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Catch up on any of the neurology news headlines you may have missed over the course of January 2025, compiled all into one place by the NeurologyLive® team.

FDA Action Update, January 2025: Approvals, Clearances, and Designations

Julie Flygare, JD, president and CEO at Project Sleep, discussed the critical role of social connections and peer support in helping individuals with sleep disorders navigate their diagnosis.

The Power of Social Support in Narcolepsy and Idiopathic Hypersomnia Care

Videos

The director of the Montefiore Headache Center at Albert Einstein College of Medicine discussed the recent FDA approval of a new migraine treatment that combines rizatriptan and meloxicam. [WATCH TIME: 5 minutes] 

Clinical Perspective on Newly Approved Combination Therapy for Migraine: Richard B. Lipton, MD

SPN-830 is a wearable subcutaneous infusion device designed to deliver continuous treatment throughout the waking day, ensuring more consistent control of OFF time.

FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment

Marco Meglio, Assistant Managing Editor for 

, has been with the team since October 2019. Follow him on Twitter 

A group of empowering women leaders in neurology explore the transformative impact of women neurologists on the medical workforce and the strides toward a more inclusive neurology field.

Episode 2: Innovative Policies That Advance Women in Neurology Departments

Deborah Benzil, MD, FAAS, vice chair of neurosurgery at Cleveland Clinic's Center for Spine Health, shared her extraordinary journey of resilience, from overcoming repeated rejections to becoming a trailblazer for women in neurosurgery, and reflects on the progress made and the challenges that remain.

Finding Happiness as a Trailblazer

Jacqueline A. French, MD, a professor of neurology at the NYU Grossman School of Medicine, provided clinical perspectives on her experiences and career growth in neurology amid National Women Physicians Day.

The Female Leaders Who Shaped an Epilepsy Icon: Reflections From Jacqueline A. French, MD

Jean Hubble, MD, a retired movement disorder specialist and consultant to the PMD Alliance, shares her reflections on National Women Physicians Day, the unique contributions of women in neurology, and the pathways to fostering future leaders in movement disorders care.

Advancing Neurology Through Compassion and Innovation: Insights from Dr. Hubble

NeurologyLive is your direct connection to the latest neurology news and interviews with expert neurologists in multimedia formats.

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Can we see changes in the brain that correspond to cognitive difficulties? Research presented at AAN 2016 offers compelling information.

Cognitive and Psychiatric Impairment in Parkinson 

How do you respond when a patient with epilepsy asks about the safety of pregnancy? The WEPOD study investigated pregnancy outcomes and deliveries.

Pregnancy and Childbirth in Women with Epilepsy

Can use of an online platform improve patient self-management in a group of Veterans with epilepsy?

Overcoming Epilepsy Self-Management Barriers

Researchers studied the prevalence and incidence of epilepsy in the elderly and confirmed three risk factors in this patient population.

Epilepsy Risk Factors in the Elderly

Interactive case study discussions highlighted the issue that treating multiple sclerosis often involves swimming murky waters.

Case-Based Considerations in MS Treatments 

To improve the quality of care for patients with epilepsy, AAN updated a set of measures and guidelines in 2014, but does a gap still exist?

Improving Epilepsy Quality of Care 

’ resubmission of its new drug application (NDA) in September 2024, the agency has approved AXS-07, an oral, rapidly absorbed, multi-mechanistic agent, as a new acute treatment of migraine with or without aura in adults. Marketed as Symbravo, the company noted that it anticipates AXS-07 to be commercially available in the United States in approximately 4 months.

AXS-07, a novel agent, is thought to act by inhibiting calcitonin gene-related peptide (CGRP) release, reversing CGRP-mediated vasodilation, and inhibiting neuroinflammation, pain signal transmission, and central sensitization. The agent, which consists of meloxicam and rizatriptan, is enabled by Axsome’s MoSEIC (Molecular Solubility Enhanced Inclusion Complex) technology, which results in faster absorption and longer plasma half-life.

"In terms of use in clinical practice, we want to treat our patients with the simplest, most effective acute treatments that we're able to offer them. So, you would not use a combination triptan and non-steroidal product in a patient whose treatment needs were fully met, either by a triptan alone or by a non-steroidal anti-inflammatory alone," 

, professor of neurology and director of the Montefiore Headache Center at 

 in a recent interview. "In fact, the design for individuals with unmet needs on single, monotherapy acute treatments was specifically a reflection of the fact that those are the patients who need combination therapy. I think that's something most prescribers would do and should do."

 for its NDA submission of AXS-07, citing issues related to the chemistry, manufacturing, and controls considerations. At the time, the agency did not request any new clinical trials to be conducted.

