Aplastic Anemia Questions & Answers

Updated: Apr 04, 2017
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Questions & Answers

Overview

What is aplastic anemia?

What is the clinical presentation of aplastic anemia?

What are the signs and symptoms of aplastic anemia?

Which lab tests are performed in the evaluation of suspected aplastic anemia?

Which procedures are performed in the evaluation of suspected aplastic anemia?

What are the treatment options for aplastic anemia?

Which medications are used in the treatment of aplastic anemia?

What is included in the nonpharmacologic management of aplastic anemia?

When is surgical care indicated in the treatment of aplastic anemia?

What is the origin of aplastic anemia?

Which organizations have issued guidelines on the diagnosis and management of aplastic anemia?

Where can more information be found about aplastic anemia?

What are the characteristics of Fanconi anemia?

What percentage of aplastic anemia is acquired?

What is the role of hematopoietic progenitors in the pathogenesis of plastic anemia?

What is the central mechanism in the pathogenesis of aplastic anemia?

What is needed for successful hematopoietic cell transplantation (HCT) in severe aplastic anemia?

What is the role of immune dysfunction in the pathogenesis of aplastic anemia?

What is the role of cytokines in the etiology of aplastic anemia?

What is the role of protein expression in the etiology of aplastic anemia?

Which transcription factors are involved in the etiology of aplastic anemia?

What is the role of telomeres in the etiology of aplastic anemia?

What is characteristic of congenital or inherited causes of aplastic anemia?

What are features of dyskeratosis congenital-caused aplastic anemia?

What causes familial aplastic anemia?

What are the characteristics of cartilage-hair hypoplasia-caused aplastic anemia?

What are the characteristics of Pearson syndrome-caused aplastic anemia?

What are characteristics of thrombocytopenia-absent radius (TAR)-caused aplastic anemia?

What are characteristics of Shwachman-Diamond syndrome-caused aplastic anemia?

What are characteristics of Dubowitz syndrome-caused aplastic anemia?

What are characteristics of Diamond-Blackfan anemia (DBA)?

What are acquired causes of aplastic anemia?

How do drugs and elements cause aplastic anemia?

How does paroxysmal nocturnal hemoglobinuria (PNH) cause aplastic anemia?

What is the incidence of aplastic anemia in the US?

What is the global incidence of aplastic anemia?

What are the racial predilections of aplastic anemia?

How does the incidence of aplastic anemia vary among males and females?

How does the incidence of aplastic anemia vary by age?

What is the prognosis of aplastic anemia?

What are the survival rates for aplastic anemia following treatment?

What are the major causes of morbidity and mortality for aplastic anemia?

Which factors increase the risk of myelodysplastic syndromes (MDS) in patients with aplastic anemia?

How does donor age affect the survival rates following bone marrow transplantation for aplastic anemia?

What effect does hematopoietic cell transplantation (HCT) have on the prognosis of aplastic anemia?

Presentation

What are the signs and symptoms of aplastic anemia?

What is necessary to identify the etiologic agent for aplastic anemia?

Which physical findings suggest aplastic anemia?

What is the physical stigmata of inherited marrow-failure syndromes that may indicate aplastic anemia?

DDX

Which conditions are most commonly included in the differential diagnosis of aplastic anemia?

What are the differential diagnoses for Aplastic Anemia?

Workup

How is aplastic anemia diagnosed?

Which CBC count findings suggest aplastic anemia?

What is the role of peripheral blood smear in the diagnosis of aplastic anemia?

What is the role of peripheral blood tests in the diagnosis of aplastic anemia?

What results of hemoglobin electrophoresis and blood-group testing suggest aplastic anemia?

What is the role of serologic testing in the diagnosis of aplastic anemia?

How is the Ham test used to diagnose aplastic anemia?

How is paroxysmal nocturnal hemoglobinuria (PNH) differentiated from aplastic anemia?

What is the role of diepoxybutane incubation in the diagnosis of aplastic anemia?

What is the role of histocompatibility testing in the diagnosis of aplastic anemia?

What is the role of microRNAs in the diagnosis of aplastic anemia?

What is the role of bone marrow aspiration and biopsy in the diagnosis of aplastic anemia?

What is the role of bone marrow culture for the diagnosis of aplastic anemia?

How is aplastic anemia differentiated from myelodysplastic syndrome (MDS)?

How do bone marrow abnormalities found in myelodysplastic syndrome (MDS) differ from those found in aplastic anemia?

How do chromosomal abnormalities found in myelodysplastic syndrome (MDS) differ from aplastic anemia?

What is the role of MRI in the diagnosis of aplastic anemia?

Which criteria are used to stage aplastic anemia?

How is severe aplastic anemia (SAA) defined?

How is very severe aplastic anemia (VSAA) defined?

Treatment

What are the treatment options for severe aplastic anemia (SAA) and very severe aplastic anemia (VSAA)?

When is inpatient care indicated for aplastic anemia?

What are the British Committee for Standards in Haematology treatment recommendations for aplastic anemia?

