Frontline Therapy for Immune Thrombocytopenia

James B. Bussel, MD:First-line treatment of ITP in almost all patients is steroids. The options in general practice, at least in the United States, are, do you use, as a steroid, prednisone or do you use high-dose dexamethasone? And the other option would be, do you give 1 or 2 doses of IV gamma globulin, or IVIg, to go with the steroids? The prednisone versus dexamethasone decision involves the fact that prednisone has been used for a very long time and is very predictable, and is a high dose but not as high a dose as high-dose dexamethasone. The advantage potentially of high-dose dexamethasone is there might be a higher rate of a curative effect in ITP. Studies on this have been conflicted in that some say yes, others say no. If you used high-dose dexamethasone, the idea is you give a huge amount of steroids for 4 days but then you stop so you try to avoid the toxicity of chronic steroid use while getting the advantage of high-dose steroids. As I said, this is not a very clear thing and most people have their own individual preferences.

The question of whether or not to add IVIg would typically depend on 2 factors. First, if there is a severe degree of bleeding, which is not the case here, then it could be used to make sure that the platelet count comes up faster to make the bleeding stop sooner. There is debate about whether prednisone actually reduces bleeding even before it increases the platelet count. But as indicated, this is debated. The other issue could be that if the woman had been admitted to the hospital, if she was given a dose of IVIg, there might be a cost offset in terms of how many days of hospitalization, depending on if she were to be kept hospitalized until her platelets increased substantially. I think certainly if IVIg shortened the hospitalization by 2 days, that would probably offset the cost. But, obviously, costs vary and are individual so that might or might not be the case.

There really are no other options for frontline treatment with the exception of IV anti-D, which has gone out of favor because of the black box warning of severe intravascular hemolysis. So, other treatments that might be thought of, such as a thrombopoietin receptor agonist, or splenectomy, or a rituximab-based regimen, or even immunosuppressive agents, are usually not given up front.

Transcript edited for clarity.

Case: A 44-year-old woman presenting with reddish-purple rash on lower legs

February 2017

• Patient presents with complaints of a reddish-purple rash on her lower legs and “constant” bruises appearing “spontaneously” without her remembering any trauma
• Physical evaluation reveals:
  • The rash to be petechiae (subcutaneous bleeding)
  • Slightly overweight (BMI = 26.5 kg/m²)
  • Patient is afebrile, with no splenomegaly
• When asked, reports her menstrual flow is unusually heavy, but says she was evaluated for and had no evidence of fibroids or endometriosis
• No personal or family history of cancer; no recent viral illnesses; no bone pain
• Current medications: no chronic medications; acetaminophen as needed; multivitamin
• Laboratory findings:
  • CBC reveals platelets 21 X 10⁹/L
  • All other findings with normal range
  • Negative forH pylori, HIV, and HCV
• Diagnosis: chronic ITP
  • Started course of prednisone 1 mg/kg X 21 days, then tapered off; at evaluation, platelets: 27 X 10⁹/L
  • Second course of prednisone 1 mg/kg X 21 days; at evaluation, platelets still <30
  • Third course of prednisone 1 mg/kg X 21 days; at evaluation, platelets still <30

February 2018

• &ldquo;Rash&rdquo; partly resolved, bruising still present
• Patient complains of weight gain on treatment and trouble sleeping
• After discussion with patient, she is started on eltrombopag (PROMACTA), at a dose of 50 mg/day