 and March 15, 2023, results showed that 82% of patients were invested in exploring more suitable treatment options and 72% felt that administering treatment at home was extremely/very important to them.

In the survey, more than half (57%) of responding patients claimed they wish their physicians better understood what they think is important in a CIDP treatment. Efficacy (95%), minimal adverse effects (97%), condition maintenance (96%), at-home administration (72%), and flexible dosing options (70%) were among the top priorities for patients’ when thinking about a treatment option.

"We know that people living with rare diseases benefit from shared decision-making with their physician about their treatment options, including how and where they receive treatment,” Jeffrey Allen, MD, associate professor of neurology at the University of Minnesota Medical School, said in a statement.1 "Having regular open discussions about the different options available, including self-administered treatment at home for CIDP maintenance for example, is particularly important for people who may find it difficult or burdensome to travel to an infusion center."

CIDP, a rare autoimmune disorder, has been traditionally treated with first-line therapies including corticosteroids, high-dose intravenous immune globulins (IVIGs), plasma exchange, or plasmapheresis. In the survey, less than half of the patients surveyed said they were very/somewhat knowledgeable about how different CIDP treatment options work (40%) or about how different CIDP treatment options are administered (45%).

Over the years, the therapeutic options for patients with CIDP has expanded. After originally receiving approval in 2010 for primary immunodeficiency (PI), CSL Behring’s immune globulin subcutaneous (human) 20% liquid (Hizentra) gained FDA-approval to treat adults with CIDP in March 2018. With the approval, the therapy became the first and only self-administered, subcutaneous immunoglobulin (SCIg) for the treatment of CIDP as maintenance therapy to prevent relapse of neuromuscular disability and impairment.

"As part of Our Promise, we are committed to listening to and understanding the concerns of people living with rare diseases and developing treatment options that address unmet needs," Bob Lojewski, senior vice president and general manager, North America, CSL Behring, said in a statement. "We are proud to be at the forefront of delivering both effective and convenient treatment options like Hizentra, which may allow those living with CIDP more flexibility by allowing them to self-administer their treatment at home. Furthermore, to help improve the treatment experience, we are proud to offer Hizentra in a range of prefilled syringe sizes, including a soon to be available 10g prefilled syringe."

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Hizentra, infused under the skin and not into a vein, is available in vials or several different syringe sizes, including 1 g, 2 g, 4, and 10 g size that will be available in early 2024. More recently, the FDA approved Takeda’s IG infusion 10% (human) with recombinant human hyaluronidase (Hyqvia), a liquid medicine, as a maintenance therapy for adults with CIDP. Hyqvia remains the only FDA-approved combination of IG and hyaluronidase, making it a facilitated subcutaneous immunoglobulin infusion.2

The approval was based on findings from the randomized, double-blinded, placebo-controlled ADVANCE-CIDP 1 trial (NCT02549170) and the single-arm, open-label, extension ADVANCE-CIDP 3 study (NCT02955355) that assessed the efficacy and safety of Hyqvia in adults with CIDP. The analysis of the primary end point among 122 with CIDP from ADVANCE-CIDP 1 showed a statistical difference between the relapse rates in the Hyqvia group (n = 57; 14.0%) compared with the placebo group (n = 65; 32.3%), which was significant (P = .0314).3

Following the approval, NeurologyLive® reached out to expert Richard Lewis, MD, a professor of neurology at Cedars-Sinai Medical Center, who provided perspective on what the new agent brings to the treatment landscape. In the video below, Lewis talked about the advantages Hyqvia offers over traditional intravenous immunoglobulin treatments for patients with CIDP.

REFERENCES
1. Harris Poll survey finds people receiving treatment for CIDP are interested in exploring more convenient treatment options. CSL Behring. January 16, 2024. Accessed January 24, 2024. https://www.prnewswire.com/news-releases/harris-poll-survey-finds-people-receiving-treatment-for-cidp-are-interested-in-exploring-more-convenient-treatment-options-302034782.html
2. U.S. FDA Approves Takeda’s HYQVIA® as Maintenance Therapy in Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). News Release. Takeda. Published January 16, 2024. Accessed January 24, 2024. https://www.takeda.com/en-us/newsroom/news-releases/2024/us-fda-approves-takedas-hyqvia-as-maintenance-therapy-in-adults-with-chronic-inflammatory-demyelinating-polyneuropathy-cidp
3. Takeda presents full data set from phase 3 ADVANCE-CIDP 1 clinical trial investigating Hyqvia as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy at PNS Annual Meeting. News release. June 20, 2023. Accessed January 24, 2023. https://www.takeda.com/en-us/newsroom/news-releases/2023/takeda-presents-full-data-set-from-phase-3-ADVANCE-CIDP-1-clinical-trial-investigating-HYQVIA%C2%AE-as-a-maintenance-therapy-for-chronic-inflammatory-demyelinating-polyneuropathy-CIDP-at-PNS-annual-meeting/
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