Clinical trial NCT00128882: Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D
Official title: Treatment of Idiopathic Thrombocytopenic Purpura in Children With Subcutaneously Administered Anti-D
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Thrombocytopenic Purpura
Keywords: Idiopathic Thrombocytopenic Purpura
Treatment
Anti-D
Subcutaneous
Clinical evaluation
Platelet count
IVIG
Lead sponsor: University of Aarhus
Sponsor class: OTHER
Collaborators: name: Kjaersgaard, Mimi, M.D.
First posted: 2005-08-10
Enrollment: 45
Sex: ALL
Minimum age (years): 1
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Idiopathic thrombocytopenic purpura (ITP)

Primary outcomes:
measure: Clinical effect evaluated on clinical score scale before and after treatment at specified intervals
timeFrame: day 0,1,3,6,14,30, 180,360
Secondary outcomes:
measure: Platelet count
timeFrame: day 0, 1, 3, 6, 14, 30, 180, 360
Other outcomes:
Locations:
city: Aalborg
country: Denmark
facility: Aalborg University Hospital, Department of Pediatrics
city: Aarhus N
country: Denmark
facility: Skejby Hospital, Aarhus University Hospital, Department of Pediatrics
city: Copenhagen S
country: Denmark
facility: Amager Hospital, Department of Pediatrics
city: Copenhagen Ø
country: Denmark
facility: Rigshospitalet, Copenhagen University Hospital, Pediatric Clinic II
city: Esbjerg
country: Denmark
facility: Esbjerg Hospital, Department of Pediatrics
city: Gentofte
country: Denmark
facility: Gentofte Hospital, Department of Pediatrics
city: Herning
country: Denmark
facility: Herning Hospital, Department of Pediatrics
city: Hjoerring
country: Denmark
facility: Hjoerring Hospital, Department of Pediatrics
city: Holbæk
country: Denmark
facility: Holbæk Hospital, Department of Pediatrics
city: Hvidovre
country: Denmark
facility: Hvidovre Hospital, Department of Pediatrics
city: Kolding
country: Denmark
facility: Kolding Hospital Department of Pediatrics
city: Nykøbing F
country: Denmark
facility: Nykøbing F, Department of Pediatrics
city: Næstved
country: Denmark
facility: Næstved Hospital, Department of Pediatrics
city: Odense C
country: Denmark
facility: Odense University Hospital
city: Randers
country: Denmark
facility: Randers Hospital, Department of Pediatrics
city: Sønderborg
country: Denmark
facility: Sønderborg Hospital, Department of Pediatrics
city: Viborg
country: Denmark
facility: Viborg Hospital, Department of Pediatrics
Study officials:
affiliation: University of Aarhus, Clinical Institute, Department of Pediatrics
name: Mimi Kjaersgaard, MD
role: STUDY_DIRECTOR
affiliation: Skejby Hospital, University of Aarhus, Department of Pediatrics
name: Henrik Hasle, MD PhD
role: PRINCIPAL_INVESTIGATOR
References: