Real-World Insights: Navigating Cardiac Myosin inhibitors in Practice 

Anjali Tiku Owens, MD, shares insights into how their institution or clinic implements the Risk Evaluation and Mitigation Strategy (REMS) program for mavacamten, detailing the requirements and responsibilities of the patient in the process. Additionally, the expert draws upon their real-world experience with the cardio myosin inhibitor (CMI) in clinical practice, providing valuable perspectives on the practical application of this novel drug class.  

Anjali Tiku Owens, MD

 We’re going to bring our discussion to a close. Thank you all for this rich and informative discussion. Before we conclude, I’d like to hear final thoughts from each of you. Dr Amann?

 As Andy said, thank you, Mike, for sharing your personal story. Thank you too, Theresa, because you shared what it’s like being a parent of a child with ADHD [attention-deficit/hyperactivity disorder]. We’re appreciative of that as well. We change lives, and I’m ever grateful to all the resources I have to do that. It’s very rewarding to treat this disorder when it’s diagnosed and treated properly. Thank you to everybody for having me join this discussion. I’ve enjoyed it greatly.

 We’ve done a terrific job summarizing some of the issues. I feel so fortunate, like many of you, my colleagues. I’ve been working in this space for over 20 years, and [it’s so rewarding] to see the evolution of the technology and the different options, the understanding of the neuroscience of ADHD. It’s so rewarding to have all these different options.

I always joke that when you’ve seen 1 patient with ADHD, you’ve seen 1 patient with ADHD. We all know that. It’s so variable, the comorbidities and the daily activity. Having these tools and options allow us to do a much better job individualizing, personalizing, and getting better outcomes overall for our patients. I agree with Mike, it’s one of the more rewarding things that we treat in psychiatry. You can really make a difference. One thing that never gets old is helping a child and feeling like you can change the course of someone’s life and put them on a different track. That’s incredible. Mike used the railroad track analogy, which I love. That’s been very rewarding. We have a lot of challenges and a long way to go, but it’s been gratifying to see the progress we’ve made so far.

 For me, as a clinician in both settings, the most positive aspect is seeing self-confidence improve and children who are frequently getting in trouble and getting sent to the principal now getting merit reports saying that they’re the student of the day, and they aren’t getting in trouble anymore. With adults, like my patient who’s an attorney, the joy of watching somebody achieve their goals because they were willing to resume treatment is one of the most gratifying things about being a clinician.

Thank you so much for asking me to be here. It’s an honor to be here with many of my colleagues who are as old as I am, who have also treated folks with ADHD through their life span and have had the pleasure of seeing all the new developments in ADHD emerge and make a difference in people’s lives.

 Twenty years ago, I used to sit in the room with you for different meetings with all the teachers, moderators, and people I looked up to. You guys brought me into the world of validating this stuff and realizing how incredible this stuff was. I’m truly honored to be on the panel with you guys because I’ve always respected you. I might have been a little obnoxious in this [discussion], or a little restless.

 Maybe I interrupted a few times. But my biggest message is that this is a blast, but I hope people watch this and buy into what we’re saying.

 We understand. Thank you. Thank you again to our viewing audience. We hope you found this 

 discussion to be useful and informative. If you enjoyed the content, please subscribe to our e-newsletters to receive upcoming 

 and other great content right in your in-box. Thanks, everyone.

ADHD experts share their final thoughts on the treatment and management of ADHD.

Final Thoughts on Management of ADHD

Birgit H. Amann, MD

Head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris-Est

Bernaudin discussed the Drepagreffe-1 and 2 studies and improvements seen over 10 years of follow-up.

Françoise Bernaudin, MD: A Decade of Follow-up Reveals allo-SCT Superiority Over SOC for Sickle Cell Anemia

Over 10 years of follow-up, allogeneic stem cell transplantation (allo-SCT) demonstrated improvements compared with standard of care (SOC) for patients with sickle cell anemia (SCA) in silent cerebral infarct (SCI) incidence and social quality of life (QoL), working memory, and processing speed.

These findings, from the DREPAGREFFE-2 study (NCT05053932), were presented at the

 66th American Society of Hematology (ASH) Annual Meeting and Exposition

, held December 7-10, 2024, in San Diego, California, by Françoise Bernaudin, MD, the head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité.

“It's important we have demonstrated that SCT does better than chronic transfusion and better than hydroxyurea, for the outcome of stenosis, to prevent SCI, and to give a better quality of life and higher cognitive performance,” Bernaudin told 

The original prospective DREPAGREFFE-1 trial (NCT01340404) showed modest improvements between allo-SCT and SOC arms, many of which have become significant over 10 years of follow-up.

Notably, Bernaudin and colleagues found that on MRI and MRA, of the 31 SOC group patients, 11 had reported SCIs—an increase of 5 incidences from 6 at enrollment—while there were no additional instances in the 28 patients in the allo-SCT group, which had 12 at enrollment. SCI were either no more visible or under 3 mm in 1 patient in the standard of care group and 4 in the allo-SCT group (

 = .010). Processing speed index (PSI) was higher for the allo-SCT group at 96.5 (SD, 21.9) compared with the standard of care group at 83.7 (SD, 14.4), which was significant (

 = .035), as well as social functioning QoL (allo-SCT: 93.8 [SD, 10.7]; standard of care: 86.4 [SD, 12.9]; 

Bernaudin F, Verlhac S, Ducros-Miralles E, et al. Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities. Presented at: ASH 2024 Annual Meeting. December 7-10, 2024; San Diego, CA. Abstract LBA-5

Françoise Bernaudin, MD

Greg Duck, PA-C is the Director of Internal Medicine and Cardiology Physician Assistant at Acclaim Physician Group in Fort Worth, Texas.

The APAC Recap: Cardiomyopathy at CAPP Live 2024 with Greg Duck, PA-C

, host Laura Ross, PA-C, clinical lipid specialist and president of the Academy of Physician Associates in Cardiology (APAC), interviewed Greg Duck, PA-C, co-chair of APAC’s heart failure committee and director of internal medicine and a cardiology PA at Acclaim Physician Group. Duck shared insights from his CAPP 2024 presentation on 

, a condition he frequently encounters in his practice with underserved populations.

Duck emphasized the importance of understanding the patient’s drug use patterns to tailor medical management and ensure effective treatment. He highlighted the need for careful use of beta blockers in patients actively using methamphetamines to avoid adverse events and promote adherence to therapy. Additionally, Duck shared key clinical pearls, such as medications like wellbutrin and mexiletine causing false-positive methamphetamine urine screens, underscoring the importance of avoiding stigma and mislabeling patients.

The conversation also touched on gaps in guidelines specific to methamphetamine-related heart failure, with Duck recommending adherence to established heart failure with reduced ejection fraction (HFrEF) protocols and regular follow-ups to optimize care. He advocated for transparency in monitoring strategies, such as routine urine drug screens, to build trust with this vulnerable population. Duck concluded with a discussion of the Heart Failure Society of America (HFSA) heart failure certification, encouraging advanced practice providers to pursue this credential to enhance patient care and professional expertise.

All episodes of The APAC Recap will be available on HCPLive.com and our 

Laura Ross, PA-C, interviewed Greg Duck, PA-C, on methamphetamine-induced cardiomyopathy, focusing on its management in underserved populations. Duck stressed understanding drug use patterns for effective treatment and highlighted the cautious use of beta blockers in active methamphetamine users. He noted medications like wellbutrin and mexiletine can cause false-positive urine screens, emphasizing the need to avoid stigma. Duck recommended following HFrEF protocols and regular follow-ups, advocating for transparency in monitoring. He encouraged pursuing HFSA heart failure certification to enhance care and expertise.

Greg Duck, PA-C shared insights from his CAPP 2024 presentation on tailoring medical management and effective treatment of methamphetamine-induced cardiomyopathy.

Greg Duck, PA-C

Part 5 of our 5-part panel discussion for CRC Awareness Month focuses on: Appropriate Guidance for CRC Screening Age/Role of Advocacy Organizations

Appropriate Guidance for CRC Screening Age, Role of Advocacy Organizations

This portion of our 5-part peer-to-peer discussion on the importance of colorectal cancer (CRC) screening focuses on appropriate guidance for screening age and the future of CRC advocacy in light of emerging technologies and evolving healthcare policies.

, as the experts discuss current and prospective age-based screening recommendations as well as the importance of both awareness and navigation of screening.

Sapienza introduces the topic and inquires about the experts’ reactions to suggestions for lowering the screening age to younger than 45 years, to which Imperiale says he is “horrified” and describes how this would likely lead to more harm than benefits. Instead, he suggests “doing things that make the most sense first,” like making patients aware of symptoms that warrant testing.

Colangelo then poses a question to Sapienza regarding his outlook on the future of CRC screening, advocacy, and support from nonprofit organizations, to which Sapienza describes the significance of both awareness and navigation efforts from the Colorectal Cancer Alliance, additionally calling attention to the important role of risk stratification.

Updates in Colorectal Cancer Screening Recommendations

Pressing Challenges in Colorectal Cancer Screening

Reaching Patients in Need of Screening for Colorectal Cancer

Navigating Patient Histories, Risk Factor Conversations in CRC Screening

 chief executive officer of the Colorectal Cancer Alliance, the largest colon cancer advocacy group in the country, and member of the American Cancer Society’s National Colorectal Cancer Roundtable steering committee.

, an internist, vice president, and chief quality officer for Premier Medical Associates, a large multispecialty group within Allegheny Health Network as well as a member of the American Cancer Society’s National Colorectal Cancer Roundtable steering committee.

 a gastroenterologist, associate division chief of the Department of Gastroenterology and Hepatology, and medical director of Endoscopic Innovations at Allegheny Health Network.

principal investigator of the BLUE-C trial, research scientist at Regenstrief Institute, and Lawrence Lumeng Professor of Gastroenterology and Hepatology at Indiana University School of Medicine.

Imperiale has no relevant disclosures to report. Colangelo has no disclosures to report. Relevant disclosures for Kochhar include CorEvitas Research, Eli Lilly, Boston Scientific Endoscopy, Olympus Endoscopy, and Pentax Endoscopy. Sapienza has no relevant disclosures to report.

In part 5 of our 5-part discussion, experts discuss their perspective on appropriate guidance for screening age and the future of CRC advocacy.

Gursimran Kochhar, MD

 What I’m hearing from you guys is that it’s important to educate your patients, to provide informed tools for them to make an informed decision based on their specific scenario. For our last question, I’m going to ask all of you: sickle cell disease has been around for over 100 years, and we’re still dealing with organ damage, acute exacerbations, quality-of-life issues, fatigue, difficulty finding a mate, difficulty finding work. What are your concerns around health equity and access to care that are persistent? What would be the 1 thing that if you could fix about sickle cell disease if you had a magic wand or a genie bottle? I’m going to have Dr Desai start, then Dr Shah and Dr Smith.

 I’m going to make the first wish. It’s to get every patient connected to some care—having somebody who will care and talk about these things, to say “I don’t know” sometimes and walk through life with them. We’ve talked about how complicated it is; they can’t do it by themselves. Everybody needs that advocate, they need the person that understands the disease. Even if they don’t understand, they’re willing to take that extra step. I wish everybody had a place that they could be connected to care.

 Excellent. Dr Shah, what’s your 1 wish from the genie bottle or magic wand?

