so hello everyone and thank you for
joining this second webinar to provide
an update on the COVID 19 outbreak
related to Duchenne and Becker
muscular dystrophy community my name is
Suzie-Ann and I'm the communications
coordinator of the World Duchenne
Organization and I'm here yet again with
Elizabeth who's the chair of the WDO and
in this webinar we invited two other
clinicians or experts who will address
the questions that arise from from the
community in regards with this
coronavirus outbreak so this webinar is
a follow-up webinar on the last given
one on March 14 and to access this
previous one you can always check the
live feed that we have and there we
also have the report of this webinar so
you can head over to worldduchenne.org
to get more information about that but
before we start I'm inviting you to join
the discussion in the comment section
because you know you will be
automatically muted upon joining your
keyboard is your voice so would you like
to ask a question you can do this via
the chat option so when you click in the
top right corner on the speak icon or
bubble on in the middle bottom if you're
on mobile so at the end of the webinar
we will collect all the questions that
haven't been answered as well so we can
try that to provide input on a follow-up
webinar so this webinar will be recorded
you will receive a link to the webinar
recording and a report in English that
you can translate and disseminate with
other clinicians and families as well so
this is the agenda for today we start
with a brief introduction where I will
discuss the outcomes from the previous
webinar and then we will head over to
the questions from experts and the
experts this time dr. Jarod Wong and
professor Annamaria De Luca and they have
provided their considerations on the
questions that we collected this time
then Nicoletta will explain something
about the show your solidarity campaign
before we call it a wrap up and move on
so last webinar we had professor Mercuri
professor Muntoni professor Finder and
professor Goemans share their opinions
and questions we gathered from the
community and you can find this
recording together with a website with a
report on our website and to summarize
these findings we created a list of 15
things we know so far about COVID 19 a
relation to Duchenne and Becker
muscular dystrophy so so far there are
no confirmed cases of the corona virus
in people affected by DMD or BMD nor
they have a higher chance of catching
the virus even if you're on non-invasive
ventilation or using a cough machine so
just like anyone families with Duchenne
and Becker are urged to stay at home as
much as possible to avoid possible
contact with the virus so any hospital
visits that are not urgent can be done
through the telephone or should be
postponed and if your country does not
have closed their schools
yet we strongly recommend to keep your
children especially when there are
steroids or who are respiratory compromised to keep them at
home so further on doctors recommend to
not to make any changes in your
medication regimens unless your
clinician advises yourself and in terms
of medication it's also advised to order
a short supply of your medicines in case
of a shortage further on if somebody
with Duchenne or Becker catches the virus
they will have a bigger chance that it
will cause a viral pneumonia and this
will always risk and this is always a
risk for respiratory failure and the
same goes for cardiac disease so
patients with heart failure
have a higher chance of being severely
impacted in case of infection so
therefore we cannot stress enough for
the rules of well really common sense
and basic hygiene so using good old
water in soap to wash your hands
regularly and thoroughly is paramount
especially for people who are caregiving
so that was a bit like these
considerations you can
read them on the website and you can
even translate them to share with your
community so then I wish to hand over
the talking stick to Elizabeth who can
then provide an update on the questions
from the community Elizabeth
yeah can your handout give me the yep
can you
yes thank you so thank you again for
dialing in logging in all and and we are
really happy today to continue what we
started last week which means informing
patient organizations around the world
as good as possible about things to
consider in the context of COVID
19 and DMD or BMD and where we last week
very much focused on prevention and also
for example for the steroids like is
there a higher risk to catch the disease
infection if we use steroids or not this
time we will also focus on what if our
child gets a fever what can be done and
so we're really happy that we have two
wonderful speakers today we will again
look at are there any what other
reliable sources and other certain drug
supplemental vitamins that can help and
what helps and what happens with
clinical trials well the first one I can
answer in that as far as saying what we
know at this moment till now it was
really uncertain that we didn't know how
EMA and FDA would consider missing data
stopping trials or all these things
which could be good happening now in
COVID times so we're very happy that
they both gave out a guidance FDA as
well as EMA we will put links on our
website as well so you can go back and
check for them and then on our website
we will have also on the newsfeed we
will have which companies send us
