Updated October 2016

Hemophilia Caring for Patients in the Home Care Environment

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This booklet is to be used as a guide to give you a better understanding of Hemophilia and the important role you play in the care of a patient with Hemophilia.

The information covered within this learning package includes: 1. What Hemophilia is and how it happens 2. Complications of Hemophilia 3. Knowing the Treatment Plan 4. Understanding Factor Concentrates 5. Nursing considerations 6. Patient teaching and support

What is Hemophilia?

Hemophilia is a type of bleeding disorder – a condition where the blood of a person with hemophilia does not clot normally. A person with hemophilia will not bleed more profusely or more quickly than other people but they will bleed for a longer time.

The reason for this is because an affected person’s blood is lacking a protein that is required for clotting.

The 2 most common types of hemophilia are;

Hemophilia A (also called Classic Hemophilia): lack a protein called Factor VIII

Hemophilia B (also called Christmas Disease): lack a protein called Factor IX

In the clotting process, tiny plasma proteins will link to form a chain called fibrin. The strands of fibrin join together to weave a mesh around the platelets, preventing the platelets from drifting back into the bloodstream. These proteins (factors I, II, V, VII, VIII, IX, X, XI and XIII) work like dominoes, in a chain reaction. This is called the coagulation cascade. If any of those proteins are removed, the chain is broken.

All About Carriers; a guide for carriers of hemophilia A and B, Canadian Hemophilia Society, 2007

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Understanding the Severity Scoring of Hemophilia

Both Hemophilia Type A (insufficient Factor VIII) and B (insufficient Factor IX) present with the same symptoms; however each type will vary in severity. Below is a collection of symptoms that are most often seen with each severity, however, each individual present a little differently.

Mild (>5% -35% clotting factor present in blood)

Infrequent bleeds Bleeding mostly occurs only when there is a procedure, surgery, or after trauma/injury Rarely have bleeding outside of injury or procedure May go long periods of time without bleeding or need of treatment Early signs of bleeding are not always easy to recognize Treatment usually only given when needed In most mild cases, patients do not self-infuse

Moderate (1-5% clotting factor present in blood)

Bleeding patterns are variable, may bleed frequently, or may not bleed for long periods of time.

Bleeds expected around time of surgery, procedures, and minor trauma Many self-infuse by the time the patient is an adult Joints and large muscles may be areas to watch closely Falls and minor bumps may lead to large areas of bruising

Severe (<1% clotting factor present in blood)

Frequent bleeds, with/without identified injury (i.e. Falls, bumps, injury) Larger factor infusion doses needed to prevent bleeding Almost always diagnosed in childhood – if parent has hemophilia pre-natal testing can be

done to determine if the fetus also has hemophilia. Home infusion program – usually self-infusing by the time the patient is an adult, but often

the family is taught to infuse children who need frequent doses. Joints and large muscles are areas of frequent bleeds and must be closely monitored

Also to consider … Von Willebrand Disease

Another type of bleeding disorder that you may see in the community and is Von Willebrand Disease (vWD). This is the most common bleeding disorder that affects both men and women and is presented as Type 1, Type 2 or Type 3.

Von Willebrand Factor (vWF) is a protein in blood which is necessary for proper blood clotting. vWF carries Factor VIII in the bloodstream and since Factor VIII is one of the proteins needed to make blood clot, low vWF levels means low levels of factor VIII. Without normal levels of factor VIII, a blood clot takes a very long time to form. For more information and resources related to Von Willebrand Disease provided by the Canadian Hemophilia Society, please click on this LINK.

There are other more rare bleeding disorders that you can read more about if interested. These include; Rare Factor Deficiencies and Platelet Function Disorders.

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What Causes Hemophilia?

Hemophilia is a condition which dates back to the 20th century. Since that time, much has been learned about the condition and several safe, effective treatments have evolved.

Hemophilia is an inherited bleeding condition that is linked to an affected X chromosome. Hemophilia is caused by a gene that has a mutation; which is simply a change in the gene sequence.

Since Hemophilia is caused carried on the X chromosome, males most often have the disorder, as they only have one copy of the X chromosome. Women can also be affected, however they most commonly are the carriers.

So, women who carry a mutation on one of their X chromosomes have a 50% chance of having a son with Hemophilia and a 50% chance of having a girl who is a carrier.

Men with Hemophilia will not pass on the disease to their sons because they transmit a Y chromosome. However if they produce a daughter, she will 100% be a carrier. This can be seen in the diagram below;

If the mutation occurs on the factor VIII gene, the person will have hemophilia A; if the mutation occurs on the factor IX gene, the person will have hemophilia B.

There are multiple types of mutations that can occur.

The type of mutation will result in the level of severity of hemophilia.

