• Tara

My NIH Doctor Presents Results of Study of New Treatment for HES

Encouraging news has been announced about the preliminary results of a study of a new treatment for hypereosinophilic syndrome in which I am a participant.

For two and one-half years I have participated in study of mepolizumab, a steroid-sparing treatment for patients with hypereosinophilic syndrome.  For me, the results of mepolizumab have been life-changing – freeing me from the harsh, toxic effects of prednisone steroids, while controlling my eosinophil count.  Turns out many other participants in the same study have also had good results, and that the treatment is “well tolerated.”

The results and preliminary conclusions from the study were presented  at a conference earlier this month by the lead researcher, Amy D. Klion, MD, of the National Institute of Allergy and Infectious Diseases (part of the National Institutes of Health, or “NIH”) in Bethesda, Maryland.  Dr. Klion is widely regarded as one of the world’s leading authorities on eosinophils. I don’t know exactly what goes on at these conferences, but I envision a group of doctors who geek out on eosinophils giving Dr. Klion the rock-star treatment when she presents.

Dr. Klion has been treating me for hypereosinophilic syndrome for nearly seven years.  For over  four years I was on a high dose of prednisone.  Although prednisone was effective in lowering my eosinophil count, the side-effects were challenging: significant weight gain, indigestion, nervousness, and mood swings, among others.  Worse, over time the efficacy (i.e. the effectiveness) of the prednisone decreases although the side effects continue.

About two and a half years ago, when I was 13, I was fortunate to be accepted into a study at the NIH to learn about the effectiveness of mepolizumab in treating hypereosinophilic syndrome.  Every three months I have traveled to NIH to receive a 30-minute intravenous drip of “mepo.”   I have experienced no side effects, my eosinophil count has been normal, and the effectiveness of the treatment has remained consistent.

This month, at the annual meeting of the American Academy of Allergy, Asthma, and Immunology, Dr. Klion shared the preliminary data and conclusions from the study.  The highlights, as reported by MedPage Today, a medical news organization, are:

  • The study included 85 patients.

  • After nearly four years of exposure to mepolizumab, 60% of patients were no longer on prednisone.

  • Side effects were similar to those seen with placebo

  • Long-term efficacy (i.e. effectiveness in lowering the eosinophil count) was maintained

  • The majority of patients continue to receive infusions at three-month intervals, while some are having the treatment every six months.  Only 10% need monthly doses.

Mepolizumab is a “humanized monoclonal antibody” that promotes inhibition of interleuken-5, a type of protein molecule that stimulates the production of eosinophils.

Mepolizumab is still an “investigational” drug not yet approved by the FDA.   It is in the approval process for treatment of asthma.  The drug is available for patients of hypereosinophilic syndrome based on “compassionate use,” meaning essentially that the participant has a life-threatening disease with no other FDA-approved treatment options.  I hope that it will receive FDA approval so that many others in need can benefit.

This study, sponsored by GlaxoSmithKline and the NIH, is still recruiting participants to locations around the US.   If you or someone you know has hypereosinophilic syndrome, it might be worth applying to join the study.  The full description of the study, including criteria for admission and locations where the study is offered, is available online.

The results of the study are preliminary, and have not been published in a peer-reviewed journal, but I am excited for Dr. Klion, her colleagues, and all those who have been diagnosed with hypereosinophilic syndrome.

The full account of Dr. Klion’s presentation as reported by MedPage Today is available here.

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