I have mailed this letter in its full form anticipating that parts of this will have to be deleted due to the "Posting Policy"...

I have no medical degree, but I have learned more in the past few years than I ever thought I would know about blood and medicine.

[PHI Redacted] was diagnosed with ITP over 4 years ago and since that diagnosis has endured numerous treatments that have failed to control his severely low platelet counts.

I am aware that this particular topic is referring to the adult use of drugs like Nplate, but please allow me to state a strong case regarding why this drug is needed, has been needed and will continue to be needed for adults and children alike. Please note that limiting access to patients by not covering its use would hinder the progress that is being made in this area of medicine.

[PHI Redacted] received his first IVIg 12 days after his initial diagnosis, to which he had a severe reaction. The 2nd infusion was less than 2 weeks later, pre-treated to ease the side effects. This form of treatment has, continued throughout the 4 years as his “standard” emergency treatment. He responds for 2-3 days and then drops back down to what is considered his “normal” platelet count of less than 10k. High dose steroids failed next, followed by a short therapy of Vincristin w/ Cyclosprine and steroids. Short because after the second dose, he suffered a life threatening paralytic ileus reaction to the Vincristin. This information is relevant because it shows his limits for future types of treatments.

7 months after his diagnosis his spleen was removed. [PHI Redacted] moved down the list of treatments, almost in clinical order. The splenectomy resulted in a recovery of counts in the 40k’s – 60k’s range for about a year before plummeting back to his normal range again. This particular year was a blessing for the mere reason that [PHI Redacted] who is an excessive, frequent and spontaneous bleeder was free from his two and a half hour nose bleeds for that year.

Once the year of “somewhat peace” was over, we were in what seemed to be worse condition than before. His body seemed to reject all treatments. That year we tried Rituximab, 6MP, Dapsone, and Cellcept.

His next treatment was a combination therapy of high dose infusions of prednisone, IVIg and WinRho injections supplemented by daily oral meds of Decadron and Azathioprine. He received the infusions every Wednesday because they only held his counts for 2-4 days before they drop dramatically. Each Wednesday appointment his counts are less than 10k.

[PHI Redacted] doctors have told [PHI Redacted] on numerous occasions that this is the worst case of ITP they have seen at their hospital. The mix of the persistently low count with the excessive bleeding and the side effects to standard treatments has been a challenge for all of us. The doctors would consistently reach out to the leading specialists. Their recommendations had all begun to turn to the new thrombopoietin lines of treatments like Nplate which gave us renewed hope.

[PHI Redacted] is now in the pediatric clinical trial for Nplate. He is showing great progress. All of the treatments he has tried before were used “off- label” to attempt to treat his ITP. THIS drug is designed TO TREAT his ITP, not some other disorder/disease/illness. Unlike the other treatments, he has had NO side effects at all. NONE. He is progressively getting his life back and [PHI Redacted].

I am a member of the Platelet Disorder Support Association. I communicate daily with parents of children with ITP as well as adults that are currently in or were in one of the clinical trials for this drug. We are excited and anxious for this desperately needed drug to become available for children and just as excited that it is now available for adults. I am aware that the “numbers” of patients with ITP are not great, that the level of attention “our” illness receives is much lower than some higher profile illnesses. Please keep in mind though that the effects are still the same. The life-threatening risk of a brain bleed is no different if a patient has low platelets due to ITP or due to another illness with low platelets as a “side effect”. The difference comes into play when you are able to treat the “other” illnesses and the platelet function returns. With ITP, there is no other alternative than to treat the platelet issue.