The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids

NCT06568913 | Recruiting

• 1 other identifier: Soh-Med-24-07-12MS
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. It leads to the accelerated removal of these cells by antigen-presenting cells (macrophages) of the spleen and sometimes the liver or other components of the monocyte-macrophage system. The bone marrow compensates for platelet destruction by increasing platelet production. ITP most often occurs in healthy children and young adults within a few weeks following a viral infection . New treatment guidelines have supported a shift from corticosteroids and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor agonists (TPO-RA), romiplostim, eltrombopag have markedly altered the treatment of ITP. The thrombopoietin receptor agonists (TPO-Ras) romiplostim and eltrombopag have shown high therapeutic activity In primary ITP. Romiplostim, a thrombopoiesis-stimulating peptibody, represents a new therapeutic option in adult refractory chronic immune thrombocytopenia (ITP). This study aimed to assess the short-term efficacy and safety of romiplostim in children withChronic ITP. The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation. TPO-RA do not need to be continued forever; about a third of patients In the first year and about another third after two years have a remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TPO-RA, their place in the treatment algorithm, unique aspects of their clinical use, adverse effects.

Conditions

Persistent or Chronic ITP Not Respnding to Steroids

Keywords

CBC

Outcome Measures

Primary Outcomes (1)

• Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists

  The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids

  12 months

Study Arms (1)

Persistent or chronic ITP not responsive to steroids

EXPERIMENTAL

The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids

Diagnostic Test: CBC

Interventions

CBC DIAGNOSTIC_TEST

Platelets counts in pediatric patient with perisistent or chronicITP unresponsive to steroids

Persistent or chronic ITP not responsive to steroids

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• pediatric patients with persistent or chronic Itp on thrombopoietin receptor agonists unresponsive to steroid.

You may not qualify if:

• Any patient with other causes of thrombocytopenia. Any patient with chronic disease . Any patient with Acute ITP . Any newly diagnosed patient with ITP.

Sponsors & Collaborators

• Sohag University: lead

Study Sites (1)

Sohag University hospitals

Sohag, Egypt

RECRUITING

Related Publications (4)

• Zaja F, Barcellini W, Cantoni S, Carpenedo M, Caparrotti G, Carrai V, Di Renzo N, Santoro C, Di Nicola M, Veneri D, Simonetti F, Liberati AM, Ferla V, Paoloni F, Crea E, Volpetti S, Tuniz E, Fanin R. Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study. Am J Hematol. 2016 May;91(5):E293-5. doi: 10.1002/ajh.24341. Epub 2016 Apr 4.

  PMID: 26910388 BACKGROUND

• Mokhtar GM, Tantawy AA, El Sherif NH. Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia. Platelets. 2012;23(4):264-73. doi: 10.3109/09537104.2011.619601. Epub 2012 Apr 3.

  PMID: 22471399 BACKGROUND

• Bussel JB, Soff G, Balduzzi A, Cooper N, Lawrence T, Semple JW. A Review of Romiplostim Mechanism of Action and Clinical Applicability. Drug Des Devel Ther. 2021 May 26;15:2243-2268. doi: 10.2147/DDDT.S299591. eCollection 2021.

  PMID: 34079225 BACKGROUND

• Giordano P, Lassandro G, Barone A, Cesaro S, Fotzi I, Giona F, Ladogana S, Miano M, Marzollo A, Nardi M, Notarangelo LD, Pession A, Ruggiero A, Russo G, Saracco P, Spinelli M, Tolva A, Tornesello A, Palladino V, Del Vecchio GC. Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP). Front Med (Lausanne). 2020 Feb 28;7:66. doi: 10.3389/fmed.2020.00066. eCollection 2020.

  PMID: 32181255 BACKGROUND

Central Study Contacts

shaimaa abdel hameed, Resident

CONTACT

01154450851; shaymaa.abdelhameed@med.sohag.eg

ALZahraa Elsayed Ahmed, Professor

CONTACT

01224340998

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Resident of pediatric and neonatology department, Sohag University Hospitals

Model Details: follow up of platelets count and The outcome of the thrombopoietin receptor agonists in pediatric patient with persistent or chronic ITP unresponsive to steroids

Study Record Dates

• First Submitted: August 21, 2024
• First Posted: August 23, 2024
• Study Start: July 14, 2024
• Primary Completion: July 14, 2025
• Study Completion: July 14, 2025
• Last Updated: August 23, 2024

Record last verified: 2024-08

Locations

EG (1)