Status:
COMPLETED
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
Lead Sponsor:
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Conditions:
Wiskott-Aldrich Syndrome
Eligibility:
All Genders
Up to 18 years
Brief Summary
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatr...
Detailed Description
Thrombocytopenia is a life-threatening symptom in WAS patients. Subjects with WAS are at increased risk of debilitating and\\ or life-threatening bleedings due to low platelet numbers. Hematopoietic s...
Eligibility Criteria
Inclusion
- Age under \< 18 years
- Subject/legal representative has signed written informed consent. ?
- Subjects diagnosed with WAS based on genetic findings.
- Subjects with thrombocytopenia (platelet count of less than 70 x 109/L).
- Subjects with a history of bleeding.
- Subjects received treatment with romiplostim 8-9 µg /kg for at least 30 days
- Available records of the points of analysis
Exclusion
- • Patients, who do not meet the inclusion criteria.
Key Trial Info
Start Date :
April 1 2012
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2020
Estimated Enrollment :
67 Patients enrolled
Trial Details
Trial ID
NCT04350164
Start Date
April 1 2012
End Date
June 1 2020
Last Update
December 28 2020
Active Locations (1)
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1
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, Russia, 117997