COVID-19 for people affected by Duchenne or Becker #2

What we know about COVID-19 part 2

What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne and Becker muscular dystrophy, the World Duchenne Organization had hosted a second webinar last Saturday for its members.  Information is provided by World Duchenne Organization.

What is the effect of steroids on the immune system?

Dr. Jarod Wong, an endocrinologist at Glasgow University – “Steroids prescribed for DMD may have some impact on lowering the immune system. Hence, routine flu vaccination is recommended. People taking steroids have been identified as an at-risk group in the current climate by some governments. However, we do not commonly see severe, unusual and serious infections in people with DMD on steroids.”

What is the effect of steroids and COVID-19 infection in DMD?

Dr. Jarod Wong, an endocrinologist at Glasgow University – “At the moment, we are not aware of any cases of people with DMD and COVID19 infection. It is theoretically possible that if infected, the infection may be more severe. However, we simply do not know. In some countries, any person on long term steroids has been classified as at-risk and recommended to isolate for a longer period of time, for instance, 12 weeks.”

Should I stop steroids in this instance then?

Dr. Jarod Wong, an endocrinologist at Glasgow University – “No, this should not happen and is not possible. One issue with anyone taking steroids for a prolonged period i.e. longer than a few months is that the adrenal glands, which make steroids naturally, are suppressed (ADRENAL SUPPRESSION LEADING TO ADRENAL INSUFFICIENCY). Even if we do want to stop steroids, a slow plan of gradual reduction over several months is essential. To cope with severe infection, extra steroids are needed – stress dosing. Otherwise, the person could become very ill and be in an adrenal crisis. One possibility of more severe infection (of all kinds) in people with adrenal suppression from taking steroids may be that steroid management during the illness is not adequate.”

Is there anything extra you need to do if on steroids during (COVID) illness?

Dr. Jarod Wong, an endocrinologist at Glasgow University – “Regardless of the type of infection in a person with DMD taking steroids, if the person has vomiting and/or diarrhea, steroids should be given in another form. If there is access to steroids in the form of hydrocortisone injection at home, this needs to be given and then presented to the hospital. In some people with DMD on steroids (especially older boys or men on lower doses of steroids), there may be a need to increase the dose of oral steroids during mild to moderate illness, which includes fever. It would be worth checking with your neuromuscular team if this is needed. In the majority of cases, this may not be necessary. Some teams have been advising all their patients to do so to be on the safe side. Generally, this should be for a period of 48 hours but maybe longer if the person is sick. Currently, if the symptoms might be COVID related and do not resolve within 48 hours, generally the advice is to contact the relevant places for COVID advice, for instance, the national hotline. For those on intermittent steroids, a steroid plan should be in place with information on what to do if the person with DMD is unwell during the days off steroids.”

Is it true steroids might have a positive effect on COVID-19?

Prof Dr. Annamaria De Luca, pharmacologist in Italy – “It has been proposed that low dosage of steroids can be useful in a so-called cytokine storm. This is a severe phenomenon that might occur in COVID-19 patients in an advanced stage of pneumonia. Normally, our immune system can combat the infection, however, at a certain stage, there could be an excessive discharge of the virus from the infected cells, leading to massive production of cytokines. China proposed that low doses of glucocorticoids such as alpha methyl prednisolone can help reduce the storm without causing immunosuppression. There are clinical trials ongoing in COVID-19 patients, but there is some debate about the real usefulness of steroids in this condition, especially in patients already on steroids. With the information and data we have, it’s important not to stop steroids unless specifically indicated. This is also valid for other Standards of Care in patients, i.e. those receiving treatment with ACE inhibitors. It’s best to maintain drugs that are effective in controlling cardiovascular function, as evidence of the potential risk of ACE inhibitors are few and controversial.”

More links

All questions and answer here: www.worldduchenne.org

Webinar #1

Webinar #2

15 THINGS WE KNOW SO FAR

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Edasalonexent and COVID-19

La Force is happy to share the latest edition of the Catabasis Connection newsletter. As we are all faced with an unimaginable situation, Catabasis wanted to reach out and share information in response to questions they have been receiving about edasalonexent and COVID-19.

Catabasis is monitoring the trial for the safety of participating boys. To date, they did not identify any safety concerns related to COVID-19. We encourage reviewing your local recommendations to reduce the risk for boys and their families, and please consult your physician regarding specific medical advice. > Catabasis Connection <

Does edasalonexent affect the immune system?

Long-term toxicology studies with edasalonexent using higher doses than those in their clinical trials have found no evidence for immunosuppression using standard clinical and anatomic physiology methods. In clinical studies, now with over 100 patient-years of exposure to edasalonexent, Catabasis has found no evidence of immunosuppression or increased infections. In the Phase 3 PolarisDMD trial of edasalonexent, as well as the GalaxyDMD open-label trial, boys are not on steroids.

