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What
type of treatment is available for FOP? What can be done to lessen
the pain?
The treatment of FOP can ultimately be divided into two categories:
1) symptom-modifying, and 2) disease-modifying therapies. At the
present time, there are no generally effective disease-modifying
therapies which can remove the bone which has formed or completely
prevent the formation of new bone. There are, however, numerous
medications that can provide some relief from acute flare ups.
Please
refer to a report in this book which reflects the authors' experience
and opinions on various classes of symptom-modifying medications,
which can serve as a guide to this
controversial area of therapeutics.
For
additional information on medications used with FOP and other treatment
issues, please consult "Medical Management
of FOP: Current Treatment Considerations."
The
following two questions deal with two relatively new possibilities
for treating people who have FOP. Accutane is a drug commonly used
in the treatment of acne that is also known for inhibiting the transformation
of connective tissue cells into cartilage and bone. Iontophoresis
is a treatment which uses electrodes and a low electric current
to deliver medication to the deep tissues including the muscle.
Some people who have FOP have found it to be helpful in controlling
pain.
What
is Accutane? What effect does it have on FOP?
Accutane is the brand name of a medication also known as isotretinoin
or 13-cis retinoic acid. It is a powerful medication commonly used
in the treatment of acne. The medication also has powerful effects
on inhibiting the transformation of connective tissue cells into
cartilage and bone. In fact, it is dangerous for women to take the
medication during pregnancy because it can interfere with the normal
formation of the skeleton in the embryo. It is this same type of
embryonic bone formation that occurs after birth in people who have
FOP.
Dr.
Michael Zasloff recognized this important effect of accutane and
wondered if this medication could be used for the benefit of patients
who had FOP. He conducted a clinical trial of the medication when
he was at the National Institutes of Health between 1984 and 1988.
Twenty-one patients took the medication at varying doses for up
to five years. Dr. Zasloff and his colleagues found that the medication
did not stop the growth of extra bone that had already started to
form in patients who had FOP. However, it appeared to decrease the
episodes of new bone formation around joints (compared to a control
group that did not take the medication) by at least 27%, as long
as there was absolutely no evidence of extra bone formation around
the joint (determined by physical examination, X-ray and bone scan)
before the medication was started.
The
medication has some unpleasant side effects-- including dryness
of the mouth and nose, minor hair loss, and cracked lips. Accutane
can also cause the fat levels (triglycerides) in the blood to rise
slightly. There is some concern that accutane may actually stimulate
bone that has already formed.
Beneficial
effects of accutane were noted at doses as low as 1-2 milligrams
per kilogram of body weight per day. The medication is taken orally,
once daily, with food. While accutane is far from a cure for FOP,
it may offer some slight benefit.
What
is iontophoresis and how can it help in treating FOP?
Iontophoresis is a treatment which uses electrodes and a low
electric current to deliver medication to the deep tissues including
the muscle. This method helps in the absorption of small molecules,
chemicals, and drugs that some patients have found helpful in easing
the discomfort of painful flare-ups. One type of treatment with
iontophoresis uses a powerful steroid called dexamethasone along
with a local anesthetic called lidocaine. The treatment can be used
intermittantly for several sessions only. There is no evidence that
this treatment changes the course of the flare-up in patients who
have FOP, though it may alleviate the pain. This is a topic you
should discuss with your FOP doctor, family doctor, and physical
therapist.
Can
injections (shots) cause problems?
Injections into the muscle (intramuscular or IM injections)
are dangerous and can cause a flare-up and bone formation at the
injection site. Immunizations and local anesthetics are two types
of shots which are given intramuscularly. Once a diagnosis of FOP
has been made, all injections into the muscle must be avoided.
Most
children already have received their usual childhood immunizations
by the time FOP is diagnosed. Approximately one-third of children
who have FOP have had flare-ups from the childhood immunization
DPT (diptheria-pertussis tetanus). In some cases, FOP was first
suspected because of a child's adverse reaction to a DPT injection.
Injections
and immunizations that are given underneath the skin (subcutaneously)
pose little risk, but deep injections into the muscle MUST
be avoided at all times.
