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.

Condition	Intervention	Phase
Idiopathic Thrombocytopenic Purpura	Drug: Anti-D	Phase II

Further Study Details: 

Primary Outcomes: Clinical effect evaluated on clinical score scale before and after treatment at specified intervals; Side effects (safety)
Secondary Outcomes: Platelet count
Expected Total Enrollment:  100

Background:

Idiopathic thrombocytopenic purpura (ITP) in children is considered a benign hematological disease. The incidence is app. 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 flue-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 flue-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 app. 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 identica. 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 form children, who receives 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

Ages Eligible for Study:  1 Year   -   14 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Idiopathic thrombocytopenic purpura (ITP)

Exclusion Criteria:

• None

Please refer to this study by ClinicalTrials.gov identifier  NCT00128882

Mimi Kjaersgaard, MD      +45 8949 6776    mimi.kjaersgaard@ki.au.dk
Henrik Hasle, MD, PhD      +45 8949 6716    hasle@dadlnet.dk

Denmark
      Amager Hospital, Department of Pediatrics, Copenhagen S,  2300,  Denmark; Recruiting
      Esbjerg Hospital, Department of Pediatrics, Esbjerg,  6700,  Denmark; Recruiting
      Gentofte Hospital, Department of Pediatrics, Gentofte,  2900,  Denmark; Recruiting
      Hjoerring Hospital, Department of Pediatrics, Hjoerring,  9800,  Denmark; Recruiting
      Holbæk Hospital, Department of Pediatrics, Holbæk,  4300,  Denmark; Recruiting
      Hvidovre Hospital, Department of Pediatrics, Hvidovre,  2650,  Denmark; Recruiting
      Nykøbing F, Department of Pediatrics, Nykøbing F,  4800,  Denmark; Recruiting
      Næstved Hospital, Department of pediatrics, Næstved,  4700,  Denmark; Recruiting
      Odense University Hospital, Odense C,  5000,  Denmark; Recruiting
      Randers Hospital, Department of Pediatirics, Randers,  8900,  Denmark; Recruiting
      Rigshospitalet, Copenhagen University Hospital, Pediatric Clinic II, Copenhagen Ø,  2100,  Denmark; Recruiting
      Skejby Hospital, Aarhus University Hospital, Department of Pediatrics, Aarhus N,  8200,  Denmark; Recruiting
      Viborg Hospital, Department of Pediatrics, Viborg,  8800,  Denmark; Recruiting
      Sønderborg Hospital, Department of Pediatrics, Sønderborg,  6400,  Denmark; Recruiting
      Aalborg University Hospital, Department of Pediatrics, Aalborg,  9100,  Denmark; Recruiting
      Herning Hospital, Department of Pediatrics, Herning,  7400,  Denmark; Recruiting

Study chairs or principal investigators

Mimi Kjaersgaard, MD,  Study Director,  University of Aarhus, Clinical Institute, Department of Pediatrics   
Henrik Hasle, MD PhD,  Principal Investigator,  Skejby Hospital, University of Aarhus, Department of Pediatrics   

Study ID Numbers:  MK-2005-1; 2003179; 2612-2447
Last Updated:  August 9, 2005
Record first received:  August 8, 2005
ClinicalTrials.gov Identifier:  NCT00128882
Health Authority: Denmark: Danish Medicines Agency
ClinicalTrials.gov processed this record on 2005-08-23