The original NDA, accepted for review in September 2021, was supported by data from 2 phase 3 randomized controlled clinical trials—the MOMENTUM trial (NCT03896009) and the INTERCEPT trial (NCT04163185). Overall, the studies revealed a statistically significant elimination of migraine pain with AXS-07 compared with placebo and active controls. 

, AXS-07 met its coprimary end points with statistical significance, first showing a greater percentage of patients achieving pain freedom (19.9%) compared with placebo (6.7%; 

 <.001) 2 hours after dosing. Second, the absence of the most bothersome symptom (36.9%) compared with placebo (24.4%) was significant (

In MOMENTUM, 1594 patients with migraine were randomly assigned 2:2:2:1 to AXS-07, 10-mg rizatriptan, MoSEIC 20-mg meloxicam, or placebo, to treat a single migraine attack of moderate or severe intensity. INTERCEPT included 302 patients with migraine who were randomly assigned to either AXS-07 (n = 152) or placebo (n = 150) at the earliest onset of migraine pain.

 “It's always good when a new treatment is approved for migraine. This particular combination tablet is something that I've been working on as an investigator for years, so I'm delighted that it got approved, and I'm looking forward to when it becomes available in the next several months for patients for the acute treatment of migraine," 

, professor of neurology at the Geisel School of Medicine at Dartmouth and vice president of the New England Institute for Neurology and Headache, told 

 in a recent interview. "The hope is that as we educate practitioners, the understanding of why combining an [nonsteroidal anti-inflammatory drugs] with a triptan should provide additional benefit in the acute treatment of migraine for patients will become more and more clear, and that is my hope for the future."

In April 2020, months after the initial readout of MOMENTUM, Axsome announced 

. All told, the coprimary end points were also identical to MOMENTUM, with a statistically significantly greater percentage of patients achieving pain freedom compared with placebo (32.6% vs 16.3%, respectively; 

 = .002) at 2 hours post dose, as well as freedom from the most bothersome symptom (43.9% vs 26.7%, respectively; 

 = .003). AXS-07 also showed efficacy in preventing progression of migraine pain beyond mild intensity and significantly reducing the use of rescue medication, with freedom of pain progression occurring in 73.5% of AXS-07-treated patients compared with 47.4% of placebo patients between 2 and 24 hours post dose (

“Today’s approval of SYMBRAVO marks an important milestone for the migraine community by providing a rationally designed novel acute treatment for this debilitating condition. Migraine attacks strike without warning, and disrupt the lives of estimated more than 39 million patients in the U.S. alone," Herriot Tabuteau, MD, chief executive officer at Axsome Therapeutics, said in a statement.

 "SYMBRAVO provides patients and clinicians an important new option which can quickly stop a migraine attack, keep it away, and allow patients to resume their normal activities, with just a single dose. SYMBRAVO demonstrates Axsome’s commitment to developing and delivering differentiated new treatments to improve the lives of patients living with difficult to treat central nervous system disorders.”

2024 American Academy of Neurology Annual Meeting

pooled analysis of the MOMENTUM and INTERCEPT trials

, with results showing that AXS-07 was effective at acutely treating migraine. In the pooled analysis, treatment with AXS-07 resulted in greater headache pain freedom (23% vs 11%; 

<.001) and absence of the most bothersome symptom (39% vs 25%; 

<.001) at 2 hours compared with placebo. Led by Stewart J. Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth, 43% of AXS-07-treated patients had reduced rescue medication use through 24 hours vs 21% of those on placebo (

<.001). Notably, a higher rate of patients on the investigational agent returned to normal functioning in comparison with placebo starting at 1-hour post-dose.

“Migraine is one of the most misunderstood and stigmatized neurological diseases, despite the fact that one in four households in the U.S. includes someone living with it. This widespread lack of understanding creates unnecessary barriers for individuals seeking proper diagnosis, care, and treatment," Susan Doughty, executive director of the Coalition for Headache and Migraine Patients (CHAMP), said in a statement.

 "CHAMP, alongside our 20 plus dedicated coalition organizations and patient advocates, is committed to empowering the migraine community by providing education, reducing stigma, and advocating for fair and equitable access to treatment options. The approval of SYMBRAVO as a new acute treatment for migraine is an important step forward, offering a new option for people seeking relief. We also see this moment as an opportunity to continue to shine a bright light on migraine, fostering greater awareness and helping to dismantle the stigma that so often surrounds this disease.”

REFERENCES
1. Axsome Therapeutics Announces FDA Approval of SYMBRAVO® (meloxicam and rizatriptan) for the Acute Treatment of Migraine with or without Aura in Adults. News Release. Axsome Therapeutics. Published January 30, 2025. Accessed January 30, 2025. https://axsometherapeuticsinc.gcs-web.com/news-releases/news-release-details/axsome-therapeutics-announces-fda-approval-symbravor-meloxicam
2. Axsome Therapeutics Receives FDA Complete Response Letter for New Drug Application for AXS-07 for the Acute Treatment of Migraine. News release. Axsome Therapeutics. May 2, 2022. Accessed January 30, 2025 https://www.prnewswire.com/news-releases/axsome-therapeutics-receives-fda-complete-response-letter-for-new-drug-application-for-axs-07-for-the-acute-treatment-of-migraine-301537093.html
3. Axsome Therapeutics Announces AXS-07 Achieves Co-Primary and Key Secondary Endpoints in MOMENTUM Phase 3 Migraine Trial in Patients with History of Inadequate Response. News release. Axsome Therapeutics. December 30, 2019. Accessed January 30, 2025. https://www.biospace.com/article/releases/axsome-therapeutics-announces-axs-07-achieves-co-primary-and-key-secondary-endpoints-in-momentum-phase-3-migraine-trial-in-patients-with-history-of-inadequate-response
4. Axsome Therapeutics announces AXS-07 achieves both co-primary endpoints and prevents migraine pain progression in the INTERCEPT phase 3 trial in the early treatment of migraine. news release. Axsome Therapeutics. April 6, 2020. Accessed January 30, 2025. https://www.finance.yahoo.com/news/axsome-therapeutics-announces-axs-07-100010869.html
5. Tepper S. Lipton R, Chhabra A, Streicher C, Parks G, Tabuteau H. Combined efficacy and safety of AXS-07 (MOSEICTM Meloxicam and Rizatriptan) in two phase 3 clinical trials. Presented at: 2024 AAN Annual Meeting; April 13-18; Denver, CO. Abstract 00519

The FDA has approved AXS-07 (Symbravo), a novel oral treatment for acute migraine in adults, developed by Axsome Therapeutics. AXS-07 combines meloxicam and rizatriptan, utilizing MoSEIC technology for rapid absorption and extended plasma half-life. Clinical trials, including MOMENTUM and INTERCEPT, demonstrated significant efficacy in achieving pain freedom and reducing migraine symptoms compared to placebo. The approval addresses unmet needs in migraine treatment, offering a new option for patients with inadequate response to existing therapies. AXS-07 is expected to be available in the U.S. within four months.

Findings from previous phase 3 trial showed that treatment with AXS-07 resulted in significant reductions in headache pain freedom, migraine symptoms, and rescue medication use.

Clinical

FDA Approves Axsome Therapeutics’ AXS-07 for Migraine Treatment

A recently published international survey reported that a small portion of patients with narcolepsy or idiopathic hypersomnia felt supported when adjusting to life with their condition. Published in 

the results of the survey highlighted the need for clinicians to assess social support and guide patients living with these sleep disorders toward resources that can foster community connections.

The online survey, conducted by Project Sleep

, gathered responses from 1308 adults with narcolepsy type 1 (48.4%), narcolepsy type 2 (28.7%), or idiopathic hypersomnia (22.9%). Participants were predominantly women (87.7%), White (88.8%), and under 40 years of age (68.7%). Participants represented 38 countries, with the majority (65.5%) residing in the United States, followed by 15.4% in Europe and 11.8% in Australia/New Zealand.

Adjusting to life with a diagnosis was challenging for the majority of respondents, with 67.6% rating the process as difficult or very difficult. Individuals with idiopathic hypersomnia reported greater difficulty compared with those with narcolepsy. Overall, only 32.3% of respondents felt supported in adapting to life with their condition.

