Outcomes in Bone Marrow Aplasia.

Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorAssiut University

About this trial

Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and fatty infiltration of the bone marrow space. Underlying mechanisms include immune-mediated attack, telomere defects, and inherent HSC compartment insufficiency. These events may occur individually or in concert, mostly involving effector T cells Historical treatment has included the use of high-dose chemotherapy and allogeneic stem cell transplantation as well as lymphotoxic immunosuppressive therapy (IST) Thrombopoietin (TPO) regulates platelet production, maturation, and release through binding of c-mpl on megakaryocytes.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • Eltrombopag

    Drug

    Treatment with eltrombopag in a dose of (50-150mg/d)

Treatment groups

3 Participants
are divided into 1 treatment group
Group A: Cases of aplastic anemia recieving EltrombopagExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Determine hematological response after 6 months

Change in CBC elements after 6 months of treatment

Time frame
6 months to 5 years
2

Determine overall survival rate.

Number if years estimated to survive after treatment

Time frame
5 years

Secondary outcomes

1

Rate of relapse in patients deemed responsers at 6 months.

Time frame
Around 1 year
2

Clonal evolution to myeloid malignancy or new chromosomal abnormality

Time frame
Around 2 years
3

Change in serum iron and ferritin over time of treatment

Time frame
From 6 months to 5 years
4

Eltrombopag efficacy in increasing platelet count

Time frame
After6 months

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

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