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Complications of Hemophilia One of the obvious complications with hemophilia is the risk of bleeding (hemorrhage), both internally and externally. Most often an external hemorrhage can be more easily managed than an internal hemorrhage.

It is important to know that most often, patients who have severe enough hemophilia will be receiving factor replacement on a prophylactic basis which reduces the risk of hemorrhage; however it is still important that the signs of hemorrhage are assessed and managed.

Signs of Internal Hemorrhage:

Changes in neurological function or sedation

Sudden weakness, long-lasting, painful headaches, double vision, seizures, confusion, unusual sleepiness, changes in behavior

Symptoms of shock (hemorrhagic) (can be internal/external bleeds)

Excessive bruising or large areas of bruising (esp. in the large muscles)

Swelling or swollen extremities, or joints (as the bleeding continues the swelling will increase)

Uncharacteristic joint stiffness, tingling, or pain especially if swelling is present

Bleeding from an injury (no matter if it is minor) that will not stop bleeding, and the patient says it is bleeding longer or heavier than normal.

Blood in urine or stool

Nausea and vomiting for prolonged periods of time (without signs/symptoms of flu or illness)

Nose bleeds with no obvious cause - may bleed profusely, and/or be difficult to stop

Excessive bleeding from a minor injury (cut, scrap, etc.) that will not stop or that stops and starts again after a time.

If a hemorrhage is undetected and untreated, significant muscle and joint damage can occur.

If a hemorrhage occurs in a vital organ (i.e. Brain) the outcome can be fatal.

Images: (Left) Swelling in the upper leg, due to a large internal muscle bleed – swelling can present with or without visible bruising; (Right) Knee swelling due to a joint bleed, most common in knees and elbows. (St. Michael’s General Hospital, Hemophilia Clinic website).

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Additional Complications

From a more long term management perspective, the three main concerns with patients who have hemophilia are;

1. The development of inhibitors 2. Joint and muscle damage 3. Pain

1. The Development on Inhibitors

Background … Factors such as age, type of hemophilia, and possibly infection arising from an implanted venous access device may increase the risk and development of inhibitors. Inhibitors typically occur within the first 50 infusions, and can take 1-2 years before “immune tolerization” is achieved with effective 80% of the time.

What this means… This complication that can occur in approximately 30% of people diagnosed with Hemophilia and are first exposed to clotting factor concentrates. When patients receive the factor, the immune system reacts to it by developing antibodies.

How this presents … The patient will not show any response to the clotting factor concentrates being given; they will have increased episodes of bleeding and bruising. This is not something that you should be seeing in the community, as patients are monitored and well-established before they are transitioned to home care. If patient does report signs of increased bleeding or bruising, it is important that they report that to their hemophilia team.

This is managed by … immune tolerance therapy which is the process of administering regularly scheduled, high doses of clotting factor concentrates. This exposure of clotting factor concentrates will wear out the antibody production and cause the immune system to stop rejecting the product. There are temporary treatments available to manage bleeding episodes at home, during this time. http://www.hemophilia.ca/files/Chapter%2008.pdf (pages 3-6)

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2. Joint and Muscle Damage

Background … Many people with Hemophilia have existing joint damage due to less advanced prophylactic treatment earlier days. In the more recent years children are treated prophylactically to ensure that joint and muscle damage does not occur.

What this means… These bleeds can eventually lead to joint or muscle damage in three ways;

1. Bleeding into the joint within 1 episode or multiple episodes 2. Inflammation of the lining of the joint (synovium) which can create a new bleed before

the first bleed has had a chance to heal. 3. If they are not treated, muscle bleeds can cause scarring and loss of flexibility of

muscles, placing additional stress on the nearby joints and increasing their risk to bleed.

How this presents … The joints that are most affected and bleed most often are the joints which work in a hinge motion, (ie. knees, ankles and elbows) due to the fact that they have very little protection from side-to-side motion.

The ball and socket type joints (ie. shoulder and hip) are well protected by large muscles, and are designed to move in many directions without being injured.

A target joint is one that bleeds more often and does not return to a healthy state between bleeds, putting it at a higher risk of developing early joint disease.

Recognizing a joint bleed and early treatment is the best way to prevent the occurrence of a target joint and development of joint disease.

Although in young children it may be difficult to tell if there is swelling in a joint, you may notice that your child refuses to use an arm or a leg, or cries if that limb is moved.

This can present with the symptoms shown on the right;

This is managed by … preventing the joint bleed as best as possible. In the meantime, if an acute bleed does happen, the joint bleed should be treated immediately so if clotting factor is given at this point, only a small amount of bleeding needs to heal. Have the patient call their Hemophilia clinic contact right away for further directions.

http://www.hemophilia.ca/files/Chapter%2008.pdf (pages 7-18)

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3. Pain

Background … Patients with Hemophilia often suffer with both acute and chronic pain. Acute pain is caused by the actual joint bleed. Chronic pain is due to the joint disease develops from previous and frequent bleeds.

How this presents … If your patient develops acute pain, this is a sign that something is wrong and needs further assessment as it is usually the earliest sign of a bleed. If untreated, the bleed will go on to stretch a joint or put pressure on muscles and other structures, making the pain intensity increase. If the patient develops pain but has no other symptoms of a bleed (ie. swelling), it is still recommended that they be assessed by the Hemophilia team and be treated with clotting factor therapy if determined as necessary.

This is managed by … treating the pain while the bleed in the joint is recovering. Once the bleed has been assessed and treated, the patient will likely still experience pain so many treatment options will be available to them. Some of these treatments include; Medications Rest Warm bath Physiotherapy Alternative treatments (ie. massage, chiropractic)

http://www.hemophilia.ca/files/Chapter%2008.pdf (pages 7-18)

Some additional resources care available at the following websites (tap the image):

You will find: Educational Resources Contact Info for all HTC’s

You will find: Educational Resources Contact Info

You will find: Educational Resources Contact Info

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Knowing the Treatment Plan

Knowing the Treatment Plan

Patients newly diagnosed with Hemophilia will be referred to a Hemophilia Treatment Center (HTC) where the primary teaching and support will happen.

The patient will have access to Hemophilia specialists, social workers, nurses, physicians, and nutritionists to empower patients to learn to live a full life with Hemophilia. This is a lifelong condition so patients or caregivers are taught to be as independent as possible with managing it.

Not every person with Hemophilia takes factor concentrates, in fact patients who do are; • severe hemophiliacs • children who are very active • hemophiliacs on prophylaxis therapy

At the HTC, patients will receive teaching on their diagnosis, precautions, treatment options, a plan of care, first doses of factor and how to administer their treatments. When working with a Hemophilia patient who is admitted to nursing service, the HTC is always the primary source of information while CarePartners provides the requested support and care in the home.

All HTCs are connected through a network and patients will receive contact information for their Hemophilia team. The provincial list is available and can be found by tapping this LINK.

Role of the Homecare Nurse

Some patients receive factor on a prophylactic basis while other receive on-demand treatment.

Prophylaxis therapy means factor concentrates are received one or more times a week to keep the factor levels high enough that bleeding is prevented in the event of an injury. This therapy is common with children with severe hemophilia and research shows that this helps to prevent children having joint damage in their adulthood. On-demand therapy means that factor concentrates are administered immediately after a bleed begins, so that the bleeding can be stopped and avoiding any damage to the joint or muscle.

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With this in mind, you may receive referrals for such situations as;

short-term factor injections before or after a procedure. Ie. Dental extractions, prostate Biopsy, Joint Arthoplasty (Hip, Knee, Elbow), Major

Surgery (Abdominal Aortic Aneurysm (AAA) repair, abdominal surgeries, etc.)

significant bleed episode, requiring long-term factor injections and patient or a caregiver is unable to administer themselves.

Ie. GI bleeds, CNS bleeds

extra support with injections, as they learn to self-inject Ie. Support for IV access, transitioning from using a port to a peripheral site

Questions to ask when you receive a referral

What type and severity of Hemophilia does the patient have?

What is the goal of care? (consider: situations above, self care, etc )

How often have they received it previously and were there any issues/side effects?

Any unique considerations? (ie. history of bleeds, immune tolerance, etc.)

What has the patient been taught and what is the expectations of the nurse?

Consider additional medical history and other social concerns that may be identified.

Role of the Patient/Caregiver

Reordering factor from HTC when home stock is low, or as needed

Pick up the factor from a hospital blood bank

Remove the factor from the fridge to allow it to warm to room temperature before use

Patient is required to record all infusions done in-home and submit this record to the HTC

when reordering factor

Monitoring symptoms and reporting the HTC as per established plan of care

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Understanding Factor Concentrate Treatment is aimed at stopping or prevent bleeding in people with hemophilia A and B by using a factor replacement therapy. (factor VIII or factor IX concentrates). These factor concentrates come from a human plasma or a genetically engineered cell line (called recombinant). Both are nearly identical to the protein that is lacking in the blood.

Following an infusion of the concentrate, the blood clotting normalizes for at least for a few hours, allowing time for a clot to form. The half-life of these concentrates is 12-24 hrs, with results lasting 2-3 days only.

Over the past several years, great improvements have been made with factor concentrates which has allowed patients the autonomy to manage their treatments safely and in the own homes. Some of these improvements include:

• Concentrate is small enough to contain enough factor activity to effectively manage bleeds. • Convenient storage for up to a year in a refrigerator or at room temperature for 3 to 12 months. • Easy reconstitution and administration within one minute. • Quick administration with the whole procedure completed within 15-20 minutes. • The previous safety resolved and no transmission of HIV or hepatitis in Canada since 1988.

For more information on how these concentrations are made and the history of Hemophilia treatments, refer to the Canadian Hemophilia Society website but clicking this LINK.

Treating Hemophilia A

The most commonly used factor in Hemophilia A is a genetically engineered (recombinant) factor VIII. The brand names you may see in Canada are Advate®, Kogenate®FS (being replaced by Kovaltry® with a longer half-life) and Xyntha®.

Patients may also receive plasma-derived factor VIII with particular situations such as; 1) Those who may be at higher risk for the development of inhibitors 2) Patients with immune tolerance who have already developed antibodies to factor VIII.

Brand names you may see in Canada are Humate P® and Wilate®. These factors are used for the treatment of VonWilldebrand Disease

Treating Hemophilia B The most commonly used factor in Canada since 1998 for Hemophilia B is the recombinant factor IX, which has a brand name of Benefix®.

A plasma-derived factor IX is also available in Canada and may be used. This factor is called ImmunineVH®.

Click on the each drug name for more information on factor, mixing instructions, videos, etc.

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Nursing Considerations

Although rare, side effects are always possible.

Prior to being referred to Care Partners patients should have an established treatment plan of care with previous exposure to the factor concentrate. If this is not the case, contact your Nurse Manager or Clinical Practice Manager.

Ensure that you have reviewed the factor concentrate information in your iPad drug book or product monograph prior to administering. If videos and other resources are available online, become familiar with that information.

Assess the patient upon each visit for signs and symptoms of bleeding and bruising, joint pain or swelling, coping, and ability to manage the procedure at home.

Assess vital signs prior to each the infusion and at 15 minutes following the infusion.

When administering the factor concentrate, be sure to use the instructions provided in every kit.

If you have any questions or concerns, contact the resource nurse at the Hemophilia Treatment Centre.

Once the factor is reconstituted, most require that it be administered within 3 hours.

The reconstituted factor is administered via IV push over a specified amount of time (as per orders and care plan – ie. 4mls/minute). The patient may have an IV accessed established or it may be administered via butterfly needle directly into the vein. Always follow the manufacturer instructions provided.

To the right is a diagram which identifies some possible reactions and how to respond to them:

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Patient Teaching and Support The patient will receive most of their Hemophilia education through the HTC, however, they may need the nurse to provide additional self-management support, , assess for and answer questions related to:

• Identifying bleeding (see “Signs and Symptoms above) and when the patient should see healthcare professional

• Identifying reactions to the factor given and what to do in case of side effects or reaction • If part of the care plan: teaching or offering support to a patient learning to self-administer • How to care for the IV site (if S/L or port)

If the nurse finds that the patient seems to have many knowledge gaps, or a poor understanding of their diagnosis, treatment or activities of daily living with Hemophilia, encourage the patient to visit or call their specific HTC.

One Last Consideration In the early 1980’s, approximately 1,000 Canadians were transfused with tainted blood –

approximately 700 Hemophilia patient were infected by HIV and approximately 20,000 Canadians were infected with Hepatitis C, many being Hemophilia patients. Testing blood products for HIV, Hepatitis C, and other blood-borne diseases was only introduced in 1990. By the mid ‘80’s testing people for HIV had been developed, and Canada realized that many people how had had blood

transfusions had been infected by HCV and HIV. (Canadian Hemophilia Society) Be aware of your patient’s blood status – HIV or HCV - especially if they received transfusions prior

to 1990 Take appropriate precautions

Resources

(1) Canadian Hemophilia Society http://www.hemophilia.ca/en/about-the-chs/case-for-support/facts-and-figures/ (2) NIH. National Heart, Lung, and Blood Institute http://www.nhlbi.nih.gov/health/health-

topics/topics/hemophilia/diagnosis (3) Comprehensive Hemophilia Program at St. Michael’s Hospital

http://www.stmichaelshospital.com/programs/hemophilia/ (4) Gulecher, C.J. Evolution of treatments for hemophilia. Journal of Infusion Nursing. 2016;39(4):218-224.

Additional information

Hemophilia Fact Page from the CCD http://www.cdc.gov/ncbddd/hemophilia/facts.html

World Federation of Hemophilia http://www.wfh.org/en/page.aspx?pid=492

Care Partners Portal link – Reconstitution instructions with pictures, and medication information https://portal.carepartners.ca/Staff/Clinical%20Nursing%20Resources/Forms/AllItems.aspx?RootFolder=%2FStaff%2FClinical%20Nursing%20Resources%2FHemophilia%20Resources&FolderCTID=0x01200058A2CA99C977FA43857E8104921D6185&View=%7B532ED8ED-36F1-40D5-BC82-29702EDC6095%7D