Case 1
A 3-month-old baby girl was referred to our hospital for thrombocytopenia. On admission, her platelet count was 12.000/microL and she had a Buchanan-Adix [9] bleeding score (B.A.b.s.) of 1 (isolated cutaneous petechiae). She was administered 2 doses of IVIg (800 mg/Kg), without success, hence it was decided to proceed with steroids as second line therapy (Prednisone 2 mg/kg for two weeks followed by three consecutive boluses of Methylprednisolone 30 mg/kg each). A bone marrow examination was performed before starting the steroid consistent with the diagnosis of ITP. Unfortunately, the prednisone-based therapy was unsuccessful (platelet count after 8.000/microL; B.A.b.s.= 3).
Given the unresponsiveness to glucocorticoid and to prevent steroid-related side effects it was decided to discontinue the therapy and start a close watch-and-wait approach.
At 3 months from diagnosis with a platelet count below 15.000/microL and persistent symptoms, after having obtained the informed consent from the parents, we started an off-label course of Eltrombopag (dose: 1.5 mg/Kg/day). The first clinical response was observed after three weeks with decreasing symptoms (B.A.b.s. 0-1). After about six weeks platelet count raised to 67.000/microL and, 9 months from the onset, it reached a value of 164.000/microL.
The patient is currently in optimal clinical condition at 11 months of follow-up and the complete blood count is normal. The current dose of Eltrombopag is 1.1 mg/Kg/die, no side effect has developed (Figure 1).