Blood
tests are obtained routinely without any problems in patients who
have FOP. Please consult the appendix of this booklet for further
information on this important subject of IM
injections and immunizations. If you or your child's physician
has any questions on this topic, please contact Dr. Kaplan, Dr.
Connor, Dr. Smith, or Dr. Zasloff.
Should
people with FOP have flu shots?
The highest risk of complications from flu occurs in infants,
the elderly, and those who are disabled. Data from a survey conducted
in 2001 strongly suggest that influenza infections serve as a trigger
for disabling flareups in patients with FOP. FOP patients who HAD
influenza had a 60% chance of having a flare-up AS A RESULT OF THE
INFLUENZA, while FOP patients who did not have influenza had only
an 11% chance of having a flare-up during the ENTIRE flu season.
These data are highly statistically significant. Preventative immunization
against viral influenza may be a reasonably safe and effective means
of preventing this unique complication of influenza in the FOP patient
population. We recommend that patients with FOP, especially those
who are older and already have severe restrictive disease of the
chest wall, consider receiving non-intramuscular influenza immunizations
(subcutaneous administration). Since the vaccine is most often given
intramuscularly, you will need to make a special request for subcutaneous
vaccination. The flu vaccine should never be given to someone who
is allergic to eggs (since the flu vaccine is developed and cultured
with eggs).
Additionally,
unaffected household members of FOP patients should also consider
being immunized annually. An intranasal spray preparation of the
influenza vaccine is currently undergoing evaluation by the U.S.
Food and Drug Administration (FDA). If and when the intranasal influenza
vaccine is approved, it would be the ideal route of administration
for patients with FOP. Anecdotal evidence from patients leads us
to recommend that influenza immunizations should not be administered
while a patient is experiencing a flare-up.
For
more information, read the article "Viral
Influenza Associated Flare-up of FOP."
Can
the extra bone be removed?
The technical answer is surprisingly "yes," but that does not
tell the whole story. Surgical intervention to correct limb position
abnormalities (which have resulted from new bone formation in patients
who have FOP) often results in a worsening of the condition. New
bone will grow back and further impair mobility. Scientists do not
yet understand the reasons this occurs. However, as a more complete
understanding of the genetic and molecular basis of FOP evolves,
they hope to be able to block renegade bone formation. At the present
time, however, that is not possible. As a result, it is extremely
important to understand that any surgical procedure to improve joint
position or remove excess bone should be avoided because of the
likelihood that it will make the condition worse.
Even
if the bone reforms, can the joint be placed in a better functional
position? Again, the answer is technically "yes." However, there
is a high risk of complications such as infection and phlebitis
(an inflammation of a vein). This is especially true with surgery
of the lower limbs. Such surgical intervention often ends in failure,
as it is difficult to reposition one set of joints in the lower
limbs without affecting posture and balance.
If
surgery for FOP is not recommended, what is the risk in having surgery
not related to FOP?
Surgical operations usually make FOP worse by causing the body
to generate more bone. Emergency situations may exist where an operation
may be necessary, as in appendicitis. Although such an operation
may lead to a flare-up, the life-threateningnature of the emergency
may justify the surgery. All parents and physicians of children
who have FOP are strongly encouraged to read, "Surgery
and FOP: When to Say Know" and "FOP
and the Life-Threatening Emergency: What to Do About Anesthesia."
What
precautions should be taken if a person with FOP breaks a bone?
Is there any difference in treatment depending upon whether the
breakage occurs in FOP bone?
A fracture in a person who has FOP needs to be treated, as a
broken bone would need to be treated in any patient who sustains
a fracture. The goal of treatment in all individuals is to allow
the bone to heal in a comfortable and functional position. People
who have FOP may not need to have their fractures immobilized for
as long as other people, as fractures generally heal rapidly. Surgery
is almost never necessary to treat broken bones in people who have
FOP. Also, a splint may suffice where a cast may otherwise be necessary.