"Clinicians see multiple patients with narcolepsy or idiopathic hypersomnia, but those patients don’t see or connect with each other. For those who did meet others, there was a wide range of experiences—some found social support quickly, within a few months or years, while others took much longer," lead author Julie Flygare, JD, president and CEO at Project Sleep, told  

 in a recent interview. "Visually, I think of social connection as opening a door. Without it, you’re behind a closed door, alone. But when that door opens, you see a world of others who share your experience. That’s powerful."

Led by senior author sleep disorder expert 

, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children's Hospital, the online survey was conducted between March 28 and April 11, 2021. The survey, composed of 30 questions, asked respondents about their diagnosis, time from symptom onset to diagnosis, ease or difficulty in adjusting to their diagnosis, whether or not they knew someone with the same diagnosis, the impact of knowing another person with the same diagnosis, methods of communicating with other people with their diagnosis and topics discussed, and sources of support.

"We weren’t really sure how to develop the survey because I hadn’t seen this question asked so directly in other communities either. So, we asked people whether they knew someone living with the same condition at the time of their diagnosis. Very few did," Flygare said. "In some cases, there are familial groupings—sometimes a child gets diagnosed because they have a parent with narcolepsy, or families become more aware of sleep disorders and recognize them more easily in their relatives. But for most, they didn’t know anyone else."

Delays in diagnosis were frequently reported, with over one-third of respondents indicating it took 10 years or more after symptom onset to receive a diagnosis, and only one-quarter were diagnosed in 3 years. Diagnostic timelines varied by condition, for example, individuals with narcolepsy type 1 were more likely to be diagnosed in 3 years (29.2%) compared with those with narcolepsy type 2 (17.9%) or idiopathic hypersomnia (17.7%). Conversely, a higher proportion of respondents with idiopathic hypersomnia (44.7%) experienced delays of 10 years or more, compared with those with narcolepsy type 2 (38.9%) and narcolepsy type 1 (35.2%).

FDA Clears Phase 2 Trial of Lorundrostat for Obstructive Sleep Apnea and Hypertension

At the time of diagnosis, most respondents (91.1 %) did not know anyone else with the same condition. This was reported by nearly all individuals with idiopathic hypersomnia (96.0%), as well as 88.8% of those with narcolepsy type 1 and 91.2% of those with narcolepsy type 2. These rates were consistent across geographic regions, with 91.0% of U.S. respondents and 91.2% of respondents from other countries reporting similar experiences. Rates were also comparable across age groups: 89.8% for ages 18–30, 92.1% for ages 31–50, and 91.3% for those over 50.

Regression analysis revealed that knowing someone with the same diagnosis was strongly associated with a greater sense of support. Younger respondents (ages 18–30) and those who were married also reported higher levels of perceived support. However, individuals with narcolepsy type 2 or idiopathic hypersomnia reported feeling less supported than those with narcolepsy type 1. Living outside the U.S. was another factor linked to lower perceived support. Additionally, respondents who identified as nonbinary, transgender, or genderfluid tended to feel less supported compared with those who identified as women (

"People with narcolepsy are diverse, and not every patient connection results in finding a new best friend. But over time, when people engage with patient advocacy groups, participate in programs, and take part in advocacy, they meet different individuals. Eventually, they often find close connections with others who truly understand their journey. They also find role models—people further along in their experience—who they can look up to. That’s different from peer support but just as important," Flygare explained in the interview.

The current study had several limitations including that diagnoses were self-reported and not verified through medical records. Additionally, the survey did not collect data on symptoms or their severity, making it impossible to analyze the relationship between social support and symptomatology. Conducted in 2021 during the COVID-19 pandemic, the survey’s findings may have been influenced by the unique social dynamics of that time, though the extent of this impact is unclear. While duplicate submissions were removed, there remains a small chance that some individuals submitted multiple responses with variations. However, this risk was deemed minimal and acceptable by researchers to preserve participant anonymity.

“I think clinicians can start by simply asking their patients, 'Do you know someone else living with this condition?' It’s a great way to begin a conversation. Social support can take many forms—not everyone wants to go to a support group, and that’s okay. But letting patients know about available patient advocacy organizations is crucial. In the U.S., there are 4 major groups, plus others worldwide. These organizations are great starting points for patients to explore what’s available to them,” Flygare highlighted in the conversation. "We often hear from people who went years after their diagnosis without knowing that Project Sleep existed, and when they finally find our resources, they’re so excited. That just feels sad because they could have had that support much earlier. So, letting patients know about advocacy organizations is a good start."