information about changing their plans
their schedules in the times of coverage
so there will also be on the WDO website
and here in the slide you see the link
where we are to find this on our website
so then already briefly Suzie-Ann
introduced today we have two experts with
a very
impressive track record in DMD one is
dr. Jarod Wong endocrinologist from
Glasgow University he has been very
active also in the field of bone health
and Duchenne so we're very happy he is
there and the other expert is professor
Annamaria De Luca
I'm sure you remember that during World
Duchenne Awareness Day last year we had
special information about supplements
and risk of supplements which was also
done by Professor de Luca so then if I give
the microphone to dr. Wong so I should
probably say bye
maybe I should have said that by the
experts there is one thing we should say
experts in COVID 19 don't exist
there are DMD expert but no COVID 19
expert and especially not a combination
of that because we haven't seen any
history now of Duchenne patient with
infected with that COVID 19 okay
dr. Wong are you unmuted okay sure can
you hear me now yeah so the question so
the first question that was posed by
Elizabeth is the question that may already
have been addressed at last week's
webinar and is the potential effect of
the use of steroids on the immune system
we know of the benefits of steroids on
the muscle in Duchenne muscular dystrophy
such high dose steroids may have some
effect on lowering the immune system and
therefore in boys with Duchenne routinely
at least one of the reasons of it is
that through vaccination is recommended
and certainly in some countries people
taking regular long term high-dose
steroids not just in Duchenne have been
identified as a risk group in the
current COVID 19 pandemic by some
and obviously there are some there may
be some differences between different
countries are not aware of all the
regulations but routinely and generally
we do not see severe unusual and serious
infections in people with DMD related on
high-dose steroids so that's the answer
for the first question Elizabeth can
you move the next slide yeah then the
next question that was posed is what is
the effect of steroid treatment in DMD
and potential COVID 19 infection as
Elizabeth has already discussed we as
far as we are aware there are no boys or men with Duchenne infected with
COVID 19 so we really don't have any
experience
theoretically it was possible that a man
or a boy infected with Duchenne that
infection may be more severe but there
are many reasons not just related to the
use of high-dose steroids because we
know our boys and men have got
respiratory issue muscle issues cardiac
issue and therefore yes potentially if
infected the infection might be more
severe but I'm not sure that it's
related directly just to the use of
high-dose steroids and the immune system
because there are many many factors in
hand or the hand hygiene etcetera and
keeping all those all those things are
extremely important
next one Elizabeth and this question has
already been discussed at last week but
we thought it was useful just to stress
again and I guess some families might
think if you are saying that they might
be some
impact on the immune system and should I
therefore then considering stopping
steroids in this current climate and
that the very clear answer is no this
should not happen as in fact is not
possible but most of you will hopefully
know of the issue of adrenal suppression
from the use of long term steroids
people who take steroids like boys with Duchenne for a long period of time they
suppress the adrenal glands which
normally make steroids so we have this
issue called adrenal suppression leading
to adrenal insufficiency
so generally out of the COVID pandemic
even if we do want to stop steroids in a
boy with Duchenne on any other condition
that means we gradually weaned over
months and sometimes even over longer
period of time because to cope with
infection extra steroids is often needed
or the person could become very ill and
be tipped into what we call an adrenal
crisis we do know that some people who
are treated with long-term steroids
infection may be more severe but one of
the possibility is that perhaps that a
management of steroids during these
acute acute illnesses may not be as
adequate and that's that's why the
response may not be as good so the clear
answer is no not to stop steroids please
do not stop steroids and and this is
generally not possible to be done
abruptly this is an this is next
question which I think if you've not
been contacted by a clinical team this
you may hear a lot more soon and it may
be there differences between where
scientists are so because of this issue
of adrenal suppression leading to
adrenal insufficiency if a boy or man
with Duchenne on long-term steroids has
a very severe illness particularly
vomiting illness and steroids to be
given in a different form and generally
as per the most recent standards of care
the recommendation is that boys with
Duchenne and men with Duchenne on steroids
should have access to a different kind
of steroid hydrocortisone which can be
injected at home or in the community
should they become unwell and before
coming to the hospital now I'm aware
that since 2018 different centers have
been trying to roll this program this is
only the injections only needed in
severe cases especially with vomiting
diarrhea illness where the boys unable
