Panelist introductions for an editorial program titled: Embracing New Therapies in Schizophrenia Management

Embracing New Therapies in Schizophrenia Management: Introductions

On September 26, 2024, the US Food and Drug Administration (FDA) approved 

 (Cobenfy), backed by evidence in phase 3 EMERGENT trials. The approval marks the start of a new era in the management of 

, as the agent, formerly known as KarXT, marks the first new pharmacological approach to treating schizophrenia since the 1970s.

Xanomeline and tropism chloride capsules act as a dual M1/M4 muscarinic acetylcholine receptor agonist in the central nervous system. Unlike previous schizophrenia treatments, this drug does not directly regulate the dopamine receptor—only indirectly modulates. 

To commemorate and offer perspective on what this approval means for the healthcare community, 

 hosted a Special Report where leading subject matter experts discussed the approval of xanomeline and tropism chloride capsule for schizophrenia. In this opening segment, moderator Sam Clark, MD, PhD, introduces panelists Rishi Kakar, MD, lead investigator of the EMERGENT trials, and Andrew J. Cutler, MD. The remainder of this panel discussion, which can be found below, focused not only on the approval of xanomeline and tropism chloride capsules but also on the latest updates and the most pressing unmet needs in schizophrenia management.

Mechanism of Action of Xanomeline and Trospium Chloride Capsules

Applying EMERGENT Trial Results to Real-World Clinical Practice for Schizophrenia

Balancing the Benefits and Risks of Xanomeline and Trospium Chloride Capsules

Unmet Needs, Future Research in Schizophrenia and Muscarinic Agonists

(Moderator) is a former practicing psychiatrist and is the founder and chief executive officer of Terran Biosciences.

is a clinical associate professor in the Department of Psychiatry and Behavioral Sciences at SUNY Upstate Medical University and the chief medical officer of the Neuroscience Education Institute.

is a psychiatrist based out of southern Florida as well as the medical director and chief scientific officer of Segal Trials. Kakar served as the principal investigator on the pivotal EMERGENT trials.

Relevant disclosures for Cutler include Karuna, Bristol Myers Squibb, and 

. Relevant disclosures for Kakar include Karuna, Bristol Myers Squibb, and others. Relevant disclosures for Clark include Terran Biosciences.

The FDA's approval of xanomeline and tropism chloride capsules (Cobenfy) on September 26, 2024, introduces a novel pharmacological approach to schizophrenia treatment, the first since the 1970s. This dual M1/M4 muscarinic acetylcholine receptor agonist indirectly modulates dopamine receptors, differing from traditional treatments. A Special Report by HCPLive Psychiatry featured experts discussing the drug's approval, its mechanism, and its application in clinical practice. The panel also addressed unmet needs and future research directions in schizophrenia management, highlighting the significance of this advancement.

In part 1 of 5, panel moderator gives an overview of the Special Report focusing on the approval of Xanomeline and tropism chloride capsules for schizophrenia.

Andrew Cutler, MD

Video content above is prompted by the following:

Overview of Spesolimab for Management of GPP

Spesolimab, an IL-36R inhibitor, is now FDA-approved to treat GPP. Can you give a brief overview of spesolimab and the efficacy and safety evidence we have seen to date.
a.	EFFISAYIL-1—IV formulation (Bachelez, 2021)
b.	EFFISAYIL-2—SQ formulation (Morita, 2023)


Panelists provide an overview of spesolimab, an IL-36R inhibitor now FDA-approved for treating GPP, discussing the efficacy and safety data from the EFFISAYIL-1 trial (intravenous [IV] formulation) and the EFFISAYIL-2 trial (subcutaneous
[SQ] formulation).


Boni Elewski, MD, FAAD 

Nearly half of all adults in the United States are living with hypertension, or blood pressure above 130/80 mmHg. Despite its prevalence, managing high blood pressure 

remains a major challenge in healthcare settings

Hypertension management can be challenging for a number of reasons, explains Benjamin Gallagher, MD, a hypertension specialist and assistant professor of medicine at Yale School of Medicine (YSM). Aside from difficulties patients may have sticking to their recommended medication regimens, clinicians 

 regarding complicated hypertension cases.

