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Vasculitis Webinar

Managing vasculitis: A patient’s perspective

In March 2017, I recorded this webinar for the Vasculitis Foundation’s Road Map to Wellness webinar series.  In the webinar, I talk about my diagnosis with EGPA/Churg-Strauss vasculitis and the first few years of my treatment course. I also talk about some of my approaches for tracking symptoms, working with doctors, and strategies for managing vasculitis. https://youtu.be/iYQWI1Wtruo For more about my disease course.  For a detailed summary of my diagnosis with EGPA/Churg-Strauss Syndrome, see here.  For a  summary of my treatment course through April 2018, see here.

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Step one: My Diagnosis with EGPA/Churg-Strauss Vasculitis

Below is a summary of the events leading to my diagnosis with EGPA/Churg-Strauss Syndrome, a rare form of vasculitis, in 2014.  Looking back, I can see worsening symptoms developing for about a year prior to my diagnosis.  Getting a definitive diagnosis can be a long and difficult road for many vasculitis patients.  I know how lucky I am to have gotten a diagnosis, and in a relatively short period of time. Before EGPA/Churg-Strauss Syndrome. Life was good before EGPA/Churg-Strauss.  I was active, worked long hours, and was always on the go.  I never got sick, never missed a day of work.  I was able to do anything and everything I wanted to do.  Then a series of strange things started to happen… Adult-onset asthma.  As a kid I was pretty healthy. I had the occasional stomach bug or respiratory infection, and some seasonal allergies, but nothing serious and no major illnesses or hospitalizations.  In my thirties, I developed asthma and a persistent annoying cough, and my allergies evolved into year round stuffiness and congestion.  My asthma doctor gave me every form of inhaler, nasal spray, antihistamine, and decongestant on the market but none of them seemed to help much. I just dealt with it. Laryngitis and vocal cord damage.  One day, I got an upper respiratory infection and an acute case of laryngitis.  I remember exactly when this happened because I was at a research conference in New York and had to give a presentation in a near whisper from the podium.  I made it through the talk, but my voice did not recover completely afterwards.  I struggled with my voice while teaching during the next semester, then I sought out an ENT who was a vocal cord specialist over the summer of 2013 to see what was going on.  He visualized my vocal cords using a camera (by laryngoscopy – not fun!) and he told me he could see exactly what the problem was – my right vocal cord was partially paralyzed!  He said this was due to a problem with my vagus nerve which controls the vocal cord nerves. My right vocal cord was too weak and would not line up with the left which created a big gap between the two cords that was making it difficult for me to speak.  I had MRIs of my brain and neck to rule out any signs of compression of the nerves controlling my right vocal cord such as a tumor or other anatomical problem, but he could not identify what was the underlying reason for the paresis. The MRI did, however, show signs of chronic rhinosinusitis, which I shrugged off.  