to take steroid medicines in some people
especially older people non ambulant and
then for instance the dose of steroid
may be slightly lower and may not be
sufficient to cope with moderate stress
and there may be a case to consider
whether there is a need to increase oral
steroids by mouth in kind of acute
illness fever or infection now the
majority of boys with Duchenne certainly
young boys this is not needed as you can
imagine that the clinical teams are now
actually very stretched and it is
possible that some clinical teams may
actually be asking all boys and men with
Duchenne to stick to us as a certain plan
so to ensure that we do not miss any
boys or men so this is something that
may need to be be to be considered so if
the boy is unwell with an acute illness
with fever there is always the question
of what to do for those who are on intermittent steroids particularly doing the period
on the ten days off or those who are on
the weekend dose only and
a plan should be in place and I think if
you've not got a plan it will be
worthwhile contacting your neuromuscular
teams with regards to that thank you
so the this is this is another important
consideration - just remember because I
think all of you are expert patients and
you know your condition
much more then the emergency treating
doctor or perhaps the intensive care
doctor it is always very good to remind
the treating doctor or your ambulance
staff that steroids cannot be stopped
and when it makes it to hospital steroid
therapy on top of all the other issues
related to COVID or chest or the
heart should not be the LAST thing you
think about in fact it may not
necessarily be the first thing that the
doctor manages but it should not be the
last thing and I recommend that those
who are English speaking let the doctors
knew that your son on yourself you're on
(L)ong term steroid you have (A)drenal
suppression you are (S)teroid depended and
in fact you need to be (T)reated with
extra steroids when unwell so don't make
this the LAST thing that we all think
about and quite often if the person is
so unwell and in intensive care in the
hospital and can take steroids by mouth
doctors should be giving those steroids
by a drip so don't make this the last
thing you think about where can we find
a lot more information so if you do
present to a hospital and the doctors
are not familiar with management of
adrenal suppression in Duchenne and the
patient organizations PPMD and clinicians
in the United States have developed a
steroid protocol the PJ Nicholoff protocol
almost all of you should know about for
management of steroids during illness
this is published in a scientific
journal in 2017 the link is below which
the doctor is able to access and a
patient friendly summary is also
available online on the PPMD website and
this is where we can point the people
who are not familiar with the treatment
protocols to the right place
but be aware that different hospitals
have their own specific protocols which
works just as well already so if they do
something slightly different totally
worried about that thank you so much and
we will make the the links available and
easy to find for everybody who's looking
for thank you so much dr. Wong for your
explanation and then we will ask then
the next expert professor Annamaria
De Luca pharmacologist in Bari in the south of
Italy about questions about drugs what
what is done and what should we do what
can we do ourselves or should we refrain
ourselves from doing anything regarding
taking drugs so the first question is
the question which is in fact asked also
last week but that question keeps coming
like can't we do anything to boost the
immune system the floor is yours
professor De Luca hello can you hear me
yes okay so this is an important point
because there is a great plethora of news
in the media about the potential
usefulness of some specific vitamins or
antioxidants or probiotics with presumed
protective or boosting effect on immune
system this every day
and mostly during these difficult days
so the first question is if any dietary
supplements can actually help in COVID
19 the answer is no the answer is no
because all vitamins minerals and other
components that allow the proper
function of our immune systems are in
our food in healthy food so the main
advice is to maintain an healthy diet
properly varied and we properly take
of fruits vegetable fish meat and
fibers of course this does not apply in
cases of specific deficiency of vitamin
as in the case of deficiency in vitamin
D or calcium which do occur in Duchenne
patients in this case of course
implementation is very important and has
to be maintained the other point is even
if a supplement is not directly
effective can anyway be used because
it's safe is natural and cannot harm
again the answer is no because the idea
that the natural compound is by
definition safe is false and dangerous
harmful effect of dietary supplements
can occur by various reason first of all
can have unwanted action for high
dosages can have harmful interaction
with other drugs use at the standard of
cares or that need to be used as case of
infection and also if supplements and
more importantly if supplements are not
purchased and in pharmacy or in any
other authorised site such as
online they can have not proper
content all principles they can have
contaminants and impurities and can have
serious consequence for health so the