For these reasons, Gallagher and his colleagues have been working over the past two years to address this issue by developing a 

 for internal medicine residents at YSM. The curriculum covers advanced topics in hypertension management, including hypertensive urgency, a condition marked by blood pressure over 180/110 mmHg but without damage to vital organs.

Recently, Gallagher, along with Donna Windish, MD, MPH, professor of medicine (general medicine) at YSM, and Natasha Cigarroa, MD, hospital resident at Yale New Haven Hospital, tested how the curriculum impacted residents’ treatment of hypertensive urgency in the outpatient setting. The 

Modifying hypertensive urgency management

The study involved 104 residents who see patients at the New Haven Primary Care Consortium. While all residents received background training on hypertension, only some of them received the hypertensive urgency curriculum, which involved a half-day workshop with lectures and educational materials. Gallagher and his colleagues then compared residents’ notes from appointments with patients diagnosed with hypertensive urgency and measured the frequency of appropriate clinical actions taken by residents from each group. The research team found that the curriculum had a positive impact on residents’ performance: those who received the new curriculum were more likely to increase their patients’ blood pressure medications and/or counsel their patients on medication adherence.

“We were able to show a small but meaningful change in clinicians’ behavior as a result of an educational intervention,” Gallagher says. “At least for a brief period of time after residents received this curriculum on hypertensive urgency, there was an uptick in appropriate management of that condition.”

Given the high incidence of hypertension and how difficult it is to manage, the curriculum could be a helpful tool not only for residents, but also for highly experienced clinicians, including those who might already feel confident in their hypertension management skills, Gallagher says. In fact, a version of the curriculum is available online through the 

Society of General Internal Medicine’s learning portal, GIMlearn

There were some challenges associated with introducing the specialized curriculum, Gallagher notes. “How do you convince people that they need to learn more about something that they think they already know how to do well—and do that without antagonizing or belittling them and making them feel like they are inexperienced?” Gallagher adds. “A lot of the aspects of the curriculum were about protocolized care, using easy-to-follow protocols and couching it in a way of trying to make things easier for clinicians.”

Hypertension affects nearly half of U.S. adults, posing significant management challenges. To address this, a specialized curriculum for internal medicine residents at Yale School of Medicine was developed, focusing on advanced hypertension management, including hypertensive urgency. A study involving 104 residents demonstrated that those who received the curriculum showed improved management of hypertensive urgency, such as increased medication adjustments and patient counseling. The curriculum, available online, aims to enhance clinicians' skills without undermining their confidence, emphasizing protocolized care to simplify clinical decision-making.

New research tested how specialized hypertennsion medical curriculum impacted residents’ treatment of hypertensive urgency in the outpatient setting.

Hypertensive Urgency: Teaching Improved Care for an Acute Form of High Blood Pressure

Eva Cornman

Gerald Appel, MD, is a professor of clinical medicine, director of clinical nephrology, and director of the Glomerular Disease Center at Columbia University.

 has been a disease without a targeted treatment, leaving patients and clinicians to deal with a devastating disease course with no clear standard of care.

Caused by dysregulation of the alternative complement pathway, C3G often leads to progressive kidney failure, with many patients ultimately requiring transplantation—only to see the disease recur.

The US Food and Drug Administration approval of Novartis’ oral iptacopan (Fabhalta) for the treatment of adults with C3 glomerulopathy (C3G) marks a turning point in C3G care, offering the first FDA-approved drug for patients with the ultra-rare kidney disease.

The decision was based on data from the phase 3 APPEAR-C3G trial, where iptacopan met the primary endpoint of reducing 24-hour urine protein-to-creatinine ratio (UPCR) by 35.1% (95% CI, 13.8% to 51.1%; 

 = .0014), with sustained reductions at 12 months.

Additional analyses revealed use of iptacopan was associated with a higher proportion of patients meeting the composite renal endpoint—defined as a ≥50% UPCR reduction and ≤15% eGFR decline—occurring in 43.5% of iptacopan-treated patients versus 25.0% of those who switched from placebo at 6 months. A favorable effect on eGFR trajectory was also observed relative to historical trends.

Not only does this approval provide C3G patients with a treatment specifically designed to address the root cause of their disease, but it also opens the door for further advancements in complement-targeted therapies. With a new tool in their arsenal, clinicians can now offer patients a chance to slow or even stop disease progression—something that was once out of reach.

For additional insight into the significance of the approval and its impact on C3G care, the editorial team of 

 spoke with Gerald Appel, MD, a professor of clinical medicine, director of clinical nephrology, and director of the Glomerular Disease Center at Columbia University; Andrew Bomback, MD, an assistant professor of medicine at Columbia University Medical Center; and Jared Hassler, MD, a pathologist at the Temple University Hospital.

Expert Perspectives: Iptacopan FDA Approval for C3 Glomerulopathy

Relevant disclosures for Appel include Aurinia, Bristol Myers Squibb, Genentech, Genzyme-Sanofi, Eli Lilly, Merck, Pfizer, Mallinckrodt, Achillion, Alexion, Reata, Travere Therapeutics, GlaxoSmithKline, Vertex Therapeutics, Novartis, Apellis, Chinook, Arrowhead, Calliditas, and Vera. Relevant disclosures for Bomback include Amgen, Novartis, Kezar, Silence Therapeutics, Genentech, Visterra, Catalyst, Q32, and Apellis. Relevant disclosures for Hassler include Travere Therapeutics, Omeros, and Novartis.

Campbell P. FDA Approves Oral Iptacopan (Fabhalta) as First C3 Glomerulopathy Therapy. HCPLive. March 20, 2025. Accessed March 21, 2025. 

https://www.hcplive.com/view/fda-approves-oral-iptacopan-fabhalta-as-first-c3-glomerulopathy-therapy

A trio of nephrology experts discuss the significance of iptacopan’s FDA approval as the first treatment for C3G.

Gerald Appel, MD

Let’s talk about the elephant in the room. We’ve been bouncing around it a little. Let’s talk about vaccine hesitancy and how you guys address that in education standpoints as well as how that can impact vaccination compliance or resistance. What’s your language? What’s your methodology around noticing that? Do you notice it? Do people say it to you? Do you feel it? How do you work down that road to try to convince people to get a vaccine?

Wendy Wright, DNP, ANP-BC, FNP-BC, FAANP, FAAN, FNAP:

 This is my baby. This is my doctoral work, so I love this stuff. A couple of things that I want people to know is that studies are pretty clear these days that even people who are vaccine proponents now feel the need to question science and vaccines. Every provider should expect what used to be a 30-second recommendation to now take 5 minutes because people feel the need to ask the questions about it. Even though they’re going to take it, they feel the need. There’s more vaccine hesitancy out there.

The other thing is that we talked about strong recommendations. We call it presumptive vs participatory. In a study, they followed providers who asked, “How do you feel about getting your shots today?” It shouldn’t surprise you that less than 10% of people took their shots. But when the provider said, “You’re due for 2 shots today. It’s okay to give them together,” 80% of people took those vaccines.

My last point on vaccine hesitancy is that you have to be prepared that people are going to ask about it. Take a deep breath and answer their questions. There are going to be the 4% of the population who are vaccine refusers. I’ve learned from all of my research that you will never move those folks. They believe in something that none of us can even fathom, but they come up with it. If they want to believe something, they’re going to google it and find research to support their beliefs. You’re never going to move them.

It’s the movable middle that we have to focus on. That’s the 20% to 40% of the population who will go based on a trusted source, as JAM [Jacinda Abdul-Mutakabbir, PharmD] mentioned. If they trust you and you make that recommendation, they’ll listen. Focus on the movable middle. The ones who march in and say, “What vaccines do I need? Give them to me,” are a provider’s dream. “Sure, give me everything I need.” With people in the middle, we have to take a breath, meet them where they’re at, be honest with them, give them what they’re asking, and don’t go into too much detail.