He performed surgery (medialization thyroplasty) to insert an implant in my right vocal cord in order to reposition the cord to improve my voice.  The surgery definitely improved my voice, but I continue to have problems, especially in low humidity and in the wintertime, sometimes when I’m having a flare, and when I need to project my voice in noisy environments or large classrooms. Lung problems and skin rashes.  A few months after my vocal cord surgery, in the fall of 2013, I started to develop lung congestion.  I kept thinking it could be a respiratory infection, or seasonal allergies, but the congestion persisted and increased to the point where I was coughing up quite a lot of sputum all day long.  At the same time, strange rashes appeared on my lower legs, which I now know were purpura. My asthma doctor gave me a course of antibiotics but the congestion did not improve.  When she gave me a short course of prednisone (my first time ever taking prednisone), I felt relief within hours and the lung congestion cleared up immediately.  The congestion quickly began to return a few weeks after I finished the course of prednisone. Bronchiectasis.  After taking a second antibiotic which did nothing for the congestion, my asthma doctor ordered a chest CT.  The chest CT showed I had bronchiectasis, an unusual lung condition, which is a form of COPD.  When I read about bronchiectasis I found that it is caused by chronic inflammation and can result in permanent damage to the airways and loss of lung capacity.  Since I am in a big city with several university hospitals, I was able to find experts in bronchiectasis nearby.  Coincidentally, the pulmonologist who specialized in bronchiectasis was at the same university as my ENT/vocal cord specialist. Time to get serious.  At this point, in March of 2014, a lightbulb went off in my head.  I had spent the last four months with persistent lung congestion and shortness of breath, as well as just feeling plain lousy all the time.  Antibiotics did not help but the prednisone worked wonders, even though the congestion kept coming back as soon as I stopped the prednisone.  I knew there was something really wrong with me and I needed to get to the bottom of it.  I would spend the next six months learning everything I could about my symptoms and going back and forth between different specialists for lots of tests. Eosinophilia.  The lung doctor ran a whole bunch of pulmonary function tests, sputum cultures, imaging, and bloodwork, including screening for cystic fibrosis.  Other than some mildly reduced lung function, everything came back normal except for one thing – my blood count showed very high levels of eosinophils.  Eosinophils (the “E” in EGPA) are a type of white blood cell which are important for controlling allergies and parasitic infections.  This turned out to be a very important clue since eosinophilia is unusual and there are a limited number of conditions that can cause high eosinophils.  The lung doctor continued with additional tests and referred me to an immunologist to help track down the cause of the eosinophilia. Peripheral neuropathy.  A few months later, in August of 2013, I began experiencing pains in my right wrist and hand, as well as numbness and tingling in my hands and fingers.  I started dropping things

