Flexibility and convenience are staples in most aspects of our lives due to new technology. Healthcare is no exception with the availability of instant access to medical records, online appointments and efforts to decrease wait times through better scheduling technology. With these advances, people expect greater flexibility and an experience centered around patient convenience.

But what about the clinical side? Niraj C. Patel, MD, Associate Professor, Pediatric Allergy and Immunology, Emory University, Children’s Healthcare of Atlanta, Atlanta, GA, who specializes in treating patients with Primary Immunodeficiency (PI), a genetic condition that impairs the immune system, participated in a Q&A with CSL Behring to discuss a recent survey centered around treatment preferences for people living with PI, and how he considers both convenience and flexibility in his practice. He emphasizes the importance of physicians and patients working together to determine the right treatment option.

How are considerations around patients’ lifestyles becoming more integrated into treatment decisions?

Today, more and more patients prefer and prioritize flexibility and convenience when it comes to their medical treatments.

People living with a rare disease like PI, or any chronic disease, require lifelong medical care and often lifelong treatments. For these persons especially, creating a patient experience that equally prioritizes flexibility, convenience and outcomes is critical.

How do you ensure you treat each patient with an individualized approach?

The physician/patient relationship is all about finding a balance between the science and the unique human needs of the patient. Both sides need to be heard and work in harmony to achieve optimal outcomes.

A physician’s day can be unpredictable as well as emotionally and physically taxing. Physicians often feel they don’t have enough time for thorough conversations with each patient, and appointments can feel rushed at times.

Before I enter an appointment with a patient, I take a few seconds to clear my mind and focus all my attention on them. I try to create an environment where my patients feel comfortable sharing what’s working well and what can be improved with their treatment plan.

What are the key factors you consider when developing a treatment plan for your PI patients?

For people living with life-long conditions like PI, finding a treatment that offers both efficacy and convenience is paramount in ensuring a favorable treatment experience and overall quality of life. People living with PI often receive regular infusions of immune globulin (Ig) replacement therapy to maintain their health, and this can interfere with their daily routine if they need to go to treatment centers to receive their medicine. A recent Harris poll survey of 104 PI patients sponsored by CSL Behring found that 94% of PI patients surveyed agree that flexibility and options are important in personalizing Ig treatment.

So, taking the time to understand a patient’s lifestyle can be an important factor when determining treatment. If a patient prioritizes flexibility and convenience, we may need to discuss whether they are candidates for subcutaneous immune globulin (SCIg), like 

 (Immune Globulin Subcutaneous [Human] 20% Liquid), which allow PI patients to self-infuse in the comfort of their home and on a schedule that is convenient for them.

During my initial conversations, I try to get a feel for how involved the patient wants to be in their treatment administration. Some patients prefer intravenous immune globulin (IVIg) – having someone else infuse their treatment – because they are nervous or unsure if they can handle sticking themselves with needles. For many patients, unfamiliarity with a treatment process can make them apprehensive. In these instances, I aim to arm my patients with information and resources, such as training videos and the reassurance that a nurse will be available for training patients to self-infuse. I believe we need to give patients the time and tools to make an informed decision and allow them to explore their options.

How often do you approach discussions about treatment options with patients?

I recommend asking patients at each appointment about their treatment experience and if there are concerns that arise, schedule a separate appointment to have a robust dialogue around all options, as well as background on their lifestyle, to inform the decision. I firmly believe that physicians must work in tandem with patients to align on a treatment plan. PI patients have a number of choices when it comes to treatment, but the thing I like to point out is whether we are talking about IVIg or SCIg, all Ig products approved by the FDA for treatment of PI meet certain standards in terms of efficacy. From there, it’s our responsibility to check in with our patients about their treatment experience to determine if there are any adjustments that need to be made. I’ve found that some issues with SCIg can be mitigated by making adjustments like changing the needle length or infusion site. We also need to ask about the impact of their treatment schedule on their quality of life – are they missing time with family or friends because of side effects or time spent infusing – and adjust treatment as needed.

According to the aforementioned survey sponsored by CSL Behring, at least 97% of surveyed PI patients using SCIg prefilled syringe reported being satisfied with the ease of administration and overall convenience. I find that patients who are busy with work, school, and family and are looking for the next thing in terms of convenience, are willing to give it a try.

At the end of the day, if a patient isn’t happy with their treatment, they are unlikely to adhere to it. Many physicians engage in some shared decision-making, but not to the extent that it should be, and it goes back to the obstacle of time.

What advice do you have for your fellow physicians to ensure their patients are on a treatment that best fits their needs?

Physicians need to take a holistic treatment approach and evaluate individual patient needs to customize treatment based on efficacy, as well as flexibility and convenience, to fit into the everyday life of those living with a life-long disease. We need to keep patients informed of any updates and new products to ensure they are on the best possible treatment for their unique needs.

Articles

WARNING: Thrombosis may occur with immune globulin products, including Hizentra. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.

For patients at risk of thrombosis, administer Hizentra at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.

Hizentra is contraindicated in patients with a history of anaphylactic or severe systemic reaction to human immune globulin (Ig) or components of Hizentra (eg, polysorbate 80), as well as in patients with immunoglobulin A deficiency with antibodies against IgA and a history of hypersensitivity. Because Hizentra contains L-proline as stabilizer, use in patients with hyperprolinemia is contraindicated.

