Learn what acid-base disorders are and how the kidneys (nephrology) manage the body’s pH balance. Understand the major types and why this balance is vital.
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The treatment of marrow failure is strictly stratified by the underlying cause (acquired versus inherited), patient age, and donor availability. The two primary pillars of treatment are Immunosuppressive Therapy (IST), which aims to halt the autoimmune destruction, and Hematopoietic Stem Cell Transplantation (HSCT), which replaces the defective organ entirely. At Liv Hospital, this algorithm is personalized, ensuring that each patient receives the modality best suited to their biological profile.
For patients with Acquired Aplastic Anemia who lack a matched sibling donor, or for older patients (typically over 40-50 years), IST is the standard of care. It is a regenerative strategy in that it preserves and allows the recovery of the patient’s residual stem cells.
HSCT is the curative standard for young patients with severe aplastic anemia and a matched sibling donor, and for all patients with inherited marrow failure syndromes.
Conditioning refers to the chemotherapy given before the transplant. In marrow failure, the goal differs from that in leukemia.
The integration of Eltrombopag (and newer agents like Avatrombopag) represents a shift towards pharmacological regeneration. By mimicking thrombopoietin’s action, these drugs directly signal the hematopoietic stem cell to proliferate. This is particularly useful in patients who cannot undergo a transplant or who have had a partial response to immunosuppression. It acts as a bridge, sustaining blood counts while the immune system stabilizes.
While regenerative therapies take effect, supportive care keeps the patient alive.
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ATG acts like a reset for the immune system. It contains antibodies that specifically target and kill T-lymphocytes, the white blood cells responsible for attacking the bone marrow in aplastic anemia. By wiping out these attacking cells, the stem cells are given a reprieve and can begin to grow again.
Eltrombopag was initially developed to boost platelet counts. However, it was discovered that it also stimulates the master hematopoietic stem cells. It is now added to immunosuppressive therapy to help kick-start the bone marrow, leading to faster and deeper recovery of blood counts.
Peripheral blood stem cells (PBSC) contain more T-cells than bone marrow. While this is beneficial in fighting leukemia, in aplastic anemia, these extra T cells increase the risk of Graft-Versus-Host Disease (GVHD). Bone marrow grafts are calmer and lead to better long-term quality of life for non-cancer patients.
Generally, yes. Because patients with aplastic anemia do not have cancer, they do not require the incredibly high, toxic doses of chemotherapy used to kill leukemia cells. The conditioning is gentler, focused mainly on immune suppression, which typically results in fewer immediate side effects and organ damage.
Immunosuppressive Therapy is not a quick fix. It typically takes 3 to 6 months to see a meaningful improvement in blood counts. Patience is key. During this time, the patient remains dependent on transfusions and careful infection prevention.
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