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Living with #raredisease EGPA: Eosinophilic Granulomatosis with Polyangiitis

Disclosure:   I received a small sum from GSK for shooting this video, which I donated to charity.  For more details and a full list of disclosures, please see the Disclaimer page. This 2 min video was recorded at GSK’s offices at the Navy Yard in Philadelphia last month as part of their educational efforts about EGPA/Churg-Strauss vasculitis.  It was filmed for a social media campaign to raise awareness about living with rare diseases like EGPA. Thanks to GSK for their outreach and educational programs on EGPA and other rare diseases, and many many thanks for their work and dedication in developing new treatments for eosinophilic disorders, including EGPA. From GSK’s website: Meet Jennifer: A science researcher whose quest became her own health As a biomedical researcher at a major medical institution, Jennifer’s happiest days are spent reading, learning, and working on discovering something that will help improve people’s health. While her research includes rare diseases, she never imagined that she’d one day put her skills to use on herself, to unlock the strange mystery going on inside her body. Aside from having allergies, Jennifer had a healthy childhood. As her allergies became progressively worse in her 30s and 40s, she also developed asthma. Then things started to get strange. She developed a host of respiratory symptoms, skin rashes, headaches and something called eosinophilia, which is a type of white blood cell in allergy and asthma, the levels of which were unusually high in Jennifer. It had taken Jennifer six months of testing, visiting multiple specialists including Respiratory and Immunology specialists when a Rheumatologist finally diagnosed her with a rare disease, Eosinophilic Granulomatosis with Polyangiitis (EGPA), formerly known as Churg-Strauss syndrome. EGPA affects about 5,000 Americans. It is characterized by asthma, an increase in eosinophils, and inflammation of blood vessels (vasculitis). “When I was diagnosed, I felt very relieved, but then quickly realized I was in this very serious situation. I had this rare disease, there was no cure, and I was going to have to face this for the rest of my life.”  -Jennifer Today, Jennifer plays a proactive role in managing her disease with her healthcare team. “My role in the team is that I am the quarterback,” said Jennifer. “My Rheumatologist is the coach and the other specialists are special teams. I am the one that has to get in there and make the plays, take the hits, and I have to get back up and get back in for the next play.” To help her prepare, Jennifer makes clear notes of what’s going on in her life from what’s working well to what’s not and she brings a summary to her doctor’s appointments. She makes sure that if she feels there’s something wrong, she raises the issue so that it can be addressed. Having a support system around her also helps her stay healthy. If Jennifer could offer advice to other patients, it’s the following: “I think it is really important to take the time to reach out to the people who are supporting you,” said Jennifer. “We know when we’re not feeling well, we know when we suspect a flare, and we know when a doctor is listening to us.  Build the people and team around you that will help you to move forward, because you can get there. Pay attention to your gut and if you are not feeling well, keep pursuing the answers.” You can view the post and video on GSK’s website at the following link:

