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Accreditation/ Credit Designation

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. These activites are not approved for AMA PRA Category 1 Credit

Acknowledgment of Commercial Support

This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.


Oncology Briefings™: Updates in Rare Hematology: Advancing Care and Improving Outcomes for Patients With Aplastic Anemia PER Pulse™ Recap

PER Pulse Recap

PER Pulse™ Recap


1 of 3
PER Pulse™ Recap

How to Identify Aplastic Anemia and Confirm Diagnosis

The online continuing medication education activity Oncology Briefings™: Updates in Rare Hematology: Advancing Care and Improving Outcomes for Patients With Aplastic Anemia provides oncologists and other health care providers with engaging instruction on how to identify aplastic anemia in their patients and current and evolving treatment standards in this rare blood disorder. Leading expert, Danielle Townsley, MD, MSc, a hematologist in Bethesda, Maryland, answers key questions, supported by the presentation of clinical science, about aplastic anemia. This first of 3 PER Pulse™ Recaps from this program focuses on the distinguishing features of aplastic anemia and how to properly diagnosis it.

Dr. Townsley discusses distinct symptoms patients may present with that suggest a diagnosis of aplastic anemia, as well as the necessary steps to take to confirm diagnosis and rule out alternative causes:

  • Aplastic anemia is characterized by a clinical presentation of pancytopenia in which 2 of the following must be present to confirm a diagnosis of severe disease: neutrophil levels: ˂0.5 x 109/L; platelet count: ˂20 x 109/L; and reticulocyte count: ˂60 x 109/L. In patients with very severe disease, neutrophil levels are even lower: ˂0.2 x 109/L.
  • Personal and family history should be taken, including comprehensive physical examination, to evaluate for an inherited syndrome. The main causes of inherited aplastic anemia include Fanconi anemia and dyskeratosis congenita (a condition of telomere syndrome), and less commonly, Shwachman-Diamond syndrome and congenital amegakaryocytic thrombocytopenia.
  • A bone marrow aspirate and trephine biopsy are both required for the confirmatory diagnosis of aplastic anemia. Myelodysplastic syndrome and leukemia must be ruled out via cytogenetic analysis and flow cytometry, respectively. Aplastic anemia can also occur in association with paroxysmal nocturnal hemoglobinuria, which is best evaluated by performing flow cytometry of peripheral blood for glycosylphosphatidylinositol-anchored proteins.

For additional information and commentary on this topic, as well as audio and supporting text, visit www.gotoper.com/online-cme-activities/oncology-briefing/oncology-briefingstrade-updates-in-rare-hematology-advancing-care-and-improving-outcomes-for-patients-with-aplastic-anemia.

For information on other topics, visit www.gotoper.com.

2 of 3
PER Pulse™ Recap

How to Treat Aplastic Anemia

The online continuing medication education activity Oncology Briefings™: Updates in Rare Hematology: Advancing Care and Improving Outcomes for Patients With Aplastic Anemia provides oncologists and other health care providers with engaging instruction on how to identify aplastic anemia in their patients and current and evolving treatment standards in this rare blood disorder. Leading expert, Danielle Townsley, MD, MSc, a hematologist in Bethesda, Maryland, answers key questions, supported by the presentation of clinical science, about aplastic anemia. This second of 3 PER Pulse™ Recaps from this program focuses on current strategies to treat aplastic anemia.

Dr. Townsley details the sequencing strategies used to treat patients with aplastic anemia, as well as important considerations to make for age and other characteristics:

  • Definitive, potentially curative, therapy typically includes hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST). Treatment is further striated based on patient characteristics, such as age, and disease characteristics, including grade and origin. Intolerability of treatment and increased risk of graft-versus-host disease following HSCT makes treatment for older patients more complex.
  • IST with horse antithymocyte globulin (ATG) and cyclosporine (CsA) is the recommended first-line treatment for patients with acquired severe or very severe aplastic anemia who lack a related human leukocyte antigen–matched donor, and for all older adults.
  • Rabbit ATG plus CsA is reserved for treatment of relapsed or refractory aplastic anemia.
  • Allogeneic HSCT is the only curative treatment for patients with inherited disease. This treatment is preferred for younger patients with histocompatible donors. Transplantation can become an option for patients 40 years or older who have failed to respond to first-line IST if a suitable donor, whether sibling or unrelated, is available.
  • Supportive care for aplastic anemia consists of careful monitoring of blood levels, appropriate red blood cell and platelet transfusions, antimicrobial prophylaxis to prevent infection, and iron chelation therapy in transfusion-dependent patients.

For additional information and commentary on this topic, as well as audio and supporting text, visit www.gotoper.com/online-cme-activities/oncology-briefing/oncology-briefingstrade-updates-in-rare-hematology-advancing-care-and-improving-outcomes-for-patients-with-aplastic-anemia.

For information on other topics, visit www.gotoper.com.

3 of 3
PER Pulse™ Recap

Emerging Treatment Strategies for Aplastic Anemia

The online continuing medication education activity Oncology Briefings™: Updates in Rare Hematology: Advancing Care and Improving Outcomes for Patients With Aplastic Anemia provides oncologists and other health care providers with engaging instruction on how to identify aplastic anemia in their patients and current and evolving treatment standards in this rare blood disorder. Leading expert, Danielle Townsley, MD, MSc, a hematologist in Bethesda, Maryland, answers key questions, supported by the presentation of clinical science, about aplastic anemia. This third and final PER Pulse™ Recap from this program focuses on emerging strategies to treat aplastic anemia.

Dr. Townsley provides key information from recent and ongoing clinical trials that are examining novel compounds to treat patients with aplastic anemia, as well as guidance how these new agents fit into current strategies:

  • Eltrombopag, a thrombopoietin receptor agonist that was initially approved by the US Food and Drug Administration for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura, has been shown to increase platelet counts and a hematologic response in patients with aplastic anemia in phase I/II trials.
  • A more recent phase I/II nonrandomized, open-label trial evaluated eltrombopag in combination with standard immunosuppressive therapy for patients with newly diagnosed, severe aplastic anemia, from the National Heart, Lung, and Blood Institute, grouped 92 patients into 3 cohorts. The complete response (CR) rate at 6 months was 36% and the overall response rate (ORR) was 80%. Overall survival, with a median follow up of 2 years, was 97%.
  • In this trial, patients in the third cohort, who received eltrombopag plus antithymocyte globulin plus cyclosporine from day 1 of treatment through 6 months had the best responses. The CR in this group of patients was 58% and ORR was 94%.

For additional information and commentary on this topic, as well as audio and supporting text, visit www.gotoper.com/online-cme-activities/oncology-briefing/oncology-briefingstrade-updates-in-rare-hematology-advancing-care-and-improving-outcomes-for-patients-with-aplastic-anemia.

For information on other topics, visit www.gotoper.com.





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