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Completed

NCT Number: NCT04350164

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

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).

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Key information

Conditions

Wiskott-Aldrich Syndrome Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytopenia Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Immune System Diseases Immunologic Deficiency Syndromes Leukocyte Disorders Leukopenia Lymphopenia Primary Immunodeficiency Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

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

Moscow, 117997, Russia

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Exclusion criteria

  • Patients, who do not meet the inclusion criteria.

Treatment and study plan

Romiplostim

Drug

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

Other names: Nplate

Primary outcomes

  1. The percentage of participants with overall platelet response (complete response + partial response)

    Time frame: 1 month (30 day +/- 14 days)

    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.

Secondary outcomes

  1. Percentage of patients with a platelet response

    Time frame: until discontinuation, from at least one month to one year

  2. Number of participants with bleeding events and severity of bleeding

    Time frame: until discontinuation, from at least one month to one year

    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).

  3. Number of participants with adverse events

    Time frame: until discontinuation, from at least one month to one year

Sponsors and collaborators

Lead sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Other

Registry information

Official study title

Retrospective Chart Review of Children With Wiskott-Aldrich Syndrome Who Received Romiplostim in Treatment of Thrombocytopenia.

Important dates

Study start
2012
Primary completion
2019
Study completion
2020
First posted
Apr 16, 2020
Registry last updated
Dec 28, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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