Immune Thrombocytopenia: First-Line Treatment Options

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 whether 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 48-year-old woman presenting with unusual bruising

October 2017

• A 48-year-old woman presents with complaints of bruising after minor bumps, bleeding gums despite regular tooth cleaning, and a recent spontaneous bloody nose; symptom onset about 1 year ago
• Physical evaluation reveals a woman of normal weight and average height, afebrile, no splenomegaly
• No personal or family history of cancer, autoimmune disease; no recent viral illnesses; no bone pain or night sweats
• Current medications: ibuprofen as needed, generic hydrochlorothiazide (HCTZ)
• Laboratory findings:
  • CBC reveals platelets 28 X 10⁹/L
  • WBCs within normal
  • Renal and hepatic function within normal
• Diagnosis: idiopathic thrombocytopenic purpura
• Patient started on a course of prednisone 1 mg/kg for 21 days, then tapered off
  • Platelets: 29 X 10⁹/L
  • Second course of prednisone 1 mg/kg for 21 days

April 2018

• After 2 courses of prednisone, patient’s platelets have not recovered
  • CBC shows platelets at 28 X 10⁹/L
• Symptoms of easy bruising and bleeding from gums continue
• After discussion with patient, she is started on the thrombopoietin receptor agonist (TPO-RA) eltrombopag (PROMACTA), at a dose of 50 mg/day
  • Dose increased to 75 mg/day; last platelet count, 65 X 10⁹/L