 For wish No. 2, I have a 2A and 2B; they’re connected. The 2A is a big shift in mindset about therapies, from being reactive to prophylactic. We have drugs that are reactive because they’re anemic or because they have pain. But why do they have to be anemic or in pain? Why can’t we move more toward prophylactic? But because therapies are expensive, there’s so much out there, and not all patients are in centers that can give you those therapies, when we get to those therapies being prophylactic, they must be accessible, easy to take, and cost-effective. Not a drug that’s so expensive that a payer isn’t going to want to support that across the country. We have patients who aren’t plugged in to care, don’t have access to health care, and don’t have insurance. Why aren’t they getting the same therapy as another patient in an area that has a comprehensive sickle cell center? So there are 2 arms: better prophylactic medications with access to as many patients as possible.

 Public health. We don’t know how many patients have sickle cell disease in the United States, but Brazil does. Brazil is supposed to be an emerging nation. That’s a travesty. If we knew how many patients had sickle cell disease in every state in the United States, then we could more appropriately advocate for resources to do everything you just heard. We can know just by having somebody snap their finger and make each state share their newborn screening registry with each other; we could do that. Eleven states are already doing it, according to the CDC [Centers for Disease Control and Prevention]. My 1 wish is to get all 50 states to do that so we can talk intelligently about how many patients have the disease. We can then make health care allocation decisions for not just doctors but community health workers, hospitals with drugs, and community-based organizations and resources to help these patients.

 Thank you so much. My 1 wish is that all 3 of your wishes—or 3½ to 4 wishes—come true. If we get there, then each individual with sickle cell disease should be able to live to be 99 years old, get to lose all their teeth, and use a walker. Having a quality of life and a quantity of life that equals that of their peers is the ultimate goal.

I want to thank you, Dr Desai, Dr Shah, and Dr Smith. Thank you for this very informative session, for all your knowledge, and for sharing your experiences. This is a transformational period in sickle cell disease care. I look forward to more drugs being approved, I look forward to a lot more research, and I look forward to the 50 states being connected and for every patient to have access to the right type of care. Thank you very much, everyone.

Concluding their discussion on sickle cell disease management, expert panelists share key unmet needs and hopes for the future.

Sickle Cell Disease: Unmet Needs and Future Directions in Care

Ifeyinwa (Ify) Osunkwo, MD, MPH, Atrium Health

 In terms of avoiding complications, a family that is well educated about the disease, the treatments available, the preventive strategies for complications, including nonmedicinal strategies explicitly, such as mindfulness, good eating habits, exercise within moderation, and good sleep habits, they are going to do far better than the family that does not have these in their lives. Most providers are not well informed about sickle cell disease. Thus, a well-informed parent or adult with the disease who provides information in a nonthreatening fashion can improve their own health care. I would stay abreast of the research as another strategy, because treatment of sickle cell disease in 2020 is vastly different than treatment of sickle cell disease in 2010, and I would wager that curative therapy options in the next decade will be better. We will have improved outcomes with less toxicity. 

Lastly, I would consider all the FDA-approved drugs as potential options for treatment. Particularly for those individuals with hemoglobin SS and S beta-zero thalassemia, with each passing year, we learn more about the benefit of this drug. In my opinion, by far this drug should be in the stable of anyone with hemoglobin SS and S beta-zero thalassemia because the benefits far outweigh any of the toxicities or complications in taking the medication. For the other drug, I think it will take time before the research will demonstrate what the specific benefits are in decreasing the rate of CNS [central nervous system] disease, heart, lung, and kidney disease, as well as potentially priapism and other complications of the disease. But I would say stay tuned because we will learn more about how these additional drugs will prevent complications in organ function in the next decade. 

