Disease State Overview Chronic inflammatory diseases cause ongoing inflammation in the body, damaging healthy cells of the digestive system, joints, skin, respiratory system, and other organs. Researchers believe that an overactive immune response may play a role in these conditions. More research is required to determine what triggers this disruption of the immune system. Atopic Dermatitis (AD) and Plaque Psoriasis are inflammatory diagnoses that mainly affect the skin. Symptoms may include red, dry patches of skin, burning, itching, swelling, blistering, pain and/or increased sensitivity. In most cases, there are periods of time when symptoms are improved called remission, and times when the disease is worse, called flares. Chronic inflammatory skin conditions like atopic dermatitis and plaque psoriasis are not contagious. Related Symptoms and Health Concerns When left untreated, chronic inflammatory skin conditions like atopic dermatitis and plaque psoriasis can be very impactful to quality of life. A few potential symptoms/experiences include:

• Rashes on the body may break open, weep fluid and bleed when scratched, making skin vulnerable to infection
• Skin may become dry and discolored, and repeated scratching may cause thickening and hardening
• Constant itching or pain may make it difficult to concentrate, taking a toll on your overall quality of life
• Unmanaged symptoms may result in poor sleep, causing daytime lethargy, mental status changes and irritation

Treatment Most chronic inflammatory diseases have no cure. Lifestyle changes and early treatment can help manage and prevent symptoms and flares. Treatment largely depends on the severity of symptoms, as well as other diseases present.  Mild cases of skin inflammation may be treated with topical creams or ointments. In many cases, a gentle daily moisturizer is an important step in maintaining skin health. If symptoms are widespread, or joint pain is present along with skin irritation, more effective treatment may be required. Depending on the diagnosis, treatment options may include immunosuppressive therapies. These are therapies like steroids, methotrexate, cyclosporine, or immunomodulators. Immunomodulators help lower the body’s overactive immune response in the skin and other organs. These are used for moderate to severe diseases. Suppressing inflammation offers relief from common symptoms. It also allows the skin to heal. Specialty medications used to treat chronic inflammatory dermatologic conditions include both oral medications and injectable options. The decision as to what option is right for you will be based upon your diagnosis, history and physical exam, past therapies, and disease severity.  Treatment Goals A few primary goals of treating atopic dermatitis are:

• Improvement in overall symptoms
• Improvement in quality of life
• Reducing need for unplanned medical visits

Strategies to Achieve Treatment Goals

• Adhere to prescribed therapy
• Keep appointments, and follow-up with physician
• Report side effects or unmanaged symptoms to your pharmacist, or healthcare team right away
• Avoid known triggers, or irritants, such as: harsh soaps/detergents, perfumes, cosmetics, cigarette smoke, heavy alcohol consumption, and food allergies
• Use appropriate skin care, such as regular use of hypoallergenic moisturizers for dry skin
• Practice stress management and relaxation techniques
• Eat a healthy diet

Additional Resources

• Download your Psoriasis guide here.
• Atopic Dermatitis | National Eczema Association | https://nationaleczema.org/Eczema/types-of-Eczema/atopic-dermatitis/
• Atopic Dermatitis: Diagnosis, Treatment, and Steps to Take | National Institute of Arthritis and Musculoskeletal and Skin Diseases | https://www.niams.nih.gov/health-topics/atopic-dermatitis/diagnosis-treatment-and-steps-to-take
• Psoriasis | Mayo Clinic | https://www.mayoclinic.org/diseases-conditions/psoriasis/symptoms-causes/syc-20355840

Clinical Disease Overview

Lysosomal storage diseases are genetic conditions that can cause a buildup of harmful materials in your body. These conditions cause people to lack certain important proteins called enzymes. Others lack certain substances that help these enzymes work well. This lack of functional enzymes prevents the body from breaking down things like sugars, fats, and other substances. When these build up in the body, they can cause harmful effects.

At present, there are more than 4 dozen different identified lysosomal storage diseases. In general, these diseases fall into one of three categories based on the type of enzyme that is missing or not working. These groups are:

• Lipidoses
  • These occur when your body doesn’t have the enzymes to break down fats. An example of a condition in this group is Wolman disease.
• Mucopolysaccharidoses
  • These types occur when your body doesn’t have the enzyme needed to break down large sugar molecules known as glycosaminoglycans. An example of a condition in this group is Hunter syndrome.
• Sphingolipidoses
  • This type of lysosomal storage disease comes from your body lacking the enzymes needed to break down a unique fatty substance called a sphingolipid. A few conditions that fall into this category include:
    • Fabry disease
    • Gaucher disease
    • Krabbe disease

Symptoms of Lysosomal Storage Disease

Symptoms vary greatly based on the type of disease present and the specific cells and organs affected. There are, however, several common symptoms across these disease types with a few including:

• Changes in your muscle
• Delayed development
• Unique facial features like a flat nose, a protruding forehead, or even very large lips
• Visceromegaly (large organs in your abdomen)

Treatment

Due to the rare nature of lysosomal storage disorders, treatment typically occurs in unique centers that specialize in this type of disease. A few types of treatment for these diseases include:

• Stem cell transplant
  • Used to help provide stem cells from donors to help produce the missing enzyme
• Substrate reduction therapies
  • These therapies help reduce the buildup of substances that aren’t being broken down by enzymes
• Enzyme replacement
  • This involves intravenous (IV) infusion of direct enzymes to replace the missing or dysfunctional ones
• Gene therapy
  • These treatments can correct the underlying genetic cause of various lysosomal storage diseases by providing a functional gene copy of the defective one/one’s present. Many are still in development.

Treatment Goals

The primary goals in the treatment of lysosomal storage diseases are to return cell and organ function to normal, improve/manage the symptoms of disease, and minimize the buildup of various molecules because of the disease. These goals help to lower the risks of organ damage and ultimately improve the quality of life of anyone living with a lysosomal storage disease.

Strategies to Achieve Treatment Goals

• Adhere to prescribed treatment plan
• Maintain regular follow-up with your healthcare team
• Report unmanaged/new symptoms or side effects to your healthcare team right away

Resources

• Lysosomal Disease Network. https://ldn.rarediseasesnetwork.org/patient-advocacy-groups
• National Organization for Rare Disorders. https://rarediseases.org/organizations/hide-seek-foundation-for-lysosomal-disease-research/

Sources

• Lysosomal storage disease overview. NIH. Accessed 12/17/2025. https://pmc.ncbi.nlm.nih.gov/articles/PMC6331358/ 

Disease State Overview

Chronic inflammatory diseases cause inflammation in the body. This may damage healthy cells of the digestive and respiratory system, joints, skin, and other organs. Researchers believe that a hyperactive immune response may play a role in these conditions. More research is required to determine what triggers this disruption of the immune system.

Crohn’s Disease (CD) and Ulcerative Colitis (UC) are inflammatory diagnoses that mainly affect the intestines and bowels. Symptoms include poor absorption of nutrients, diarrhea, rectal bleeding, abdominal pain and cramps, urgency, and/or constipation.  Diet and stress may intensify these symptoms, but do not cause the disease.  In most cases, there are periods of time when the disease is worse, called flares, and there are periods when symptoms improve, called remissions.

Related Symptoms and Health Concerns

If left untreated,

• Constipation may lead to bowel obstruction
• Severe diarrhea may lead to dehydration
• Fissures may develop, which cause pain and bleeding
• Body wide issues may develop, including malnutrition, swollen and painful joints, swelling in the extremities, sores and rashes on the skin, osteoporosis, and kidney stones
• Unmanaged symptoms may result in loss of appetite, weight loss, delayed growth and development, and poor sleep, causing daytime lethargy, mental status changes and irritation

Treatment

Most chronic inflammatory diseases have no cure. Lifestyle changes and early treatment may help manage and prevent issues. Treatment largely depends on the severity of symptoms.

Mild cases of inflammatory GI disease are treated with anti-inflammatory medications. These medications include corticosteroids and amino salicylates. If symptoms are widespread, stronger, more direct medications are used.

Depending on the diagnosis, treatment options may also include immunosuppressive therapies or immunomodulators.  Immunomodulators are therapies that help target key processes that change the immune response and are used to treat moderate to severe disease. This can help lower specific parts of a hyperactive immune system and greatly improve disease control and lower inflammation. Reducing inflammation offers relief from common symptoms and allows intestinal tissues to heal.

Treatment Goals

The primary goals of treating inflammatory GI conditions are to:

• Decrease bleeding and urgency
• Lower inflammation
• Lower risk of cancer
• Maintain remission
• Prevent flares
• Prevent hospitalizations and/or surgery
• Reduce or eliminate symptoms of disease
• Reduce risk of long-term problems caused by disease

Strategies to Achieve Treatment Goals

• Adhere to prescribed therapy
• Keep appointments, and follow-up with physician
• Report side effects or unmanaged symptoms to your pharmacist, or healthcare team
• Avoid known disease triggers
• Eat a healthy diet, and avoid certain foods and beverages that may aggravate symptoms

Resources

• https://www.crohnscolitisfoundation.org/what-is-crohns-disease
• https://www.mayoclinic.org/diseases-conditions/crohns-disease/symptoms-causes/syc-20353304

Disease State Overview Growth Hormone Deficiency (GHD) is a rare health condition caused when the pituitary gland doesn’t make enough growth hormone. Growth hormone is essential for healthy growth in children and also plays an important role in metabolism, bone strength, and overall well-being. GHD can occur in both children and adults. Childhood-onset GHD is often present at birth and may be due to genetic mutations that affect the development or function of the pituitary gland. Adult-onset GHD typically develops later in life and may result from brain tumors, autoimmuneconditions, or headinjuries. Related Symptoms and Health Concerns In children, symptoms often relate to height and physical maturity. Common signs include:

• Slow growth or shorter height than expected
• Delayed or absent puberty
• Poor nail or hair growth
• Higher-pitched voice
• Low blood sugar
• Fatigue or low energy
• Increased body fat

If left untreated, children may not reach their expected adult height and may experience delayed development or bone weakness.

