Treatment Overview

Living with aplastic anemia can be deeply challenging, as its symptoms of fatigue, weakness, and frequent infections disrupt daily life and cause anxiety. Aplastic anemia is a rare but serious condition where the bone marrow stops producing enough new blood cells, including red cells (causing anemia), white cells (increasing infection risk), and platelets (increasing bleeding risk). This deficiency requires specialized and timely intervention.

Treatment is critical to restoring normal blood cell counts, thereby relieving symptoms and preventing life-threatening complications like severe infection or uncontrolled bleeding. Since aplastic anemia is often caused by the body’s own immune system mistakenly attacking the bone marrow, medication choices depend on whether the patient is eligible for a bone marrow transplant or if immunosuppressive therapy is needed. Treatment plans are highly individualized based on the patient’s age and the severity of the condition (National Heart, Lung, and Blood Institute, 2022).

Overview of treatment options for Aplastic Anemia

The main goal of treating aplastic anemia is to revitalize the bone marrow to produce healthy blood cells or to replace the faulty bone marrow entirely. Treatment is generally split into two key approaches:

  1. Stem Cell Transplantation (SCT): This procedure offers a potential cure, especially for younger patients with a matching sibling donor.
  2. Immunosuppressive Therapy (IST): This medication-based approach is used when a transplant is not possible, focusing on calming the immune system attack on the bone marrow.

Both approaches are often supported by transfusions and antimicrobial drugs to manage the immediate risks associated with low blood counts. Medications are the primary route for patients over the age of 50 or those lacking a suitable donor.

Medications used for Aplastic Anemia

The core drug classes used to treat moderate to severe aplastic anemia include Immunosuppressants and Stimulants.

1. Immunosuppressive Agents: These drugs treat the underlying immune attack. The primary combination typically involves Antithymocyte Globulin (ATG) and Cyclosporine. ATG is given intravenously in a hospital setting and works rapidly. Cyclosporine is an oral medication taken long-term to keep the immune system quiet.

2. Bone Marrow Stimulants: Thrombopoietin Receptor Agonists, such as eltrombopag, are a newer class of oral medication used either alone or in combination with IST. They help spur the bone marrow to increase blood cell production.

3. Supportive Medications:

  • Antibiotics, Antivirals, and Antifungals: These are essential to prevent and treat infections, which are common due to the low white blood cell count.
  • Androgens: Rarely used today, but certain male hormones may sometimes stimulate blood cell production.

Response to immunosuppressive therapy is not immediate; recovery often takes several weeks or months. Blood counts may stabilize or improve gradually, with platelet counts usually taking the longest to recover (Mayo Clinic, 2023).

How these medications work

The major medications used in aplastic anemia work by directly addressing the immune system or by providing a powerful signal to the bone marrow stem cells.

Immunosuppressive Agents (ATG and Cyclosporine): T-cells mistakenly attack bone marrow stem cells. ATG destroys these T-cells to stop the faulty immune response. Cyclosporine suppresses remaining T-cells, preventing a new attack.

Thrombopoietin Receptor Agonists (Eltrombopag): These newer agents stimulate the thrombopoietin receptor, which normally regulates platelet production. This stimulation boosts the growth and differentiation of all blood cell lines (red, white, and platelets) from stem cells (MedlinePlus, 2021).

Side effects and safety considerations

Given the potency of these drugs, side effects are common and require close monitoring by a hematologist.

Immunosuppressants risk allergic reactions (e.g., during ATG) and heightened infection risk due to immune suppression. Cyclosporine can cause kidney issues (requiring lab checks), high blood pressure, or increased hair growth.

Bone Marrow Stimulants may cause liver toxicity (requiring monitoring), headache, or nausea. Since treatment may fail to fully restore counts, patients are monitored for clonal evolution, a rare but serious bone marrow change necessitating a therapy switch. Patients should seek immediate care for high fever, unusual bruising, or uncontrolled bleeding.

Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.