IgA-deficient patients with anti-IgA antibodies are at greater risk of severe hypersensitivity and anaphylactic reactions. Thrombosis may occur following treatment with Ig products, including Hizentra.

Monitor patients for aseptic meningitis syndrome (AMS), which may occur following treatment with Ig products, including Hizentra. In patients at risk of acute renal failure, monitor renal function, including blood urea nitrogen, serum creatinine and urine output. In addition, monitor patients for clinical signs of hemolysis or pulmonary adverse reactions (eg, transfusion-related acute lung injury [TRALI]).

Hizentra is derived from human blood. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.

The most common adverse reactions (observed in ≥5% of study subjects) were local infusion-site reactions, as well as headache, diarrhea, fatigue, back pain, nausea, extremity pain, cough, upper respiratory tract infection, rash, pruritus, vomiting, upper abdominal pain, migraine, arthralgia, pain, fall, and nasopharyngitis.

The passive transfer of antibodies can interfere with response to live virus vaccines and lead to misinterpretation of serologic test results.

Hizentra®, Immune Globulin Subcutaneous (Human), 20% Liquid, is indicated for:

Treatment of primary immunodeficiency (PI) in adults and pediatric patients 2 years and older.

Maintenance therapy in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to prevent relapse of neuromuscular disability and impairment.

Limitation of Use: Maintenance therapy in CIDP has been systematically studied for 6 months and for a further 12 months in a follow-up study. Continued maintenance beyond these periods should be individualized based on patient response and need for continued therapy.

To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 

Striking the Right Balance: Shared Decision-Making in Primary Immunodeficiency Care

Vitiligo is a chronic autoimmune condition resulting in depigmented lesions of skin that can appear on any part of the body. The condition, which has been diagnosed in approximately 1.5 million people in the United States

, occurs with similar frequency in all ethnic groups and can occur at any age, although initial symptoms often appear before the age of 30

Given the visual nature of the disease, vitiligo is sometimes viewed as a cosmetic issue rather than as an autoimmune condition

. Because of this misconception, the severity and impact of vitiligo – both physical and emotional – can often be downplayed or overlooked. In addition, the limited treatment options currently available for vitiligo can lead people living with this condition to believe they have little to no choices

More than cosmetic: The role of the immune system in vitiligo

Research suggests vitiligo develops in genetically-susceptible individuals when environmental triggers – e.g., sun exposure, chemical exposure, physical skin trauma or psychological strain – induce stress responses in the skin

.This causes a localized autoimmune response in which melanocytes are targeted and destroyed by CD8+ T-cells

An environmental trigger combined with genetic factors leads to melanocyte stress, causing an inflammatory response

Innate immune cells activate CD8+ T-cells that target melanocytes and release IFN-γ

JAK-STAT activation and inflammatory cycle triggering: 

IFN-γ activates the JAK-STAT pathway (by binding to JAK 1 and 2) to induce production of immune mediators (CXCL9/10) by keratinocytes. More autoreactive T-cells are attracted to the site, where they continue the inflammatory cycle by producing more IFN-γ in a positive feedback loop

The T-cell attack creates a hostile environment resulting in melanocyte death, which leads to depigmentation

People living with vitiligo would benefit from additional options to consider with their health care team.

Vitiligo has a significant burden of disease

The social stigma around vitiligo, and the idea that it is a cosmetic issue rather than an autoimmune condition, can significantly impact people with vitiligo. Findings from the global 

ernational Communities (VALIANT) study underscore the profound impact vitiligo has on people’s lives. This was an observational, cross-sectional evaluation of the burden of vitiligo from the perspective of patients aged 18 years and older with vitiligo and health care professionals who treat patients with vitiligo

Of the more than 3,500 adults with vitiligo surveyed, nearly 60% said they had been diagnosed with mental health conditions, including anxiety (28.8%) and depression (24.5% )

49% said having vitiligo made them feel less confident and more self-conscious

Some of those surveyed also said they experience sleep disturbance and approximately half said the progression of their disease worries them

Additionally, a patient-focused meeting held by the U.S. Food and Drug Administration (FDA) in March 2021 explored the health effects and daily impact of vitiligo as well as management goals

. Among the key findings was that vitiligo can lead to feelings of loss of identity, particularly among African Americans

. Some participants expressed that others are unable to see past their condition, making it hard to establish successful friendships or secure employment

As such, it is critical for health care providers to approach discussions about vitiligo with their patients as an autoimmune condition and to consider the full impact that vitiligo can have on a person’s daily life. By educating people with vitiligo about the science behind their condition and forming a doctor-patient relationship based on trust, an individualized management plan tailored to a patient’s specific needs and goals can be established.

Supporting patients and managing vitiligo

As a result of their disease, people with vitiligo may feel frustrated, misunderstood and unheard, and their health care teams may have a challenging time supporting their needs. It is essential that dermatologists and other health care providers who diagnose and manage vitiligo discuss the science behind the condition and the impact it may have with their patients. Approaches to engage in these conversations can include:

Explain the autoimmune nature of vitiligo

Establish a regular cadence of appointments to ensure appropriate follow up

It is important that dermatologists take the time to understand how the condition impacts their patients’ quality of life and emotional well-being. Management of vitiligo is a process, and expectations need to be set based on each patients’ individualized management plan. We need to form open lines of communication with patients early on and provide easily understood information about the science behind the condition. We can thereby help people living with vitiligo see it as an autoimmune rather than cosmetic issue and support informed decisions about their health and the overall management of their disease.