1. Adults with GHD usually do not experience height-related symptoms. More common symptoms in adults include:

• Fatigue and reduced stamina
• Increased body fat, especially around the waist
• Decreased muscle mass and strength
• Higher risk of bone fractures due to low bone density.
• Reduced exercise capacity
• Changes in cholesterol or blood sugar levels
• Emotional changes, such as low mood or irritability
• Poor concentration or memory concerns.

2. If left untreated, GHD can affect heart health, metabolic balance and overall quality of life.

Treatment Children with GHDmay receive injectable synthetic human growth hormone through a specialty pharmacy.  These injectable medications are administered just under the skin , by the childor caregiver at home. Formulations may require the patient to inject daily, however newer optionsallow for once-weekly injections to improve adherence and convenience. Children are usually able to stop treatment once they finish growing.  It may take 3-6 months to see noticeable improvement in growth after starting therapy. Adults with GHD may also receive growth hormone therapy to improve energy, metabolism, bone density, and overall quality of life. Treatment Goals The main goals of treating GHD are to:Replenish the lack of growth hormones

• Improve quality of life
• Help children obtain normal or near-normal adult height
• Restore healthymetabolism
• Increase bone strength
• Minimize the likelihood of cardiovascular events

Strategies to Achieve Treatment Goals

• Adhere to prescribed therapy
• Keep appointments, and follow-up with physician
• Report side effects or unmanaged symptoms to your pharmacist, or healthcare team
• Avoid cigarette smoke, heavy alcohol consumption, and recreational drug use
• Exercise regularly to promote healthy lifestyle
• Eat a balanced, nutritional diet

Resources

• Download your Growth Hormone Deficiency guide here .
• Boston Children’s Hospital – Growth Hormone Deficiency
• Johns Hopkins Medicine – Growth Hormone Deficiency
• Endocrine Society – Growth Hormone Deficiency
• National Organization for Rare Disorders – GHD
• Barrow Neurological Institute – Adult Growth Hormone Deficiency

Disease State Overview

Hemophilia is a bleeding disorder that prevents blood from clotting appropriately. The most common forms of hemophilia are inherited through the X-chromosome, and as a result affects mostly males. Hemophilia occurs in approximately 1 in 5,000 male births in the U.S. and approximately 400 babies are born with hemophilia A each year. 400,000 people worldwide are living with hemophilia and about 30,000 are living with it in the United States alone. All races and economic groups are affected equally. People with hemophilia who have access to factor replacement therapy have a more normal life expectancy.

Hemophilia is treated differently depending on what protein is missing in the blood. There are 4 types that are classified as follows:

• Hemophilia A – Also called classic hemophilia, it is 4 times more common than hemophilia B, and it occurs when factor VIII levels are deficient.
• Hemophilia B – Also called Christmas disease, occurs when factor IX levels are deficient.
• Hemophilia C – occurs when factor XI levels are deficient
• Acquired hemophilia – A person can develop hemophilia as a result of their body’s immune system attacking clotting factors. An underlying cause can be identified in approximately 50% of patients.

von Willebrand Disease (VWD)

VWD is the most common inherited bleeding disorder, affecting up to 1% of the U.S. population. It occurs when the body has low levels or defective von Willebrand factor (VWF), a protein essential for blood clotting. VWF helps platelets stick together and helps your body form a stable clot. People with VWD have difficulty forming clots and tend to take longer to stop bleeding.

There are three inherited types and one acquired form:

• Type 1 VWD: Reduced levels of VWF (20%-50% of normal), causing mild symptoms. A subtype, Type 1C, leads to faster clearance of VWF, prolonging bleeding.
• Type 2 VWD: Normal VWF levels, but the factor does not function properly. This type has four subtypes (2A, 2B, 2M, 2N) with mild to moderate symptoms.
• Type 3 VWD: Very low or no VWF, sometimes with low FVIII, leading to severe bleeding, including spontaneous bleeds into joints and muscles.
• Acquired VWD: Develops in adults due to immune diseases (e.g., lupus), heart disease, cancer, or certain medications.

Related Symptoms and Health Concerns

In general, people with hemophilia or VWD bleed longer than those without. A person with hemophilia can bleed inside or outside of the body. The most common types of internal bleeds are in the joints and muscles.

