Eltrombopag in Children With Idiopathic Aplastic Anemia

• Status: Completed
• Trial end date: 2020-01-05
• Target enrollment: 0
• Participant gender: All

Summary

Aplastic anemia is a rare disorder characterized by pancytopenia and a hypo cellular bone marrow.but,It is very serious disease causing morbidity and mortality.

Aplastic anemia can be treated effectively with haematopoietic stem cell transplantation and immunosuppressive drug regimens but haematopoietic stem cell transplantation has limitations due to its cost and many patient are unsuitable. Immunosuppressive drug has a significant number of patients have persistent cytopenias. Currently, the treatment of these patients is regular transfusion, which are expensive, inconvenient, and associated with serious side effects related to iron overload and transfusion.

Eltrombopag is an oral thrombopoietin mimetic that selectively binds at the transmembrane and juxtamembrane domains of the thrombopoietin receptor, at sites distinct from the binding site of thrombopoietin therefore it does not compete for binding with the native molecule. It promoting thrombopoiesis and release of platelets from mature megakaryocytes. Also, promote other hematopoietic stem cell as well as in thrombopoiesis .

• Phase: Phase 4
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Assiut University

Criteria

Inclusion Criteria:

Current diagnosis of sever and moderate Aplastic anemia

• Diagnosis of sever Aplastic anemia is established if Bone marrow cellularity <25% or and at least two of the following criteria are met:- (i) absolute neutrophil count less than 0.5 × 109/L, (ii) platelet count less than 20 × 109/L, and (iii) reticulocyte count less than 20 × 109/L

• Moderate aplastic anemia is defined as bone marrow cellularity <50 percent and depression of at least two out of three blood counts below the normal values: criteria are met:- (i) absolute neutrophil count less than 1200/mm3, (ii) platelet count less than 70,000/mm3, and (iii) anemia with hemoglobin less than or equal to 8.5 g/dL and absolute reticulocyte count less than or equal to 60,000/mm3 in transfusion-dependent patients but not fulfilling the criteria of sever aplastic anemia

• No, evidence of viral or drug suppression of the marrow, dysplasia, or underproduction anemias secondary to B12, folate, iron or other reversible causes.

• Age equal to 1 years old to 18 years old

• Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.

• Hematopoietic stem cell transplantation is not available or suitable as a treatment option or has been refused by the patient.

• Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag

Exclusion Criteria:

Prior and/or active medical history of:-

• Fanconi anemia (via chromosomal breakage test or growth arrest by flow cytometry). Other known underlying congenital/inherited marrow failure syndromes.

• Symptomatic Paroxysmal Nocturnal Hemoglobinuria

• Other known or suspected underlying primary immunodeficiency

• Any malignancy

• Active infection not responding to appropriate therapy

• Any out of range lab values Creatinine >2.5 mg/dL× the upper limit of normal, Total bilirubin >1.5 × the upper limit of normal mg/dL ,Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5 × the upper limit of normal

• Hypersensitivity to eltrombopag or its components

• Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely.