Lacey Noel Scott
Lacey is 9 years old and was diagnosed with Juvenile Dermatomyositis & Juvenile Rheumatoid Arthritis July 27, 2012.
Every year children die because of the lack of research. Because this disease is an orphan disease and is not popular in the media and affects so few people it is not profitable for research facilities to conduct research or try and find a cure. Myositis patients also need more FDA approved medications for this disease and this can only be accomplished by funding research.
Every year children die because of the lack of research. Because this disease is an orphan disease and is not popular in the media and affects so few people it is not profitable for research facilities to conduct research or try and find a cure. Myositis patients also need more FDA approved medications for this disease and this can only be accomplished by funding research.
What is Juvenile Dermatomyositis
Juvenile Dermatomyositis (JDM), is an autoimmune disease in which the body’s immune system attacks its own cells and tissues.
Weak muscles and skin rash are the primary symptoms of JDM.
Even within these designations, JDM affects every child differently. Some of the more onerous secondary symptoms are calcinosis, vasculitic ulcers and contractures.
Incidence of JDM
JDM is a rare disease and its exact incidence is unknown. Estimates from various studies suggest that between 1 and 3 children per million will develop this disease each year.
Genesis of the Disease
The immune system is a group of cells that normally protects the body from infection and other environmental factors. In an autoimmune disease like JDM, however, the cells get active and they cannot stop. The immune system ends up attacking previously healthy tissues, harming the body instead of protecting it. In JDM, the immune system mistakenly targets muscles, skin and other tissues.
The process that occurs when the immune system attacks healthy tissue is called inflammation. In JDM sufferers, the inflammation primarily occurs in the blood vessels that lie under the skin and in the muscles. This inflammation causes the weak muscles and skin rashes. If blood vessels or muscle cells in other parts of the body are inflamed, other systems of the body can be affected, such as the digestive tract, heart and lungs.
What are the primary symptoms of JDM?
• Weak muscles
The muscles that are affected the most are near the trunk of the body: the stomach, thigh muscles, neck and upper arm muscles. Other muscles, however, can become weak as well. Sometimes there is inflammation in the swallowing tube or esophagus, which makes it difficult for the child to swallow. Other times, there is inflammation in the intestinal tract, which causes bowel difficulties and stomach pain. A child with JDM has great difficulty climbing stairs and just getting up from a sitting position. Walking and running become very challenging and exhausting. Some children will literally roll out of bed, because sitting up and lifting the head becomes too difficult. In addition to weakness, some children with JDM experience pain in their muscles.
• Skin rash
The skin rash in JDM usually occurs on the eyelids, knuckles, elbows, knees and ankles. The rash may appear before, after or at the same time as the muscle weakness. Sometimes the rash is so faint that it is not noticeable.
• Fatigue
With JDM, the child becomes tired easily and can only walk short distances. The child needs to rest often and lacks the energy for normal activities. It becomes difficult for the child to keep up with friends.
What are other possible symptoms?
• Calcinosis
Researchers believe approximately 20% to one-third of the children with JM will develop calcinosis. Calcinosis is the development of small lumps or linear deposits of calcium under the skin or in the muscle. They may feel like rocks under the skin and can range in size from a small pebble to a large softball. They can also form in a sheet-like appearance.
• Vasculitic Ulcers
Vasculitic ulcers are holes in the skin or gastrointestinal tract caused by inflammation of the blood vessels. When they occur in the skin, they look like open sores and can extend deep into the tissues. The ulcers can be very painful and generally are associated with a more severe course of JDM. When the ulcers occur in the gastrointestinal tract, they can harm the digestive organs and result in very serious illness. The warning signs of this condition are severe stomach pain, dark black stools or blood in the stools.
• Contractures
Contractures are shortened muscles that cause a joint to stay in a bent position or have limited movement. Contractures can occur due to inflammation, muscle weakness, or calcinosis crossing over a joint.
• Lipodystrophy
Lipodystrophy is a loss of body fat. In areas affected by lipodystrophy,
the fat cells become damaged, and if widespread and extensive, this can result in problems with storing fat and sugar, resulting in high cholesterol and diabetes.
What is the treatment for JDM?
Although medications can help alleviate the symptoms of JDM, the disease has no known cure. The primary medications used to treat the symptoms of JDM are corticosteroids, immunosuppressants and chemotherapy. These medications themselves can cause severe side effects, making JDM challenging to treat. Many researchers believe that early and aggressive treatment is usually the best predictor of a better outcome of this disease.
High dose oral Prednisone or other corticosteroids often coupled with intravenous corticosteroids (Solumedrol) are usually the first line of treatment for JDM. Since the side effects of corticosteroids can be very troublesome, Methotrexate (a chemotherapy drug administered at much lower doses than used for cancer patients) is usually introduced early to allow for tapering of the corticosteroids.
Second-line treatments include Cyclosporine and Intravenous Immunoglobulin (IVIG). Plaquenil is often used to control the rash.
For patients who do not respond to the first and second line of
treatments, or in patients severely affected by the disease, Rituximab, Cytoxan and CellCept can be added.
Protection from UVA & UVB rays from the Sun is essential. Even limited exposer to the sun can be harmful.
These medications all have their own side effects, but the most common ones for Prednisone are:
• Increased appetite & weight gain • Rounded face
• Mood change • High blood pressure
• Stretch marks • Fragile bones and bone damage
• Cataracts • Slow growth
In addition to medications, physical therapy is generally prescribed to increase strength and flexibility in muscles and joints.
What is the prognosis
There is no cure for JDM, but with advances in early diagnosis and aggressive treatment, the outcome has continued to improve. Some children experience a mild form of the disease and may go into remission. Others follow a more severe and potentially debilitating course that can be life-long. Some JDM patients will have a loss of range of motion. Some will battle an array of serious complications, resulting in the inability to walk, ongoing pain, disfigurement and even death. Whether the course of the disease is mild or severe, JDM is life-changing for all of these children and their families.
Weak muscles and skin rash are the primary symptoms of JDM.
Even within these designations, JDM affects every child differently. Some of the more onerous secondary symptoms are calcinosis, vasculitic ulcers and contractures.
Incidence of JDM
JDM is a rare disease and its exact incidence is unknown. Estimates from various studies suggest that between 1 and 3 children per million will develop this disease each year.
Genesis of the Disease
The immune system is a group of cells that normally protects the body from infection and other environmental factors. In an autoimmune disease like JDM, however, the cells get active and they cannot stop. The immune system ends up attacking previously healthy tissues, harming the body instead of protecting it. In JDM, the immune system mistakenly targets muscles, skin and other tissues.
The process that occurs when the immune system attacks healthy tissue is called inflammation. In JDM sufferers, the inflammation primarily occurs in the blood vessels that lie under the skin and in the muscles. This inflammation causes the weak muscles and skin rashes. If blood vessels or muscle cells in other parts of the body are inflamed, other systems of the body can be affected, such as the digestive tract, heart and lungs.
What are the primary symptoms of JDM?
• Weak muscles
The muscles that are affected the most are near the trunk of the body: the stomach, thigh muscles, neck and upper arm muscles. Other muscles, however, can become weak as well. Sometimes there is inflammation in the swallowing tube or esophagus, which makes it difficult for the child to swallow. Other times, there is inflammation in the intestinal tract, which causes bowel difficulties and stomach pain. A child with JDM has great difficulty climbing stairs and just getting up from a sitting position. Walking and running become very challenging and exhausting. Some children will literally roll out of bed, because sitting up and lifting the head becomes too difficult. In addition to weakness, some children with JDM experience pain in their muscles.
• Skin rash
The skin rash in JDM usually occurs on the eyelids, knuckles, elbows, knees and ankles. The rash may appear before, after or at the same time as the muscle weakness. Sometimes the rash is so faint that it is not noticeable.
• Fatigue
With JDM, the child becomes tired easily and can only walk short distances. The child needs to rest often and lacks the energy for normal activities. It becomes difficult for the child to keep up with friends.
What are other possible symptoms?
• Calcinosis
Researchers believe approximately 20% to one-third of the children with JM will develop calcinosis. Calcinosis is the development of small lumps or linear deposits of calcium under the skin or in the muscle. They may feel like rocks under the skin and can range in size from a small pebble to a large softball. They can also form in a sheet-like appearance.
• Vasculitic Ulcers
Vasculitic ulcers are holes in the skin or gastrointestinal tract caused by inflammation of the blood vessels. When they occur in the skin, they look like open sores and can extend deep into the tissues. The ulcers can be very painful and generally are associated with a more severe course of JDM. When the ulcers occur in the gastrointestinal tract, they can harm the digestive organs and result in very serious illness. The warning signs of this condition are severe stomach pain, dark black stools or blood in the stools.
• Contractures
Contractures are shortened muscles that cause a joint to stay in a bent position or have limited movement. Contractures can occur due to inflammation, muscle weakness, or calcinosis crossing over a joint.
• Lipodystrophy
Lipodystrophy is a loss of body fat. In areas affected by lipodystrophy,
the fat cells become damaged, and if widespread and extensive, this can result in problems with storing fat and sugar, resulting in high cholesterol and diabetes.
What is the treatment for JDM?
Although medications can help alleviate the symptoms of JDM, the disease has no known cure. The primary medications used to treat the symptoms of JDM are corticosteroids, immunosuppressants and chemotherapy. These medications themselves can cause severe side effects, making JDM challenging to treat. Many researchers believe that early and aggressive treatment is usually the best predictor of a better outcome of this disease.
High dose oral Prednisone or other corticosteroids often coupled with intravenous corticosteroids (Solumedrol) are usually the first line of treatment for JDM. Since the side effects of corticosteroids can be very troublesome, Methotrexate (a chemotherapy drug administered at much lower doses than used for cancer patients) is usually introduced early to allow for tapering of the corticosteroids.
Second-line treatments include Cyclosporine and Intravenous Immunoglobulin (IVIG). Plaquenil is often used to control the rash.
For patients who do not respond to the first and second line of
treatments, or in patients severely affected by the disease, Rituximab, Cytoxan and CellCept can be added.
Protection from UVA & UVB rays from the Sun is essential. Even limited exposer to the sun can be harmful.
These medications all have their own side effects, but the most common ones for Prednisone are:
• Increased appetite & weight gain • Rounded face
• Mood change • High blood pressure
• Stretch marks • Fragile bones and bone damage
• Cataracts • Slow growth
In addition to medications, physical therapy is generally prescribed to increase strength and flexibility in muscles and joints.
What is the prognosis
There is no cure for JDM, but with advances in early diagnosis and aggressive treatment, the outcome has continued to improve. Some children experience a mild form of the disease and may go into remission. Others follow a more severe and potentially debilitating course that can be life-long. Some JDM patients will have a loss of range of motion. Some will battle an array of serious complications, resulting in the inability to walk, ongoing pain, disfigurement and even death. Whether the course of the disease is mild or severe, JDM is life-changing for all of these children and their families.