Note: Dr. Batra has been compensated for her time.

Bowcock AM, Fernandez-Vina M. Targeting skin: vitiligo and autoimmunity. 

. https://www.sciencedirect.com/science/article/pii/S0022202X1535452X. Accessed June 23, 2022.

National Library of Medicine. Medline Plus. Vitiligo. 

https://medlineplus.gov/genetics/condition/vitiligo/

Frisoli M, Essien K, Harris JE, et al. Vitiligo: mechanisms of pathogenesis and treatment. 

U.S. Food and Drug Administration. The voice of the patient: A series of reports from the U.S. Food and Drug Administration’s (FDA’s) patient-focused drug development initiative. https://www.fda.gov/media/155068/download. Accessed May 4, 2022.

Manga P, Elbuluk N, Orlow SJ. Recent advances in understanding vitiligo. 

Rodrigues M, Ezzedine K, Hamzavi I, Pandya AG, Harris JE; Vitiligo Working Group. New discoveries in the pathogenesis and classification of vitiligo. 

Strassner JP, Harris JE. Understanding mechanisms of autoimmunity through translational research in vitiligo. 

Damsky W, King BA. JAK inhibitors in dermatology: the promise of a new drug class. 

Howell MD, Kuo FI, Smith PA. Targeting the Janus kinase family in autoimmune skin diseases. 

Bibeau K, Hamzavi IH, Ezzedine K, et al. Mental health and psychosocial burden among patients living with vitiligo: findings from the global VALIANT survey. Presented at Maui Derm for Dermatologists, January 2022.


More Than a Cosmetic Concern – Increasing Understanding of and Addressing Challenges and Care in Vitiligo, an Autoimmune Condition

COVID-19 has disrupted healthcare practices and challenged Primary Care Providers (PCPs) to rapidly innovate to deliver care. It has also upended patients’ lives and forced many to skip preventive care checkups and screenings, as well as highlighted gaps in chronic diseases and disparities in outcomes. In the last two years, cancer screening rates have plummeted in nearly all age groups

despite a rising incidence of colorectal cancer (CRC) in younger adults.

 Colorectal cancer is the second leading cause of cancer deaths in the United States;

 however, when caught in early stages, it is treatable in ~90% of people.

Regular screening can help catch cancer early.

 So, as we consider patient care in light of the ongoing pandemic, how can we encourage eligible adults to get screened when they’re due? We have an opportunity to redesign our approach – action is needed across the healthcare spectrum to ensure health systems, community centers, and PCPs partner to prioritize health literacy and ensure patients have the necessary support and resources to make informed decisions.

Increasing health literacy is critical to combatting colorectal cancer. With the recommended colorectal cancer screening age starting at 45 for average-risk Americans

, there should be a sense of urgency in finding new ways to engage with patients around preventive care measures.

New data from The Harris Poll** highlighted a lack of awareness about the risk*** of colorectal cancer. Many of those surveyed perceived themselves to be at minimal risk. In fact, 73% of respondents aged 45-49 believed they are at low risk for colorectal cancer. This demonstrates a critical and concerning gap in health literacy. We have a responsibility to ensure screening-eligible Americans, particularly adults aged 45-49 and those in vulnerable communities, have the resources and information they need to understand why screening should be a priority.

So, what steps can we take to drive progress? While I would love to see sweeping public health changes on initiatives for self-management of preventive care, where colon cancer screenings could be as accessible and convenient as a home-delivered rapid COVID antigen test, taken after using a validated screening calculator to self-assess their risk level for colorectal cancer, I believe that there are a few simple actions that can make a large impact now. 

We could reframe how we engage in cancer screening conversations. It is easy to lead with, “Now you’re 45, it’s time for your colonoscopy.” But perhaps start with, “Do you ever think about getting cancer?” It might be a surprising question to hear, even alarming, but it could give you some insights on your patient’s fears or willingness to screen for the recommended cancers or only specific types and why. Primary care providers are a trusted resource and can start the conversation about personal risk and share the best screening options to meet patients where they are.

Another option is to offer a choice in colon cancer screening options to patients first, before waiting to hear if there is any hesitancy about colonoscopy. While a colonoscopy is considered the primary screening option for colorectal cancer, offering a choice in screening can encourage those who have hesitancy to get scoped.

 Many respondents to the Harris Poll noted that their discomfort – physical or stigma-related – with colorectal cancer screening has prevented them from staying up to date, indicating an opportunity for more screening options. In fact, among those who have ever skipped a colorectal cancer screening after a referral, 38% of those surveyed cite having done so due to discomfort with the idea of invasive procedure. Additionally, 74% of adults surveyed agreed that they would get screened for colorectal cancer if it were a non-invasive procedure, while 72% of respondents said that they would be more willing to get screened if they could use an at-home test instead of going to a doctor's office.

Despite these results, only 4 in 10 respondents (40%) were familiar with at-home DNA based stool tests. Each type of screening test has its strengths and shortcomings when comparing effectiveness, accessibility, and ease of use, but the best screening is the one that gets done. When considering health disparities in populations where paid sick time is not available, or patients are primary caregivers to children or elders and unable to take time away for the colonoscopy prep and procedure, providing resources that clearly delineate various screening options is critical.