Other symptoms include:

• Nose bleeds
• Prolonged bleeding from minor cuts
• Bleeding that stops and resumes after stopping for only a short time
• Blood in the urine
• Blood in the stool
• Large bruises
• Easy bruising (unexplained bruising)
• Excessive bleeding with dental work or tooth extraction
• Heavy periods and/or periods lasting more than 7 days

Treatment

While there’s no cure for hemophilia or VWD, most people with these diseases can lead normal lives. Treatment will depend on the type and severity of the conditions. Factor replacement products are common disease treatments for patients with bleeding disorders. Each case is treated uniquely with a product and strategy to best meet the patient’s needs. For example, some patients only use a factor product when they are experiencing bleeding, while other patients may take them proactively to help prevent a bleed. A few non-factor therapies used in bleeding conditions include the following: 

• Desmopressin: used for mild bleeding in the joints, muscle, nose, mouth or proactively before and after a planned surgery
• Aminocaproic acid: helps to treat mouth and mucosal bleeds
• Tranexamic acid: approved for treatment of heavy menstrual bleeding
• Hormone therapies: can be effective in reducing and treating heavy menstrual bleeding
• Gene therapy: used to treat the underlying genetic condition that causes hemophilia. The provider at the hemophilia treatment center will help determine eligibility and discuss the risk and benefits of therapy.

For minor wounds, if you or your loved one experiences a small cut or scrape, using pressure and a bandage will generally take care of the bleeding. For small areas of bleeding beneath the skin, use an ice pack. Ice pops can be used to slow down minor bleeding in the mouth.

Treatment Goals

The primary goals of treatment are to prevent bleeding, manage active bleeding, and prevent long term damage to provide a near-normal life. This is achieved in many patients by: 

• Raising factor levels
• Quickly treat active bleeds
• Injury prevention and self-care measures
• Appropriate training and education for administration of treatment
• Maintaining optimal therapy adherence
• Preventing, minimizing, and managing side effects

Strategies to Achieve Treatment Goals

• Adhere to drug therapy
• Monitoring and follow-up with care team
• Prevent, minimize, and manage side effects

Resources

• Hemophilia Federation of America
• Rare Diseases – Acquired Hemophilia

Sources:

• National Bleeding Disorders Foundation (NBDF) Medical and Scientific Advisory Council (MASAC) 2024 guidelines in Document 290 – MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System: https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-290-masac-recommendations-concerning-products-licensed-for-the-treatment-of-hemophilia-and-selected-disorders-of-the-coagulation-system
• World Federation of Hemophilia (WFH) 2020 Guidelines for the Management of Hemophilia: https://www1.wfh.org/publications/files/pdf-1863.pdf 
• NBDF MASAC Document 290 – MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System: https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-290-masac-recommendations-concerning-products-licensed-for-the-treatment-of-hemophilia-and-selected-disorders-of-the-coagulation-system
• NBDF MASAC Document 266 – MASAC Recommendations Regarding the Treatment of von Willebrand Disease: https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-266-masac-recommendations-regarding-the-treatment-of-von-willebrand-disease 

Disease State Overview Hepatitis C is a liver disease caused by the hepatitis C virus. Many people with hepatitis C have no symptoms and don’t know they are infected. It is estimated that around 2.4 million people currently have chronic hepatitis C in the US. If left untreated, hepatitis C can lead to serious liver problems such as cirrhosis and liver cancer. Hepatitis C is spread through direct contact with infected blood. This transmission could occur through needle sharing, drug injection, healthcare needle sticks, sexual contact, or child birth to name a few. Hepatitis C has two distinct phases, acute and chronic. 

• Acute hepatitis C occurs when a person is first infected with the virus. Symptoms can vary from person to person. Some may experience things like fatigue, dark urine, nausea, or abdominal pain while others have no symptoms at all. Less than half of those infected clear the virus on their own in the first 6 months without treatment. 
• Chronic hepatitis C is the long term liver infection phase of hepatitis C. In this stage, the disease is often silent causing no symptoms. Over time however, liver damage can occur as described previously.

Testing and Treatment Hepatitis C is easily tested for using an initial antibody test. This test detects the past exposure to the hepatitis C virus. All adults 18 years of age and older are recommended to have this test complete at least once. In addition, anyone with a past history of injection drug use, known exposure to hepatitis C, hemodialysis, or abnormal liver test results should also have this initial antibody test conducted. If positive, a follow up test to detect hepatitis C virus RNA is conducted to confirm positivity. Thankfully, hepatitis C infection can be treated and cured in nearly 95% of cases. There are several different options for treatment using various direct-acting-antiviral therapies. Treatments range from typically 8-12 weeks often resulting in cure. Treatment Goals The primary treatment goal of hepatitis C is a sustained virological response. This means the virus has not been detected in your blood 12 weeks after completing your direct-acting antiviral treatment. This helps prevent some of the long term negative effects of hepatitis C such as liver damage, liver scarring, and liver cancer ultimately improving length of life. Once cure has been achieved, this also stops the transmission of the virus through blood. Strategies to Achieve Treatment Goals

• Adherence to direct-acting antiviral therapy
• Monitoring and follow-up with care team

Resources

• American Liver Foundation Support for Patients with Hepatitis C. https://liverfoundation.org/liver-diseases/viral-hepatitis/hepatitis-c/support-for-patients-with-hepatitis-c/
• HepC.com. https://www.hepc.com/how-hep-c-transmitted

Sources

• Hepatitis C treatment guidelines. Accessed 12/15/2025. https://www.hcvguidelines.org
• Clinical Overview of Hepatitis C. Accessed 12/15/2025. https://www.cdc.gov/hepatitis-c/hcp/clinical-overview/index.html