The first recognition for decreasing opioid dependency is acknowledgment that pain occurs. There should be trust between the provider and the patient, and the patient needs to develop a strategy to manage the pain that is not dependent on opioids but is dependent on first, understanding the triggers, and understanding how to use mindfulness and other strategies to decrease the severity and intensity of the pain. Then, the goal is to learn enough about their disease and enough about their treatment to use a stepwise approach to managing their disease when it comes to acute vaso-occlusive pain. That would include the use of nonsteroidal drug, the use of hydrocodone as a stepwise approach, and then eventually morphine. After the pain subsides, it’s important to stop the medications I just mentioned so the use of pain medications will not result in other complications.

Strategies to avoid complications of sickle cell disease and ensure non-dependency on opioids when treating patients for pain as a result of the disease. 

Unmet Needs in Sickle Cell Disease Management 

Michael R. DeBaun, MD, MPH, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease 

Columbia University College of Physicians and Surgeons

For what it’s worth, I interview a lot of doctors. I’ve done a lot of panels. What struck me about you folks—the folks who deal with diabetics every day, or people with diabetes—I’m informed that diabetics is not the best term.

Not good—so, people with diabetes, people dealing with this disease. What I noticed is that you listen. Every 1 of you talks about listening to people. Doctors are bad at that, as a group, but not you. Did you select this because you like listening, or did you learn to listen because that’s what you do? I don’t know. It was just an observation. Call it a hunch. Look, we have come to the end of this discussion, but I don’t want to leave without giving each of you an opportunity to say something that you feel we may have omitted or something that you want to leave our audience with. Why don’t we go around, and each of you can take about 30 seconds and give us some final thoughts. Why don’t we start with you, Elaine?

 I’m just thinking back on the patients that I saw today. One of the biggest challenges of diabetes, and dealing with highs or lows, is that you could do everything perfectly Monday to Monday. With other things like saving money, or training for a marathon, you have something to build on. Maybe on the eighth day you can take a day off. That’s not the case with diabetes. You’ve got to start all over again that eighth day and do it all over again, and it may be the worst day of the eight. It’s such a marathon to run, and you don’t have an end line, or point, to look forward to. As a provider, you have to place yourself in that reality. You have to see through the patient’s eyes every day, and if you don’t do that, then you won’t understand their struggles and you miss the boat. That’s what I strive to do every day for them.

Wow, that was well said. Thank you. We really need to understand that, as Elaine has said, diabetes never gives the patient a break; it’s 24-7. Hypoglycemia is incredibly scary and really does limit so much of what the patient can and can’t do if we aren’t careful with educating our patients, assisting with quality of life, and making sure patients have a good understanding of their medications. We need to provide good education on prevention and treatment, and the use of continuous glucose monitoring [CGM] to try and help patients prevent hypoglycemia. We all need to take it seriously and make sure that we ask the right questions every time we touch the patient, so that hopefully we can have a better outcome for them.

I’m going to say something completely different, but I agree with what Elaine and Davida said. I want a system in which the pharmacist tells the patient and me every 2 years when their glucagon is going to expire, so I know to write a new prescription. Because what makes me crazy is when I’ve forgotten and the patient has forgotten to ask, and then they have a bad low and they don’t have their glucagon. Why can’t the pharmacists do this? I get bugged all the time about everything. Why can’t the pharmacist just say, “Patient X needs a prescription for glucagon”? That’s no skin off my back. They should probably just do it without a prescription. Why does this even have to require a prescription? That’s another issue. We need a system so patients and providers are reminded to do the refills. I don’t know why it doesn’t exist, so we need to create that solution.

Could that be built into the EMR [electronic medical record]?

When you click the prescription, it should pop up in 2 years.

 It should just say, “This is due.” Why not?

Jay Shubrook, DO, FACOFP, FAAFP, BC-ADM: 

Hypoglycemia is certainly something that’s very important to our patients, and most providers underestimate the frequency of it. Please ask your patient and the family at every visit about hypoglycemia. There’s no better time than today because we have plenty of tools to identify hypoglycemia, such as CGM, or continuous glucose sensors. We have ways that we can help patients have less hypoglycemia—particularly patients with type 2 diabetes—with novel therapies, and we have very effective treatments that can abort a hypoglycemic episode. Making sure you use the right treatment at the right time really is going to empower your patients not to be paralyzed by hypoglycemia but to be an informed and active crusader to prevent it and treat it earlier.