main point is not to use the dietary
supplement as auto medication and
without any control of health
specialists this applies also to
melatonin which is claimed to have a
wide variety of effect including effect
as antioxidant and anti-inflammatory
this is an endogenous compound mostly
useful for regulating the sleep sleep
time and recently there has been
proposed the relationship between
melatonin levels and the severity of the
infection again this is not supported at
the moment by very strong scientific
evidences and the use of melatonin
especially if not under control of
doctors has to be really not encouraged
at at all thank you thank you shall we
go to the next question yes please
so the next question is we also have
heard a little bit last time there the
might also be in aspects on the steroids
that probably probably could have a
positive effect in COVID 19 could you
explain a little bit more about that yes
in fact there was it has been proposed
that low dosage of steroids can be useful
in the so called cytokine storm
cytokine storm is a severe phenomenon
that may occur in COVID 19 patients at
advanced at stage of pneumonia normally
the activity of our immune system is
very important to contrast infection
however at a certain stage of the
disease and in some patients
there could be an excessive discharge of the
virus from the infected cells leading to
a massive production of cytokines and
this reaction has to be controlled
because it's very very serious so among drugs that can have interest for
against the cytokine storm
there are steroids in China especially
it has been proposed that low doses of
glucocorticoids such as alpha methyl prednisolone can help to reduce the storm
without causing a massive
immunosuppression and at the moment
some clinical trials are ongoing in
COVID 19 patients but I have to say
that there is some debate about about
the usefulness of steroids in this in
this context by the way in this frame I
think this is another reason not to stop
steroids in in patients of course it has
to be proved that the low steroids doses
are really interesting and important for
the cytokine storm and what happens to
patients that are already under chronic
treatment with steroids but for
considering the information we have right
now and all the data in the information
that dr. Wong provided it is
important not to stop the steroid
treatment unless specifically indicated
and this is valid also for other
standard of care in patients also the
treatment with ACE inhibitors has been
questioned at but there is no scientific
evidence that this therapy can be
actually not useful can lead to worsen the infection by the virus
and actually it is not proved so
it's better to maintain drugs that are
effective in controlling cardiovascular
function in Duchenne patients thank
you
yes please and there have been some
discussions also on fora
about what is the best drug to treat
fever in the case of COVID 19
which could you have some information
about that yes indeed there have been
many reports about the preferential use
of paracetamol rather than other
non-steroidal anti-inflammatory drugs
with idea that these latter can
actually have a too extensive
anti-inflammatory action that could be
not useful in case of virus infection by
the way this has not be confirmed and
recently the European Medicines Agency
actually said that patients with COVID 19
can use any anti-inflammatory drugs
according to what the doctor decide in
relation to patients and to the drug and
the risk benefit ratio said that the
specific situation for Duchenne or Becker
patients can be different considered that
many non-steroidal anti-inflammatory
drugs are not advised because the
concomitant use of steroids so in this
setting
paracetamol is for sure the first choice
drug and importantly it has to be used
for at low dosage and for the shortest
time possible because I remember I would
like to underline that fever is also a
symptom is important to have a diagnosis
for the COVID 19 so it's important
to understand if there is fever because
doctors have to make a proper diagnosis I'm
ready for the next questions coming this
is one who is we saw like half an hour
ago the Trump tweeted something about it
yes this is a very hot topic yes the
chloroquine useful for treating the
infection it's important to say that at
the moment there is no approved therapy
for COVID 19 unfortunately and there
is a lot of efforts in in searching for
effective drugs and it's also very
dynamic field because we we try to learn
from what we live every day so it
happens often that we have a lot of
impressive news and we have to
understand the balance between hope and
reality so for chloroquine this
is very interesting because chloroquine
is an old drug it is used as
anti-malaria drug since its many years
along with its metabolite hydroxy
chloroquine there are evidences
that this compound can reduce virus
entry in cells and in this case could be
somehow interesting to assess is use
as prophylactic drugs in infected and also
its usefulness in infected people studies
are ongoing so there is no clear proof
that chloroquine can be actually effective
but what we have to learn in this case
and to think about is that chloroquine
even if it's safe is not totally safe it
may have important side effects
especially at heart level because it
can lead to prolongation of QT
intervals in the heart and this can
