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Smart Thinking blog

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World Castleman Disease Day

Thursday 23 July marks World Castleman Disease Day. Castleman disease (CD) is the name given to a group of rare and life-threatening lymphoproliferative disorders that affect the lymph nodes and related tissues.

Gary and Stacey

Gary Gravina (pictured above), 45, is a former marine, and had been a carpenter for 27 years at the time he was diagnosed with idiopathic Multicentric Castleman Disease (iMCD).

He described himself as healthy and active guy for a long time, taking up vigorous vocations and hobbies, but days after his diagnosis was at the point of struggling for his life on a ventilator and feeding tube in the ICU, with every major organ in his body failing, including his liver, kidney, spleen and lungs. Doctors told his wife Stacey, that they didn’t think he’d make it through the night.

He was treated at the Hospital at U Penn in the US, where his doctors could consult directly with Dr David Fajgenbaum, who has pioneered research in Castleman disease, and has recently marked three years in remission.

Gary’s story

I was a very healthy, active guy for a long time and had been a carpenter for 27 years when Castleman Disease knocked me down in just a few days to the point of struggling for my life on a ventilator and feeding tube in the ICU, with every major organ in my body failing. The fight from that hospital room back to a life I can enjoy and still laugh as I do today has been the hardest I’ve ever known.

Dr. David Fajgenbaum and the Castleman Disease Collaborative Network (CDCN) were directly responsible for the diagnostic criteria that my doctors used to take my critical state from defensive and stabilising measures to direct treatment of the problem. That diagnostic criteria was published on the very day I was admitted to the hospital and, earlier on the same day I began treatment, I began donating blood and tissue to the CDCN.

As a former Marine, I know what it's like to volunteer for something that could kill me. What I'd never experienced was volunteering for something that could save my life, and it was David and the CDCN who first offered me the opportunity to do that.

GaryAt that critical state lying in the ICU, just off the ventilator, tied by needles and tapes to a litany of chemicals and machines, venturing into my first tenuous bites of solid food in nearly a month, my mind was at times frantic, wanting so badly to just tear it all loose and get back to the very most basic blessings of being alive: breathing, walking, eating, hugging without assistance.

When David first visited me in my room, I immediately clutched at the hope that I could get there, where he is: vital, alert, driven. In my foremost mind, I was continually going back to the state of fitness and ability I'd willfully enjoyed for decades. I wanted the nurses and doctors to see that I was not this frail, swollen body lying helpless in my bed, laid out by this deadly co-pilot of my immune system.

I was a guy who took up vigorous vocations and hobbies, fit and laughing and downright hard to hurt. This is what was fighting its way to the surface when I told my physical therapist: "I AM an avid cyclist!!" So it was difficult to find words when David asked if I would be willing to donate samples for research.

It was so much more than just 'yes'. This was a quietly thrilling opportunity to fight, not just to survive, but to fight back, to strike a blow against sickness and fear and being singled out by the bad luck of the draw. An opportunity to step up from my fight to Our Fight.

I recently passed three years in remission, and during this time, the CDCN has continued to make substantive and impactful advancements in identification, treatment and increased understanding of the genetics of Castleman Disease.

From researchers to patients' families, the CDCN has helped connect us all. It makes me very hopeful, getting to know other patient's stories at various stages of Castleman diagnosis and treatment, and following the progress of the world’s medical researchers and data scientists engaged in the battle.

If you're a patient who might donate samples and medical data, I hope you will. This isn't just the CDCN's fight or my fight. It's Our Fight.

About Castleman disease

Castleman disease (CD) is the name given to a group of rare and life-threatening lymphoproliferative disorders that affect the lymph nodes and related tissues.

One subtype of CD is idiopathic Multicentric Castleman Disease (iMCD), whereby the cause is not known. iMCD accounts for between 33-58% of published MCD cases globally. There are approximately 540-960 new cases of iMCD diagnosed every year in the US, with a similar number estimated in Europe.

iMCD has the worst prognosis of all subtypes, where historically one third of patients have died within five years of diagnosis. The disorder is just as serious and life-threatening as many rare blood cancers, including progressive non-Hodgkin’s lymphoma.

The symptoms of iMCD are often attributed to other, more well-known conditions. Patients can experience inflammatory symptoms such as enlarged lymph nodes, weight loss, fatigue and fever. In severe cases, patients risk a life-threatening overproduction of immune cells (cytokine storm), which can lead to organ failure and, ultimately, death. Less commonly (<10% of cases), patients will have no signs or symptoms of the condition.

While the exact cause of iMCD is unknown and there are no known risk factors, increased IL-6, a pro-inflammatory cytokine, has been established as a key driver of symptoms and may play a role in its development. Identifying the immune factor released in iMCD is key to the development of better treatments and diagnostic tests, which will ultimately help improve patient outcomes.

Prior to 2014, treatment options for patients with iMCD were limited to chemotherapies and corticosteroids, and there were no treatment guidelines in place, making it challenging for HCPs to determine the best treatment for patients. The emergence of therapies that target specific pathways, such as IL-6, have been shown to reverse symptoms and improve long-term outcomes for patients.

Developing treatments for rare diseases

EUSA Pharma works at the forefront of developing treatments for rare diseases. Their goal is to transform the treatment landscape for underserved diseases and improve outcomes in areas where there are few patients yet high unmet medical needs.

Through their existing portfolio, which supports patients with iMCD, neuroblastoma, renal cell carcinoma (RCC) and oral mucositis (dryness of the mouth and throat) as a common side effect to anticancer therapy, they have proven dedication to improving survival outcomes and quality of life (QoL) in diseases where there has been high unmet need.

EUSA Pharma are a sponsor of ACCELERATE, the first international CD registry developed by the Castleman Disease Collaborative Network (CDCN), and are committed to working in collaboration with HCPs, the CD patient community and other organisations to better understand CD and the needs of patients.

EUSA Pharma take their responsibility to act as a true partner with patient communities seriously, and are committed to working with HCPs who have determined that their patient has an unmet medical need for their serious, life-threatening disease.

For some of their treatments, including in iMCD, they have mechanisms in place allowing HCPs to prescribe, within their professional responsibility, their medical products that are not yet approved or available for their eligible patients in their country.

23rd July 2020

From: Healthcare

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