REFERENCES
1. Flygare J, Oglesby L, Parthasarathy S, et al. Social support and isolation in narcolepsy and idiopathic hypersomnia: An international survey. Sleep Med. 2025;125:65-73. doi:10.1016/j.sleep.2024.11.013
2. Project Sleep. Homepage. Accessed January 23, 2024. https://project-sleep.com/

A recent international survey highlights the challenges faced by patients with narcolepsy and idiopathic hypersomnia in finding social support. Conducted by Project Sleep, the survey involved 1308 participants, revealing that only 32.3% felt supported in adapting to their condition. Delays in diagnosis were common, with many taking over a decade to receive a diagnosis. Knowing someone with the same condition was linked to increased perceived support. The study underscores the need for healthcare providers to facilitate connections and guide patients toward supportive resources, despite privacy concerns.

In a recent survey, key factors associated with perceived support included knowing someone who had the same diagnosis of their sleep disorder, younger age, and marital status. 

Survey Highlights Patients’ Need for Support in Adapting to Life in Narcolepsy or Idiopathic Hypersomnia

New topline results from the randomized, double-blind, placebo-controlled phase 2 trial (NCT05109091) of Alterity Therapeutics’ ATH434 revealed that treatment with the investigational agent was well tolerated and demonstrated clinical as well as biomarker-based benefits, particularly at the 50 mg dose, among patients with early-stage multiple system atrophy (MSA).These findings support ATH434’s potential as a disease-modifying therapy for MSA to slow disease progression and reduce iron accumulation in key brain regions.

The study enrolled 77 participants across 23 sites in 6 countries, who received either 50 mg or 75 mg of ATH434 or placebo twice daily for 12 months. Findings showed that ATH434 was well tolerated and effectively reduced iron accumulation in brain regions impacted by MSA. The 50 mg dose demonstrated the greatest benefit, slowing clinical progression by 48% at 52 weeks compared with the placebo (

 = 0.03). Additional analyses also suggested trends in improved motor function and preserved brain volume.

"I was impressed with the effects, that appear durable over 52 weeks in the 50mg dose. As a clinical signal, it supports progressing to a larger phase 3 trial. We want to see a therapy have a chance to change the way MSA progresses. This is a step in the right direction. We see a clear signal for disease modification which is truly exciting," coordinating investigator 

"We see a clear signal for disease modification which is truly exciting. This is a new concept for MSA. There is a clear antioxidant mechanism, an effect on labile iron, and indication that MSA is important for clinical development of this compound."

The primary end point of the trial assessed brain iron content via MRI. All told, ATH434 reduced or stabilized iron accumulation in affected brain regions, with the 50 mg dose showing significant reductions in the putamen at 26 weeks (

 = 0.025) and approaching significance in the globus pallidus at 52 weeks (

 = 0.08). Clinical efficacy was assessed using the Unified MSA Rating Scale Part I (UMSARS I), a common MSA assessment that measures disability and disease severity.

 Overall, patients on the 50 mg dose experienced a 48% slower disease progression at 52 weeks (

 = 0.03) compared with placebo, and the 75 mg dose slowed progression by 29% (

= 0.2). At 26 weeks, the 75 mg dose showed a 62% slowing of disease progression (

Syn-One Test Displays Significant Clinical Utility for the Diagnosis of Synucleinopathies

“We are thrilled that ATH434 has demonstrated significant slowing of clinical progression and an excellent safety profile in this rare, rapidly progressive disease,” David Stamler, MD, chief executive officer at Alterity, said in a statement.

 “Currently, there are no approved treatments that slow the progression of MSA and these results show that ATH434’s targeted iron engagement may truly have a disease modifying effect.”

Additional secondary end points included improved motor performance, with the 50 mg dose showing significant benefit on the Clinical Global Impression of Severity scale (mean change, -0.81; 

= 0.009). Wearable sensors also indicated increased step count, walking time, and standing time in treated patients. ATH434 was well tolerated, with adverse event rates comparable with placebo. Most adverse events were mild to moderate, and no treatment-related serious adverse events were reported. The 50 mg dose had the lowest rate of discontinuations because of adverse events (n = 0), compared to the 75 mg dose (n = 5) and placebo (n = 3).

