The second session in PPMD’s Becker Muscular Dystrophy webinar series focused on cardiopulmonary care. Our clinical experts reviewed what every person living with Becker and their families should know about care for the heart and lungs.
Speakers:
- Dr. Andreas Barth – Medical Director, Center for Inherited Heart Diseases; Associate Professor of Clinical Medicine and Genetic Medicine, Division of Cardiology (Cardiac Electrophysiology), Johns Hopkins University
- Dr. Seth Hollander – Medical Director of Heart Transplantation; Clinical Professor of Cardiology, Stanford Medicine Children’s Health
- Dr. John Pascoe – Associate Program Director, Pulmonary Fellowship Program; Associate Professor, UC Department of Pediatrics, Cincinnati Children’s Hospital
Heart Health in Becker — Dr. Andreas Barth
- Heart problems are common in Becker, even when muscle weakness seems mild.
- Everyone with Becker should have regular heart checkups to watch for heart weakness or rhythm problems.
- Heart tests may include:
- An ECG (looks at heart rhythm)
- Wearable monitors if symptoms come and go
- Long-term monitors if concerns continue
- Treatments for heart rhythm issues can include:
- Medications to steady the heartbeat or support heart function
- Procedures to fix abnormal heart rhythms
- A defibrillator device (ICD) if the heart is weak or shows scarring. This can prevent dangerous heart rhythms
- Families should reduce general heart-health risks, like high blood pressure, high cholesterol, sleep apnea, diabetes, smoking, and obesity.
- Some cholesterol medicines (statins) can worsen muscle symptoms. Other options, such as a class of medicines called PCSK9 inhibitors and bempedoic acid (marketed as Nexletol in the US) may be safer.
Heart Transplant & Advanced Therapies — Dr. Seth Hollander
Last fall, PPMD gathered top cardiac, surgery and other key experts to engage in dedicated conversations around cardiac transplantation across the spectrum of dystrophinopathy, creating a path forward for transplantation as a potential option for patients living with Duchenne and Becker. Through this meeting, a detailed protocol for cardiac transplantation was developed. This effort has already driven real-world progress, including a dystrophinopathy patient successfully receiving a heart transplant.
Severe heart weakness is the main reason people with Duchenne or Becker may need advanced care.
Artificial heart pumps (ventricular assist devices, or VADs) and heart transplants are possible and successful for carefully selected individuals with Becker.
Key considerations for successful VAD placement or transplantation include:
- Careful evaluation to choose the right candidates
- A coordinated, experienced medical team
- Education and support for families
With expert care before, during, and after transplantation, people with Becker can be listed and receive donor hearts quickly with good outcomes.
Lung Health in Becker — Dr. John Pascoe
People with Becker often maintain stronger lung function for a longer time, but regular breathing tests are still important, and careful monitoring and support during times of illness is crucial.
Families should keep track of:
- Forced vital capacity (FVC), a key breathing measure
- Use of cough-assist machines
- Use of nighttime breathing support (like BiPAP)
When managing respiratory illness at home:
- Use cough assist regularly, or more often as needed, to help clear mucus
- Use nighttime breathing support as usual and even during the day if needed
- Avoid cough suppressants. Plain guaifenesin is okay to use.
Call your care team or go to the ER if:
- You see signs of breathing trouble:
- Faster breathing or heart rate
- Increased tiredness or shortness of breath
- Trouble clearing mucus
- Chest, shoulder, or stomach working harder to breathe
- Oxygen levels stay below 95% on room air after using cough assist
- Breathing gets worse or extra breathing support is needed
- The individual is too tired to clear mucus
In the hospital:
- Breathing issues in Becker usually need breathing pressure support like BiPAP, not just oxygen alone
- Bring cough assist and breathing machines from home
- Tests may include blood gas, chest X-ray, and basic labs
- When recovering, breathing support should be reduced slowly, and if a breathing tube is used, it’s safest to come off the tube directly onto full breathing support with BiPAP (not CPAP alone)
Everyone living with Becker should have regular heart and lung monitoring, as early recognition and response to symptoms can be lifesaving. Managing cardiovascular risk factors and keeping an updated emergency plan are critical parts of care. Always bring your home machines and care plan to hospital visits, and remember that strong teamwork between families and medical providers is essential for the best outcomes.
For additional information and resources on Becker, please visit PPMD’s website.
Don’t forget to keep PPMD’s Safe and Unsafe Medication List on hand! Be sure to contact your primary care provider and/or neuromuscular team directly for medical advice. If you are worried about your child, please do not wait to call. You can find a list of CDCC emergency numbers here.
Watch the recorded webinar
The post Becker Muscular Dystrophy Series – Part 2: Cardiopulmonary Care appeared first on Parent Project Muscular Dystrophy.