 Yes. I’ve also seen people talk about how sometimes it takes more than 1 interaction with a patient to convince them. Maybe they come in and you tell them, “You need this vaccine today,” and they say, “Absolutely not.” But by the end of the appointment, they say, “Tell me more about it and I’ll think about it for next time.” We see this with patients in the clinic with other vaccines as well. “I’m not ready to get 5 vaccines today, but I’ll get 1 next time.” We think, “OK, we’ll take what we can get. Let’s just start off on the right foot.” Sometimes it takes persistence and patience, which we don’t all have at this point in the pandemic. But there are people who can be swayed if we take the time, ask them multiple times, and not give up on them.

 Yes. We use a Z code in our charts for vaccine refusal by patient or parent. That’s a signal to the nurse practitioner who sees them next that there’s been a vaccine that they didn’t get the last time, and to take a look at the note and try to follow up on that conversation.

Wendy Wright, DNP, ANP-BC, FNP-BC, FAANP, FAAN, FNAP, and Madeline King, PharmD, BCIDP, provide insight on vaccine hesitancy among patients and the importance of increasing vaccine uptake to protect against SARS-CoV-2.

Addressing Vaccine Hesitancy and Uptake for SARS-CoV-2

Jacinda Abdul-Mutakabbir, PharmD

We have heard the statistics many times. There are more than 7000 

 and for more than 90%, there are no treatments available. Our organization supports a community affected by one of the 90%—Niemann-Pick Disease including ASMD and NPC, both rare genetic, progressive, and often-fatal lysosomal storage disorders.

There are fewer than 2000 known cases worldwide, many affecting children. Symptoms include cognitive impairment, clumsiness and difficulty walking, enlarged spleen and liver, difficulty swallowing and eating, fatigue, increased fracture risk, and early satiety. There are currently no approved treatment options in the US.

The great news for the Niemann-Pick community is that promising treatments are in the later stages of clinical development with the potential for regulatory approval in the coming year. Achieving this milestone would be a significant victory for patients and their families who face significant challenges living with Niemann-Pick. But this potential good news also highlights an important consideration in planning for the introduction of new treatments for patients who are in desperate need. How do you prepare patients and families to transition from an era of no hope to one where treatment is available?

Much of our work at the National Niemann-Pick Disease Foundation (NNPDF) in recent years has focused on meeting this challenge head on and our approach in many ways serves as a template that other rare disease communities might consider.

From our earliest days, NNPDF has provided support services to patients living with Nieman-Pick Disease and their families. These efforts have focused on building broader awareness of the disease and helping to identify and expand the services and resources that families need.

We have also taken steps to keep families updated about the range of clinical research programs that have been initiated in recent years to try to develop new treatments. Our recent annual conference included a full schedule of presentations and updates on these clinical research programs, and we had more participants in our virtual meeting this year than ever before.

As we continue our focus on disease awareness and patient support, we have also in recent years had to expand our efforts to include both information and education to prepare the Niemann-Pick community to transition to an era when treatment might be available. In addition to helping families learn about and understand the process for developing and approving drugs in the US, we are also now providing resources to help them better understand and manage issues related to their healthcare coverage, reimbursement, patient access and industry-sponsored financial assistance programs.

We are helping them understand that there will be indications and eligibility criteria for different treatments and that NNPDF is working to ensure that access to treatment through healthcare coverage is available to as many patients as possible. We are also developing new resources that outline the important questions and topics that patients and families should discuss with their medical provider to empower them to take a more active role in often complex treatment decisions.

While availability of treatment is good news, many of the issues associated with patient access can cause stress and confusion. An important goal in developing programs related to the introduction of an approved treatment is to help reduce anxiety and outline a clear path forward in accessing care. For many rare disease communities, another daunting but welcome challenge for many families may be the need to reconsider many longstanding plans for the future once approved treatment is available.

In many cases, an approved treatment will mean major changes in how patients think about their own health, plan for their care, and engage with their medical teams. Treatment may present new challenges that require careful planning and monitoring. Patients and caregivers might need to reassess plans related to education, careers, job training, and living independently. Once again, our goal must be to provide the insights and support services that can help reduce any challenges associated with making important life decisions at every stage.