References

  1. National Heart, Lung, and Blood Institute. https://www.nhlbi.nih.gov
  2. Mayo Clinic. https://www.mayoclinic.org
  3. MedlinePlus. https://medlineplus.gov
  4. National Organization for Rare Disorders. https://rarediseases.org

Medications for Aplastic Anemia

These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Aplastic Anemia.

Found 9 Approved Drugs for Aplastic Anemia

MethylPREDNISolone

Brand Names
Solu-Medrol MethylPREDNISolone, Solu-Medrol, Medrol

MethylPREDNISolone

Brand Names
Solu-Medrol MethylPREDNISolone, Solu-Medrol, Medrol
Form: Injection, Tablet
Method of administration: Oral, Intravenous, Intramuscular
FDA approval date: October 24, 1957
Classification: Corticosteroid
When oral therapy is not feasible, and the strength, dosage form, and route of administration of the drug reasonably lend the preparation to the treatment of the condition, the intravenous or intramuscular use of Methylprednisolone Sodium Succinate for Injection, USP, is indicated as follows: Allergic states Control of severe or incapacitating allergic conditions intractable to adequate trials of conventional treatment in asthma, atopic dermatitis, contact dermatitis, drug hypersensitivity reactions, perennial or seasonal allergic rhinitis, serum sickness, transfusion reactions. Dermatologic diseases Bullous dermatitis herpetiformis, exfoliative erythroderma, mycosis fungoides, pemphigus, severe erythema multiforme (Stevens-Johnson syndrome). Endocrine disorders Primary or secondary adrenocortical insufficiency (hydrocortisone or cortisone is the drug of choice; synthetic analogs may be used in conjunction with mineralocorticoids where applicable; in infancy, mineralocorticoid supplementation is of particular importance), congenital adrenal hyperplasia, hypercalcemia associated with cancer, nonsuppurative thyroiditis. Gastrointestinal diseases To tide the patient over a critical period of the disease in regional enteritis (systemic therapy) and ulcerative colitis. Hematologic disorders Acquired (autoimmune) hemolytic anemia, congenital (erythroid) hypoplastic anemia (Diamond-Blackfan anemia), idiopathic thrombocytopenic purpura in adults (intravenous administration only; intramuscular administration is contraindicated), pure red cell aplasia, selected cases of secondary thrombocytopenia. Miscellaneous Trichinosis with neurologic or myocardial involvement, tuberculous meningitis with subarachnoid block or impending block when used concurrently with appropriate antituberculous chemotherapy. Neoplastic diseases For the palliative management of leukemias and lymphomas. Nervous System Acute exacerbations of multiple sclerosis; cerebral edema associated with primary or metastatic brain tumor, or craniotomy. Ophthalmic diseases Sympathetic ophthalmia, uveitis and ocular inflammatory conditions unresponsive to topical corticosteroids. Renal diseases To induce diuresis or remission of proteinuria in idiopathic nephrotic syndrome or that due to lupus erythematosus. Respiratory diseases Berylliosis, fulminating or disseminated pulmonary tuberculosis when used concurrently with appropriate antituberculous chemotherapy, idiopathic eosinophilic pneumonias, symptomatic sarcoidosis. Rheumatic disorders As adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in acute gouty arthritis; acute rheumatic carditis; ankylosing spondylitis; psoriatic arthritis; rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy). For the treatment of dermatomyositis, temporal arteritis, polymyositis, and systemic lupus erythematosus.