I want to thank all of you for being with us. This has been eye-opening for me because I don’t treat diabetes on a daily basis. I always thought, “It’s about sugar, for sure; usually it’s about hyperglycemia.” This hypoglycemic problem is real, significant, and tricky. These new therapies that I’ve learned about from you are strikingly interesting. Thank you all for being here.

I want to thank all of you at home for watching this 

. If you enjoyed the content, I want you to subscribe to our e-newsletter to receive upcoming Peer Exchanges and other great content right in your in-box.

Thought leaders in diabetes provide closing remarks about the importance of continuous awareness of glycemic control in patients as well as the need for effective communication between patients and providers. 

Final Thoughts: Awareness and Communication in Diabetes 

Peter Salgo, MD, Columbia University Irving Medical Center 

Advancing the State of Precision Medicine in Rheumatoid Arthritis

01:29 Precision Medicine in Rheumatoid Arthritis (RA)

03:34 Biomarkers and Diagnostic Tools in RA

07:08 Science Behind Molecular Signature Response Classifier (MSRC) Test

08:30 Accelerate Information of Molecular Signatures (AIMS) Study

10:33 Experience with MSRC Test in Clinical Practice

12:46 Heterogeneity of RA and Development of Other Biomarkers in RA

14:49 MSRC Test Coverage by Medicare and Future of Precision Medicine in RA

17:54 Safe Step Act in RA Management and Success in Utilization of Precision Medicine

20:48 Final Thoughts and Recap of Discussion

Expert rheumatologists discuss precision medicine in rheumatoid arthritis. In particular, they discuss available data and the value of various tools in guiding treatment selection in routine clinical practice.

Advancing the State of Precision Medicine In Rheumatoid Arthritis

Vibeke Strand, MD, MACR, FACP

 I’ve enjoyed the opportunity to talk with you because I’ve learned a ton. Your experience and acknowledge is outstanding. To wrap this up, what would be your take-home messages for health care providers treating opioid-induced constipation?

 One would be to open the conversation if you see it on their list. I find that being a PCP [primary care physician] these days is the hardest job. The patients are living longer and in and out of the hospital. With the amount of record review, interaction with the charts, and certain care measures, this may be 10th on the list. They may be doing well from a pain relief perspective. We ask that question, but it’s hard when they have a million other medical problems. But it’s a simple question.

Patients oftentimes don’t want to bring it up or talk about it. It’s embarrassing. But it can affect their quality of life. We’re happy to help. I’m happy to see patients and have the longer discussion with them, especially if we can initiate the appropriate-line therapies to start. It’s important to open that conversation and ask them the simple question: “I see you’re on opiates. How are your bowel habits?” If there’s an issue, then start to get them on the right track to treatment, because it’s very efficacious and safe, as we talked about. They don’t have to bear this burden. We can help fix it for them.

 Yes, the conversation or even the prompt to have that conversation is important. We transitioned to electronic intake forms, and we have smart responses, where if somebody is taking opioids, it starts to ask about all the things related to opioids, including feeling sleepy, being able to function at work, and driving. Constipation came up there, and we were surprised by the number of people who were responding to this. We had never thought to have that discussion. With the time that you have to be able to discuss this, it might be very low on the list. But if you’re able to screen and have the conversation based on that response, it cuts out a lot of time, and thankfully we have some great options to choose from that will make the conversation go quickly and smoothly.

 Excellent. Thank you once again for participating in 

Expert Perspectives in the Management of Opioid-Induced Constipation

A panel of experts discuss their key takeaways and the future of treating opioid-induced constipation. 

Authors