Acute Immune Thrombocytopenia (ITP) in Childhood: Retrospective and Prospective Survey in Germany

Abstract

Treatment of acute childhood immune thrombocytopenia (ITP) is controversial. For information on the present situation in Germany, a retrospective and a prospective survey were carried out. In the retrospective survey, questionnaires were sent to all German pediatric hospitals asking about local policies for handling ITP and whether in the preceding year (starting on October 1995) death or ICH had occurred; 86% answered. In the prospective survey, 94% responded to the mailings ("have you seen a case of ITP?") sent in monthly intervals between October 1, 1996 and September 30, 1997; 89% of the questionnaires were retrieved. In the retrospective survey, no deaths and no ICH were reported. If only mild bleedings, such as skin bleeds alone (or additional mild mucous membrane bleeding) were present, 20.5% (26.4%) preferred the "watchful waiting" regimen (supportive care), irrespective of the platelet count; 79.5% (73.6%) would treat if the platelet counts were <5 x 10(9)/L, 73.5% (67.9%) if < 10, 35.9% (33.6%) if < 20, and 4.2% (2.6%) if <30. Of the treaters, 50.5% would prefer immunoglobulins (Ig), 24.4% glucocorticosteroids (GC), and 20.5% GC and/or Ig. Generally, a rise in platelets, most frequently >50 x 10(9)/L was considered as therapeutic success. In the prospective survey, from the reported 323 children an annual incidence of 2.16 per 100,000 children was calculated. The incidence depended on age and gender, being highest for boys younger than 2 years with 5.8 (girls 3.42) and low with 0.44 for boys (girls 0.89) older than l4 years. About 60% of the patients had a preceding infection. Although 83% had a platelet count <20 x 10(9)/L (56% <10 x 10(9)/L), almost all (97.5%) had only mild bleeding symptoms; 2.5% had serious bleeding symptoms requiring blood transfusion or nasal packing, none had ICH, and no death was reported. The mean platelet count on admission was 11.348 (lowest count 8.253) x 10(9)/L. Sixty-one percent received Ig, 19% GC (both either alone or as first choice), 6% Ig plus GC, and 14% no treatment. Side effects were reported in 22% of the children treated with Ig. The retrospective survey mirrored the uncertainty in regard to treatment. The prospective survey provided new aspects on incidence, age, and gender distribution. Although almost all patients had only mild bleeding symptoms, most received Ig and/or GC. The decision to treat depended mainly on the platelet count. From these surveys, conclusions about the effectiveness of treatment cannot be drawn. Recommendations based primarily on platelet counts must be reconsidered.

Newly diagnosed immune thrombocytopenia in children and adults: a comparative prospective observational registry of the Intercontinental Cooperative Immune Thrombocytopenia Study Group

“…Our data support and confirm some previously reported observations, including the predominance of males in the pediatric age group, whereas female gender dominates among adult patients. [13][14][15] This analysis suggests that autoimmune disorders, which are associated with female gender, 16 may be an explanation for the greater number of adult women with ITP. Reasons for the observed high proportion of males in childhood ITP remain unknown.…”

“…Reasons for the observed high proportion of males in childhood ITP remain unknown. 10,[14][15] An important finding of this analysis is the huge overlap in the clinical and laboratory features of ITP at first diagnosis among children and adults. Similarities are observed in presenting platelet counts, incidence and type of bleeding when platelet counts are less than 20¥10 9 /L, and reported family history of thrombocytopenia.…”

Rituximab in the treatment of autoimmune haematological disorders

“…Idiopathic thrombocytopenic purpura is an autoimmune bleeding disorder with an incidence of approximately 30 per million persons per year in adults (Frederiksen & Schmidt, 1999;Neylon et al, 2003) and a similar incidence in children (Sutor et al, 2001;Zeller et al, 2005). The pathogenesis of ITP is not entirely clear, but patients develop autoantibodies against platelet surface glycoproteins.…”

Use of Recombinant Factor VIIa in a Pediatric Patient With Initial Presentation of Refractory Acute Immune Thrombocytopenic Purpura and Severe Bleeding

“…1 It is postulated that platelet destruction in ITP is caused by autoantibodies that target glycoprotein complexes (IIb/IIIa and/or Ib/IX) 10 or by T-cell mediated cytotoxicity. 11 Although rare, intracranial hemorrhage is the most severe complication of ITP.…”

“…1,2 It often follows a recent history of viral illness and typically resolves within 6 months. 3 Severe bleeding is a rare complication of ITP, with the incidence of intracranial hemorrhage ranging from 0.1% to 1%.…”