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Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.

NCT ID: NCT04350164

Last Updated: 2020-12-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

67 participants

Study Classification

OBSERVATIONAL

Study Start Date

2012-04-01

Study Completion Date

2020-06-30

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Wiskott-Aldrich Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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treatment

romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.

Romiplostim

Intervention Type DRUG

romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.

Interventions

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Romiplostim

romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.

Intervention Type DRUG

Other Intervention Names

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Nplate

Eligibility Criteria

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Inclusion Criteria

* 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
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Federal Research Institute of Pediatric Hematology, Oncology and Immunology

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Anna Shcherbina, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

National Research Center for Pediatric Hematology , Moscow, Russian Federation

Locations

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Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

Moscow, , Russia

Site Status

Countries

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Russia

Other Identifiers

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NCPHOI-2020-02

Identifier Type: -

Identifier Source: org_study_id