NeuroVoices: Kayla Scippa, on Recognizing Unmet Need in Chronic Demyelinating Polyneuropathy


The associate director, Patient Reported Outcomes, Johnson & Johnson, provided clarity on a poster presentation from AAN 2024 examining the most critical aspects to CIDP disease severity.

Kayla Scippa, associate director of Patient Reported Outcomes at Johnson & Johnson

Kayla Scippa

Chronic demyelinating polyneuropathy (CIDP) is a neuromuscular disorder characterized by progressive weakness and impaired sensory function in the arms and legs. It is an autoimmune neuropathy usually caused by damage to the myelin sheath (the insulation covering the nerve and protecting the underlying nerve fibers) of the peripheral nerves. Diagnosing CIDP can be difficult, as a broad range of diseases can affect nerves and muscles, often producing similar symptoms, such as weakness and numbness.

For years, CIDP has been treated with corticosteroids or intravenous immunoglobulin (IVIG). Less commonly, plasma exchange has also been used. Earlier this year, the treatment options expanded with the approval of Takeda’s liquid IG infusion 10% with recombinant human hyaluronidase (Hyqvia), a maintenance therapy to prevent relapse of neuromuscular disability and impairment. Hyqvia remains the only FDA-approved combination of IG and hyaluronidase, making it a facilitated subcutaneous immunoglobulin infusion.

Because of the heterogeneity of the disease, developing effective therapies has been a challenge for CIDP. At the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, investigators presented data from a preliminary conceptual model of CIDP based on targeted literature review sources. Using interviews from 12 patients with the disease, the most frequently reported symptoms were muscle weakness (n =12), neuropathic sensations (n = 12), numbness (n = 10), fatigue (n = 10), difficulties with balance, and pain (n = 6). CIDP most commonly negatively impacted mobility (n = 12), activities of daily living (n = 12), emotional wellbeing (n = 11), work (n = 9), hobbies/leisure (n = 9), and fine motor skills (n = 8).

In a new iteration of NeuroVoices, lead investigator Kayla Scippa, associate director, Patient Reported Outcomes, Johnson & Johnson, sat down with NeurologyLive® following the meeting to provide greater clarity on the data and the major unmet needs for patients with CIDP. She spoke about how these findings will help inform drug development for the future, gearing efforts towards the symptoms that are most prevalent in CIDP. In addition, she touched upon some of the research goals and how to expand on this data going forward.

NeurologyLive: What were the greatest takeaways from your research?

Kayla Scippa: From this research, what we really understand is that the patient experience of living with CIDP is heterogeneous, and many patients experience numerous symptoms that have a negative impact on their ability to function and also their ability to participate in their lives to the fullest extent. Most frequently, we saw patients reported experiencing muscle weakness, neuropathic sensations, like tingling, or those pins and needles sensations, numbness, fatigue, balance difficulties, and pain. I should also note that muscle weakness was rated as one of the topmost bothersome symptoms by the majority of patients in our research sample, which makes sense when you think about the functional impact that muscle weakness can have on patients' ability to be mobile. When you can't move around, and you can't participate and do those core activities of daily living, which many times can involve the use of needing assistive equipment or the need to even adapt their homes to the kind of the changes in environments that are needed to their mobility levels, it can have a real impact on patients' quality of life. What we also saw from this research is the extensive impact that CIDP has on daily life beyond just those symptom experiences and their functional abilities. We saw patients describe extensively how CIDP impacted their ability to maintain relationships with others, how it impacted their emotional well-being, as well as even their ability to kind of go to work or go to school.

Are there underappreciated aspects to CIDP that clinicians aren’t fully aware of?

One of the main reasons why we did this research is to our knowledge, there wasn't a conceptual model that described the relationship between the symptoms of CIDP and its impacts on patients. There was a real unmet need in order to understand and characterize how patients living with CIDP feel and function and to try to visualize the connections between symptoms and impacts. But outside of this specific research, and in the context of rare disease, one of the main challenges can be that the disease itself is unknown and underrecognized.

We heard from our research that many patients experienced a delay in diagnosis because providers may not necessarily be familiar with a disease that's this rare, and it can be also a bit of a process to get to the right diagnosis. And to your point, I think one of the main gaps our research really aimed to address is to try to understand that patient perspective of the disease, and what areas are most important and most impacted in their lives. Patients are uniquely positioned to be one of the best recorders of their disease experience. In CIDP, a clinician can't look at a patient and discern the severity of pins and needles sensations that the patient is experiencing, that's a symptom that only patients feel. We need to pay very close attention to how patients are describing their disease experience. I think that's an area that's under recognized more broadly in rare disease, although there's greater emphasis lately, and making sure that we have patients' voices captured in that way.

What are the next steps following this research?

As somebody who has spent a lot of time thinking about patient quality of life and working in patient-reported outcomes, what we know generally is that patients need better treatment options that can help better address the core CIDP symptoms and get them back to participating in daily life as they wish. Like I said, the impact of CIDP is vast in that muscle weakness is a core symptom. It impacts people's ability to be mobile, to do daily activities like go to the grocery store, and even participate in leisure activities and hobbies. For example, you take someone who previously was out in their garden every day working on their flower beds and taking great joy in that activity. After their CIDP symptoms emerge, they can't walk around unassisted, or even on treatment, they have real trouble being able to participate and to do those activities. It can be a real strain on patients' lives. In terms of improvements in quality of life, I think what we're really looking at is filling a gap for that unmet need for patients so that they can have better treatment options that get them back to functioning the way that they would wish.

Are there certain types of research we need to conduct for this patient population?

With respect to our planned research, what we're interested in and the next steps for our work is really to understand what matters most to patients living with CIDP so that we can have a better understanding of those outcomes of priority. It's really important that we're measuring what matters to patients, and that the treatment options available are addressing their core disease experience. That can extend even beyond those core symptoms and functional impacts. But really, that's the point of having a comprehensive understanding of disease experience, because it may not be enough for patients living with CIDP just to get a little improvement of muscle weakness if that doesn't change their daily life and how they function within it.

Are there anything else noteworthy?

I think the last thing that I would say is really the point of our research with this conceptual model is to reinforce the outcomes that matter to patients. And how can we make sure that treatments that are under development or that are on the market are really addressing that patient unmet need. In addition, we want to have a great understanding of not only how patients experience the symptoms of CIDP, but where we need to go to make sure that patients can get back to their daily activities and to participate to that extent that they would like.

Transcript edited for clarity. Click here for more coverage of AAN 2024.

1. Scippa K, Macey J, Batista A, et al. Recognizing unmet need in chronic inflammatory demyelinating polyneuropathy: development of a conceptual model characterizing patient experiences and perspectives. Presented at: 2024 AAN Annual Meeting; April 13-18; Denver, CO.
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