February 25th, 2014

Honoring Rare Disease Day: Spotlight on Multicentric Castleman’s Disease

by Joanne Vanak, RN, MSN; Senior Director of Medical Affairs Scientific Advocacy, Janssen Scientific Affairs, LLC

Rare Disease Day

Imagine living with a disease so rare it’s hard to get the proper diagnosis. Picture life as a patient with a condition so unique you can barely find a physician with the experience to treat it. Unfortunately, for the nearly 25 to 30 million Americans who suffer from one of the approximately 6,800 rare diseases recognized in the United States,[1] this uncertainty is a reality. And it’s one of the primary reasons Johnson & Johnson and Janssen remain committed to uncovering treatment options for those facing unmet medical needs.

In the United States, a disease is considered rare if it affects fewer than 200,000 Americans.[1] Some rare diseases affect only a fraction of that. For example, in the case of a disease called Multicentric Castleman’s disease, or MCD, the patient numbers are so low that it’s difficult to get an accurate estimate of the number of people impacted. However, based on approximate calculations, experts believe there are fewer than 2,000 Americans with this condition.[2]*

So what is MCD? It is a disorder in which lymphocytes, a certain type of white blood cells, are over-produced, causing enlargement of lymph nodes or other internal organs containing lymphoid tissue. This can cause a variety of symptoms and weaken the immune system, making it hard to fight infections,[3] which can be very serious and even be fatal in some patients with MCD.[3] Because MCD is so rare and the symptoms are often like those caused by other diseases,[3] it may take time to find the right diagnosis, which can leave patients feeling alone and frustrated.

Those who suffer, however, are not alone. There are a number of advocacy groups dedicated to rare disease, including the National Organization for Rare Disorders (NORD). This year, on February 28, rare disease patients, families, caregivers, and groups will unite to observe Rare Disease Day, an international advocacy day to bring widespread recognition of rare diseases as a global health challenge and celebrate the motto set forth by NORD: “Alone we are rare. Together we are strong.TM

Johnson & Johnson and Janssen will join the world in honoring the 7th annual Rare Disease Day. On this day – and every day – Janssen will “raise and join hands” to support people affected by thousands of rare diseases. While Janssen doesn’t have a specific focus on rare diseases, we are committed to developing compounds in areas of unmet medical need, which can extend to include rare diseases. Our rare disease work includes MCD, mantle cell lymphoma, multiple myeloma, myelodysplastic syndrome, and soft tissue sarcoma, among others.

Please join us and help raise awareness of rare diseases. To participate in an event, visit the Rare Disease Day USA page for activities in your community.

*Estimate from a U.S. claims database


Joanne VanakFor the past four years, Joanne Vanak has been a Senior Director of Medical Affairs Scientific Advocacy for Janssen Scientific Affairs, LLC, overseeing Oncology and Immunology Advocacy. She has been with Johnson & Johnson for 15 years, beginning her career as a Medical Science Liaison in the Northeast Region and has performed at every clinical level within the team.


References

[1] National Center for Advancing Translational Sciences (NCATS). National Institutes of Health (NIH). FAQ About Rare Diseases. Available from: http://www.ncats.nih.gov/about/faq/rare/rare-faq.html.

[2] Mehra M et al. Use of a Claims Database to Characterize and Estimate the Incidence of Castleman’s Disease. Poster presented at: 54th American Society of Hematology (ASH) Annual Meeting and Exposition; Dec. 8-11, 2012; Atlanta, GA. Abstract available from: https://ash.confex.com/ash/2012/webprogram/Paper51578.html.

[3] American Cancer Society. Castleman disease. Available from: http://www.cancer.org/acs/groups/cid/documents/webcontent/003093-pdf.pdf.

One Response to “Honoring Rare Disease Day: Spotlight on Multicentric Castleman’s Disease”

  1. Sandra Roth says

    My son has a very rare disease – with less than 200 people in the world identified as having Progressive Osseous Heteroplasia. This article is very appreciated – to recognize the struggle that we have to not only identify, but treat, these rare conditions.

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