Disease State Overview Human immunodeficiency virus (HIV) is a virus that attacks parts of the body’s immune system. It is spread by coming in contact with HIV contaminated body fluids like: blood, semen, rectal fluids, or vaginal fluids. Without effective treatment, the virus can deplete the immune system causing acquired immunodeficiency syndrome (AIDS). AIDS makes the body’s immune system much weaker making it easier to get infections even from the body’s natural bacteria. At this time, there is no cure to HIV. Prevention HIV infection can be prevented in many ways. A few include:

• • Practicing safe sex (using a condom)
  • Never sharing needles
  • HIV testing to know if positive, effective treatment prevents transmission
  • Pre-Exposure-Prophylaxis (PrEP)

PrEP is a highly effective prevention method where people at high-risk of getting HIV infection take medication to prevent the virus from infecting them if they are exposed. PrEP has been shown to lower the risk of infection from exposure by up to 99%. PrEP is available as both a once daily medication or injections every 1-6 months. Protection begins within the first 21 days of therapy. Related Symptoms and Health Concerns Symptoms of HIV vary from person to person and based upon if the person is in the acute phase or chronic phase of infection. Typically, the acute phase symptoms first begin within 2-4 weeks of virus exposure. During this time, a flu-like illness can appear with symptoms including:

• Fever and chills
• Skin rash
• Stomach issues like diarrhea and nausea
• Swollen lymph nodes

Chronic phase of the infection can lead to more serious and noticeable side effects as the immune system is depleted. As this phase progresses, it can eventually lead to AIDS. A few serious effects include:

• Chronic diarrhea
• Frequent sometimes serious infections
• Night sweats
• Persistent fatigue
• Skin issues
• Unexplained weight loss

Treatment The treatment of HIV is known as antiretroviral therapy (ART). ART combines multiple medications that work in different ways to treat HIV and stop its growth. When undergoing treatment, the amount of virus in your blood will be checked known as “viral load”. Both injectable and oral medication options are available for treatment. Treatment Goals The main goals of HIV treatment are to obtain undetectable viral levels in the body. This undetectable viral level will help restore immune function protecting health and length of life, reduce the number HIV associated illness and complications, and prevent transmission to others. Individuals on successful ART treatment can lead near normal lives. Strategies to Achieve Treatment Goals

• Adherence to ART treatment
• Ongoing monitoring and follow-up with your care team

Resources

• AIDS Healthcare Foundation. https://www.aidshealth.org/
• National Alliance of State and Territorial AIDS Directors PrEP and PEP Assistance Programs: https://nastad.org/prepcost-resources/prep-assistance-programs

Sources

• About HIV. Accessed 12/16/2025. https://www.cdc.gov/hiv/about/index.html
• HIV treatment guidelines. Accessed 12/16/25. https://clinicalinfo.hiv.gov/en/guidelines

Disease State Overview

Multiple sclerosis (MS) is a condition where the immune system mistakenly attacks the myelin sheath, the protective covering around the nerves in your brain and spinal cord. When this covering is damaged, the signals slow down or get disrupted, which leads to MS symptoms.

People with MS may experience one of four main types of the disease.  Each type can range from mild to severe and affects people differently.

1. Clinically isolated syndrome (CIS). CIS is the first episode of MS-like symptoms, caused by inflammation and loss of myelin and lasting at least 24 hours.  A person may have one symptom or several, and symptoms may fully or partially go away.  Not everyone who has CIS will develop MS.  An MRI can help predict the chance of developing MS.  If lesions are present, there is a 60-80% chance of having a second episode and being diagnosed with MS.  If no lesions are seen, the chance of developing MS drops to about 20%.
2. Relapsing-remitting MS (RRMS). RRMS is the most common form, with about 85% of people diagnosed with this type.  RRMS is characterized by clearly defined relapses (also called flare-ups or exacerbations) when symptoms suddenly worsen and period of remission, when symptoms improve or disappear and the disease does not get worse.
3. Secondary progressive MS (SPMS). Some people who start with RRMS will eventually transition to SPMS.  In this phase, symptoms and disability gradually worsen over time, with or without occasional flare-ups or periods of stability.  Before modern treatments were available about 50% of people with RRMS developed SPMS within 10 years.  Today, medications may help delay or reduce progression.
4. Primary-Progressive MS (PPMS). About 15% of people are diagnosed with PPMS. This type involves steady worsening of symptoms from the start, without early relapses.

Related Symptoms and Health Concerns

MS can cause many symptoms, and every person’s experience is different.  Symptoms may vary in intensity and can change over time.

Common symptoms:

• Fatigue
• Numbness or tingling 
• Balance or walking problems
• Bladder or bowel changes
• Vision problems
• Dizziness or vertigo
• Sexual dysfunction
• Emotional changes
• Pain and itching
• Depression
• Muscle stiffness or spasms (spasticity)
• Weakness
• Memory or cognitive problems
• “MS hug” (tight chest/torso sensation)

Less common symptoms include speech problems, swallowing issues, tremors, hearing loss, seizures, and breathing difficulties.

If you experience new or worsening symptoms, it’s important to contact your healthcare provider for guidance.

Treatment

There is no cure, but many treatments can help you live well with MS.  Care is usually led by a neurologist and may include:

• Modifying the disease course through disease-modifying medications.
• Treating relapses (or exacerbations)
• Managing symptoms with medication and/or rehabilitation strategies
• Improving or maintaining function through rehabilitation
• Providing emotional support with mental health professionals

Treatment Goals

The primary goals of MS treatment are:

• Modify or reduce relapses
• Delay progression and help prevent long-term disability
• Treating relapses
• Managing symptoms
• Increasing survival and long-term health
• Maintain good adherence to therapy
• Preventing, minimizing, and managing symptoms

Strategies to Achieve Treatment Goals

• Adhere to drug therapy
• Monitoring and follow-up
• Prevent and treat relapses
• Minimize disruption to daily life due to progression of MS
• Prevent, minimize, and manage side effects

Resources and References:

• National MS Society https://www.nationalmssociety.org/
• https://www.ninds.nih.gov/health-information/disorders/multiple-sclerosis

Background:

Cancer is a disease where some cells in the body begin to grow uncontrollably and even spread to other parts of the body. It can start nearly anywhere, and the risk of development tends to increase with age. At present, there are an estimated 18 million people living with cancer in the United States. Cancer is a general term given to more than 100 different types of disease. It is often grouped into what is known as solid tumor, blood cancers, or mixed type.

There are several different classifications of cancer, each describing the starting location or cell type where the disease begins:

• Carcinomas: These types of cancer start in the skin, or tissues that line organs. A few examples of cancers that follow this classification are breast, colon, and lung.
• Leukemias: This type of cancer involves the blood-forming components of the body. These cancers can grow large numbers of abnormal cells quickly.
• Lymphomas: These cancer types begin in the immune system cells and lymphatic system.
• Myelomas: Begin in a white blood cell known as a plasma cell. 
• Sarcomas: These cancer types begin in the bone, connective tissues, fat, and muscle.

In addition to this classification, it’s also important to be familiar with a few general terms used to describe things in cancer care. This is not a comprehensive list, however a few key terms to understand include:

• Benign: noncancerous
• Biopsy: to take a tissue sample
• Malignant: cancerous
• Metastasis: spread of cancer from the starting site to other parts of the body
• Neoplasm: abnormal growth/tumor
• Partial remission: significant improvement in disease, but still detectable
• Prognosis: the disease outlook or likely outcome
• Remission: minimal to no cancer symptoms/no detectable disease
• Recurrence: cancer has returned

How does cancer start?

Cancer is a disease of genetics. Meaning, it is caused by changes to genes that control the way cell’s function, grow, and divide. Each cell in the body has a process for how it should grow and divide correctly. When cancer starts, it is usually because of a mistake during the cell division process, of damage to DNA from exposures in the environment (chemicals, UV rays, etc), or from inherited genetic changes from parents that cause a breakdown in the correct process. Most of the time, the body finds and eliminates cells with damaged DNA before a cancerous process can even begin. However, the body’s ability to do this lowers as we get older. 

Symptoms:

The signs and symptoms of cancer are vastly different from person to person and dependent on the location and type of cancer. A few symptoms possible with cancer include things like:

• Changes in bowel or bladder patterns
• Difficulty swallowing
• Fatigue
• Lump or area of hardness felt under the skin
• Trouble breathing
• Unexplainable fevers, bruising, or weight change

Most people with cancer will lose weight at some point. When you lose weight for no known reason, it’s called unexplained weight loss. An unexplained weight loss of 10 pounds or more may be the first sign of cancer.

Fever is very common with cancer, but it more often happens after cancer has spread from where it started. Almost all patients with cancer will have fever at some time, especially if the cancer or its treatment affects the immune system. Less often, fever may be an early sign of cancer, such as blood cancers like leukemia or lymphoma.

Pain may also be an early symptom with some cancers like bone cancers or testicular cancer. Most often, pain due to cancer means it has already spread (metastasized) from where it started.

Along with skin cancers, some other cancers can cause skin changes that can be seen. These signs and symptoms include:

• Darker looking skin 
• Excessive hair growth
• Itching
• Reddened skin
• Yellowish skin and eyes

Diagnosis:

• Physical exam. Your doctor may feel areas of your body for lumps that may indicate a tumor. During a physical exam he or she may look for abnormalities, such as changes in skin color or enlargement of an organ that may indicate the presence of cancer.
• Laboratory tests, such as urine and blood tests, may help your doctor identify abnormalities that can be caused by cancer. For instance, in people with leukemia, a common blood test called complete blood count (CBC) may reveal an unusual number of white blood cells.
• Imaging tests. Imaging tests allow your doctor to examine your bones and internal organs. Imaging tests used in diagnosing cancer may include computerized tomography (CT) scan, bone scan, magnetic resonance imaging (MRI), ultrasound and X-ray, among others.
• Biopsy. During a biopsy, your doctor collects a sample of cells for testing in the laboratory. In most cases, a biopsy is the only way to definitively diagnose cancer. 
• Genetic testing. During the process of diagnosis many patients undergo genetic testing to identify genetic causes of illness.

Treatment:

Cancer treatment is highly specific to the needs of every patient. The decisions regarding treatment take into account the cancer type, staging, and patient specific needs and health. A few examples of different treatment options include but are not limited to:

• Surgery. Surgery can be used to diagnose, treat, or even help prevent cancer in some cases. 
• Chemotherapy. Chemotherapy (chemo) is the use of medicines or drugs to kill fast-growing cells in cancer. It is a systemic treatment meaning, the therapy travels throughout the body.
• Radiation Therapy. Radiation Therapy uses high-energy particles or waves to destroy or damage cancer cells. It is one of the most common treatments for cancer, either by itself or along with other forms of treatment.
• Targeted Therapy. Targeted therapy is the process of using drugs that target specific changes or mutations in cancer cells that help them grow. These treatments often are more effective in cancers that display the targeted mutation with some providing less side effects compared to traditional chemotherapy. 
• Immunotherapy. Immunotherapy works by helping your own immune system recognize and fight cancer.
• Stem Cell Transplant (Peripheral Blood, Bone Marrow, and Cord Blood Transplants)
• Hormone therapy. Certain cancer types grow more quickly when exposed to your body’s hormones. Some treatments work by blocking or turning off these hormones to stop the cancer cells from growing.

Goals of cancer treatment

Setting appropriate goals and expectations in cancer care is one of the most important steps in the treatment process. It helps provide a roadmap for where care is heading. Goals of treatment are designed together with the doctor by weighing the risks and benefits for cancer treatments and likelihood for success. A few potential goals of your cancer treatment may include:

• Adherence to treatment regimen
• Cure disease
• Delay progression of disease
• Improve quality of life
• Manage cancer symptoms
• Prevent and manage side effects to treatment

Supportive care

Appropriate support during the cancer journey is vital to long term success and wellbeing. Cancer care can be an overwhelming, and long journey leaving many in poor health due to side effects and cancer symptoms, and often depressed. During treatment it is likely that you will require some assistance for:

• Physical symptoms
• Emotional support
• Social support

Key supportive care services include things like pain management, nutrition support, advanced care planning, and exercise programs. The overarching goals of supportive care are to enhance your quality of life, improve treatment tolerance, and to provide holistic care during your journey.

Additional Resources

• Patient Resources. NCCN | https://www.nccn.org/patientresources/patient-resources
• Online Support Groups | https://www.dailystrength.org/

Sources

1. Clinical Care Guidelines. NCCN. Accessed 12/09/25. https://www.nccn.org/
2. Guidelines. ASCO. https://www.asco.org/practice-patients/guidelines

Rheumatology – Chronic Inflammatory Disease 

Ankylosing Spondylitis (AS), Gout, Psoriatic Arthritis (PsA), Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE), Systemic Juvenile Idiopathic Arthritis (sJIA)

Disease State Overview

Chronic inflammatory diseases cause long-lasting inflammation in the body. This inflammation can damage healthy cells and tissues over time.  Many areas of the body can be affected including, but not limited to, the digestive system, joints, skin, and respiratory system. Researchers believe that these conditions may be related to an overactive immune system, but more research is needed to understand what triggers this disruption of the immune system. 

The term rheumatism is used to describe many conditions that cause inflammation in the joints, muscles, and connective tissues.  It includes a wide range of diseases such as ankylosing spondylitis (AS), psoriatic arthritis (PsA), rheumatoid arthritis (RA), and systemic lupus erythematosus (SLE).

The most common symptom of rheumatologic inflammatory diseases is joint pain. Joints may also feel warm, tender, stiff, or swollen. Other symptoms may include feeling unusually tired, having low energy, occasional fevers, loss of appetite, skin rashes and vision changes.  In most cases, these conditions can be cyclical.  There are periods where symptoms are worse, called flares.  When symptoms ease and feel better, it is called remission.

Related Symptoms and Health Concerns

If left untreated,

• Inflammation can lead to permanent joint damage or deformity
• physical disability, making everyday activities and work more difficult
• negative impact on mental and social well-being
• patients may be at an increased risk of heart, lung and kidney problems
• patients may be at an increased risk of uveitis, which can cause eye pain, sensitivy to light, and blurry vision

Treatment

Most chronic inflammatory diseases have no cure. Lifestyle changes and early treatment may help manage and prevent complications. Treatment largely depends on the severity of symptoms. Research shows that people with rheumatoid arthritis who are diagnosed early and treated aggressively do better over the long-term.