At-home screening options such as Cologuard® can provide a non-invasive, effective, and convenient screening option for adults 45 and older, who are at average risk for colorectal cancer. In the effort to improve health literacy, it is important to discuss with patients what makes them eligible and ineligible for Cologuard. Small practices and solo PCPs with limited resources have found it helpful that Cologuard also has a built-in, 24/7 patient support team to follow-up on and encourage test completion in over 240 languages which can help make the process more convenient and contact free, particularly as screening rates have dropped amidst COVID-19.

As we navigate this complex healthcare landscape, action is needed to ensure health systems, community centers, and PCPs partner to prioritize health literacy and engage patients with the meaningful resources and necessary support to make informed decisions. To do so, we must reframe our approach as we face increasingly challenging areas of care. With colorectal cancer incidence rates on the rise in younger populations,

 screenings remain the most important tool to detect preventable fatalities. Meeting patients where they are with solutions that fit their specific needs is critical to catching up on missed screenings and encouraging future screenings.

Indication and Important Risk Information


Cologuard is intended to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use if you have had adenomas, have inflammatory bowel disease and certain hereditary syndromes, or a personal or family history of colorectal cancer. Cologuard is not a replacement for colonoscopy in high-risk patients. Cologuard performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. False positives and false negative results can occur. In a clinical study, 13% of people without cancer received a positive result (false positive) and 8% of people with cancer received a negative result (false negative). Rx only.

** Data is according to a national survey of 5,021 U.S. adults aged 45-75 conducted by The Harris Poll on behalf of Exact Sciences between March 25 and April 15, 2021.

*** Average risk in this survey was defined as those who do not have a personal history of: colorectal cancer, Inflammatory Bowel Disease, adenomas, a positive result from a colorectal cancer screening within the last 6 months, a family history of: colorectal cancer, Familial adenomatous polyposis (FAP, Hereditary non-polyposis colorectal cancer syndrome (HNPCCC or Lynch Syndrome), Peutz-Jeghers Syndrome, MYH-Associated Polyposis (MAP), Gardner’s Syndrome, Turcot’s (or Crail’s) syndrome, Cowden’s syndrome, Juvenile Polyposis, Cronkhite-Canada syndrome, or Neurofibromatosis, and are not experiencing rectal bleeding.

Chen RC, Haynes K, Du S, Barron J, Katz AJ. Association of Cancer Screening Deficit in the United States With the COVID-19 Pandemic. JAMA Oncol. 2021;7(6):878–884. doi:10.1001/jamaoncol.2021.0884

Wolf AMD, Fontham ETH, Church TR, et al. Colorectal cancer screening for average‐risk adults: 2018 guidelines update from the American Cancer Society. CA Cancer J Clin. 2018;68(4):250‐281. doi:10.3322/caac.21457.

Siegel RL, Miller KD, Fuchs HE, Jemal A. Cancer statistics, 2021. CA Cancer J Clin. 2021;71:7-33. doi:10.3322/caac/21654.

National Cancer Institute. Cancer stat facts: colorectal cancer. Accessed November 10, 2021. https://seer.cancer.gov/statfacts/html/colorect.html.

Davidson KW, Barry MJ, Mangione CM, et al. Screening for colorectal cancer - US Preventive Services Task Force recommendation statement. JAMA. 2021;325(19):1965-1977. doi:10.1001/jama.2021.6238

Redesigning our Approach to Combating the Rising Incidence of CRC in Young Adults

For decades, limited understanding and a lack of available treatment options made neuromyelitis optica spectrum disorder (NMOSD) an especially difficult diagnosis for neurologists to make and deliver. A rare and lifelong autoimmune disease that impacts the optic nerve(s) and spinal cord,

 NMOSD affects up to 15,000 people in the U.S.,

 and can cause lasting damage to the nervous system, including blindness, muscle weakness and even paralysis.

 Due to its overlapping characteristics with multiple sclerosis (MS), NMOSD is often misdiagnosed and can worsen if treated improperly with MS medication or if left untreated.

The importance of proper diagnosis and treatment for NMOSD cannot be underestimated. Without treatment, in just five years from diagnosis, up to half of people with NMOSD will likely require a wheelchair, and more than 60 percent may become functionally blind.

 Until recently, there were no medicines specifically evaluated and approved to treat NMOSD, leaving healthcare professionals with limited options for treatment. Given the progressive nature of this debilitating condition, the medical community was in need of targeted treatments.

Fortunately, the understanding of NMOSD has improved substantially, thanks to continued investment in neuroscience research and development, and collaboration between biotechnology companies, healthcare providers and patient advocacy organizations. These advancements have paved the way for Genentech, a member of the Roche Group, to better understand NMOSD and advance treatment.

Meaningful Advancements in Understanding the Science of NMOSD

Although it’s not clear what causes NMOSD, there is strong scientific evidence that interleukin-6 (IL-6), a protein made by immune cells, may play a key role in the inflammation associated with the condition. IL-6 helps regulate the body’s immune response to inflammation, including the production of aquaporin-4 (AQP4) antibodies, which target and damage cells found in the optic nerves, brain and spinal cord. In response, these cells produce even more IL-6. In fact, IL-6 has been found in excess in the blood and brain of people diagnosed with NMOSD. Increased IL-6 can lead to more inflammation, which can lead to relapses, one of the clinical hallmarks of the condition. These unpredictable, severe relapses may cause cumulative, irreversible, neurological damage and disability for those living with NMOSD.

