Patient Story: Sara Ceresa

A Lucky Conversation

“My introduction to von Willebrand disease was completely random.” Sara was at a business dinner with her husband, chatting with the wife of a colleague. The woman spoke about her nephew who had VWD, and because Sara had never heard of the disease, she asked for more information. The woman described her nephew's symptoms – prolonged nose and mouth bleeds, often accompanied by bruises with lumps under the skin. “I thought, whoa! This sounds just like my kids!” “I told my husband, who told his dad, who said ‘Oh yeah, my sister has that.’ Nice to know!” Sara had had nosebleeds as a child; she thought everyone experienced them. So, she wasn't overly concerned about her daughters' frequent and lengthy nose bleeds. On one occasion before their VWD diagnosis, one of her daughters bit her tongue and it bled for twelve hours. Sara called their pediatric dentist and, as he advised, just continued applying ice and pressure while waiting for the bleed to stop. Her twin daughters got tested at age 5 and came out positive, diagnosed with moderately severe VWD and designated Type 2. Her husband, undiagnosed until his 40's, was also tested and initially diagnosed as a mild Type 1 case. They surmised that the disease was inherited from Sara's father–in–law, although he was never tested. A bit later, while battling cancer, he developed a GI bleed. Because the doctors had no documentation about his possible VWD, he was treated with whole blood transfusions but was bleeding out. Sara tried to intervene with information about the factor replacement treatment recommendations that had been made for her husband, but it was too late. Her father–in–law died. “That put a fire in my belly to learn everything I could about VWD to protect my husband and my kids. This is not going to happen to them!”

Preparation and Passion

Sara was a science major in college and had studied genetics for a year in graduate school. In addition, her work as a study coordinator for clinical trials in an ophthalmology clinic made her very comfortable with scientific research and analysis. And she had training in phlebotomy. “Add my concern for my family to my scientific background and you will understand that I am both passionate and uniquely prepared to deal with VWD. Plus, I am good at speaking with doctors – I understand that they're very busy people with a lot of patients to see. Often hematologists are also oncologists, and they focus more of their energy on cancer than on bleeding disorders. So, I believe that you have to make sure that you are asking questions and really communicating in order to get the care you need. You have to advocate for yourself or, in my case, for your family.” Sara went through a time of frustration that her family's VWD type couldn't be conclusively determined. Despite taking advantage of all current tests for VWD including genetic testing, her husband's and daughters' diagnoses didn't fit cleanly in one particular subtype. When Sara's daughters were first prescribed medication to treat their VWD, she was unaware that there were several options available to her. “My daughters were first prescribed Stimate^® nasal spray, but I didn't like it because it can mess with your electrolyte levels. Later we switched to Humate–P^®, then tried Alphanate^®, which lasted for exactly one infusion.” Sara wasn’t pleased with the results of either product. Sara knew it was time to seek another alternative. “I started attending events and learning more myself. In my research, I discovered wilate^®, and was attracted to the 1:1 balance of FVIII and VWD factor, and avoidance of buildup in the system of FVIII. Based on my family's factor levels, it made sense to me that wilate would be a good fit. Eventually I was able to enroll my daughters in a free product trial. Wilate performed beautifully for us, stopping bleeds in minutes.” Now that her family has an effective treatment plan, Sara has more patience in waiting for VWD subtyping labels for her family. When her daughters reached puberty, Sara's research led her to the option of suppressing menstrual bleeding altogether. Both daughters take continuous oral hormone pills to completely stop menstruation. “It was a very personal decision. Everyone has unique symptoms and needs – even my twin daughters differ in their experience with the disease. Because we had no family history of breast cancer and we were not concerned about blood clots, we opted for this approach to managing menstrual bleeds. It has worked for us, but it is not for everyone.”

Stimate^® is a registered trademark of Ferring Pharmaceuticals. Humate-P^® is a registered trademark of CSL Behring GmbH. Alphanate^® is a registered trademark of Grifols.

Infusing at Home

Typically, only some Type 3 VWD patients infuse factor replacement prophylactically on a regular basis. This can mean that Type 1 and 2 patients have very limited experience with infusion, particularly self–infusion, and rarely have factor available at home. Sara wanted proactive protection in case of emergencies, and she wanted to be able to respond quickly and effectively when needed. She asked her hematologist to prescribe a supply of wilate. Essentially self–trained in infusion, she feels comfortable responding if and when required. “I feel super lucky to be able to infuse at home. It can take hours at the hospital to get this done. When my daughter recently had a 4 am nosebleed that went on for a few hours, I was so thankful that I had product at home. She is afraid of sticks. We used relaxation techniques and my husband sat with her and watched a show while I infused her. We treated her and the bleeding stopped. She wasn't even late for school! What would that have looked like if we had had to go to an ER? Sometimes the doctors and nurses there have no experience with factor. They may not know how to mix an infusion, really understand dosing, or have product readily available. It can take forever. Yes, you can request a home health nurse from your specialty pharmacy as an alternative. But I'm not even sure how long that would take.” Sara routinely keeps factor on hand at home and when traveling if she and her daughters are going to be away from home for any significant amount of time. Her daughters require infusions only about three times a year; her husband less often than that.

