Soliris (eculizumab) For the Treatment of PNH to Reduce Hemolysis

Transcription

1 Soliris (eculizumab) You have done well to manage your PNH with Soliris. Living with a rare disease, like PNH, can be challenging, but by sticking with your Soliris therapy, you are doing a lot to help yourself. Soliris works to reduce the signs, symptoms, and health risks associated with PNH. This brochure provides helpful tips for ongoing management of your PNH with Soliris therapy. For the Treatment of PNH to Reduce Hemolysis

2 Living with a rare disease, like PNH, can be challenging, but by following your prescribed management plan for Soliris therapy, you are doing a lot to help yourself. Soliris works to reduce the signs, symptoms, and health risks associated with PNH. This brochure provides helpful tips for managing your PNH with Soliris therapy.

3 Indication and Usage Paroxysmal Nocturnal Hemoglobinuria (PNH) Soliris is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: SOLIRIS ( ) or at Common side effects in people with PNH treated with Soliris include headaches, runny nose and colds, sore throat, back pain, and nausea.

4 What are the long-term consequences of PNH? Our commitment Alexion s objective is that every patient with PNH who can benefit from Soliris will have access to Soliris. PNH is a lifelong disease. Once it develops, most patients have it for life 1 In PNH, the symptoms you can t see or feel can hurt you the most. Blood clots (potentially leading to stroke or heart attack) 2-4 Kidney disease 2 Damage to your organs 5 PNH is constant. Even though you can t see it, destruction of blood cells, or hemolysis, is always happening. Ongoing hemolysis is the leading cause of the signs, symptoms, and serious health problems of PNH 1 PNH is manageable. Work with your doctor on a management plan that may include Soliris 1 If you would like more in-depth information about PNH, for yourself or to share with others, call OneSource TM at SOLIRIS ( ). An Alexion Nurse Case Manager can assist you with additional education and helpful resources. Speak to your physician about your signs, symptoms, and lab results for a complete picture of your PNH. 2

5 What are the implications of hemolysis? Because of changes in your bone marrow, your red blood cells lack important proteins that protect them from a part of the immune system called complement 6 Hemolysis leads to the serious and potentially life-threatening consequences of PNH 6 Without these key proteins, your red blood cells can be attacked by complement 6 This leads to hemolysis the destruction of your red blood cells 6 PNH clone size 7 The % of your blood cells that lack these protective proteins is sometimes called a PNH clone size. Your doctor may routinely monitor your clone size, as it can change over time. Blood clots (thrombosis) in different places in your body 2,4 Anemia and the need for blood transfusions 5 Other bone marrow disorders Chronic kidney disease and/or damage to your other organs 1,2,5 Additional clinical manifestations, including 2,5 : Fatigue Abdominal pain Difficulty swallowing Shortness of breath Erectile dysfunction Difficulty performing daily activities PNH may overlap with other conditions such as aplastic anemia (AA) and myelodysplastic syndrome (MDS), sometimes called bone marrow failure disorders. 8 Your doctor will talk to you about the best approach to manage PNH and any symptoms you may have if there are issues with your bone marrow. 3

6 What is Soliris (eculizumab)? Soliris is the only drug approved by the Food and Drug Administration (FDA) for the treatment of patients with PNH to reduce hemolysis. 9 You ve decided to take an active role in the management of your disease and have started treatment with Soliris, the only drug approved by the FDA for the treatment of PNH to reduce hemolysis. 9 Soliris reduces hemolysis. Soliris works by inhibiting complement, a part of your body s defense system, from attacking red blood cells that are missing an important protein. This can help reduce hemolysis and relieve the symptoms of PNH 9 Soliris provides clinical benefits. In clinical trials, patients with PNH experienced significant reductions in hemolysis and fewer episodes of thrombosis. They also generally experienced improvements in their fatigue and other signs and symptoms of PNH 9,10 Soliris should be taken at the recommended dosing schedule to reduce hemolysis. A well-managed therapy schedule keeps Soliris at the levels necessary to inhibit the destruction of your red blood cells 9 You must receive meningococcal vaccines at least 2 weeks before your first dose of Soliris; consult your physician regarding appropriate vaccination guidelines 9 Vaccination is important because Soliris may increase your chance of developing certain kinds of infections, such as meningitis. 9 In addition to receiving the vaccines recommended by your doctor, stay alert and notify him/ her if you develop any of these symptoms while on Soliris 9 : Headache with nausea or vomiting Headache and a fever Headache with a stiff neck or stiff back Fever Fever and a rash Confusion Muscle aches with flu-like symptoms Eyes sensitive to light 4

7 What are the benefits of staying on your dosing schedule? For Soliris to inhibit hemolysis, the drug needs to stay above a certain level in your blood. However, like most drugs, Soliris is broken down and eliminated by your body over time. Therefore, it is important to maintain a regular therapy schedule to achieve optimal results. 9 What could happen if I missed a dose? If you miss a Soliris infusion, call your doctor right away. Soliris needs to be infused according to the recommended dosing schedule for you to get the most out of your treatment. If the level of Soliris in? your body gets too low, hemolysis can occur. 9 Hemolysis is the underlying cause of the MYTH: major health TRUTH: problems in patients with PNH,1 and missing doses may cause hemolysis to worsen.9 Follow the recommended dosing schedule, and work closely with your doctor to best manage your PNH. MYTH: TRUTH: How is Soliris administered? Soliris is administered as an infusion into a vein, either via gravity feed, a syringe-type pump, or an infusion pump, to make sure it quickly gets to where it works your blood. 9 The infusion lasts approximately 35 minutes in adults. 9 Helpful Tip Planning for Travel Remember, your Nurse Case Manager can help when you plan to be away from home. As soon as you have booked your travel, let your Nurse Case Manager know and he/she will try to ensure that your Soliris infusions continue without interruption. Regular infusions are important to get the most from your Soliris therapy. Just call SOLIRIS about a month before you go. 5

8 How have your symptoms changed since you started receiving Soliris (eculizumab)? Individual results for patients with PNH on Soliris may vary, so the improvements you see in your health and your experiences with your therapy may differ from those of other people. 11 Consider keeping a journal of signs and symptoms to share with your doctor. When you track your signs and symptoms over time, you and your doctor can be more aware of changes in your health. Take note of how you re feeling every day! In clinical trials, patients with PNH taking Soliris experienced the following: 87% reduction in hemolysis as measured by lactate dehydrogenase (LDH) 12,b 92% fewer blood clots one of the serious health problems with PNH 10,b 73% reduction in the average number of blood transfusions 13,c Reduction in fatigue and improvements in health-related quality of life 9 In Soliris clinical trials, most patients received concomitant anticoagulants. The effect of anticoagulant withdrawal during treatment with Soliris has not been studied. Therefore, treatment with Soliris should not alter anticoagulant management. 9 For me, managing my PNH means feeling less tired and having the energy to spend quality time with my husband. Remembering my life with PNH before I started taking Soliris reminds me never to take everyday things like that for granted again. Abby a a Adapted from real patient story. b Based on a sample size of 195 patients. c Based on a sample size of 87 patients. 6

9 What are the benefits of continued Soliris therapy? EARLY BENEFITS LONG-TERM BENEFITS At 3 weeks 6,13,14 Improvement in fatigue and healthrelated QoL At 6 months 13 Stabilization of hemoglobin levels Reduction in transfusion requirements At 36 months and up to 4.5 years 12 Sustained reduction in hemolysis and blood clots a Sustained reduction in transfusion requirements Sustained improvement in fatigue and health-related QoL Consistent safety profile Reduction in blood clots with Soliris over time 12 A measure of hemolysis One way that your doctor can check your progress with Soliris is to order a simple LDH blood test. LDH measures hemolysis and helps determine optimal treatment intervals. 13,15 When your blood has less LDH, it means Soliris is working. Soliris can lower LDH in people with PNH right away and can maintain that level over time. 13 Common side effects in patients with PNH treated with Soliris include headaches, runny nose and colds, sore throat, back pain, and nausea. 9 a In Soliris clinical trials, most patients also received concomitant anticoagulants. 7

10 What can I do to stay committed to Soliris (eculizumab) therapy? Remember quick facts about Soliris infusion therapy Hemolysis is the cause of serious health problems in patients with PNH 1 Ongoing Soliris infusion therapy offers sustained results over time 9,12 To get the most from your treatment, maintain a regular Soliris therapy schedule 9 It is possible to successfully manage your PNH 9,12 UTH: Stay on schedule! Infusions can be a challenge to fit into your busy schedule, but it s important to remember that it is possible to successfully manage your PNH with sustained Soliris therapy. 9,12 Contact OneSource TM for information on the possibility of receiving Soliris infusions in the comfort of your own home. Home infusion must be approved by your health care provider Home infusion must be approved by your insurance provider If approved by your health care provider and insurance provider, OneSource TM Nurse Case Managers can help facilitate the arrangements Dealing with a rare disease can be lonely. But OneSource put me in touch with other people managing their PNH, just like me. It s been great to be able to share my experiences with my new friends they understand what it s like to live with PNH. Dan a a Adapted from real patient story. 8

11 How can I help maximize the benefits of my Soliris therapy? Helpful Tip Consider additional resources to help you remain on track You have been working hard to manage your PNH. To maximize the benefits of your Soliris therapy, it is important to keep up with your regular infusion schedule. If you aren t already doing so, consider utilizing resources such as a planner MYTH: or an electronic TRUTH: app to help MYTH: remind you of TRUTH: your infusion appointments. Keep up the great work! By maintaining a regular infusion schedule, tracking your symptoms, and effectively communicating with your doctor and Nurse Case Manager, you are taking the right steps to protect yourself against the possible health risks associated with PNH. Taking care of your health is something to be proud of!? If you are ever away from home, your Nurse Case Manager can review your insurance coverage with you and if your insurance allows help create a personalized plan that will fit into your travel schedule. 9

12 Indication and Important Safety Information 9 WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: SOLIRIS ( ) or at Indication and Usage Soliris is indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. Important Safety Information Contraindications Soliris should not be used in: Patients who have meningococcal infection Patients who have not been vaccinated against meningitis infection unless the doctor decides that urgent treatment with Soliris is needed Warnings and Precautions Soliris increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early. 1. You must receive meningococcal vaccination at least 2 weeks before your first dose of Soliris unless you have already had this vaccine. If your doctor decided that urgent treatment with Soliris is needed, you should receive meningococcal vaccination as soon as possible. 2. If you had a meningococcal vaccine in the past, you might need additional vaccination before starting Soliris. Your doctor will decide if you need additional meningococcal vaccination. 3. Meningococcal vaccines do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: 10

13 Indication and Important Safety Information (Continued) 9 Warnings and Precautions (Continued) Headache with nausea or vomiting Headache and a fever Headache with a stiff neck or stiff back Fever Fever and a rash Confusion Muscle aches with flu-like symptoms Eyes sensitive to light Monitoring Disease Manifestations After Soliris Discontinuation (Continued) Kidney problems Blood clots Difficulty breathing Thrombosis Prevention and Management Treatment with Soliris should not stop you from taking your anticoagulant medicine (medicine that keeps your blood from clotting). Helpful Tip Other Infections Soliris may also increase the risk of other types of serious infections. If your child is treated with Soliris, make sure that your child receives the following vaccinations: Streptococcus pneumoniae Haemophilus influenza type b (Hib) Monitoring Disease Manifestations After Soliris Discontinuation If you have PNH, your doctor will need to monitor you closely for at least 8 weeks after stopping Soliris. Stopping treatment with Soliris may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: Drop in the number of your red blood cell count Drop in your platelet count Confusion Chest pain Serious Allergic Reactions Serious allergic reactions can happen during your Soliris infusion. Tell your doctor or nurse right away if you get any of these symptoms during your Soliris infusion: Chest pain Trouble breathing or shortness of breath Swelling of your face, tongue, or throat Feel faint or pass out If you have an allergic reaction to Soliris, your doctor may need to infuse Soliris more slowly, or stop Soliris. MYTH: TRUTH: MYTH: TRUTH: Adverse Reactions Common side effects in people with PNH treated with Soliris include: Headaches Runny nose and colds Sore throat Back pain Nausea? Carry your Patient Safety Card with you at all times. Show it to any doctor or nurse who treats you. PATIENT SAFETY INFORMATION CARD Important Safety Information for Patients Taking Soliris Soliris can lower the ability of your immune system to fight infections, especially meningococcal infection, which requires immediate medical attention. If you experience any of the following symptoms, you should immediately call your doctor or seek emergency medical care, preferably in a major emergency medical care center: headache with nausea or vomiting headache and a fever headache with a stiff neck or stiff back fever fever and a rash confusion muscle aches with flu-like symptoms eyes sensitive to light! Get emergency medical care right away if you have any of these signs or symptoms and show this card. Even if you stop using Soliris, keep this card with you for 3 months after your last Soliris dose. Your risk of meningococcal infection may continue for several weeks after your last dose of Soliris. 11

14 Remember, your Nurse Case Manager is here for you to provide continued support. Call SOLIRIS ( ) for answers to any questions you may have or for information about: Health Insurance Navigation: Health insurance can be overwhelming, and OneSource TM can help you navigate the process Personalized Patient Support: OneSource TM can help you if you are traveling, changing your insurance, or experiencing a major life event that could affect your regular Soliris schedule, such as a change in family, job, provider, or insurance status Community Connections: if you would like to speak with someone who knows what you are going through, your Nurse Case Manager can put you in touch with another person also living with PNH References: 1. Sahin F, Ozkan M, Mete N, et al. Multidisciplinary clinical management of paroxysmal nocturnal hemoglobinuria. Am J Blood Res. 2015;5(1): Brodsky RA. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R, Benz EJ Jr, Silberstein LE, et al, eds. Hematology: Basic Principles and Practice. Philadelphia, PA: Saunders/Elsevier; 2013: Audebert HJ, Planck J, Eisenburg M, et al. Cerebral ischemic infarction in paroxysmal nocturnal hemoglobinuria: report of 2 cases and updated review of 7 previously published patients. J Neurol. 2005;252(11): Hill A, Kelly R, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013;121(25): Weitz I, Meyers G, Lamy T, et al. Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria. Intern Med J. 2013;43(3): Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4): Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18): Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. Hematology Am Soc Hematol Educ Program. 2011;2011: Soliris [Prescribing Information]. New Haven, CT: Alexion Pharmaceuticals; Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12): Sahin F, Yilmaz A, Ozkan M, et al. PNH is a debilitating, fatal but treatable disease: same disease, different clinical presentations. Am J Blood Res. 2015;5(1): Hillmen P, Muus P, Roth A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013;162(1): Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12): Hill A, Rother R, Wang X, et al. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010;149(3): Hill A, Hillmen P, Richards S, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005;106: WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: SOLIRIS ( ) or at com. Soliris is a registered trademark of Alexion Pharmaceuticals, Inc Alexion Pharmaceuticals, Inc. All rights reserved. US/SOL-PNH/17/0015a