Ashish Pradhan, M.D., Executive Director of Neuroimmunology at Genentech, explained that this improved understanding of the connection between IL-6 and NMOSD inspired researchers at Chugai, another member of the Roche Group, to explore what happened when IL-6 receptor activity was blocked. “They hypothesized that by blocking IL-6 signaling pathways, it could reduce the inflammation commonly seen in people with NMOSD. A novel recycling antibody technology was used so that after binding to the IL-6 receptor, the treatment being designed could be released back into circulation, allowing it to bind to other IL-6 receptors. This continued investment in research and development paved the way for our team at Genentech to better understand the condition and help deliver a new, innovative treatment.”

It has been a little over one year since EnspryngTM (satralizumab-mwge) was approved by the U.S. Food and Drug Administration in August 2020 as the first and only subcutaneous treatment option for adults with AQP4 antibody-positive NMOSD, which is detectable in approximately 80 percent of those living with the NMOSD.

 It is also the only approved NMOSD therapy designed to target and inhibit the IL-6 receptor.

Enspryng was studied in one of the largest pivotal clinical trial programs undertaken for this rare neurological disease. The two studies -- SAkuraSky and SAkuraStar -- found that Enspryng significantly reduced the risk of relapse in AQP4 positive patients at 96 weeks compared to treatment with a placebo by 74% and 78% respectively [HR = 0.26, 0.22; CI = (0.11, 0.63), (0.06, 0.82); p = 0.0014, 0.0143].

 Enspryng is contraindicated in patients with a known hypersensitivity to satralizumab or any of the inactive ingredients, an active hepatitis B infection, or active or untreated latent tuberculosis. The warnings and precautions for Enspryng are infections, including hepatitis B virus reactivation and tuberculosis, elevated liver enzymes, decreased neutrophil counts, and hypersensitivity reactions. Appropriate precautions should be taken by the healthcare provider when considering vaccinations.

“For decades, we relied on unproven or off-label therapies in a sort of ‘blunt force’ approach to treat NMOSD,” said Geoffrey Eubank, M.D., System Medical Chief, General Neurology at OhioHealth Neurological Physicians and one of the Enspryng clinical trial investigators. “Exploring the role of IL-6 in NMOSD and discovering a treatment to block those chemical messengers and reduce attacks has filled a significant gap in how NMOSD is treated and provided much-needed hope for patients and those of us who treat them.”

In addition to its IL-6 inhibition, Enspryng was designed using novel recycling antibody technology that allows for longer duration of antibody circulation and subcutaneous dosing every four weeks.

 Instead of needing to go to a healthcare facility for infusion therapies, Enspryng can be self-administered by a person living with NMOSD or a caregiver at home, after an initial loading dose and following training from a healthcare provider, if deemed appropriate by a physician.

Continuing to Advance the Science of NMOSD

While Genentech has made a tremendous advancement in NMOSD, its work isn’t over yet. The discovery of the role of IL-6 role in NMOSD has redefined the company’s understanding of the condition and it remains committed to advancing NMOSD clinical research, including ongoing safety and efficacy studies with Enspryng. Genentech is also conducting research to gain a deeper scientific understanding of NMOSD in diverse patient populations that could help provide better care for those living with the condition.

Dr. Pradhan added, “We are proud of the progress we have made in addressing many key challenges that have long eluded the neuroscience community, including NMOSD and other neurological conditions that limit human potential. To sustain this forward trajectory, we must continue to follow the science, push the boundaries of scientific understanding, monitor the safety and efficacy of our treatments and keep patients where they belong -- at the heart of our research. We can then look to the future of neuroscience with more confidence, knowing that we will continue to focus on areas with the highest unmet needs and provide patients with the support and care needed to live their best lives beyond their disease.”

For more information on Enspryng, please visit 

Important Safety Information & Indication

ENSPRYNG is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

ENSPRYNG is contraindicated in patients with a known hypersensitivity to satralizumab or any of the inactive ingredients, an active hepatitis B infection, or active or untreated latent tuberculosis.

An increased risk of infections, including serious and potentially fatal infections, has been observed in patients treated with IL-6 receptor antagonists, including ENSPRYNG. The most common infections reported in a randomized clinical trial of patients treated with ENSPRYNG who were not on other chronic immunosuppressant therapies and that occurred more often than in patients receiving placebo, were nasopharyngitis (12%) and cellulitis (10%). The most common infections in patients who were on an additional concurrent immunosuppressant, and that occurred more often than in patients receiving placebo, were nasopharyngitis (31%), upper respiratory infection (19%), and pharyngitis (12%). Delay ENSPRYNG administration in patients with an active infection, including localized infections, until the infection is resolved.

Risk of HBV reactivation has been observed with other immunosuppressant therapies. Patients with chronic HBV infection were excluded from clinical trials. Perform HBV screening in all patients before initiation of treatment with ENSPRYNG. Do not administer ENSPRYNG to patients with active hepatitis. For patients who are chronic carriers of HBV [HBsAg+] or are negative for HBsAg and positive for HB core antibody [HBcAb+], consult liver disease experts before starting and during treatment with ENSPRYNG.

