Idiopathic Thrombocytopenic Purpura Clinical Trial
Official title:
Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D
The purpose of this study is to describe the effect of anti-D on symptoms and platelet count in children suffering from unexplainable low platelet counts, when anti-D is administered as an injection under the skin. The hypothesis is that the injection with anti-D under the skin is as effective as anti-D given in a vein.
Background:
Idiopathic thrombocytopenic purpura (ITP) in children is considered a benign hematological
disease. The incidence is approximately 50 cases a year in Denmark. Approximately 25 % will
experience chronic disease. Follow up and treatment of these patients is not centralized.
The drug of choice is intravenous IgG (IVIG) for treatment of ITP. The side effects are
flu-like symptoms, and in rare cases aseptic meningitis. Another option is intravenous
anti-D, if the child is rhesus positive. Anti-D is primarily used in North America. The
effect of Anti-D is comparable with IVIG when considering the time it takes to bring the
platelet count above 50,000/μL. Anti-D also causes flu-like symptoms. Establishing an i.v.
access is a disadvantage to both IVIG and anti-D. For both treatments mechanism of action is
not finally described.
Subcutaneous IgG substitution therapy is used for patients suffering from
agammaglobulinaemia. It is therefore known, that immunoglobulin uptake is possible after
subcutaneous administration. Subcutaneous anti-D has been tried in few patients suffering
from chronic thrombocytopenia with positive results.
IVIG treatment is expensive compared to anti-D. Treatment of a 20 kg child costs
approximately 17,000 Dkr for IVIG and 2,500 Dkr. for anti-D.
Hypothesis:
- Subcutaneous administered anti-D is as effective as IVIG/i.v. anti-D;
- Subcutaneous administered anti-D has fewer less severe side effects than IVIG/i.v.
anti-D.
Purpose:
- To document the effect of subcutaneous anti-D;
- Describe complications;
- Describe aspects of the mechanism of action.
Material and Methods:
Children are eligible if admitted to a pediatric department in Denmark for diagnosis,
observation or treatment of acute or chronic ITP. Examination and diagnostic work up is
similar throughout the country, but not identical. No specific tests are required for
diagnosis. If treatment is indicated rhesus positive children are treated with subcutaneous
anti-D. Rhesus negative children are treated according to local guidelines. Specified
follow-up on all children is mandatory. For research purposes one blood sample form all
children is collected, and from children, who receive medical treatment, several blood
samples are collected. Analysis for changes in immunological signaling peptides will be
performed with special attention to the mechanism of action of anti-D.
;
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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