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

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

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).

Study Overview

• Status: Completed
• Conditions: Wiskott-Aldrich Syndrome
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 67

Contacts and Locations

Study Locations

• Russian Federation
  • Moscow, Russian Federation, 117997
    • Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

patients of the immunology department

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	1 month (30 day +/- 14 days)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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).	until discontinuation, from at least one month to one year
Number of participants with adverse events		until discontinuation, from at least one month to one year

Collaborators and Investigators

• Sponsor: 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 record dates

Study Major Dates

• Study Start (Actual): April 1, 2012
• Primary Completion (Actual): December 27, 2019
• Study Completion (Actual): June 1, 2020

Study Registration Dates

• First Submitted: April 10, 2020
• First Submitted That Met QC Criteria: April 15, 2020
• First Posted (Actual): April 16, 2020

Study Record Updates

• Last Update Posted (Actual): December 28, 2020
• Last Update Submitted That Met QC Criteria: December 24, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immunologic Deficiency Syndromes; Immune System Diseases; Disease; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Blood Platelet Disorders; Leukopenia; Leukocyte Disorders; Primary Immunodeficiency Diseases; Lymphopenia; Syndrome; Thrombocytopenia; Wiskott-Aldrich Syndrome
• Other Study ID Numbers: NCPHOI-2020-02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No