Topical creams or ointments, as well as nonsteroidal anti-inflammatory drugs (NSAIDs), may help with pain and reduce inflammation. If symptoms are widespread, more aggressive treatment may be required. Depending on the diagnosis, treatment options may include immunosuppressive therapies, such as steroids, disease-modifying antirheumatic drugs (DMARDs), or immunomodulators.  Immunomodulators may help decrease the body’s hyperactive immune response in the skin, joints and other organs.  Suppressing inflammation not only offers relief from common symptoms, but it also allows the tissues to heal.

Specialty medications used to treat chronic inflammatory rheumatologic conditions include, but are not limited to:

• Oral immunomodulators
• Injectable immunomodulator therapies

Treatment Goals

The primary goals of treating inflammatory rheumatic conditions are to:

• Reduce or eliminate symptomatic disease activity
• Manage exacerbating conditions, such as depression, anxiety, or sleep loss due to disease symptoms
• Prevent flares (maintain remission)
• Reduce risk of long-term complications, such as bone deformities
• Maintain as normal a lifestyle as possible.

Strategies to Achieve Treatment Goals

• Adhere to therapy
• Keep appointments, and follow-up with physician
• Report side effects or unmanaged symptoms to your pharmacist, or healthcare team
• Avoid known triggers, such as cigarette smoke, heavy alcohol consumption, and food sensitivities
• Practice stress management and relaxation techniques
• Participate in regular exercise and physical activity programs
• Eat a healthy diet, and maintain a healthy weight

Sources

• https://www.cdc.gov/arthritis/rheumatoid-arthritis/
• https://www.niams.nih.gov/health-topics/rheumatoid-arthritis
• https://www.niams.nih.gov/health-topics/ankylosing-spondylitis
• https://www.hopkinsmedicine.org/health/conditions-and-diseases/ankylosing-spondylitis
• https://www.niams.nih.gov/health-topics/psoriatic-arthritis
• https://www.niams.nih.gov/health-topics/juvenile-arthritis
• https://www.cdc.gov/lupus/about/index.html

Disease State Overview

Asthma is a long-term condition that affects the airways in your lungs.  These airways can become inflamed and narrowed.  This makes it harder to move air in and out of the lungs.  It can affect people of all ages, although it often begins in childhood.  Certain things – called triggers – can worsen asthma symptoms.  When symptoms suddenly get worse, you may have an asthma attack.

There are several types of asthma, and each may require different treatment or management strategies.  These include:

• Allergic asthma
• Aspirin-induced asthma
• Cough-variant asthma
• Exercise-induced asthma
• Nighttime (or nocturnal) asthma
• Steroid-resistant asthma
• Occupational asthma (related to workplace exposure)

Experts have started to recognize the impact of inflammation in asthma.  Four biological pathways of inflammation have been identified: eosinophilic, neutrophilic, mixed eosinophilic and neutrophilic, and non-inflammatory.  

When asthma does not improve with long-term controller medications, it may be a sign of severe asthma.  There are 2 main types of severe asthma: Type-2 inflammation and Non-Type-2 inflammation.  Those patients with Type-2 severe asthma respond well to biologic medications.  These treatments target specific parts of the immune system and may be obtained at your specialty pharmacy.

Related Health Symptoms and Health Concerns

Asthma can cause a variety of breathing problems.  Common symptoms include:

• Wheezing (a whistling sound when you breathe)
• Coughing
• Shortness of breath
• A feeling of tightness in your chest

For some people, an asthma attack may begin with a cough that doesn’t go away.  Your chest may start to feel tight — some people describe it as having the air “sucked out” of them.  You may also begin to wheeze or feel lightheaded. 

Having and following your asthma action plan is very important.  It helps to know what steps to take when symptoms start, when to use your rescue medication, and when to seek emergency care. 

Call 9-1-1 right away if you are struggling to breathe, your symptoms are getting worse quickly or your rescue inhaler is not helping.  

Treatment

While there is no cure for asthma, there are many treatments that can help you manage your symptoms and live a healthy, active life.  Treatment usually includes monitoring your breathing, avoiding triggers, and using medications to both prevent and treat symptoms.

Most people use a combination of medications to keep their asthma under control. You may have a quick relief (rescue) medication to help stop symptoms and a long-term control medication to help prevent symptoms from happening in the first place.

The types of medications include:

• Bronchodilators
• Corticosteroids (steroids)
• Anticholinergies
• Biologics
• Antibiotics

Treatment Goals

The goals of treating asthma include:

• Improved breathing
• Reduce airway inflammation
• Reduce asthma symptoms
• Improve physical function and quality of life
• Maintaining optimal therapy adherence
• Preventing, minimizing, and managing side effects of therapy

Strategies to Achieve Treatment Goals

• Have an asthma action plan
• Adherence to therapy
• Monitoring and follow-up with physician
• Know and reduce asthma triggers
• Reducing the risk of additional lung damage through smoking cessation
• Immunization against flu and pneumonia
• Appropriate management of other underlying diseases such as allergies, high blood pressure, anxiety, etc.

Additional resources

• https://www.lung.org/lung-health-diseases/lung-disease-lookup/asthma/learn-about-asthma/types/severe-asthma
• https://www.nhlbi.nih.gov/health/asthma