The final focus in supporting patients is often related to helping them manage expectations. In many cases, treatments for rare diseases will have some limitations in areas including efficacy, safety and cost. Advocacy leaders can play a role in helping their communities work with their healthcare teams to assess, understand, and address all the factors related to treatment so that patients and caregivers can make informed decisions.

In some cases, patients and caregivers may need very targeted services to support their psychological and emotional health. Peer-to-peer connections can be especially effective in helping patients handle the emotional impact of the transition to treatment.

Of course, none of these challenges should dampen the new hope that a first approved treatment can bring to a rare disease community. A first treatment is both a milestone and a sign that research is advancing and could lead to more personalized treatment options or combination therapies in the years ahead.

By recognizing the needs of patients and families whenever a new treatment becomes available, advocacy organizations can continue to lead in bringing them the targeted information and support they need to access care and thrive. The key is recognizing that an approved treatment and a new standard of care is not the end of the road in patient advocacy. It is a new direction and the beginning of a new era of hope that requires careful planning, new resources and very customized support.

Joslyn Crowe, MSW, MA, is executive director of the National Niemann-Pick Disease Foundation, Inc., a non-profit, unbiased patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research.

The views expressed in this piece are those of the author, not necessarily those of the publication.

Healthcare experts and clinicians interested in contributing to HCPLive may contact the editorial staff 

Getting a Treatment Is Not the End of the Road in Rare Diseases

Joslyn Crowe

Lauren Blieden, MD, is a professor of ophthalmology at Baylor College of Medicine.

Long-Term Considerations in Angle Closure Glaucoma with Lauren Blieden, MD

 research evolves, long-term patient outcomes remain a key focus, particularly in angle closure cases. With critical gaps in long-term data, clinicians are refining management strategies while awaiting further research to guide best practices.

, Lauren Blieden, MD, a professor of ophthalmology at Baylor College of Medicine, highlighted critical updates in patient management, the role of patient education, and the importance of advocacy in ophthalmology. Blieden presented a talk entitled "Glaucoma/Narrow Angles” at the 

Texas Ophthalmological Association (TOA) Annual Meeting

Long-term follow-up data from the EAGLE trial remains a key area of interest in glaucoma research, particularly regarding the progression of angle closure as patients age. With nearly a decade since its publication, ophthalmologists are eager for updates to guide long-term management strategies. Blieden shared insights from her clinical experience, noting that while stable patients can often be monitored less frequently, uncertainties remain about disease progression in older age groups.

Blieden emphasized the importance of patient education, especially in diverse populations with a higher prevalence of angle closure. Early symptom recognition—such as sudden headaches or vision changes—can prevent delays in care. She also highlighted the need for patient awareness regarding medication-related risks and the importance of seeking specialized ophthalmic care rather than general emergency services in acute cases.

Blieden noted the Texas Ophthalmological Association (TOA), recently renamed the Texas Society of Eye Physicians and Surgeons, continues to serve as a vital platform for both clinical education and ophthalmic advocacy. Smaller meetings facilitate in-depth discussions on emerging research, case studies, and legislative issues, including scope-of-practice regulations and liability concerns.

Blieden LS. Glaucoma/Narrow Angles. Presented at Texas Ophthalmological Association (TOA) Annual Meeting. Houston, Texas. March 21-22, 2025

Blieden discusses long-term patient management in glaucoma, the importance of follow-up care, and the role of advocacy in ophthalmology.

Lauren Blieden, MD

Maliha Ahmed, DO

 I want to give us all an opportunity to give some advice or practice pearls to all of our viewers including other hematologists, maybe focusing a bit on red blood cell health or other areas that you think are important including decreasing long term organ damage. I’ll start with you Elna. What advice or practice pearls do you have for our audience?