Tuberculosis has occurred in patients treated with other IL-6 receptor antagonists. Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating ENSPRYNG. Consider anti-tuberculosis therapy prior to initiation of ENSPRYNG in patients with a history of latent or active tuberculosis in whom an adequate course of treatment cannot be confirmed, and for patients with a negative test for latent tuberculosis but having risk factors for tuberculosis infection. Consult infectious disease experts regarding whether initiating anti-tuberculosis therapy is appropriate before starting treatment. Patients should be monitored for the development of signs and symptoms of tuberculosis with ENSPRYNG, even if initial tuberculosis testing is negative.

Live or live-attenuated vaccines should not be given concurrently with ENSPRYNG because clinical safety has not been established. Administer all immunizations according to immunization guidelines at least 4 weeks prior to initiation of ENSPRYNG for live or live-attenuated vaccines and, whenever possible, at least 2 weeks prior to initiation of ENSPRYNG for non-live vaccines.



Mild and moderate elevations of liver enzymes have been observed in patients treated with ENSPRYNG at a higher incidence than in patients receiving placebo. ALT and AST levels should be monitored every 4 weeks for the first 3 months of treatment, followed by every 3 months for one year, and thereafter, as clinically indicated.

Decreases in neutrophil counts were observed in patients treated with ENSPRYNG at a higher incidence than placebo. Neutrophil counts should be monitored 4 to 8 weeks after initiation of therapy, and thereafter at regular clinically determined intervals.

Hypersensitivity reactions, including rash, urticaria, and fatal anaphylaxis, have occurred with other IL-6 receptor antagonists.

There are no adequate data on the developmental risk associated with the use of ENSPRYNG in pregnant women. In an animal reproduction study, no adverse effects on maternal animals or fetal development were observed in pregnant monkeys and their offspring, with administration of ENSPRYNG at doses up to 50 mg/kg/week. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2%-4% and 15%-20%, respectively. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

No information is available on the presence of ENSPRYNG in human milk, the effects of ENSPRYNG on the breastfed infant, or the effects of ENSPRYNG on milk production. ENSPRYNG was excreted in the milk of lactating monkeys administered ENSPRYNG throughout pregnancy. Human IgG is excreted in human milk and the potential for absorption in the infant is unknown. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ENSPRYNG and any potential adverse effects on the breastfed infant from ENSPRYNG or from the underlying maternal condition.

Safety and effectiveness in pediatric patients have not been established.

Clinical studies of ENSPRYNG did not include sufficient numbers of patients aged 65 years and older to determine whether they respond differently from younger patients. However, population pharmacokinetic analyses in patients with NMOSD did not show that age affected the pharmacokinetics of ENSPRYNG. In general, caution should be used when dosing the elderly, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant diseases or other drug therapy.

The most common adverse reactions (≥15% in either trial) were nasopharyngitis (31%), headache (27%), upper respiratory tract infection (19%), rash (17%), arthralgia (17%), extremity pain (15%), gastritis (15%), fatigue (15%), and nausea (15%).

For additional safety information, please see the full 

REFERENCES
1. Wingerchuk DM, Lennon VA, Lucchinetti CF, et al. Lancet Neurol 2007;6:805-815.
2. Guthy Jackson Foundation. NMO FAQs. https://guthyjacksonfoundation.org/nmo-faqs.
3. Kessler RA, et al. Neurol Neuroimmunol Neuroinflamm 2016;3:e269.
4. Williamson_Poster_2018_ ICPE_Epidemiologic Temporal Trends of NMOSD
5. Chihara N, Aranami T, Sato W, Miyazaki Y, Miyake S, Okamoto T, et al. Interleukin 6 signaling promotes anti‐aquaporin 4 autoantibody production from plasmablasts in neuromyelitis optica. Proc Natl Acad Sci USA. 2011;108:3701–6.
6. Bradshaw M, Kimbrough D. Neuromyelitis Optica Spectrum Disorders. Practical Neurology. February 2019;76-84.
7. Traboulsee A et al. Safety and efficacy of satralizumab monotherapy in neuromyelitis optica spectrum disorder: a randomised, double-blind, multicentre, palcebo-controlled phase 3 trial. Lancet Neurology. 2020;19:402-412.
8. Yamamura T et al. Trial of Satralizumab in Neuromyelitis Optica Spectrum Disorder. N Eng J Med. 2019;381:2114-2124.
9. Our Proprietary Technologies: About Chugai.” Chugai Pharmaceutical Co, Ltd. www.chugai-pharm.co.jp/english/profile/rd/technologies.html.
10. Igawa T. Nat Biotechnol. 2010; 28:1203 -07.

Breakthroughs in NMOSD: Better Understanding the Science and Treatment

Atopic dermatitis (AD) is one of the most common skin conditions managed by primary care clinicians.

 For doctors who care for infants and children, early education in skin care management is vital for the long-term success of patients who struggle with AD and sensitive skin. Eczema Awareness Month is a prime opportunity to equip our patient care teams with evidence-based skin care advice and tips for AD prevention.

Nearly all pediatricians can identify the red, itching rash of AD. Despite this intimate clinical familiarity, the exact cause of AD is unknown. Research has clearly shown a strong association with family history and over 45 genes are implicated in AD.

 However, the heritability of the disease is largely unexplained. Research has also suggested structural deficits and a disordered immune response as causes of AD, but these have never been proven.

