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

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology (Other)

Overall Status

Completed

CT.gov ID

NCT04350164

Collaborator

(none)

Enrollment

Location

Actual Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

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 pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS).

Condition or Disease	Intervention/Treatment	Phase
Wiskott-Aldrich Syndrome	Drug: Romiplostim

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 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.

Study Design

Study Type:

Observational

Actual Enrollment :

67 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

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

Actual Study Start Date :

Apr 1, 2012

Actual Primary Completion Date :

Dec 27, 2019

Actual Study Completion Date :

Jun 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
treatment 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. Other Names: Nplate

Arm

Intervention/Treatment

treatment

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.

Other Names:

Nplate

Outcome Measures

Primary Outcome Measures

The percentage of participants with overall platelet response (complete response + partial response) [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 Outcome Measures

Percentage of patients with a platelet response [until discontinuation, from at least one month to one year]
Number of participants with bleeding events and severity of bleeding [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).
Number of participants with adverse events [until discontinuation, from at least one month to one year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology	Moscow		Russian Federation	117997

Sponsors and Collaborators

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Investigators

Principal Investigator: Anna Shcherbina, MD, PhD, National Research Center for Pediatric Hematology , Moscow, Russian Federation

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

ClinicalTrials.gov Identifier:

NCT04350164

Other Study ID Numbers:

NCPHOI-2020-02

First Posted:

Apr 16, 2020

Last Update Posted:

Dec 28, 2020

Last Verified:

Dec 1, 2020

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Thrombocytopenia

Wiskott-Aldrich Syndrome

Syndrome

Study Results

No Results Posted as of Dec 28, 2020