“The fact that we achieved statistical significance on the UMSARS is extremely meaningful because it assesses the functional areas affected in MSA and is the endpoint needed to support drug approval by the U.S. Food and Drug Administration (FDA),” Stamler said in a statement.

 “Based on the strength of these Phase 2 data, we look forward to engaging with the FDA as quickly as possible to discuss the path forward for accelerating the development of ATH434 given the tremendous unmet need for treating MSA. We are very grateful for the invaluable contributions of the study participants and the clinical sites who contributed to the study.”

Stamler concluded in a statement, “We now have evidence that targeting excess labile iron in neurodegenerative disease can be achieved. By redistributing this reactive form of iron that contributes to disease pathogenesis, not only can we target α‑synuclein aggregation, but we can also break the vicious cycle underlying disease progression. This has implications for developing disease modifying treatments for orphan diseases such as MSA and Friedreich’s ataxia as well as major neurodegenerative disorders such as Parkinson’s disease and Alzheimer’s disease.”

REFERENCES
1. Alterity Therapeutics Announces Positive ATH434 Phase 2 Trial Results in Multiple System Atrophy Led By Robust Clinical Efficacy. News Release. Alterity Therapeutics. Published January 30, 2025. Accessed January 30, 2025. https://alteritytherapeutics.com/investor-centre/news/2025/01/30/alterity-therapeutics-announces-positive-ath434-phase-2-trial-results-in-multiple-system-atrophy-led-by-robust-clinical-efficacy/
2. Geser F, Wenning GK, Seppi K, et al. Progression of multiple system atrophy (MSA): a prospective natural history study by the European MSA Study Group (EMSA SG). Mov Disord. 2006;21(2):179-186. doi:10.1002/mds.20678

ATH434, an investigational agent by Alterity Therapeutics, showed promise in a phase 2 trial for early-stage multiple system atrophy (MSA). The trial demonstrated that ATH434 was well tolerated and reduced iron accumulation in key brain regions, with the 50 mg dose significantly slowing disease progression by 48% over 52 weeks. The study's primary endpoint was brain iron content, with secondary endpoints including motor performance improvements. These findings suggest ATH434's potential as a disease-modifying therapy for MSA, with implications for other neurodegenerative disorders.

Recent results from a phase 2 trial of ATH434 revealed its potential in reducing brain iron accumulation and preserving motor function in early-stage multiple system atrophy.

Multiple System Atrophy Agent ATH434 Shows Promising Topline Efficacy in Phase 2 Trial

Delays in initiating immunotherapy for chronic inflammatory demyelinating polyneuropathy (CIDP) led to worsened axonal damage and higher disability, according to a newly published study from the Immune-mediated Neuropathies Biobank (INHIBIT). Published in the 

, researchers also observed that comprehensive therapeutic approaches could potentially improve long-term outcomes in patients with CIDP.

"The clinical course of [patients with CIDP] is the most important marker for treatment decisions. As the disease course is slowly progressive in many patients, [nerve conduction studies] and ultrasound findings are important additional aspects that can lead to increased vigilance or support a treatment decision," senior author Thomas Grüter, MD, professor of neurology at St. Josef Hospital Ruhr University Bochum in Germany, told 

Investigators included 30 patients diagnosed with CIDP within 1 year prior to enrollment for the study. Authors used clinical, therapeutic, and diagnostic data including nerve conduction studies and ultrasounds to analyze outcomes. Findings showed that delays in starting therapy correlated with axonal damage (r

 = 0.467) and increased disability (R² = 0.200). Conversely, early and combined first- or second-line therapies significantly reduced disability progression (r = 0.773) and improved axonal damage (r = 0.467).

The findings in the current study emphasized the need for prompt treatment strategies to minimize disability. Axonal damage, which was linked to higher overall disability sum scores (r

 = 0.581), emerged early in the disease course, highlighting the importance of early intervention. Researchers also noted the results from the study suggested that multimodal approaches, combining therapeutic and diagnostic tools, may optimize patient outcomes.

"If the diagnosis is uncertain, the available diagnostic tools should be exhausted, including nerve biopsy in cases of doubt," Grüter explained. "As intravenous immunoglobulins and cortisone are well tolerated first-line treatments, they should be started as soon as possible. The treatment response can also be used as a diagnostic tool."