lead to dangerous arrhythmias we know
that many Duchenne patients may have
prolongation of the QT intervals and
these unwanted effect can actually be
more important in association with other
drugs so it's a it's fundamental to
remember that the chloroquine hydroxide
chloroquine have to be used at under
strict medical control and monitoring
very carefully heart function and of
course again any any possibility to get
drug from non authorized source on
internet and self medication is
absolutely discouraged because can be
seriously dangerous thank you so much I
think this is really what we all need it
to hear thank you so much and then the
last question to you at least at this
moment is but what are the drugs which
are currently used when a subject is
infected with COVID 19 as I was saying
this new corona virus is a new virus
therefore no specific drugs are
available however during the last
decades a lot of progresses have been
made in the field of antiviral drugs and
we now have drugs effective against the
virus for HIV infection hepatitis and
SARS Ebola so those viruses are somehow
similar to corona virus in that they are all RNA viruses and so even if they are
different what is hoped is that some
basic mechanism between the viruses is
maintained and so a drug developed for
another virus can help in case of
infection by COVID 19 so in this general
frame some
virus some sorry some drugs used for HIV infection or Ebola are used
off-label in infected patients and off
labels as you know very well means that
there are there is no specific drugs
against the main the main cause of the
disease but they can help for the
mechanism of action so I can I can
mention a few example of drugs that gave
promising results in China and in Italy
and you probably heard in in in the
media one is Remdesivir it is a
nucleoside analog that has been
developed with some controversial
results for Ebola and it seems promising
for patients affected by the new corona
virus some clinical trials are ongoing
in China another treatment is with the
combination of interferon alpha with
inhibitors of viral proteases is
lopinavir/ritonavir this is a
classical cocktail used for HIV
infection and is also possible in
children older than two years other
classical antiviral drugs such as ribavirin with other inhibitors of reverse
transcriptase use it again for HIV
infection have been considered with
different level of enthusiasm and
another promising drugs is favipiravir this
is an antiviral drug used for infection
by influenza virus is available
in Japan and some use its ongoing off-label and with clinical trials for
having a better idea of
efficacy similarly some antibacterial
drugs this has been mentioned also for chloroquine and in
association with azithromycin
can be used according to the general
state of the patients considering that
some opportunistic infection bacterial
infection can also occur I would mention
also tocilizumab sorry
tocilizumab is an inhibitor of
interleukin 6 approved for rheumatoid
arthritis and with a great potential to
control cytokine storm which has been
used with success in Italy on some few
patients and the clinical trial has been
is ongoing now of course all these drugs
are drugs that needs to be prescribed
and have to be used under medical
control and many of them have to be used
only in hospital settings so these are
no drugs for auto medication again auto
medication is really really dangerous
in these conditions especially in
patients with or that already have a
complex standard of care as like
Duchenne patients so another point is
that intensive work is ongoing to try to
obtain a vaccine as you probably heard
worldwide and there is also the attempt
to use plasma extract of patients to try
to obtain compounds that can be helpful
for infected patients so for the moment
this is the situation again it's a very
dynamic situation and things change
rapidly
day after day because it's really
unclear which is the best strategy so we
have to rely on daily experience and
scientific basis thank you thank you
really thank you so much and when I
listening to both of you I realize how
fortunate we are in the Duchenne field
to have people like you not only with
knowledge you have but also that you're
willing on Saturday afternoon to show up
for us and help us to respond to all the
questions
the families at home have really thank
you so much
probably you've seen that some questions
came in I merely for dr. Wong the
first is if somebody if someone is
supposed to start steroids now or soon
does it make sense to wait until the
pandemic is over can you unmute dr. Wong
yeah I'm not sure that there is so I
suppose I think you say so I'm a
pediatric endocrinologist and this is
sort of a neuromuscular sort of decision
I know I'm not entirely sure that there
is actually a potential benefit or
disadvantage but I suppose in the
current climate we also need to consider
what's practical I certainly know in the
UK the GPS and pharmacists are
completely overwhelmed and new
prescriptions are either very slow or
not being attended to so I'm not sure
that there is a particular sense to
either delay or or start straightaway
but it may just be determined by what's
actually possible in this current
alignment
thank you so much and there was another
question which I think Duchenne
families all have and suffer from and
that is that it is in it's so difficult
to have clinicians listen to you if