Eltrombopag

Brand Names
Promacta, Alvaiz

Eltrombopag

Brand Names
Promacta, Alvaiz
Form: Tablet, Powder
Method of administration: Oral
FDA approval date: April 01, 2016
Classification: Thrombopoietin Receptor Agonist
PROMACTA is a thrombopoietin receptor agonist indicated: for the treatment of thrombocytopenia in adult and pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. PROMACTA should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

Felbatol

Generic Name
Felbamate

Felbatol

Generic Name
Felbamate
Form: Tablet, Suspension
Method of administration: Oral
FDA approval date: July 29, 1993
Classification: Anti-epileptic Agent
Felbamate oral suspension is not indicated as a first line antiepileptic treatment. Felbamate oral suspension is recommended for use only in those patients who respond inadequately to alternative treatments and whose epilepsy is so severe that a substantial risk of aplastic anemia and/or liver failure is deemed acceptable in light of the benefits conferred by its use. If these criteria are met and the patient has been fully advised of the risk, and has provided written acknowledgment, felbamate oral suspension can be considered for either monotherapy or adjunctive therapy in the treatment of partial seizures, with and without generalization, in adults with epilepsy and as adjunctive therapy in the treatment of partial and generalized seizures associated with Lennox-Gastaut syndrome in children.

Ferriprox

Generic Name
Deferiprone

Ferriprox

Generic Name
Deferiprone
Form: Tablet, Solution
Method of administration: Oral
FDA approval date: November 25, 2011
Classification: Iron Chelator
FERRIPROX Tablets are an iron chelator indicated for the treatment of transfusional iron overload in adult and pediatric patients 8 years of age and older with thalassemia syndromes.

Solu-Cortef

Generic Name
Solu-Cortef

Solu-Cortef

Generic Name
Solu-Cortef
Form: Injection
Method of administration: Intravenous, Intramuscular
FDA approval date: April 27, 1955
Classification: Corticosteroid
When oral therapy is not feasible, and the strength, dosage form, and route of administration of the drug reasonably lend the preparation to the treatment of the condition, the intravenous or intramuscular use of hydrocortisone sodium succinate for injection is indicated as follows: Allergic states: Control of severe or incapacitating allergic conditions intractable to adequate trials of conventional treatment in asthma, atopic dermatitis, contact dermatitis, drug hypersensitivity reactions, serum sickness, transfusion reactions. Dermatologic diseases: Bullous dermatitis herpetiformis, exfoliative erythroderma, mycosis fungoides, pemphigus, severe erythema multiforme (Stevens-Johnson syndrome). Endocrine disorders: Primary or secondary adrenocortical insufficiency (hydrocortisone or cortisone is the drug of choice; synthetic analogs may be used in conjunction with mineralocorticoids where applicable; in infancy, mineralocorticoid supplementation is of particular importance), congenital adrenal hyperplasia, hypercalcemia associated with cancer, nonsuppurative thyroiditis. Gastrointestinal diseases: To tide the patient over a critical period of the disease in regional enteritis (systemic therapy) and ulcerative colitis. Hematologic disorders: Acquired (autoimmune) hemolytic anemia, congenital (erythroid) hypoplastic anemia (Diamond Blackfan anemia), idiopathic thrombocytopenic purpura in adults (intravenous administration only; intramuscular administration is contraindicated), pure red cell aplasia, select cases of secondary thrombocytopenia. Miscellaneous: Trichinosis with neurologic or myocardial involvement, tuberculous meningitis with subarachnoid block or impending block when used concurrently with appropriate antituberculous chemotherapy. Neoplastic diseases: For the palliative management of leukemias and lymphomas. Nervous System: Cerebral edema associated with primary or metastatic brain tumor, or craniotomy. Ophthalmic diseases: Sympathetic ophthalmia, uveitis and ocular inflammatory conditions unresponsive to topical corticosteroids. Renal diseases: To induce diuresis or remission of proteinuria in idiopathic nephrotic syndrome, or that due to lupus erythematosus. Respiratory diseases: Berylliosis, fulminating or disseminated pulmonary tuberculosis when used concurrently with appropriate antituberculous chemotherapy, idiopathic eosinophilic pneumonias, symptomatic sarcoidosis. Rheumatic disorders: As adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in acute gouty arthritis; acute rheumatic carditis; ankylosing spondylitis; psoriatic arthritis; rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy). For the treatment of dermatomyositis, temporal arteritis, polymyositis, and systemic lupus erythematosus.
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