 I think it is the shifting paradigm that yes sickle cell is an anemia and it causes pain, but the thing that reduces survival and still has maintained the early mortality in sickle cell disease patients compared to the general population is not necessarily the pain or the pain episodes. It is the progressive organ dysfunction and so we should slowly move away from the deception of sickle cell being a mild disease. We should consider it like diabetes or hypertension and that the organ damage is also a silent killer. If we shift that paradigm the way we approach them and how we approach our practice even towards them, we will shift an emphasis in embracing and offering disease-modifying agents to almost all of our patients and having the discussion of the impact of end organ dysfunction and damage in overall longevity and mortality.

 Thank you so much, Elna. Nirmish, you’re next.

 I would just extend the conversation in the sense that educate and be real, continue to educate. I love the descriptions that came up in our conversation today. As I told Matt, I’m going to use some of those. I’m very graphic about drawing pictures and being in the room and getting them to really understand and see what’s happening inside of them because that gives them a little bit of control of knowing what’s happening to themselves. Otherwise, it’s a chronic disease that they have no control of. What control can you give them? It’s educating them about the disease and being able to tell others about what’s happening to them. I think the pearl for me is to educate them and that includes education about these new therapies, and the new drugs that are available now. I even actually started to talk to them about the new drugs that potentially could come.

When we talk about gene therapy, I still kind of give a teaser about it. We’re not there yet. It’s all about being real with them. We don’t have the data. I’m trying to get them to understand that’s why research studies are occurring; this is what we hope that patients can be involved enough with. The patients, I can’t say this enough, they’re really smart. They’re really savvy about connecting to others and they want the information. They just take it up and soak up this information and I think it’s good for the community. Those are some of the pearls. And I mentioned it before, talk about these new medicines when they don’t need it. Talk about everything but pain until you have to talk about pain. Elna brought this up, that there might be the pediatrician inside of us trying to think about the whole world and not the adult side, but I think that our adult colleagues, and I know Biree you do this as well, I think we’re understanding that the quality of life is a big deal. Thus, I think those are some things to consider as we have our colleagues continue to see patients with these new drugs.

 I’ll leave you with 2 more images or analogies that I use because I think that there’s nothing sexy about having a chronic disease and a lot of it is just persistence of the patients, families, and providers in meeting the treatment goals and treatment plan. As I tell families when they’re going for their annual ophthalmology visit or their annual TCD [Trans Cranial Doppler], there’s nothing sexy about this. It’s like changing the oil in your car. It’s a pain. It’s annoying. It costs a little money. But if you don’t do it, what happens to the car? All the kids know the answer to that. It breaks down. Thus, that’s why [there is] this constant persistent pressure to keep on the plan is really very important.

The other one that I find useful to try and sometimes improve adherence to the treatment plan is the idea that organs are like a rock on the beach and the waves every day hit the beach, and they hit that rock and every day you don’t see anything. It’s just like the damage which is done every day with sickling even though you have no symptoms. There’s some clinical inexorable accrual of organ damage is what my mentor used to call it. There’s a little bit of damage every day that’s imperceptible. When you come back to that beach at the end of high school and you see the rock there, it’s still getting pounded by the wave and then you go away to college and then you have kids and you come back and all of a sudden there’s no rock on the beach. It’s just sand. It’s that daily pounding of that rock by a wave that you don’t notice the rock changes, but it’s becoming sand slowly and that’s what we’re trying to prevent with the daily damage of sickle cell disease on your organs.

Hydroxyurea and other disease-modifying agents are trying to prevent that from the daily point of view so that, as Nirmish pointed out or Elna pointed out, we’re trying to improve the overall health and organs of our patients who we present to our adult colleagues so that they can ultimately hopefully have a prolonged life and that sickle will no longer be an either immediately life-threatening or life-limiting disease, but hopefully have normal lifespan and quality of life. That’s really the goal. It’s hard. It’s hard to do that as a child, as a teen, and as an adult with so many other pressures on us but to encourage them to continue to follow the treatment plan with what we have while also being cognizant of what’s coming and what might be until something better comes along, as I say. Let’s follow this plan now. My promise is to keep them informed of what’s potentially available, both experimental and approved, and then they can choose collaboratively with us to potentially take on some of those new therapies.