What is the efficacy of immunosuppressive drugs for the treatments for aplastic anemia?

How are pregnant women with aplastic anemia treated?

In which patient group is monotherapy with hematopoietic growth factors not recommended for the treatment of aplastic anemia?

Why is frequent outpatient follow-up needed for patients with aplastic anemia?

Which specialists should be responsible for treating patients with aplastic anemia?

What are the British Committee for Standards in Haematology recommendations for blood transfusions to treat aplastic anemia?

When is the use of irradiated blood products recommended by the British Committee for Standards in Haematology for the treatment of aplastic anemia?

What is the importance of a transfusion plan in the treatment of aplastic anemia?

What are the risk factors for infections in patients with aplastic anemia?

What are the British Committee for Standards in Haematology guidelines for use of prophylactic antibiotic and antifungal agents in the management of aplastic anemia?

What is the role of cytokine support in the treatment of aplastic anemia?

When is central venous placement indicated in the treatment of aplastic anemia?

What is the treatment of choice for a young patient with severe or very severe aplastic anemia (SAA or VSAA)?

What is the difference between bone marrow and peripheral blood progenitor cell (PBPC) harvesting for the treatment of aplastic anemia?

How is the pain associated with bone marrow donation characterized?

What is a major limitation of hematopoietic cell transplantation (HCT) for treatment of aplastic anemia?

What is a commonly used conditioning regimen for transplantations in aplastic anemia?

What effect do reduced-intensity conditioning (RIC) regimens have on outcomes in severe aplastic anemia (SAA)?

What drugs are used in standard conditioning regimens for transplantation in aplastic anemia?

What increases the risk of graft versus host disease (GVHD) following hematopoietic cell transplantation (HCT) for aplastic anemia?

What are the differences between horse antithymocyte globulin (ATG) and rabbit antithymocyte globulin (ATG) for immunosuppressive therapy in aplastic anemia?

What is the benefit of early referral to a transplantation center following diagnosis of aplastic anemia?

How is fertility affected by high-dose cyclophosphamide conditioning in patients with aplastic anemia?

When is unrelated-donor hematopoietic cell transplantation (HCT) indicated for aplastic anemia?

What has improved outcomes in unrelated-donor hematopoietic cell transplantation (HCT) for aplastic anemia?

How likely is graft versus host disease (GVHD) following hematopoietic cell transplantation (HCT) for the treatment of aplastic anemia?

What can decrease the risk of severe graft versus host disease (GVHD) following hematopoietic cell transplantation (HCT) for aplastic anemia?

How do outcomes compare between horse antithymocyte globulin (ATG) and rabbit antithymocyte globulin (ATG) for conditioning in patients with aplastic anemia?

What is the likelihood of graft failure for unrelated matched hematopoietic cell transplantation (HCT) versus mismatched transplants for the treatment of aplastic anemia?

What can reduce the risk of graft rejection in unrelated donor transplantation for aplastic anemia?

What is the optimal regimen for unrelated-donor hematopoietic cell transplantation (HCT) for aplastic anemia?

Which conditioning regimens have shown good results in aplastic anemia (SAA)?

What is a rescue therapy option for aplastic anemia?

What is the role of umbilical cord blood transplantation (CBT) for the treatment of aplastic anemia?

What are the response and survival rates for aplastic anemia treated with immunosuppressive therapy (IST)?

Which immunosuppressive therapy (IST) is administered as first-line therapy in patients with severe or very severe aplastic anemia (SAA or VSAA)?

What is the role of central venous catheter placement for immunosuppressive therapy (IST) for aplastic anemia?

What is the difference in response to horse or rabbit antithymocyte globulin (ATG) in immunosuppressive therapy (IST) for aplastic anemia?

How is complete responses (CRs) and partial responses (PRs) to immunosuppressive therapy (IST) defined for aplastic anemia?

What is the preferred choice of immunosuppressive therapy (IST) for aplastic anemia?

What is the treatment response in aplastic anemia?

What is the treatment outcome in most cases of aplastic anemia?

What is the role of high-dose cyclophosphamide in the management of aplastic anemia?

What is the relationship between paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia?

What immunosuppressive therapies (ISTs) are being investigated for aplastic anemia?

What are the dietary recommendations for patients with aplastic anemia?

What are the activity recommendations for patients with aplastic anemia?

When is the likelihood of infections increased in patients with aplastic anemia?

What are the treatment options for aplastic anemia refractory to immunosuppressive therapy (IST)?

Medications

What are the goals of drug treatment in patients with aplastic anemia?

What are the treatment options for aplastic anemia?

Which medications in the drug class Chelators are used in the treatment of Aplastic Anemia?

Which medications in the drug class Antineoplastic Agent, Antimetabolite (purine) are used in the treatment of Aplastic Anemia?

Which medications in the drug class Hematopoietic Growth Factors are used in the treatment of Aplastic Anemia?

Which medications in the drug class Immunosuppressive Agents are used in the treatment of Aplastic Anemia?