The decision on exactly what form of treatment is necessary for
a fracture in a person with FOP depends on numerous factors which
include: the type of fracture, the bone that is fractured, whether
the injury is open or closed, the age of the individual, the degree
of deformity, and the functional status of the limb prior to the
fracture. The decision on the best type of treatment must be made
on an individual basis by the individual's physician.
Fractures
can also occur through the heterotopic bone in a person with FOP.
If the joint is already locked, no further immobilization may be
necessary. Pain medication may be necessary as with any fracture.
Should
milk be provided in the diet if the body already generates too much
bone?
Milk is a good food that contains calcium, which is an important
element for every person's body, even if one has FOP. While it is
well-known that the calcium in milk helps build strong bones, it
is not calcium that makes FOP bones grow. FOP bones grow because
of a genetic signal in the body. Milk will not make the FOP worse,
and it will keep the rest of the body healthy. While the body needs
calcium for the bones to grow and stay healthy, it is vital for
other body functions, too. Calcium allows the nerves to function
properly, the heart to beat, and other important metabolic functions
to occur. The body cannot live without calcium. In addition, people
who have FOP need to fulfill a recommended daily allowance for calcium
because the increased risk of falls necessitates a strong skeleton.
An individual who has FOP does not need to intake more calcium than
anyone else, but should drink one or two glasses of milk each day.
In summary, FOP is not a problem of too much calcium; it is a problem
of too much bone tissue.
What
can be done to prevent skin breakdown over a bony or difficult-to-reach
area?
Skin breakdown and pressure sores are very common and troublesome
problems in people who have FOP, especially in adults. Skin breakdown
can occur from increased pressure over a normal bony area or an
extra piece of bone. If there is excessive pressure on the skin,
breakdown can occur rapidly over the course of hours. Proper padding
over bony prominences or frequent changes in position can prevent
such an occurrence. Special well-padded seating should be considered
in all adults who spend prolonged time in the seated position. Skin
breakdown is much less of a problem in adult patients whose body
position prevents them from sitting.
Another
common cause of skin breakdown is residual moisture and perspiration
in difficult-to-reach areas, such as skin creases and skin folds
near joints or where the arm or leg is fixed against the body wall.
As perspiration occurs and moisture builds up, bacterial and fungal
organisms can develop. Skin infection and breakdown can result.
These problems are often difficult to prevent and even more difficult
to treat.
Surprisingly,
drying agents, such as alcohol and powders, make the problem worse.
The irritated areas should be kept clean with soap and water. Sterile
gauze moistened with saline solution and applied over the irritated
area is better than drying solutions, powders, or antibiotic creams
as it allows the regenerating skin cells to migrate into this area.
If
an individual with FOP has such difficult-to-reach areas, s/he needs
to consult a physician about proper skin care. If skin irritation
or breakdown begins to occur in such a difficult-to-reach area,
consult a physician immediately.
How
should people who have FOP take care of their teeth in order to
minimize dental procedures?
FOP flare-ups involving the jaw can cause the jaw to fuse and,
therefore, make eating and performing dental procedures difficult.
Preventive care is extremely important. The same basic rules about
dental hygiene which apply to everyone are even more important for
people who have FOP. One should brush the teeth after each meal,
avoid sweets between meals, and see the dentist at least twice a
year. It is also a good idea to use fluoride and plaque rinses.
Dental sealants also may be useful. Electric tooth brushes, small
tooth brushes, or water pics can be used to reach difficult areas.
The Braun Interclean electric flossing system may also be helpful.
In cases where the jaw is fused, it may be easier for a dental hygienist
to use a prophylactic jet to clean the teeth. A prophy jet uses
a high pressured stream of water with a cleansing agent (such as
baking soda) to clean the teeth. As a result, it can reach areas
that may otherwise be difficult to reach because of fusion of the
jaw.
In
some cases, dental procedures may lead to FOP flare-ups. Stretching
of the jaw or intramuscular/intraoral injections should be avoided
as these have led to jaw fusion in some people who have FOP. Interligamentary
injections can be used, as these are less likely to cause a flare-up.
Please consult "Minimizing Risk:
Dental Precautions for People with FOP" and "A
Dental Guide for People with FOP" for more information.
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