Timely Diagnosis and Monitoring Key to Preventing CIDP Disability, Study Shows

This research included patients from the INHIBIT registry, with a diagnosis-to-enrollment time averaging 22 weeks. Patients were diagnosed based on the European Federation of Neurological Societies/Peripheral Nerve Society diagnostic criteria.

 Clinical and diagnostic data were assessed at baseline and after 12 months. Patients were systematically selected to ensure early disease representation, with equal monitoring of therapeutic interventions. Nerve conduction study scores, pathological spontaneous activity, and Heckmatt scale readings were key indicators, which were all significantly associated with disability progression.

"The outcome of the [patients with CIDP] in our cohort was analyzed using various clinical and instrumental methods. Clinically, we analyzed the patients using established scores such as the mRS, INCAT-ODSS and vigorometry. Additionally, we used [nerve conduction studies] and nerve ultrasound," Grüter noted in the interview. "Reduced amplitudes in [nerve conduction studies], pathological spontaneous activity in myography, and a fibrotically remodeled muscle in muscle ultrasound are indications of axonal damage. This was done by the same operator in a prospective approach as part of the INHIBIT rigestry."

Overall, therapeutic insights from the study revealed that early initiation of therapy was crucial for reducing axonal damage and preventing disability progression. First-line therapies were most strongly associated with mitigating disability (r = 0.773), demonstrating their significant role in improving patient outcomes. Second-line therapies also provided notable benefits, particularly in addressing axonal damage (r = 0.467). Importantly, the study found no significant adverse events among any of the treatment groups, suggesting that these therapeutic approaches are not only effective but also well-tolerated.

"Many people with CIDP are sufficiently stable with available first-line therapy. However, there is a significant proportion of patients who experience rapid progression despite this therapy. In these patients, escalation of therapy, such as B-cell depletion, should be considered, even if it is not yet approved. In this case, the potential risks and side effects must be discussed very thoroughly with the patient," Grüter added. 

Authors noted that the study's single-center design ensured consistency but might have introduced biases related to patient demographics and disease presentation, limiting generalizability. By focusing on early CIDP, the researchers may have also excluded patients with severe cases who had already undergone multiple treatments. Additionally, the study's follow-up period may not have captured long-term effects or changes in treatment responses. Despite established criteria used to assess axonal damage and were validated, potential influences from very proximal conduction blocks and collateral sprouting could have impacted the accuracy of the results.

"Multimodal monitoring is essential in [patients with CIDP] because the slowly progressive nature of the disease makes monitoring difficult. Axonal damage is the most important predictor of clinical disability. It should therefore be assessed multimodally," Grüter said. "Monitoring with [nerve conduction studies] is often not possible, especially in cases of severe axonal damage. Nerve and muscle ultrasound offers new possibilities."

REFERENCES
1. Schumacher A, Hieke A, Spenner M, et al. Early therapy initiation is crucial in chronic inflammatory demyelinating polyneuropathy: prospective multimodal data from the German INHIBIT registry. J Neurol. 2025;272(1):100. Published 2025 Jan 7. doi:10.1007/s00415-024-12860-w
2. Van den Bergh PYK, van Doorn PA, Hadden RDM, et al. European Academy of Neurology/Peripheral Nerve Society guideline on diagnosis and treatment of chronic inflammatory demyelinating polyradiculoneuropathy: Report of a joint Task Force-Second revision [published correction appears in Eur J Neurol. 2022 Apr;29(4):1288. doi: 10.1111/ene.15225]. Eur J Neurol. 2021;28(11):3556-3583. doi:10.1111/ene.14959

Delays in initiating immunotherapy for CIDP result in worsened axonal damage and increased disability. Early and combined therapeutic approaches significantly reduce disability progression and improve axonal damage. The study emphasizes the importance of early intervention and multimodal approaches to optimize patient outcomes. Axonal damage is identified as the primary cause of clinical disability, necessitating further research to identify therapeutic options that reduce neurodegeneration. The study's single-center design may limit generalizability, but it highlights the potential of axonal damage as a biomarker for disease severity and treatment outcomes.

A new study highlighted the importance of initiating immunotherapy promptly in chronic inflammatory demyelinating polyneuropathy to prevent axonal damage and disability progression.

Lee Nourse

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