Thriving Amid The Turbulent Ride

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1 Thursday, April 3 7:00 8:45 AM Rosen Shingle Creek Panzacola F 1/2 A Symposium Held in Conjunction with the 2014 NHIA Annual Conference & Exposition 2014 NHIA Annual Conference & Exposition Thriving Amid The Turbulent Ride

2 05 S. Hemophilia Management in Home Infusion Thursday, April 3 7:00 8:45 AM Rosen Shingle Creek Panzacola F 1/2 Pharmacist, Pharmacy Technician and Nurse Continuing Education Contact Hours: 1.5 ACPE Pharmacist and Pharmacy Technician Program #: L01 P & T Knowledge Based Learning Activity Education Overview: Hemophilia is a rare but serious X linked congenital bleeding disorder estimated to affect 20,000 people in the U.S. The primary goal of care in this patient population is to prevent and treat bleeding with the appropriate factor replacement therapy, following evidence based dosing algorithms. Self or caregiver administration of prophylactic factor concentrate in the home has become the standard of care for patients with mild, moderate, and severe hemophilia and von Willebrand disease. Prevention of bleeding, and its resultant joint damage, is an important strategy in the preservation of normal musculoskeletal function, but requires active patient involvement paired with insightful clinical monitoring and nuanced patient education. This program will provide a comprehensive overview of hemophilia, from pathophysiology to genetic inheritance, treatment guidelines and special considerations for different patient groups based on hemophilia type and severity, and evolving research. The collaborative role of the home infusion team (pharmacist, nurse, dietitian, social worker, and prescriber) in the treatment and management of these complex patients will be explored in a case based approach with a discussion of strategies for optimizing patient outcomes. Faculty: Donna Haffler, RN, BSN, Regional Care Coordinator for the East, Bleeding Disorders Program, Walgreens Infusion Services, Lexington, KY; and Kirstin Schmidt, RN, National Clinical Director, Bleeding Disorders Program, Walgreens Infusion Services, Deerfield, IL Faculty Biographical Statement: Donna Haffler, RN, BSN, has over 25 years of nursing experience in a range of clinical settings. She joined the Walgreens Bleeding Program in 2013 as the Regional Care Coordinator for the East. Prior to that, Donna was a Clinical Nurse Coordinator for the University of Kentucky s Hemophilia Treatment Center and provided comprehensive care for families in Kentucky. In addition to her Hemophilia Treatment Center experience, Donna spent six years at an infusion pharmacy as the Director of Nursing and Patient Care. Donna s top priorities are empowering patients and their families by providing patient education and ensuring families receive the best possible care. In her role as part of the Walgreens team, Donna provides consultation, coordinates family education events, facilitates networking and develops customized care plans to address a family s specific needs. Kirstin Schmidt, RN, manages the Walgreens Bleeding Disorder program, as Clinical Director, by providing oversight, direction, education and training; including clinical staff development, leading research, providing in services, speaking at special events and writing educational articles. Kirstin joined the Walgreens Bleeding Disorder team in Previously she worked for the Mayo Clinic in Rochester, MN, where she was a hemophilia nurse coordinator at the Mayo Comprehensive Hemophilia Center for 14 years. She has been an advocate for the bleeding disorders community for more than 16 years and has been an active member of the National Hemophilia Foundation as well as her local foundation, serving on a number of committees, including the development of the Health Center Standards for Camps Serving Persons with Bleeding Disorders (NHF), Annual Meeting Planning Committees and Women with Bleeding Disorders Planning Committee. She has also been very involved with camp planning and has served as a camp nurse for 15 summers. In addition to her work with the NHF and local chapter, Kirstin has spoken on a wide variety of topics, authored several articles and has served as study coordinator for numerous studies while at the Mayo Clinic. Pharmacist, Pharmacy Technician and Nurse Education Objectives: 1. Describe the coagulation pathway and the steps to clot formation. 2. Explain the genetic basis of hemophilia. 3. Describe the incidence, type and severity levels of hemophilia A & B. 4. List complications associated with hemophilia, and strategies to minimize their occurrence. 5. Discuss the concept of inhibitor development and its impact on the prevention and/or treatment of bleeding episodes. 6. Describe the clotting factors and medications currently used in the treatment of hemophilia, as well as the promising agents in the research pipeline. 7. Describe guidelines for dosing and administration of factor products. 8. Describe the components of a comprehensive hemophilia management program. 9. Describe operational considerations in clotting factor management NHIA Annual Conference & Exposition 1

3 Learning Assessment Questions: 1. A female carrier of hemophilia A has which of the following? a. A 50% risk of having an affected daughter b. A 50% risk of having a carrier daughter c. A 50% risk of having an affected son d. B and C 2. Spontaneous bleeding is most likely to occur in a patient with which of the following? a. Mild hemophilia b. Moderate hemophilia c. Severe hemophilia d. All of the above 3. Factor VIII is given by which of the following? a. Bolus dose b. Self administered home infusion c. Continuous infusion d. All of the above 4. Key factors to consider when managing a patient with hemophilia include which of the following? a. Access for prompt treatment of bleeding episodes b. Access to nursing/pharmacy support c. Immediate replacement of product/supplies d. All of the above 5. Inhibitor development is the most significant risk factor associated with hemophilia. a. True b. False Answers can be found on the last page of this booklet NHIA Annual Conference & Exposition

4 Disclaimer The information within this CME/CE activity is for continuing education purposes only, and is not intended to substitute for the medical judgment of the healthcare provider. Recommendations for use of any particular therapeutic agents or methods are based upon the best available scientific evidence and clinical guidelines. Reference in this activity to any specific commercial products, process, service, manufacturer, or company does not constitute its endorsement or recommendation 2014 NHIA Annual Conference & Exposition 3

5 I (we) do not intend to discuss an off-label use of a product during this activity. I (we) have not had any relevant financial relations during the past 12 months to disclose. Objectives Upon completion, the participant should be able to: Describe the genetics of bleeding disorders Discuss the incidence, severity and consequences of hemophilia A and B Describe the different factor products Discuss the concept of inhibitor development and its consequence in hemophilia Describe bleeds, their assessment, and potential complications NHIA Annual Conference & Exposition

6 2014 NHIA Annual Conference & Exposition 5 Demographics Hemophilia is a rare genetic bleeding disorder caused by the absence or deficiency of a clotting protein necessary for proper clot formation Approximately 20,000 cases of hemophilia in the United States One of the most expensive chronic diseases Total average annual cost of factor concentrate can range from $60,000 to $150,000 Presence of inhibitor drives costs much higher From the late 1970s to the mid 1980s, approximately half of people with hemophilia in the United States were infected with HIV 3 Normal Clotting Process After an injury to skin or other body tissue blood usually clots within three to five minutes Blood clot creates a patch at the site of injury to stop active bleeding into surrounding tissues The body begins to repair itself immediately Three major steps in the normal clotting process Vasoconstriction Platelet plug: formed by clotting factors Fibrin clot formation In the presence of a bleeding disorder there is incomplete or delayed formation of the fibrin clot

7 Coagulation Cascade Clotting in Bleeding Disorders One factor is absent or deficient The blood clot does not form completely or at all Leads to excessive amounts of blood loss or leakage into surrounding tissues Bleeding continues or rebleeding occurs Bleed longer, not faster NHIA Annual Conference & Exposition

8 2014 NHIA Annual Conference & Exposition 7 Most Common Bleeding Disorders Hemophilia A Factor VIII Deficiency Classic Hemophilia Royal Disease Hemophilia B Factor IX Deficiency Christmas Disease von Willebrand Disease (VWD) Hemophilia A & B

9 2014 NHIA Annual Conference & Exposition 8 Hemophilia A & B Hemophilia A Factor VIII deficiency More common ~80% (8 out of 10) of those with hemophilia Hemophilia B Factor IX deficiency Approximately 3,300 people in the US living with hemophilia B ~10-20% of those with hemophilia Clinical manifestation in hemophilia A and B are the same Genetics X-linked recessive genetic disorder Inherited (genetic) = family history Spontaneous mutation (30%) = no family history Expressed in males (one X chromosome) All daughters are carriers sons affected Carried by females (two X chromosomes) 50% risk for carrier daughter 50% risk for affected son

10 2014 NHIA Annual Conference & Exposition 9 Bleeding following circumcision Head bleed following traumatic birth Bleeding / oozing from umbilical cord / heel stick / injections Bleeding of the mouth and gums Bleeding into the joints (usually after 2 years of age) Nose bleeds that are frequent and hard to stop Excessive bruising Excessive/uncontrolled bleeding following an invasive procedure Family history 1/3 born with no family history (spontaneous mutation) Lab Testing Special coagulation tests are performed Identifies the type and severity of bleeding disorder Prenatal Diagnosis Genetic counseling Individualized

11 Severity of Hemophilia Severe Hemophilia (60% of those affected) < than 1% factor in circulation Moderate Hemophilia (15% of those affected) 1%-5% factor in circulation Mild Hemophilia (25% of those affected) 6%-49% factor in circulation Can bleed without injury (spontaneous bleeds) Can bleed with trauma or surgery or incidental injury Bleeding with trauma or surgery, dental procedures, injections Usually develop joint problems ~ 1 bleeding event weekly unless treated prophylactically (Normal Level = %) Possible joint problems ~ 4-6 bleeding events annually Rarely develop joint problems Bleeding events uncommon The goal of treatment is to prevent or control bleeding in persons with hemophilia A and B Factor replacement Administration of the missing factor (FVIII or FIX) Bolus infusion or Continuous Infusion Quality Patient Care Hemophilia Treatment Center (HTC) Knowledgeable physician Home infusion company/specialty pharmacy with clinicians who specialize in bleeding disorders NHIA Annual Conference & Exposition

12 2014 NHIA Annual Conference & Exposition 11 Treatment Options Factor Replacement Prophylaxis On Demand DDAVP (Desmopressin Acetate/ Stimate) Synthetic form of vasopressin that stimulates factor VIII and helps release von Willebrand factor Mild hemophilia A, vonwd (mild-moderate Type I) IV, SC, Nasal Stimate /7 Antifibrinolytic Agents Factor Replacement Products Derived from human plasma Products virally inactivated Blood donors now screened for HIV, hepatitis C and hepatitis B Derived from DNA technology Manufactured synthetically Classified as recombinant products Long-term effects not really understood or known

13 2014 NHIA Annual Conference & Exposition 12 Factor Products Factor VIII Products Plasma derived products First generation recombinant FVIII product (Human albumin) Second generation products (stabilized with sucrose so less human albumin) Third generation products (plasma free) Factor IX Products Plasma Derived Recombinant Other Factor Products Bypassing Agents FCT VII FCT XIII Prothrombin Complex Concentrates (PCCs) Fibrinogen Concentrates vwf products Administration of Factor Factor can be replaced via IV bolus infusion or continuous infusion Factor may be given at home Self-administered Home care support should include the following: Infusion products and supplies 24-hour on-call nursing, pharmacy, and delivery support Immediate replacement of factor Nursing support for venous access issues, patient education for self infusion, unnecessary ER avoidance, and infusions for patients unable to self infuse

14 2014 NHIA Annual Conference & Exposition 13 Dosing of Factor Products The dose of Factor is based on the patients weight Factor VIII One unit/kg of Factor VIII concentrate will increase the blood level by 2% Factor IX One unit/kg of Factor IX concentrate will increase the blood level by 1% Prophylactic Treatment Patient outcomes are more favorable if factor is circulating prior to injury Goal of scheduled administration of factor is to maintain high factor levels to prevent most, if not all, episodes of uncontrolled bleeding Prevents long-term complications and joint deterioration May be used in infants through adults Usually infused three times per week for hemophilia A and twice per week for hemophilia B Reduces ER and hospitalization costs Concluded from a multicenter study ( )

15 2014 NHIA Annual Conference & Exposition 14 Disease Management Keys: Prompt treatment Nursing and pharmacy support Immediate replacement of factor and supplies Patients can live relatively normal lives with access to adequate medical treatment and home support Self-management skills are essential High costs can be managed by involving the patient/family and establishing goals at the outset of treatment Inhibitors

16 2014 NHIA Annual Conference & Exposition 15 Inhibitors Antibodies against infused factor Antibodies neutralize infused factor Alternate therapy necessary Incidence of inhibitor developmentº 33% with severe hemophilia A 1%-6% with severe hemophilia B Spontaneous remission rare Immune Tolerance Therapy (ITT) Inhibitor Risk Factors Age Often within the first infusions (73%) Can develop at any age Challenges of the immune system Infection Immunizations Genetics Family history Genetic mutation (Inversion 22) Race Higher incidence in non- Caucasian patients African-American Asian Hispanic Product choice/

17 2014 NHIA Annual Conference & Exposition 16 When to Screen for an Inhibitor Annual screening at Comprehensive Health Exam (CHE) Prior to surgical /invasive procedures Regular intervals during initial 50 treatment days When inhibitor is suspected Clinically, bleeds no longer respond to factor treatmentº Suspected if factor VIII level no longer increases to the expected level following FVIII infusion Measurement of an Inhibitor Inhibitor titer assay(bethesda units) Low-responding Inhibitor Titer < 5 BU BU does not increase with exposure to additional factor Requires higher dosing of factor to achieve the desired level Transient Inhibitor Titer usually < 5 Bethesda Units 10% of low titer inhibitors are considered transient and disappear in a matter of weeks-months High-responding Inhibitor Titer > 5 BU Strong response to infused factor Requires alternate therapy

18 2014 NHIA Annual Conference & Exposition 17 Treating a High Responding Inhibitor Treatment with Bypassing agents Bypass the deficient clotting factor Recombinant activated VII (rfviia- Novoseven ) Due to short half-life repeat dosing is needed every 2-3 hours Activated Prothrombin Complex Concentrates apccs (FEIBA )º Plasma derived (virally inactivated) Contains both FVIII and FIX in small amounts Half-life ~ 4-7 hours Antifibrinolytic therapy is contraindicated while using appcs Treatment of Inhibitors Immune Tolerance Therapy (ITT) Eradicate the inhibitor Frequent high doses of factor repeatedly over a long time period Other types of therapy Usually used if ITT fails or in conjunction with ITT Immunosuppressants Steroids

19 2014 NHIA Annual Conference & Exposition 18 Immune Tolerance Therapy Immune tolerance can be achieved in ~70% of patients who receive regular and prolonged infusions of factor VIII The best means to achieve tolerance is still unknown Recommended ITT Algorithms An international workshop on ITI convened in North America and England in 2006 to formulate consensus recommendations for patients with hemophilia A and hemophilia B. Algorithms for severe hemophilia A When to start ITI Dose Product Prophylaxis during ITI CVAD use/issues

20 2014 NHIA Annual Conference & Exposition 19 Factor IX Inhibitor 5-10 times less common than in hemophilia A Clinically more difficult to treat Many patients with FIX inhibitors develop anaphylactic type reactions to the infused factor during or shortly after an infusion 80% are high responder type Management becomes increasingly challenging Greater risk of morbidity Lack of established guidelines for treatment of FIX antibody Prophylaxis for Inhibitors Prophy with bypassing agents Prior to the start of ITI rfviia is recommended to prevent an anamnestic increase while waiting for inhibitor titer to decrease Prophylaxis should be considered if a patient continues to have bleeding episodes while getting ITI Monitor FVIII recovery once BU<10 Discontinue bypass therapy at any level of FVIII recovery

21 2014 NHIA Annual Conference & Exposition 20 Surgery With an Inhibitor Surgery should be approached with caution Both rviia (NovoSeven ) and FEIBA can be used to manage hemostasis during surgery Both products have been shown to control at least 80% of bleeding episodes associated with high- titer inhibitors Hemostatic efficacy is difficult to predict J (18yo) Diagnosed w/ inhib at age 12m BU <10 Port placed and ITI initiated Remained on ITI x 2.5yrs Port remained in place x 5 yrs Began self PIV at age 8 No hx of joint bleeds, 1 thigh bleed Current regimen rfviii 3x/wk HS Senior Missed total of 5 days of school Case Study: A Tale of Two Brothers

22 2014 NHIA Annual Conference & Exposition 21 C (8yo) Diagnosed w/ inhibitor at age 11m BU Port Placed NovoSeven prn bleeds FEIBA prn at age 4y Port replaced 4 times Developed F8 allergy (IgE) age 7y R knee target joint nd grade - missed school days this year Wheelchair 95% of time Quality of Life issues for family Case Study continued Used with permission by family

23 2014 NHIA Annual Conference & Exposition 22 Complications of Bleeding Disorders History and Assessment What are the symptoms? How long have symptoms been present? Treatment given Did an injury happen before the symptoms started? Has a similar problem occurred in the past? Get details of actions taken at home including infusions, immobilization, ice, etc.

24 Joint Bleeds Types of Bleeds Muscle Bleeds Soft Tissue Bleed Joint Bleeds Most common in knees, ankles, elbows Symptoms collection of blood in joint space may cause joint to feel hot Tingling or bubbling sensation Reluctance to move Swelling and joint pain as bleed progress Repeated bleeding in and around the joint cavity results in damage to the joint 2014 NHIA Annual Conference & Exposition 23

25 2014 NHIA Annual Conference & Exposition 24 Synovectomy Radioactive synovectomy A radioactive isotope is injected into a target joint Arthroscopic synovectomy Removal of the synovium through small incisions Open synovectomy The joint is opened surgically and the synovium removed Correction of factor level to 100% prior to, during and following the procedure is necessary to prevent re-bleeding into the joint space If pain and decreased range of motion (ROM) become severe and interfere with the patients quality of life, joint replacement is an option Factor replacement is essential Clotting factor levels are kept at 100% usually by continuous intravenous infusion for 10 days or more Specific management must be done by the physician familiar with hemophilia, with access to clotting factor and to a special coagulation laboratory to monitor factor levels frequently

26 2014 NHIA Annual Conference & Exposition 25 Risks of Surgery Intra-operative and post-operative bleeding Transfusion with blood products may be necessary Infection Infection may occur early or develop weeks or months after surgery Infection requires antibiotic therapy usually by intravenous route and in-hospital An infection may not clear up in the presence of an artificial joint Post procedure bleeding Second most common type of bleeding Common sites of muscle bleeds: (hip) iliopsoas, (thigh) quadriceps, (calf) gastrocnemius muscles, and forearm Early signs and symptoms: Reluctance to move extremity Pain / Swelling Change in mobility and range of motion Symptoms worsen as bleeding progresses Treatment Replacement Factor RICE (rest-ice-compression-elevation) Physical Therapy

27 2014 NHIA Annual Conference & Exposition 26 Risks Compartment Syndrome A bleed into a muscle can spread through the muscle length When muscle bleeding occurs in the forearm, calf or groin, swelling can create pressure on the nerves which can result in numbness, and subsequent pain and inability to move the limb Complaint of a "pulled muscle" may be confused with a deep muscle bleed A pulled muscle complaint is always to be considered a deep muscle bleed until proven otherwise Life Threatening Bleeds Central Nervous System Bleeds (Head / Intracranial) Highest risk to life Neck / Throat Abdominal / GI Evaluation necessary Iliopsoas Muscle

28 Other Types of Bleeds Mouth and Gum Bleeds Nosebleeds Menorrhagia Hematuria The earlier bleeding is identified and treated, the better the prognosis for the patient NHIA Annual Conference & Exposition 27

29 2014 NHIA Annual Conference & Exposition 28 Program Overview Empowering independence Nursing support across life stages Therapy management and medication oversight Local nursing and pharmacy Ethics and integrity Control total cost of care through Assay Management Dose Management (including doses on hand) ER avoidance Pre and post op support Conclusion Optimal outcomes require Medical care by a comprehensive Hemophilia Treatment Center Prompt access to medications, infusion supplies, and adjunctive therapy Patient and family empowerment with goal of family/patient infusion Patient education on potential bleeding episodes and how to treat Understanding disease sequelae Collaboration between HTC, patient, and home infusion provider

30 Questions? References 2014 NHIA Annual Conference & Exposition 29

31 2014 NHIA Annual Conference & Exposition 30 References DiMichele DM. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia. 2009; 15: Hay CM. The epidemiology of factor VIII inhibitors. Haemophilia. 2006: 12(Suppl 6): DiMichele DM. Inhibitor treatment in haemophilias A and B: inhibitor diagnosis. Haemophilia. 2006:12(suppl 6): Canadian Hemophilia Society Web site. Accessed January 7, Astermark J, Altisent C, Batarova A, et al: European Haemophilia Therapy Standardisation Review Board. Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia 2010;16(5): References

32 NOTES: 2014 NHIA Annual Conference & Exposition 31

33 Answers: 1. D. Both b (50% risk of having a carrier daughter) and c (50% risk of having an affected son) are correct 2. C. Severe hemophilia 3. D. All of the above 4. D. All of the above 5. A. True NHIA Annual Conference & Exposition

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