Newly Diagnosed, just wondering

[Richard R]

I was diagnosed on Mar 31, 2014 with SAA. A bone marrow biopsy was done with only 5% left, and the Doctor ordered a cytogenetic check. I was hospitalized to stabilize my blood counts, discharged, and referred to the Hematologist. He started HATG on the 11th of April and I had a one day interruption when my PICC line developed a clot and a fever ensued so instead of taking 4 days, it took 5. My WBC <.1 and in the time since then it rose to .61, but has steadily gone back down on each lab which are on a Mon/Wed/Fri schedule. Platelets have been infused on about a 5 day schedule with a low of 7 and a high of 56. It seems like I use about 7 - 10/day. Hgb goes down from a high of 10.5 and I have had 2 transfusions since discharge from the hospital on the 22nd of April.

I realize that it has only been 18 days since my discharge and I haven't seen any threads on how long it can take for an increase in some of my numbers. So, when is it reasonable to say it either is or isn't helping? Do patients get multiple HATG treatments? Is Eltrombopag a usual treatment and is it available outside of the NIH NCT basis? My age is 63 and I have been in excellent health prior to this. Never hospitalized and never sick.

Thanks for your help.

[Neil Cuadra]

Hi Richard.

Dr. Ramon Tiu of the Cleveland Clinic said recently that you shouldn't rely on blood counts to judge the successfulness of ATG treatment until 6 months after the treatment. I know that waiting that long for results can tax your patience, and require regular transfusions that you hoped to be freed from. I hope you'll see positive results much sooner, which is the case for some patients.

So far eltrombopag (Promacta) has received FDA approval for two conditions:

• chronic idiopathic thrombocytopenic purpura (ITP) -- in 2008
  
  
• thrombocytopenia during treatment for chronic hepatitis C -- in 2012

In February 2014 the FDA gave eltrombopag breakthrough therapy status, which provides expedited development and review.