Thank you, Matt. I really love the imagery in those analogies. I think we’ll all start to use them. I just wanted to add that I think when I speak to colleagues, clinicians of any specialty, patients, the community, family members who help to support their loves ones living with sickle cell disease; I really tell them that the tide is changing in sickle cell disease and it’s changing pretty quickly. Not only do we have more therapies available now, but our understanding of new potential targets is also growing exponentially and we’re seeing that playing out in the clinical trials that are available and that will be emerging in the near future. I think that sickle cell disease as we know it is changing. It is changing. Our patients may not feel that way today, but I do talk to my pediatric colleagues including some of you on the webinar today and I’m always heart-warmed when I hear the hydroxyurea story, for example. My colleagues say that ever since we’ve been giving it to babies starting at 9 months of age, we have such a healthier kindergarten class when we see those 5-year-old’s go off. That just warms my heart.

I think as an adult practitioner I’ve not yet begun fully to that extent to see the effects of novel agents or disease-modifying therapies or even transplant for that matter because we don’t do enough of it in the adult world. But I think that that tide is really changing. Just like hemophilia, for example, where we’ve come a long way, the expectation in hemophilia management is no leads. When we think about HIV care and we were making people toxic, sick with therapy as we knew it a few decades ago, and now people can be on 1 pill once a day. I think that there’s the potential in the very near future, hopefully in all of our lifetimes, to see that amazing impact on those affected by sickle cell disease. I’d say the future is definitely bright. I want to thank Elna, Nirmish, and Matt. And I want to thank you all for watching this 

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A panel of experts in hematology discuss the future directions for sickle cell disease and share practice pearls for disease management. 

Practice Pearls for the Management of SCD

Nirmish Shah, MD

Colorectal cancer is the third most common cancer and a leading cause of cancer death in the United States. Colonoscopy, recommended for all adults aged 45 or older, is a crucial screening tool where gastroenterologists remove polyps to prevent them from becoming cancerous. Recognizing the importance of detecting these polyps, the FDA authorized the first AI-assisted device for colonoscopies in 2021.

Dr. Gyanprakash Ketwaroo at the West Haven VA emphasizes the value of AI in providing a "second set of eyes" during colonoscopies. This technology, known as computer-aided detection (CADe), has shown promise in increasing adenoma detection rates, according to a meta-analysis led by Dr. Dennis Shung. The analysis, which reviewed 44 studies, found that CADe systems improved adenoma detection compared to standard colonoscopies, although the increase in advanced colorectal neoplasia detection was slight.

Shung's meta-analysis suggests further research is needed to assess AI's impact on interval colorectal cancer rates and recommends that future studies should randomize colonoscopists to understand better where CADe can be most effective. Yale has implemented this AI technology at the West Haven VA campus, providing additional support for endoscopists and trainees during procedures.

The American Gastroenterological Association (AGA) is also preparing new clinical guidelines for using AI in colonoscopies, for which Dr. Shung is the co-first author. These draft guidelines conditionally recommend CADe for polyp detection in adults, considering the technology's safety and efficiency. The AGA is gathering feedback from both providers and patients on AI use in screening, aiming to publish the final guideline in March 2025, with regular updates as the technology evolves.

Overall, AI-assisted colonoscopies represent a significant advancement in colorectal cancer screening, with the potential to enhance detection rates and outcomes for patients. Clinicians are advised to stay informed on evolving guidelines and technological developments to offer the best care possible.

Colorectal cancer ranks as the third most prevalent cancer and a major cause of cancer mortality in the U.S. Colonoscopy is vital for early detection, with AI-assisted devices enhancing polyp detection. The FDA approved the first AI device for colonoscopies in 2021, and studies show improved adenoma detection rates with AI. Further research is needed to evaluate AI's impact on interval cancer rates. The American Gastroenterological Association is developing guidelines for AI use in colonoscopies, with final recommendations expected in March 2025, emphasizing the importance of staying updated on advancements.

Computer-aided detection during colonoscopies with AI may help increase adenoma detection rates.

Authors