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Tapers, treatments, and flares: My treatment course for EGPA/Churg-Strauss Vasculitis (Oct 2014 – Apr 2018)

NOTE:  This is not a typical treatment course for patients with EGPA/Churg-Strauss (not that there is such a thing as a “typical” EGPA patient!).  It has taken several years and much trial and error to find a combination of medications to try and keep my immune system under control and it’s a work in progress.  One of the most critical factors is to work with clinicians who are experts in treating patients with your form of vasculitis, especially for rare forms of vasculitis like EGPA/Churg-Strauss.  If you are newly diagnosed or having setbacks in your treatment, please do not be discouraged by all of my ups and downs.  Instead, I hope what you will see from my treatment course is that: 1) there are always more options for treatment; 2) progress is possible if you work with experienced clinicians and develop a treatment plan you can commit to; and 3) have some patience, it may take a while. Disclosure:  I was a patient in a GSK-sponsored clinical trial from March 2015 to May 2016 testing mepolizumab in EGPA/Churg-Strauss patients. For more details and a full list of disclosures, please see the Disclaimer page. Flare #7.  I am in my seventh flare since I started treatment for EGPA/Churg Strauss three and a half years ago, so I thought the Zen proverb above suits the occasion.  Below is a summary of my prednisone tapers and vasculitis flares, and the steps taken to find a combination of immunosuppressive treatments to help control the disease activity.  I’ve summarized all of this information in the graph to give a better sense of the literal ups and downs that I’ve been through during my treatment so far.  Even though, as my rheumatologist says, “past performance is no indicator of future results”, I’m pretty sure that this is not going to be my last flare. And that’s okay. Figure legend.  Treatment course showing prednisone tapers, clinical symptoms, disease flares, and treatments received from October 2014 – April 2018.  The graph shows my prednisone dose (mg/day) in red.  I scored my clinical symptoms using the Birmingham Vasculitis Activity Score (BVAS) and BVAS scores are shown on the graph in black.  The dotted line across the graph shows the level of 20 mg prednisone per day – my flares always occurred when I reached this point during my first two years of treatment.  Flares are noted by red arrows at the top of the graph.  The addition of methotrexate, mepolizumab, rituximab, and IVIG treatments are shown at the top of the graph.  Note that the first three mepolizumab injections were received while I was enrolled in a double blind clinical trial in which I was randomized to the placebo control group so these injections were saline only, but subsequent monthly mepolizumab injections are the actual drug. Diagnosis.  I was diagnosed with EGPA/Churg-Strauss Syndrome, a rare and potentially life threatening form of vasculitis, in the fall of 2014.  At the time, I thought that getting the diagnosis was going to be the hard part.  Boy, was I wrong.  I was diagnosed by a rheumatologist who is one of the world experts in EGPA/Churg-Strauss and just happens to be located in Philadelphia where I live. My symptoms included chronic rhinosinusitis, adult-onset asthma, pulmonary congestion, bronchiectasis, eosinophilia, mononeuritis (vocal cord paresis), peripheral neuropathy, purpura, hip joint pain, fatigue. Except for eosinophilia, my bloodwork was otherwise normal for inflammatory markers or antibodies, i.e., a normal ESR, normal CRP, and a negative ANCA titer. Initial treatment approach – prednisone, azathioprine, methotrexate.  I was extremely fortunate to get an early diagnosis so I had only suffered some mild nerve and lung damage, but the potential for further damage to my lungs, nerves, and possibly other organs, was high.  For this reason, my rheumatologist felt it was important take an aggressive approach to control the inflammation.  I would begin a treatment course familiar to many patients with vasculitis – starting with high doses of prednisone, a very powerful and very fast acting anti-inflammatory corticosteroid, followed by the addition of a second drug for maintenance immunosuppression, such as azathioprine or methotrexate. The prednisone would then be tapered gradually once the second drug was on board to control further inflammation. I began at 60 mg prednisone (a target of 1 mg/kg is the typical induction dose and I was 52 kg).  I also started taking Bactrim to help prevent any possible respiratory infection due to having a suppressed immune system.  Azathioprine was then added, but had to be discontinued within a few weeks due to severe GI intolerance.  We switched to methotrexate and chose to use weekly subcutaneous injections to minimize possible GI side effects. After reaching maximum therapeutic doses of methotrexate for a month, we began tapering the prednisone.  When I got down to 20 mg per day of prednisone, I had a flare, meaning that symptoms of active disease returned which included peripheral neuropathy, hip joint pain, fatigue, and purpura on my lower legs.  Due to the flare, my prednisone was increased to 50 mg per day for one month. Mepolizumab clinical trial.  At this point, it seemed the methotrexate was not enough to control the vasculitis, so my next option would be to add a stronger immunosuppressant such as rituximab, which depletes B cells.  Another alternative was an experimental drug, mepolizumab, which was being tested in a Phase III clinical trial to specifically target the eosinophils (the “E” in EGPA).  My rheumatologist was participating in the trial and offered me a chance to enroll. This was the first trial of its kind for EGPA.  It was a randomized double blind study, meaning that I would be randomly selected to receive mepolizumab or placebo control (saline) but neither my doctor nor I would know whether I was receiving the drug or the placebo control. After discussion with my rheumatologist, I decided to enroll in the trial for three reasons:  1) because the previous clinical trials testing mepolizumab in asthma patients showed a good safety profile

Tapers, treatments, and flares: My treatment course for EGPA/Churg-Strauss Vasculitis (Oct 2014 – Apr 2018) Read More »

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How do the IL-5 inhibitors Cinqair, Fasenra, and Nucala work?

The IL-5 inhibitors Cinqair (reslizumab), Fasenra (benralizumab), and Nucala (mepolizumab) block IL-5 and help control eosinophils.  How do they work and what are the differences between them? Why target IL-5? IL-5 is a cytokine, a small signaling molecule.  IL-5 is produced and secreted by T cells and mast cells.  The IL-5 molecules can then signal to other blood cells including eosinophils.  IL-5 binds to the IL-5 receptor present on the surface of eosinophils and activates them.  IL-5 is the major activator of eosinophils so modulating IL-5 is a good way to try to control eosinophils. Available IL-5 inhibitors.  There are currently three commercially available IL-5 inhibitors in the United States.  These include reslizumab (trade name Cinqair), benralizumab (trade name Fasenra), and mepolizumab (trade name Nucala).  All three of these medications are humanized monoclonal antibodies in the class of medications known as biologics.  The “zumab” at the end of their names stands for humanized monoclonal antibody.  All three medications have been approved by the FDA for the treatment of severe eosinophilic asthma.  In addition, mepolizumab (Nucala) has also been approved for the treatment of EGPA/Churg-Strauss Syndrome, but at a higher dose than the dose used for the treatment of eosinophilic asthma. Figure legend – Inhibiting the lL-5 pathway.  Left:  IL-5 molecules normally bind to the IL-5 receptor on eosinophils, which causes activation or stimulation of the eosinophils.  Center:  Reslizumab (Cinqair) and mepolizumab (Nucala) bind to the IL-5 molecules and prevent the IL-5 molecules from binding to the IL-5 receptor.  This sequesters the IL-5 molecules and prevents them from activating their target receptor.  Right:  Benralizumab (Fasenra) works by a different mechanism.  Benralizumab (Fasenra) binds to the IL-5 receptor and prevents the IL-5 molecules from binding to the IL-5 receptor. Download a pdf of the image above here. What is the effect of inhibiting the IL-5 pathway?  Reslizumab (Cinqair) and mepolizumab (Nucala) bind to IL-5 directly.  Benralizumab (Fasenra) works by a different mechanism by binding to the IL-5 receptor and preventing IL-5 from binding.  The end result is similar in that all three medications inhibit the IL-5 receptor from being activated.  This inhibits eosinophil stimulation and activation. The absence of eosinophil stimulation and activation causes eosinophil levels to decrease.  All three IL-5 inhibitors have been shown to decrease eosinophil levels in the blood of patients with eosinophilia. Comparing efficacy of IL-5 inhibitors.  There have been no clinical trials comparing these medications to each other for the control of eosinophilia and these trials are not likely in the future.  Decisions on which inhibitor to use for eosinophilic asthma are therefore likely to be empirical.  If one inhibitor does not seem to be effective in controlling eosinophilia, or causes side effects, then patients may have better results with one of the others.  In particular, if reslizumab (Cinqair) or mepolizumab (Nucala) are not effective or cause side effects, then benralizumab (Fasenra) is a possible alternative as it works by a slightly different mechanism.  Side effects can sometimes be caused by the formulation or additives during the manufacturing process, so a patient with side effects when taking mepolizumab (Nucala) may better tolerate the reslizumab (Cinqair), or vice versa. View a video of this post on YouTube here.

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The MIRRA trial: Mepolizumab (Nucala) or placebo for the treatment of EGPA/Churg-Strauss Syndrome

Wechsler et al. Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis. N. Engl. J. Med. 2017 376(20):1921-1932. Disclosure:  I was a patient in this clinical trial from March 2015 to May 2016 and I am still taking mepolizumab to treat my EGPA. For more details and a full list of my disclosures, please see the Disclaimer page. Game changer:  Why is this study important? This clinical trial was the first randomized controlled trial ever conducted for EGPA. It is also one of the largest clinical trials conducted for EGPA, requiring the participation of 31 trial sites to enroll enough patients with this rare disease.  Mepolizumab is the first medication to be approved by the FDA for the treatment of EGPA.  The results from this clinical trial supported the application for FDA approval which was granted in December 2017. Trial sponsorship.  The trial was sponsored by a collaboration between GlaxoSmithKline and the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health. NIAID funded an investigator initiated clinical trial application, which provided support for five of eight clinical trial sites in the United States.  Disclosures related to the MIRRA trial and the published manuscript can be found in the supplemental data on the manuscript publisher’s website at the links provided at the bottom of this post. The big picture.  This Phase III double blinded clinical trial tested mepolizumab (Nucala), a monoclonal antibody which targets IL-5, an important regulator of eosinophils, for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA).  The study focused on patients with EGPA who had relapsing disease (flares) or difficult to control symptoms (refractory disease). Purpose of the study and study endpoints.  Patients with EGPA have high levels of eosinophils. Mepolizumab is a monoclonal antibody which targets IL-5, an important regulator of eosinophils, and reduces eosinophil levels in the blood, so it may be useful to help control symptoms related to EGPA.  The purpose of the study was to measure whether mepolizumab could lead to better remission in patients with EGPA compared to controls. The end points of the study were the number of weeks patients were in remission (no evidence of disease activity) and the number of patients in remission at weeks 36 and 48 of the trial. Patient population.  Patients diagnosed with EGPA who had relapsed (had a flare) or were unable to go into remission (refractory disease) and were at least 18 years of age were eligible to be enrolled in the study.  136 patients were enrolled at 31 sites across nine countries between February 2014 and September 2016. Outline of the study.  This was a Phase III clinical trial studying patients diagnosed with EGPA who were receiving typical treatment for their EGPA and who had relapsed (had a flare) or were unable to go into remission (refractory disease).  136 adult EGPA patients were randomized into two groups of 68 patients per group.  One group received their regular treatments with prednisone and other drugs like methotrexate or azathioprine (standard of care), plus monthly injections of mepolizumab (300 mg subcutaneously). The second group received their regular treatments plus a monthly subcutaneous injection of saline (placebo control).  Treatments were given monthly for one year (52 weeks).  The study was double-blinded, meaning that neither the patients nor their doctors knew whether they were receiving injections of the placebo or the drug.  Patients were monitored monthly and their EGPA symptoms were measured and scored.  Patients began a controlled taper of their daily prednisone dose one month after their first injection and continued tapering their prednisone doses according to a defined schedule.  The level of the symptoms and the number of weeks in remission (i.e. no major relapses or worsening symptoms) were compared between the mepolizumab and placebo control groups. Mepolizumab led to more patients in remission than placebo.  Patients who received mepolizumab had more weeks with no EGPA symptoms (remission) than the patients who received the placebo, which allowed the patients to reduce the amount of prednisone they were taking.  However, only about half of the patients who received mepolizumab had their symptoms reduced enough to go into remission.  This means that mepolizumab could control symptoms and lead to remission in about half of the patients, but mepolizumab did not completely control symptoms in the other half of the patients.  Here is a quote from the abstract’s “conclusions” which summarized these results: mepolizumab resulted in significantly more weeks in remission and a higher proportion of participants in remission than did placebo, thus allowing for reduced glucocorticoid use. Even so, only approximately half the participants treated with mepolizumab had protocol-defined remission Mepolizumab helped patients remain in remission compared to patients in the placebo group. Patients taking mepolizumab had more weeks in remission than patients on placebo (28% vs. 3%).  The patients taking mepolizumab were also more likely to be in remission at the end of the trial than patients in the placebo group (32% in remission for the mepolizumab group vs. 3% for the placebo group at week 48 of the trial). As stated in the abstract’s “results” section: mepolizumab treatment led to significantly more accrued weeks of remission than placebo (28% vs. 3% of the participants had ≥24 weeks of accrued remission; odds ratio, 5.91; 95% confidence interval [CI], 2.68 to 13.03; P<0.001) and a higher percentage of participants in remission at both week 36 and week 48 (32% vs. 3%; odds ratio, 16.74; 95% CI, 3.61 to 77.56; P<0.001) Mepolizumab also helped patients reduce the total dose of prednisone they were taking.  About half of the patients receiving mepolizumab were able to reduce their daily dose of prednisone to 4 mg or less per day by the end of the study, while only 7% of patients in the control group reached 4 mg or less of prednisone per day. This was stated in the abstract’s “results” section: a total of 44% of the participants in the mepolizumab group, as compared with 7% of those in the placebo group, had an average daily dose of prednisolone or prednisone of 4.0

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The GiACTA trial: Tocilizumab in Giant Cell Arteritis