 Regardless of the primary cause, patients with AD share the consequences of a complex interaction between internal and external factors that result in a poorly functioning outer skin layer.

The abnormality in patients with AD is a dysfunctional epidermis. Abnormal skin structure and function result in increased water loss, chronic inflammation, increased skin pH, and microbial colonization. In addition, cytokines and other immune modulators interact with neural fibers resulting in chronic itch.

 These changes result in patients with AD experiencing cycles of itching rash with intermittent flares requiring medical care.

Most patients with AD begin to have flares by the age of five years.

 Trouble spots in infancy are the cheeks, scalp, and extensor surfaces of the arms and legs. As kids age, the commonly affected areas are the flexor surfaces of the elbows and knees, ankles, wrists, and neck. Acute flares involve the development of intensely itchy, dry, red papules, and vesicles. Chronic lesions present as thickened skin with altered pigmentation.

 Affected areas change over time due to the natural changes in skin architecture as a person ages.

Infant skin is fully functional at birth but carries some significant differences from mature skin. The transport and distribution of water are notably different in infant versus adult skin, with newborn skin being initially drier and of neutral pH. Over 2-3 months, newborn skin becomes slightly acidic as the acid mantle forms.

 Water-binding factors and free fatty acids increase on the surface of the skin, improving the function of the outermost skin layer as the pH drops. The acid mantle is necessary for proper epidermal function and is influenced by products used on the skin.

Beginning in the labor and delivery suite, care decisions can promote the development of healthy infant skin. At delivery, term infants are covered in a thick substance called vernix. Vernix is thought to be critical to the development of the outmost skin layer while in utero, as well as protective against the unavoidable insults to newborn skin. Although vernix is primarily water, it also contains enzymes and proteins with antimicrobial and water-binding properties. Due to these protective factors, it's recommended that vernix stays on infant skin for at least six hours after birth. Gentle dry wiping after birth allows some vernix to remain in place,

 and delaying the first infant bath until 24 hours of life is preferred.

Use the right products and timing for infant bathing. Routine bathing can begin after the first week of life without disrupting the skin barrier.

 Infant cleansing products should contain a low amount of surfactants and have a neutral to slightly acidic pH. Most research suggests using a liquid-based, mild synthetic detergent, followed by a thorough rinse.

 Immersion bathing is preferred, but typically avoided until the umbilical stump is gone. However, there is little to no evidence of increased umbilical infection with immersion bathing.

 A quick, warm water bath (less than 10 minutes), 2-3 times per week is typically enough for most babies.

After the bath, maintaining and replacing skin moisture is key to routine care, especially for infants at risk of developing AD. Beginning daily, full-body emollient treatments at three weeks of life is one of the few evidence-based AD prevention strategies.

 Emollients should be fragrance-free and contain few preservatives. Additives like ceramides or colloidal oatmeal may be increasingly helpful to hydrate infant skin and are typically well-tolerated.

 After the bath is the best time to apply emollients, trapping water in the skin while preventing water loss.

Although AD classically spares the warm and humid diaper environment, the presence of debris on sensitive infant skin can lead to breakdown and diaper rash. In addition, friction caused by excessive wiping, or the diaper itself, can damage the skin surface and make the area prone to inflammation and infection. For children with sensitive skin, commercially available disposable diapers are preferred to cloth diapers. Disposable diapers do a superior job removing wetness and maintaining proper skin pH during wear.

In addition to disposable diapers, parents should be encouraged to use effective diaper wipes and creams. Diaper wipes with minimal ingredients, like WaterWipes, are preferred to diaper wipes containing surfactants or other unnecessary ingredients. In a large clinical trial, babies cleansed with wipes with the fewest ingredients had less incidence of diaper rash and shorter duration of the rash.

 In turn, WaterWipes was awarded the National Eczema Association seal of acceptance. Finally, applying a barrier ointment or zinc-based diaper cream will protect infant skin from the chemicals in the urine and feces that begin skin breakdown, preventing skin damage that makes babies prone to diaper rash.

Above all else, one of the most powerful care tools for patients with AD is education. At every well visit, reinforcing the message to parents about routine skin care will help families maintain skin integrity and limit AD flares. Providing a written plan for families augments the office conversation and solidifies verbal instruction. Free AD plans to use with patients and their families are available online from reputable organizations, such as the American Academy of Allergy, Asthma & Immunology

 and the American Academy of Dermatology.

Education is not just for the exam room. Our patients need support from all members of our care teams, including the nurses who help with in-office or phone advice. Keeping the medical team up-to-date with current recommendations helps all patients receive a consistent and accurate skin care message. A free, one-hour course on infant skin care is available online from the 

. The course features evidence-based infant skin care recommendations and information on the most common infant rashes seen in the primary pediatric office. This CE-accredited course is ideal for clinical nursing teams, care providers, and interested parents.

Healthy skin care is important for all infants, especially those at risk for AD. Providing evidence-based education to families and caregivers about care for this chronic condition, including best practices and products for bathing and diapering. Additionally, every office visit is an opportunity to remind families about the importance of limiting irritants and replacing skin moisture to prevent AD flares. The efforts of every member of the health care team will ensure each patient has the tools needed for a lifetime of healthy skin.

