Clinical trial NCT05846529: Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP
Official title: Assessment of Response Rate and Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP in Assiut.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Thrombopoietin Receptor Agonist
Lead sponsor: Assiut University
Sponsor class: OTHER
First posted: 2023-05-06
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Observational model: CASE_CONTROL
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants and children between age of (2 year to \<18 years old)

Brief summary: This study aimed to evaluate the response rate and safety of oral thrombopoietin receptor agonist (Eltrombopag drug) used for children with chronic immune thrombocytopenia in patients attending Assiut university children's hospital.

Detailed description: Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by decrease number of the platelets due to destruction of the platelets by antiplatelet antibodies. Also, platelet production can be reduced because the antiplatelet antibodies can also damage megakaryocytes . Its prevalence being approximately 8 per 100,000 children It is characterized by temporary or persistent reduction of the platelet number to less than 100 × 109/liter.

All new cases at diagnosis are defined as acute ITP. While, Persistent ITP is defined as ITP lasting between 3 and 12 months from diagnosis while chronic ITP is defined as the presence of ITP for more than one-year ITP Patients have an increased hazard of bleeding that may have no effect on patient's life or have life-threatening danger the goal of treatment in chronic ITP is to increase and then keep platelets in a sufficient number to prevent bleeding

. Meanwhile most life-threatening bleeding occurs with platelet number less than 30 × 109/liter.

New guideline recommend that treatment constricted on symptomatic patients with number lower than 30 × 109/liter (3,6) new Guidelines recommended that corticosteroids as first-line therapy and splenectomy considered as one of several second-line therapies. Other choices exist for ITP patients with insufficient response or who do not undergo to splenectomy including IVIg, IV anti-D, Rituximab, danazol, azathioprine, Vinca alkaloids and cyclophosphamide New consensus statements and guidelines recommend thrombopoietin-receptor (TPO-R) agonists as second and third line of treatment Eltrombopag consider the first oral, nonpeptide TPO-R agonist accepted for the treatment of chronic ITP in patients with insufficient response to at least one other therapy It rises platelet number by increase platelet production by binding to the transmembrane domain of the TPO-R. It does not compete with endogenous TPO in vitro and it enhance production and differentiation of BM progenitor cells in the megakaryocytic lineage

Eligibility criteria: Inclusion Criteria:

* Subjects between 2 year and \<18 years of age in both sex
* Confirmed diagnosis of chronic ITP
* all patient received oral thrombopoietin receptor agonist (Eltrombopag) for 6 months or more

Exclusion Criteria:

* age of the patient \< 2years and \>18 years
* secondary thrombocytopenia
* Concurrent or past malignant disease, including myeloproliferative disorder
* patient with platelet agglutination abnormality that prevents reliable measurement of platelet counts
* patient with bone marrow aplasia

Primary outcomes:
measure: response to oral thrombopoietin receptor agonist
timeFrame: baseline
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
References:
citation: Kuter DJ. The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Rev. 2022 May;53:100909. doi: 10.1016/j.blre.2021.100909. Epub 2021 Nov 16.
citation: Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. doi: 10.1111/bjh.15573. Epub 2018 Sep 7.