Raising Awareness

Managing VWD can take a huge amount of preparatory work, including dealing with insurance and specialty pharmacy companies. “It takes a lot of mental energy. I do everything. I can get burned out – that's why I want to help people take better care of themselves.” As an Octapharma Patient Educator, Sara makes sure she's aware of the current needs of the community so that she can address relevant issues. “I am a caregiver and I approach VWD from that standpoint.” Sara gives talks, does women's retreats, conducts art therapy with kids, and is active on Facebook – always endeavoring to educate people about VWD and how both patients and caregivers can thrive. “I do everything I can to get the word out about VWD, bleeding disorders, and caregiving – at school, at church, in my community, and online. I knew nothing about any of this until ten years ago – now I am committed to raising awareness.”

Indications and Important Safety Information for NUWIQ^® [Antihemophilic Factor (Recombinant)].

Please see NUWIQ full Prescribing Information. Instructions For Use

Indications and Usage

NUWIQ^® is a recombinant antihemophilic factor [coagulation factor VIII (Factor VIII)] indicated in pediatric and adult patients with Hemophilia A for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes. NUWIQ^® is not indicated for the treatment of von Willebrand Disease.

Contraindications

NUWIQ is contraindicated in patients who have manifested life-threatening hypersensitivity reactions, including anaphylaxis, to the product or its components.

Warnings and Precautions

Hypersensitivity reactions, including anaphylaxis, are possible with NUWIQ. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, or pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

The formation of neutralizing antibodies (inhibitors) to Factor VIII can occur following the administration of NUWIQ. Monitor all patients for the development of Factor VIII inhibitors by appropriate clinical observations and laboratory tests. If the plasma Factor VIII level fails to increase as expected, or if bleeding is not controlled after NUWIQ administration, suspect the presence of an inhibitor (neutralizing antibody).

Adverse Reactions

The most frequently occurring adverse reactions (>5%) in clinical trials were upper respiratory tract infection, headache, fever, cough, lower respiratory tract infection, rhinitis, chills, abdominal pain, arthralgia, anemia, and pharyngitis.

Please see NUWIQ full Prescribing Information. Instructions For Use

Indicaciones e Información de seguridad importante de NUWIQ^® [factor antihemofílico (recombinante)].

Consulte la Información de prescripción completa de NUWIQ. Instrucciones de uso

Indicaciones y uso

NUWIQ^® es un factor antihemofílico recombinante [factor VIII para la coagulación (factor VIII)] indicado en pacientes pediátricos y adultos con hemofilia A para el tratamiento y el control a demanda de los episodios hemorrágicos, el control perioperativo de las hemorragias y profilaxis de rutina con el fin de reducir la frecuencia de los episodios hemorrágicos. NUWIQ^® no está indicado para el tratamiento de la enfermedad de von Willebrand.

Contraindicaciones

NUWIQ está contraindicado en pacientes que hayan manifestado reacciones de hipersensibilidad al producto o sus componentes, como anafilaxia, que ponen en riesgo la vida.

Advertencias y precauciones

Es posible que NUWIQ cause reacciones de hipersensibilidad, como anafilaxia. Los primeros signos de reacciones de hipersensibilidad que pueden progresar y derivar en anafilaxia incluyen angioedema, opresión en el pecho, disnea, sibilancias, urticaria o prurito. Si se producen reacciones de hipersensibilidad, suspenda la administración de inmediato e inicie el tratamiento adecuado.

Se pueden formar anticuerpos neutralizantes (inhibidores) del factor VIII luego de administrar NUWIQ. Controle a todos los pacientes para detectar el desarrollo de inhibidores del factor VIII mediante observaciones clínicas y análisis de laboratorio adecuados. Si el nivel del factor VIII en plasma no aumenta según lo esperado o si la hemorragia no se controla luego de la administración de NUWIQ, se debe sospechar la presencia de un inhibidor (anticuerpo neutralizante).

Reacciones adversas

Las reacciones adversas más frecuentes (>5%) que tuvieron lugar en ensayos clínicos fueron infección del tracto respiratorio superior, dolor de cabeza, fiebre, tos, infección del tracto respiratorio inferior, rinitis, escalofríos, dolor abdominal, artralgia, anemia, y faringitis.

Consulte la Información de prescripción completa de NUWIQ. Instrucciones de uso

Indications and Important Safety Information for wilate^® [von Willebrand Factor/Coagulation Factor VIII Complex (Human)].

Please see wilate full Prescribing Information.

Indications

wilate^® is a von Willebrand Factor/Coagulation Factor VIII Complex (Human) indicated in children and adults with von Willebrand disease for on-demand treatment and control of bleeding episodes; for perioperative management of bleeding; and for routine prophylaxis to reduce the frequency of bleeding episodes in children 6 years of age and older and adults with VWD. wilate^® is also indicated in adolescents and adults with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes; and for on-demand treatment and control of bleeding episodes.

Contraindications

wilate^® is contraindicated in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reactions, to human plasma-derived products, any ingredient in the formulation, or components of the container.

Warnings and Precautions

Hypersensitivity Reactions

Hypersensitivity reactions may occur with wilate^®. Signs and symptoms include angioedema, burning and stinging at the infusion site, chills, flushing, generalized urticaria, headache, hives, hypotension, lethargy, nausea, restlessness, tachycardia, tightness of the chest, tingling, vomiting, and wheezing that may progress to severe anaphylaxis (including shock) with or without fever. Closely monitor patients receiving wilate^® and observe for any symptoms throughout the infusion period.

Because inhibitor antibodies may occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.

Thromboembolic Events

In VWD, continued treatment using a FVIII-containing VWF product may cause an excessive rise in FVIII activity, which may increase the risk of thromboembolic events. Monitor plasma levels of VWF:RCo and FVIII activities in patients receiving wilate to avoid sustained excessive VWF and FVIII activity levels.

Neutralizing Antibodies

VWD

Neutralizing antibodies (inhibitors) to FVIII and VWF in patients with VWD, especially type 3 patients, may occur. If a patient develops inhibitor to VWF (or to FVIII), the condition will manifest itself as an inadequate clinical response. Thus, if expected VWF activity plasma levels are not attained, or if bleeding is not controlled with an adequate dose or repeated dosing, perform an appropriate assay to determine whether a VWF inhibitor is present.
In patients with antibodies against VWF, VWF is not effective and wilate administration may lead to severe adverse events. Consider other therapeutic options for such patients.

Hemophilia A

Monitor plasma Factor VIII activity by performing a validated test (e.g., one stage clotting assay), to confirm that adequate Factor VIII levels have been achieved and maintained.
Monitor for the development of Factor VIII inhibitors. Perform a Bethesda inhibitor assay if expected Factor VIII plasma levels are not attained, or if bleeding is not controlled with the expected dose of Wilate. Use Bethesda Units (BU) to report inhibitor levels.

Transmissible Infectious Agents

wilate^® is made from human plasma. Because this product is made from human blood, it may carry a risk of transmitting infectious agents, e.g., viruses, and theoretically, the variant Creutzfeldt-Jakob disease (vCJD) agent. There is also the possibility that unknown infectious agents may be present in the product. The risk that wilate^® will transmit viruses has been reduced by screening plasma donors for prior exposure to certain viruses, by testing for the presence of certain current virus infections, and by inactivating and removing certain viruses during manufacture. Despite these measures, it may still potentially transmit disease.

Record the batch number of the product every time wilate^® is administered to a patient, and consider appropriate vaccination (against hepatitis A and B virus) of patients in regular/repeated receipt of wilate^®. ALL infections thought by a physician possibly to have been transmitted by this product should be reported by the physician or other healthcare provider to Octapharma USA, Inc., at 1-866-766-4860.

Monitoring and Laboratory Tests

Monitor plasma levels of VWF:RCo and FVIII activities in patients receiving wilate^® to avoid sustained excessive VWF and FVIII activity levels, which may increase the risk of thromboembolism, particularly in patients with known clinical or laboratory risk factors.
Monitor for development of VWF and FVIII inhibitors. Perform assays to determine whether VWF and/or FVIII inhibitor(s) is present if bleeding is not controlled with the expected dose of wilate^®.

Adverse Reactions

The most common adverse reactions to treatment with wilate (≥ 1%) in patients with VWD were hypersensitivity reactions, urticaria, chest discomfort and dizziness. The most common adverse reactions to treatment with wilate (≥ 1%) in previously treated patients with hemophilia A was pyrexia (fever).

Please see wilate full Prescribing Information.

Indicaciones e Información de seguridad importante de wilate^® [complejo de factor de von Willebrand/factor VIII de la coagulación (humanos)].

Consulte la Información de prescripción completa de wilate.

Indicaciones

wilate^® es un complejo de factor de von Willebrand/factor VIII de la coagulación (humanos) indicado en niños y adultos con enfermedad de von Willebrand para el tratamiento y el control a demanda de los episodios hemorrágicos, para el control perioperativo de las hemorragias y para profilaxis de rutina con el fin de reducir la frecuencia de los episodios hemorrágicos en niños de 6 años de edad en adelante y adultos con EvW. wilate^® también se indica en adolescentes y adultos con hemofilia A para profilaxis de rutina con el fin de reducir la frecuencia de los episodios hemorrágicos; y para el tratamiento y el control a demanda de los episodios hemorrágicos.

Contraindicaciones

wilate^® está contraindicado en pacientes con reacciones de hipersensibilidad conocidas, incluidas las reacciones anafilácticas o las reacciones sistémicas graves a productos derivados de plasma humano, cualquier ingrediente de la formulación o los componentes del envase.

Advertencias y precauciones

Reacciones de hipersensibilidad

Pueden producirse reacciones de hipersensibilidad con el uso de wilate^®. Los signos y síntomas incluyen angioedema, ardor y picazón en el lugar de la infusión, escalofríos, sofoco, urticaria generalizada, dolor de cabeza, ronchas, hipotensión, letargo, náuseas, inquietud, taquicardia, opresión en el pecho, hormigueo, vómitos y sibilancias que pueden progresar y derivar en anafilaxia grave (incluido shock) con o sin fiebre. Controle de cerca a los pacientes que reciban wilate^® y preste atención a cualquier síntoma durante todo el período de infusión.

Debido a que los anticuerpos inhibidores pueden desarrollarse de forma concomitante con las reacciones anafilácticas, evalúe a los pacientes que experimenten una reacción anafiláctica para detectar la presencia de inhibidores.

Eventos tromboembólicos

En la enfermedad de von Willebrand, el tratamiento continuo con un producto de FvW que contiene FVIII puede provocar un incremento excesivo en la actividad del FVIII, y eso puede aumentar el riesgo de eventos tromboembólicos. Controle los niveles en plasma de las actividades del FvW:RCo y el FVIII en los pacientes que reciben wilate para evitar niveles de actividad excesivos y sostenidos del FvW y el FVIII.

Anticuerpos neutralizantes

EvW

Pueden desarrollarse anticuerpos neutralizantes (inhibidores) del FVIII y del FvW en pacientes con enfermedad de von Willebrand, especialmente en los pacientes de tipo 3. Si un paciente desarrolla un inhibidor del FvW (o del FVIII), la afección se manifestará como una respuesta clínica inadecuada. Por lo tanto, si no se alcanzan los niveles en plasma esperados de la actividad del FvW o si la hemorragia no se controla con una dosis adecuada o repetida, realice la prueba correspondiente para determinar la presencia de un inhibidor del FvW.
En los pacientes con anticuerpos contra el FvW, este no es eficaz y la administración de wilate puede provocar eventos adversos graves. Considere otras opciones de tratamiento para dichos pacientes.

Hemofilia A

Controle la actividad del factor VIII mediante un análisis validado (por ejemplo, una prueba de coagulación de una etapa), para confirmar que se hayan alcanzado y mantenido los niveles del factor VIII.
Controle el desarrollo de inhibidores contra el factor VIII. Realice una prueba del inhibidor Bethesda si no se alcanzan los niveles en plasma esperados del factor VIII o si la hemorragia no se controla con la dosis esperada de Wilate. Utilice la unidad Bethesda (UB) para informar los niveles del inhibidor.

Agentes infecciosos transmisibles

wilate^® está hecho a partir de plasma humano. Debido a que este producto está hecho de sangre humana, puede implicar un riesgo de transmisión de agentes infecciosos, como virus y, en teoría, el agente de la variante de la enfermedad de Creutzfeldt-Jakob (vCJD). También existe la posibilidad de que haya agentes infecciosos desconocidos en el producto. El riesgo de que wilate^® transmita virus se ha reducido mediante la realización de pruebas de detección a los donantes de plasma para detectar la exposición previa a determinados virus, mediante análisis para detectar la presencia de ciertas infecciones virales actuales y mediante la desactivación y eliminación de determinados virus durante la fabricación. A pesar de estas medidas, aún existe la posibilidad de que transmita enfermedades.

Registre el número de lote del producto cada vez que wilate^® se administre a un paciente y considere aplicar las vacunas adecuadas (contra los virus de la hepatitis A y B) a los pacientes que reciban wilate® de manera regular o repetida. TODAS las infecciones que un médico considere que se pueden haber transmitido a través de este producto deben ser informadas por el médico u otro proveedor de atención médica a Octapharma USA, Inc., al 1-866-766-4860.

Control y análisis de laboratorio

Controle los niveles en plasma de las actividades del FvW:RCo y el FVIII en los pacientes que reciban wilate® para evitar niveles de actividad excesivos del FvW y el FVIII, que pueden aumentar el riesgo de tromboembolia, especialmente en pacientes con factores de riesgo clínicos o de laboratorio conocidos.
Realice controles para detectar el desarrollo de inhibidores del FvW y el FVIII. Realice pruebas para determinar la presencia de inhibidores del FvW o el FVIII si la hemorragia no se controla con la dosis esperada de wilate^®.