Dr. Natasha Burgert is a board-certified pediatrician who practices at Pediatric Associates in Overland Park, KS. She is also a spokesperson for the American Academy of Pediatrics and routine contributor to Forbes Health. She also serves as a medical advisor for WaterWipes.

1.Fishbein AB, Silverberg JI, Wilson EJ, Ong PY. Update on Atopic Dermatitis: Diagnosis, Severity Assessment, and Treatment Selection. J Allergy Clin Immunol Pract. 2020;8:91-101.

2.Waldman AR, Ahluwalia J, Udkoff J, Borok JF, Eichenfield LF. Atopic Dermatitis. Pediatr Rev. 2018;39:180-193.

3.Oetjen LK, Mack MR, Feng J et al. Sensory Neurons Co-opt Classical Immune Signaling Pathways to Mediate Chronic Itch. Cell. 2017;171:217-228.e13.

4.Gerner T, Haugaard JH, Vestergaard C et al. Disease severity and trigger factors in Danish children with atopic dermatitis: a nationwide study. J Eur Acad Dermatol Venereol. 2021;35:948-957.

5.Blume-Peytavi U, Hauser M, Stamatas GN, Pathirana D, Garcia Bartels N. Skin care practices for newborns and infants: review of the clinical evidence for best practices. Pediatr Dermatol. 2012;29:1-14.

6.Visscher MO, Adam R, Brink S, Odio M. Newborn infant skin: physiology, development, and care. Clin Dermatol. 2015;33:271-280.

7.Johnson E, Hunt R. Infant skin care: updates and recommendations. Curr Opin Pediatr. 2019;31:476-481.

8.Blume-Peytavi U, Cork MJ, Faergemann J, Szczapa J, Vanaclocha F, Gelmetti C. Bathing and cleansing in newborns from day 1 to first year of life: recommendations from a European round table meeting. J Eur Acad Dermatol Venereol. 2009;23:751-759.

9.Silverberg NB. Atopic dermatitis prevention and treatment. Cutis. 2017;100:173;177;192.

10.Price AD, Lythgoe J, Ackers-Johnson J, Cook PA, Clarke-Cornwell AM, MacVane Phipps F. The BaSICS (Baby Skin Integrity Comparison Survey) study: A prospective experimental study using maternal observations to report the effect of baby wipes on the incidence of irritant diaper dermatitis in infants, from birth to eight weeks of age. Pediatr Neonatol. 2021;62:138-145.

11. Aaaai.org. 2021. [online] Available at: <https://www.aaaai.org/Aaaai/media/MediaLibrary/PDF%20Documents/Libraries/AtopicDerm-English.pdf> [Accessed 4 October 2021].

12.aad.org. 2021. [online] Available at: <https://assets.ctfassets.net/1ny4yoiyrqia/ZPT1WHhZHcxlpbDgq3hZX/3973a527e55d064306e40509d5004bb7/eczema-action-plan.pdf> [Accessed 4 October 2021].

Atopic Dermatitis Prevention: Evidence-Based Infant Skin Care for the Primary Care Office

Join Linda Stein Gold, MD and Larry Eichenfield, MD for an informative presentation about recent developments in atopic dermatitis (AD). They will review the evolving understanding of AD, diagnosis, management, the patient journey, and new novel treatment option.

To learn more, watch the live broadcast on Tuesday, October 19, 2021, 6:00 pm EDT and 8:30 pm EDT. To register for the event, visit  

https://studio.mjhassoc.com/reach/incyte-broadcast

The Burden of Atopic Dermatitis and a Novel Topical Pathway

The complexity and heterogeneity of asthma reflects its wide range of potential triggers, clinical manifestations, and underlying drivers. Various disease pathways – spanning both innate and adaptive immunity – can serve as tributaries to the chronic inflammatory state at the core of the disease.

In many individuals with severe, uncontrolled asthma, pathogenic processes can overlap dynamically in ways that can change over time.

 This can makes it difficult to base clinical management decisions the apparent predominance of a specific inflammatory pathway and its constituent mediators.

 As a result, the estimated 1 million people in the U.S. contending with severe, uncontrolled asthma face ongoing challenges that can evolve into exacerbations, hospitalizations and consequent use of health care resources.

In recent years, new insights into the role of the airway epithelium in asthma have provided a framework for understanding how diverse and changeable inflammatory signals can converge to promote severe clinical disease. The significance of airway epithelium in asthma derives not only from the well-known structural damage it undergoes but from the proinflammatory signals it initiates.

As the first point of contact between potential asthma triggers and the lungs, the epithelium sits at the headwaters of multiple distinct inflammatory cascades. Whether the immediate trigger is a pathogen, allergen, or irritant, the epithelium can respond by releasing proinflammatory cytokines including IL-25, IL-33, and thymic stromal lymphopoietin (TSLP).

 First identified as a growth factor in the early 1990s, TSLP has more recently attracted considerable scientific interest because of its capacity to drive multiple downstream innate and adaptive immune responses.

TSLP Action Across Major Inflammatory Categories

Despite its vast phenotypic diversity, asthma can be categorized at a high level based on major patterns of inflammation. About two-thirds of individuals with severe asthma exhibit type 2 (T2) inflammatory patterns.

 T2 inflammation includes classic allergy, where antigen presentation to type 2 helper T (Th2) cells promotes the production of allergen-specific IgE antibodies, mast cell degranulation, and release of proinflammatory mediators.

Sponsored

Latest News