Stone et al. Trial of Tocilizumab in Giant-Cell Arteritis. N. Engl. J. Med. 2017 377(4):317-328. NEJM Quick Take:  Click on the following link from the New England Journal of Medicine to view a 2 minute video summarizing the study results: video. Game changer:  Why is this study important? Tocilizumab is the first treatment, other than corticosteroids, shown to be effective for giant cell arteritis.  Giant cell arteritis can cause very serious problems including blindness and aortic aneurysms. Corticosteroids like prednisone cause lots of side effects, especially when used long term, so it is important to limit the amount of prednisone. This clinical trial was the first large scale randomized controlled trial to test tocilizumab for the treatment of giant cell arteritis.  Tocilizumab was approved by the FDA for the treatment of giant cell arteritis in May of 2017 based on the results of this trial. Trial sponsorship.  The trial was sponsored by Hoffman-La Roche (Roche), which provided the study drug and support for the clinical trial sites.  Disclosures related to the GiACTA trial and the published manuscript can be found in the supplemental data on the manuscript publisher’s website at the links provided at the bottom of this post. The big picture.  The Giant Cell Arteritis Actemra (GiACTA) trial was a Phase III double blinded clinical trial testing tocilizumab (Actemra) for giant cell arteritis.  Tocilizumab is a monoclonal antibody which binds to the IL-6 receptor alpha and blocks the cytokine IL-6 from binding to its receptor.  IL-6 is a pro-inflammatory cytokine that is elevated in several inflammatory diseases including giant cell arteritis.  The study focused on patients with giant cell arteritis who were newly diagnosed with giant cell arteritis or who had relapsing disease (flares) when tapering prednisone. Purpose of the study and study endpoints.  Patients with giant cell arteritis have high levels of inflammation markers such as erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP).  They also have high levels of some specific cytokines including IL-6, which is a cytokine that is known to cause inflammation (pro-inflammatory cytokine) and can induce CRP. Patients with giant cell arteritis are usually treated with prednisone to control the inflammation, but often have relapses or flares when they try to taper their prednisone levels.  Tocilizumab is a monoclonal antibody which blocks the function of IL-6 by binding to its receptor (IL-6 receptor alpha) so it may be useful to help control symptoms related to IL-6 induced inflammation.  The purpose of the study was to measure whether tocilizumab could lead to better remission in patients with giant cell arteritis compared to patients receiving prednisone alone. The end points of the study were the number of patients who were in remission in each study group at the end of the 52 week trial. Remission was defined as a normal CRP and the absence of a flare (a flare was defined as a return of signs and symptoms and an elevated ESR). Patient population.  Patients who were newly diagnosed with giant cell arteritis or who had relapsed (had a flare) and were 50 years or older were eligible to be enrolled in the study.  251 patients were enrolled at sites in fourteen countries starting in July 2013 and the study was completed in April 2016. Outline of the study.  This was a Phase III clinical trial studying patients diagnosed with giant cell arteritis who were newly diagnosed or who were treated with prednisone and had relapsed (had a flare) when they tried to taper their prednisone.  The study was scheduled to last 52 weeks.  251 adult patients with giant cell arteritis were randomized into four groups.  The first group (100 patients) received weekly subcutaneous injections of tocilizumab (TCZ) plus high dose daily prednisone and began a controlled taper of their daily prednisone dose down to 0 according to a defined schedule over 26 weeks.  The second group received subcutaneous injections of tocilizumab every two weeks while tapering their prednisone over 26 weeks.  The third group (50 patients) received prednisone but no tocilizumab while tapering their prednisone over 26 weeks, and the fourth group of patients (51 patients) received prednisone but no tocilizumab and tapered their prednisone over 52 weeks. Patients were followed for one year (52 weeks).  Patients in the second, third, and fourth groups received subcutaneous injection of saline (placebo control) on weeks they were not scheduled to receive tocilizumab.  The study was double-blinded, meaning that neither the patients nor their doctors knew whether they were receiving injections of the placebo or the drug.  Patients were monitored monthly and their prednisone doses and giant cell arteritis symptoms were measured and scored.  The number of patients in remission (i.e. no major relapses or worsening symptoms) and the cumulative prednisone dose were compared between the two tocilizumab groups and the two placebo control groups. Tocilizumab led to more patients in remission than prednisone alone.  More patients who received tocilizumab were in remission at 52 weeks than the patients who only received prednisone.  A little more than half (53-56%) of the patients who received tocilizumab had their symptoms reduced enough that they were in remission without prednisone at the end of the study, while only 14-18% of patients taking prednisone alone were in remission at the end of the study.  This means that tocilizumab alone could not control symptoms enough to keep the other half of the patients (44-47%) in remission at the end of the study.  Here is a quote from the abstract’s “results” which summarized these results: Sustained remission at week 52 occurred in 56% of the patients treated with tocilizumab weekly and in 53% of those treated with tocilizumab every other week, as compared with 14% of those in the placebo group that underwent the 26-week prednisone taper and 18% of those in the placebo group that underwent the 52-week prednisone taper (P<0.001 for the comparisons of either active treatment with placebo). Tocilizumab also helped patients reduce the total dose of prednisone they were taking.  The patients receiving tocilizumab who went into remission were able to reduce the

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