Should trial participants still go to the hospital for their assessments?

They are fortunate that site visits are relatively infrequent during the Phase 3 PolarisDMD trial with assessments every 3 months. Currently, they are focused on ensuring that patients have uninterrupted drug supply as well as safety monitoring. Catabasis is working closely with its clinical trial sites with frequent communication.

Will the outcome of ongoing trials be endangered by not being able to carry them out as per protocol?

Catabasis is actively monitoring the situation and has plans in place to address potential disruptions. Fortunately, they designed their clinical trial so that visits are relatively infrequent. Catabasis is working with sites to support drug supply, as well as safety and efficacy assessments.

About Catabasis

The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families. There lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and the Phase 3 trial, please visit www.catabasis.com.

About La Force DMD

The Force’s mission is to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate in the funding of promising research projects. Where access to treatments for rare diseases is concerned, it is essential that our community be strong: each member must be an active spokesperson who helps raise awareness for DMD among the general public, as well as for the challenges associated with access to treatment.

 

Edasalonexent is an investigational drug that is not yet approved in any territory.

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COVID-19 for people affected by Duchenne or Becker

WHAT WE KNOW ABOUT COVID-19

What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne and Becker muscular dystrophy, the World Duchenne Organization had hosted a webinar last Saturday for its members.  All these information are provided by World Duchenne Organization

General information

  • It’s a respiratory virus that can be spread by aerosols: little droplets when you cough or sneeze.
  • Symptoms are coughing, having fever, shortness of breath and difficulty breathing.
  • The virus can survive for hours on hard surfaces, so you don’t have to see the person who is symptomatic and spreading.
  • The highest risks are the older population above 60 and vulnerable people.
  • This Wednesday, the WHO officially declared COVID-19 a pandemic.

Stanford University School of Medicine Webinar ‘Coronavirus for non-virologists’

And DMD/BMD

  • There is no expertise about Duchenne / Becker muscular dystrophy and the coronavirus as we don’t know any DMD/BMD patient affected by it
  • We have asked DMD experts to give a reaction to the questions of our families
  • Situation and national rules will be different in all countries

 

How to follow instructions if resources are scarce?

Prof. Dr. Jonathan Finder –“The best way to protect your sons is the avoidance of crowds and careful handwashing with soap and water.”

Prof. Dr. Nathalie Goemans –“We cannot stress enough the rules of common sense and hygiene, applicable to the general population and even more important for the helpers and caregivers.”

Elizabeth Vroom –“Next to washing your hands often, it’s necessary to clean surfaces, door handles and touch screens regularly.”

 

What impacts do steroids have on the immune system?

Prof. Dr. Jonathan Finder –“Steroids are a mild immunosuppressant and reduce the activity of lymphocytes, and these are the cells that help fight off viruses.”

 

Do people DMD/BMD patients have a higher chance of catching the virus?

Prof. Dr. Jonathan Finder – “No, and possibly they have a lower risk given that they are less likely to be touching doorknobs and handles and shake hands and the like. Those in schools or just out and about have the same risk from respiratory droplets.”

Prof. Dr. Nathalie Goemans – “We cannot stress enough that containing this epidemic is everyone’s responsibility, we should all temporarily restrict our contacts and stay as much as possible at home, respecting strict measures of hygiene.”

 

When infected, will it take them longer to fight it off?

Prof. Dr. Jonathan Finder –“We have no information about this. Presuming that steroids are being used, it is likely that the illness will be a bit harder to fight since steroids are mildly immunosuppressant. This is NOT to say that one should stop steroids: DO NOT STOP STEROIDS, as this is dangerous and riskier than the possible risks of COVID-19.”

Prof. Dr. Nathalie Goemans –“Yes, it is known that a severe course of COVID-19 can cause permanent damage to the lungs. On a positive note: although steroids are known to reduce immunity, it might well be that steroids could have a protective role in the pathophysiology (cytokine-storm) of severe ARDS in COVID-19 but we don’t know yet.”

 

Are they at higher-risk or ‘vulnerable people’ most likely to die?

Prof. Dr. Jonathan Finder – “They are at higher risk to be sure as the illness is a viral pneumonia, and having pneumonia is a risk for respiratory failure in this population. But as for “more likely to die” I would say NO as these patients are younger and for the most part do not have underlying lung disease. Those with chronic lung disease are the highest risk group, along with the elderly.

On the other hand, cardiac disease is a risk factor, and there is a great deal of cardiac disease in the DMD population. Thus I do have concerns about the risk of COVID-19 infection for those patients with heart failure.”

 

More answer here < WDO Webinar: COVID 19 and Duchenne & Becker muscular dystrophy >

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