An observational study in Wiskott-Aldrich Syndrome, sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. Completed at 1 site in Russian Federation. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2020-12-28.
Sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Observational
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 pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS).
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 stem cell transplantation is an effective treatment of WAS and all its symptoms yet requires time for donor search and is not widely utilized in cases with mild WAS with isolated thrombocytopenia. TPO-RAs have been used in individual WAS patients, wherein publications describing large WAS cohorts treated with TPO-RAs are lacking.
Based on the previous reports, WAS patients in our Center have been receiving treatment with TPO-RA romiplostim since 2012.
The aim of the study is to retrospective analyze patients' data in order to asses treatment efficacy and safety of romiplostim in WAS thrombocytopenia.
The study will collect and analyze information that is already in the patients' medical records. Information about clinical data (assessment of bleeding tendency with a modified World Health Organization (WHO) Bleeding Scale), laboratory values (such as clinical and biochemical analysis of blood) will be included.
Evaluation of the efficacy therapy was based on the results of physical examination, including bleeding events at the time of diagnosis and after 6-month TPO-RA was initiated and platelet response. A complete response was defined as a platelet count >100 x 109/L in the absence of bleeding symptoms, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. Non-response was defined as not achieving a platelet count of > 30 x 109/L from the baseline count.
697 studies on the registry are indexed under Thrombocytopenia; 153 are open to participants now.
This study's enrollment of 67 is below the median of 120 across 183 observational studies indexed under Thrombocytopenia.
Browse Thrombocytopenia studies →Federal Research Institute of Pediatric Hematology, Oncology and Immunology is the lead sponsor of 55 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
patients of the immunology department
Exclusion Criteria:
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
Drug: Romiplostim
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
Also known as: Nplate
The percentage of participants with overall platelet response (complete response + partial response)
A complete response defined as a platelet count \>100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L.
Time frame: 1 month (30 day +/- 14 days)
Percentage of patients with a platelet response
Time frame: until discontinuation, from at least one month to one year
Number of participants with bleeding events and severity of bleeding
The incidence and severity of bleeding events evaluated with a modified World Health Organization (WHO) Bleeding Scale. (G1=Petechiae, epistaxis \<30 min, G2=Mild blood loss, hematomas, epistaxis \>30 min, melanotic stool G3=Gross blood loss, requiring blood transfusions, G4=Fatal bleeding).
Time frame: until discontinuation, from at least one month to one year
Number of participants with adverse events
Time frame: until discontinuation, from at least one month to one year
This study is completed, as verified in Dec 2020. You cannot join it, but the record below documents what was studied.
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Federal Research Institute of Pediatric Hematology, Oncology and Immunology