Acute immune thrombocytopenia (ITP) in childhood: retrospective and prospective survey in Germany
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Published on: July 9, 2014
A H Sutor1, A Harms, K Kaufmehl
1Universitäts-Kinderklinik, Freiburg, Germany. sutor@kikli.ukl.uni-freiburg.de
Insights
Treatment guidelines for childhood immune thrombocytopenia (ITP) remain uncertain. Studies in Germany show most children have mild bleeding, yet often receive treatment, primarily based on platelet counts, warranting reconsideration of current recommendations.
Area of Science:
- Pediatrics
- Hematology
- Epidemiology
Background:
- Treatment of acute childhood immune thrombocytopenia (ITP) lacks standardized guidelines.
- Existing treatment strategies are controversial, necessitating an evaluation of current practices in Germany.
Purpose of the Study:
- To assess the current situation and treatment policies for childhood ITP in Germany.
- To determine the incidence, age, and gender distribution of ITP in children.
- To evaluate treatment modalities and outcomes in pediatric ITP cases.
Main Methods:
- Conducted a retrospective survey of German pediatric hospitals regarding ITP management and outcomes.
- Performed a prospective survey over one year to track ITP cases, incidence, and treatment approaches.
- Collected data on patient demographics, bleeding symptoms, platelet counts, and treatments administered (Immunoglobulins, Glucocorticosteroids).
Main Results:
- No deaths or intracranial hemorrhages (ICH) were reported in either survey.
- The prospective survey calculated an annual ITP incidence of 2.16 per 100,000 children, varying by age and gender.
- Despite predominantly mild bleeding symptoms (97.5%), most children with low platelet counts received treatment, mainly Immunoglobulins (Ig) or Glucocorticosteroids (GC).
Conclusions:
- Current treatment decisions for childhood ITP appear heavily influenced by platelet counts, often irrespective of bleeding severity.
- The effectiveness of treatments like Ig and GC cannot be concluded from these surveys.
- Recommendations for ITP management based solely on platelet counts require re-evaluation.
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.
