A Randomized Study of IVIG vs. IVIG With High Dose Methylprednisolone in Childhood ITP.

• The rapidity of rise in Platelet Count [ Time Frame: The first 24 hours following the administration of therapy ] [ Designated as safety issue: No ]
  
• Time taken to reach a Platelet Count of less than or equal to 20, less than or equal to 30, and less than or equal to 50 x 10^9/L. [ Time Frame: Time frame determined by outcome ] [ Designated as safety issue: No ]
  

• Frequency of response to therapy at different time points [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  
• Days to PC falling to < 20 x 109/L [ Time Frame: Time frame determined by outcome ] [ Designated as safety issue: No ]
  
• Adverse Effects of therapy [ Time Frame: 1 week ] [ Designated as safety issue: No ]
  
• Quality of life changes over time and between the treatment groups [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  

Rarely children with immune thrombocytopenia purpura (ITP) can present with severe or life-threatening bleeding. In these cases it is very important that the platelet count be raised as quickly as possible. Several studies have shown that IVIG and corticosteroids on their own can raise platelet counts, but few studies have examined how the combination of IVIG and corticosteroids compares to IVIG alone in raising platelet counts in childhood ITP. Yet despite the lack of conclusive evidence to indicate that steroids given together with IVIG is more effective, this combination treatment is often given when children present with a life-threatening bleed, e.g. intracranial bleed. In addition to presumed greater effectiveness of giving the two agents together there is also evidence to show that the combination of IVIG with steroids may have other beneficial effects, in addition to greater effectiveness at raising platelet counts. This can include reducing side effects of IVIG.

We propose to compare the effectiveness of the combination of IVIG with corticosteroids to IVIG alone in raising platelet counts in children with ITP and a platelet count less than 20 x 109/L. Patient will be eligible only if they in conjunction with their treating physician have decided to be treated with IVIG. In this way they will require an intravenous regardless of study participation. The primary outcome is the rise in platelet count as reflected by the platelet count at 24 hours.

Hypothesis:

IVMP and IVIG, administered together, will

1. increase the PC faster, and
2. minimize the adverse effects of IVIG, and
3. lead to a more sustained increase in PC (longer time before needing retreatment) If it is shown that the combination of these agents does result in a quicker rise in PC, this would support and justify the use of the combination therapy in emergency situations.

Study Proposal and Methods:

We propose to prospectively evaluate 2 treatment regimens in patients with childhood ITP:

Regimen A: Placebo followed by IVIG 1 g/kg (Gamunex® Immune Globulin Intravenous [Human], 10%; Bayer)* x 1 dose Regimen B: Combination therapy (IV MP (Solu-Medrol®, Upjohn) 30 mg/kg (max. 1 g) over 30 min followed by IVIG 1 g/kg (Gamunex® Immune Globulin Intravenous [Human], 10%; Bayer)* x 1 dose

*Gamunex will be given according to manufacturer's guidelines. Gamunex has been demonstrated to be safely and effectively administered by means of a rapid infusion protocol whereby it can be given over a period of 2 hours (although in some cases it needs to be given at a slower rate over a longer period of time).