A prospective, randomized trial of conventional, dose-accelerated corticosteroids and intravenous immunoglobulin in children with newly diagnosed idiopathic thrombocytopenic purpura

To determine the minimal essential treatment for childhood acute idiopathic thrombocytopenic purpura (ITP), a prospective, randomized trial was conduc ted focusing on hemorrhagic manifestation as well as platelet count. Subjec ts with a platelet count of <10 x 10(3)/<mu>L or 10 to 29 x 10(3)/muL and m ucosal bleeding (group 1) were randomly assigned to receive intravenous imm unoglobulin (IVIg) at 1 to 2 g/kg, conventional oral prednisolone (o-PSL) ( 2 mg/kg for 2 weeks), parenteral methylprednisolone (mPSL) (5 mg/kg for 5 d ays), or pulsed parenteral methylprednisolone (PmPSL) (30 mg/kg for 3 days) . Subjects with a platelet count of 10 to 29 x 10(3)/muL without mucosal bl eeding (group 2) were randomized to receive either o-PSL or no treatment. I n subjects with a platelet count of 30 x 10(3)/muL or higher (group 3),pati ents undergoing no specific treatment were monitored. In group 1, Mg offere d faster platelet enhancement compared with o-PSL and mPSL, although neithe r mPSL nor PmPSL showed any advantage, even over o-PSL. Platelet response w as uniformly excellent when pretreatment platelet count was greater than or equal to 10 x 10(3)/muL. Furthermore, the presence or absence of mucosal b leeding in subjects with a platelet count <10 x 10(3)/<mu>L had no effect o n the response to treatment. In group 2, platelet increase was indifferentl y attained with or without o-PSL. These data suggest that childhood acute I TP with a platelet count greater than or equal to 10 x 10(3)/muL may be lef t untreated or may be treated with o-PSL when mucosal bleeding is evident, whereas for those with a platelet count <10 x 10(3)/<mu>L, IVIg is the most predictable platelet enhancer. Thus, a platelet count of 10 x 10(3)/muL se ems to be informative enough to decide whether to treat childhood acute ITP . Int J Hematol. 2000;72:376-383. (C) 2000 The Japanese Society of Hematolo gy.