you end up in a hospital in a
crisis and families have information or
want to point out something it's really
very hard to get the attention you you
need for your problem so somebody asks
here could we would there be a
possibility to have a phone number of an
expert so the doctor could call the
expert and and hear from them what needs
to be done so I guess it really depends
on the system in your hospital
indeed in the country addressing
specifically just on the steroid
management certainly in my hospital and
in a lot of uk-based Hospital there can
be alerts that can be put up there other
non Duchenne relate that doctors can view
but of course sometimes they still don't
view it and some of us will over the
week quite soon be sending out specific
instructions and letters for the
patients with regards to steroid
management plans and what's needed so I
guess that can be shown to families I am
a way that different patient
organizations have different alerts that
I suppose can be used and I guess you
just need to clarify with your
neuromuscular team it's there is a bit
specific contact person that they can
mention that they encourage or ask the
emergency treating doctors to to contact
so I guess that's generally the advice
and in a just occur depending on the
setup in your hospital or your country
thank you so much thank you so then this
is kind of the end of the part with the
questions to the experts and then we
will a little bit like last week say
what what you as patient organisations
who are online now can do to change the
situation in your country or help
support and one of the things is that as
patient organization as a Duchenne
patient organization we are in a
fantastic position to tell everybody and
use all our tools and social media and
maybe emails to donors to volunteers to
tell them stay at home and do it for us
for our children it's extremely
important that you stay at home and help
us to slow down the spread of the
disease so I would recommend everybody
to start a campaign we could use the
hashtag home4Duchenne
because it's really there and home4Becker as
well of course because it's really
important that we slow down the
progression
disease and we have the best story there
are not many people have such good
stories as we have to tell why it's so
important strictly adhere to national
and local authorities and guidelines
avoiding the spread of fake news at your
community if you doubt about some news
come back to us and like now we will
find the experts who can explain to you
what is what is real news and what is
fake news share this webinar and the
report we will make a report again with
the main topics from this webinar spread
the best information you can follow the
next webinar
Nicoletta will announce that later if
you have questions or you hear relevant
questions please send them to us that
helps us to prepare the next webinar
which will be next week and what can we
do for you well it is that we record
again this web recorded again this
webinar we will again make a report
translate you can go to the our website
and look at the live feed where you see
the news we update that as we said
earlier also about what clinical trials
what is changing in the field of
clinical trials or might happen there we
will have the weekly webinar and we
really would like to support everyone
where we can so don't hesitate to
contact us then now I am leaving you and
Nicoletta is the next will speak to you
hello everyone so we are all happy to
have you here online even today and is
nice to have these weekly call all
together on this emergency because now
we are all on these together and we can
move forward only together I just wanted
to show with you soon as pictures that
arrived at this week with the home4Duchenne
and home4Becker hashtag
because as you as Elizabeth told you
it's really really important to
continuing sharing photographs of your
selves and of you at home with this
hashtag because it's simple
to raise the awareness of you know of
the importance about staying at home
especially for people with severe
diseases like Duchenne but for everyone
because the impact of one person can be
really really important for the
community so here we have three
fantastic children showing how they are
living the quarantine time and they come
from different places from Spain and
from United States and it's really
really important that this campaign will
move forward and we need your help and
your support in your countries from our
side as Elizabeth told you we are
always here to help you to listen to
your needs and your question and
continuing about information and
supporting your groups so we really
thank you for being here today and we
wait for you for the next webinar next
week
okay so I think that's it thank you all
so much for joining this webinar I'm
sure we have touched a lot of questions
but not all of them so if you have any
questions that are not discussed that
you would like to have more information
for just contact either Nicoletta or me
at the email address as shown here in
the bottom we will share the recording
of the webinar soon we will upload it
and we will make sure that you will
receive the report so you can further
disseminate the learnings we had during
this webinar so thank you very much for
for tuning in I giving you back 15
minutes of your time and I wish you all
a very happy weekend so thank you all
and speak to you soon goodbye
