MDS and AML Risk Groups and Treatments

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What is one of the primary goals of therapy for patients with Myelodysplastic Syndromes (MDS)?

  • Curing the disease completely
  • Minimizing the overall cost of treatment
  • Eliminating all comorbid conditions
  • Improving quality of life (correct)

What is the role of evaluation of comorbid conditions in MDS management?

  • It is crucial for assessing treatment tolerability and outcomes (correct)
  • Only relevant if the patient is in a high-risk category
  • It does not influence treatment decisions
  • It is required solely for administrative procedures

Which treatment is considered curative for MDS?

  • Immunosuppressive therapy
  • Intensive chemotherapy
  • Allogeneic hematopoietic stem cell transplantation (correct)
  • DNA hypomethylating agents

What characterizes patients with lower-risk MDS in terms of treatment?

<p>Therapy based on transfusion needs (D)</p> Signup and view all the answers

Which therapy is commonly recommended for patients with higher-risk MDS?

<p>Intensive chemotherapy or allogeneic stem cell transplantation (A)</p> Signup and view all the answers

What percentage of newly diagnosed MDS patients have one or more comorbidities?

<p>Approximately 50% (D)</p> Signup and view all the answers

Which treatment strategy is recommended for patients with lower-risk MDS?

<p>Focus on reducing transfusion requirements (B)</p> Signup and view all the answers

What is the significance of having comorbid conditions in MDS management?

<p>It complicates treatment tolerability and outcomes (B)</p> Signup and view all the answers

What type of CEBPA mutation is linked to an overall survival benefit?

<p>Double mutation (D)</p> Signup and view all the answers

Which gene's mutation is most commonly associated with the residue R882?

<p>DNMT3A (B)</p> Signup and view all the answers

What prognostic influence does a CEBPA double mutation confer?

<p>Overall survival benefit (D)</p> Signup and view all the answers

What was the overall survival (OS) rate for allogeneic transplantation in the study?

<p>23% (C)</p> Signup and view all the answers

How is lack of complete remission after the first induction therapy related to prognosis?

<p>Poorer prognosis (B)</p> Signup and view all the answers

What does ECOG stand for, which was mentioned as being associated with improved outcomes in the study?

<p>Eastern Cooperative Oncology Group (D)</p> Signup and view all the answers

What is the prognostic outcome associated with a remission duration of less than 6 months?

<p>Poor prognosis (C)</p> Signup and view all the answers

Which treatment did not show a significant difference in achieving complete remission (CR) compared to best supportive care (BSC)?

<p>Decitabine (C)</p> Signup and view all the answers

What was the relative risk (RR) of complete response (CR) for Decitabine compared to best supportive care?

<p>17% (D)</p> Signup and view all the answers

In the multivariate analysis, which factor had the highest hazard ratio indicating poorer outcomes?

<p>ECOG score (B)</p> Signup and view all the answers

What was the purpose of reporting a 0.3 hazard ratio in the context of transplant outcomes?

<p>To demonstrate less non-relapse mortality (B)</p> Signup and view all the answers

How many patients achieved complete response (CR) in the Decitabine group?

<p>17% (B)</p> Signup and view all the answers

What was the main characteristic of all patients in the cohort discussed?

<p>IPSS Int-2 or higher category (D)</p> Signup and view all the answers

What is the minimum percentage of blasts required for the diagnosis of Acute Myeloid Leukemia (AML)?

<p>20% (A)</p> Signup and view all the answers

Which of the following karyotypes are considered diagnostic for AML, regardless of the percentage of blasts present?

<p>t(8;21) (B), t(15;17) (D)</p> Signup and view all the answers

What patient-specific factor is associated with decreased overall survival in AML?

<p>Age ≥ 60 years (A)</p> Signup and view all the answers

What role does karyotype play in the prognosis of AML?

<p>It is the strongest prognostic factor for response to therapy and survival. (C)</p> Signup and view all the answers

Why is age ≥ 60 years a significant factor in AML prognosis?

<p>Older patients often have higher comorbidities and may struggle with therapy. (B)</p> Signup and view all the answers

Which of the following factors is not considered a prognostic factor for AML?

<p>Ethnicity (B)</p> Signup and view all the answers

What is the clinical significance of identifying specific genetic abnormalities in AML?

<p>They can influence treatment options and prognosis. (B)</p> Signup and view all the answers

What factors can influence the prognosis in AML, according to the World Health Organization classification?

<p>Presence or absence of karyotype abnormalities and molecular mutations. (C)</p> Signup and view all the answers

What is the recommended timeframe for beginning consolidation therapy after hematologic recovery?

<p>Within 2 weeks (B)</p> Signup and view all the answers

Which of the following criteria is NOT part of the complete remission (CR) definition for AML?

<p>Disappearance of a karyotype abnormality (A)</p> Signup and view all the answers

Why might low-intensity remission induction therapy be preferred for certain patients?

<p>To minimize the need for transfusions and improve quality of life (C)</p> Signup and view all the answers

What primary metric is used to determine the success of low-intensity therapy?

<p>Hematologic improvement (C)</p> Signup and view all the answers

What is the typical treatment approach for patients with AML who are ineligible for a transplant?

<p>Consolidation therapy (B)</p> Signup and view all the answers

Which of the following describes a characteristic of intensive remission induction therapy for newly-diagnosed AML?

<p>Results in a complete response rate regardless of blast counts (B)</p> Signup and view all the answers

For patients receiving low-intensity therapy, when does the treatment typically continue?

<p>Until progression of disease occurs (C)</p> Signup and view all the answers

What is the significance of a bone marrow evaluation showing less than 5% blasts in defining complete remission?

<p>It is a critical criterion for defining complete remission (B)</p> Signup and view all the answers

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Study Notes

Risk Groups in MDS and AML

  • Low-risk groups: IPSS low, IPSS intermediate-1, IPSS-R very low, IPSS-R low.
  • High-risk groups: IPSS intermediate-2, IPSS high, IPSS-R intermediate, IPSS-R high, IPSS-R very high.

Treatment Goals for MDS

  • Alter natural disease progression.
  • Decrease reliance on red blood cell transfusions.
  • Enhance quality of life.

Comorbidities in MDS

  • About 50% of newly diagnosed MDS patients have one or more comorbidities, complicating treatment options.
  • Evaluating comorbid conditions is crucial for effective management.

Curative Options

  • Only allogeneic hematopoietic stem cell transplantation offers a potential cure for MDS.
  • Lower-risk patients benefit from:
    • Hematopoietic growth factors.
    • DNA hypomethylating agents.
    • Immunosuppressive therapies.
  • Higher-risk patients may benefit from:
    • Intensive chemotherapy.
    • Allogeneic hematopoietic stem cell transplantation.

Management of Lower-Risk MDS

  • Treatment depends on transfusion independence; typically monitored until transfusion dependence arises.
  • Transfusion support is provided with packed RBCs and platelets as necessary.

Diagnostic Criteria for AML

  • Diagnosis requires ≥20% peripheral blasts or bone marrow biopsy findings consistent with AML.
  • Karyotype abnormalities can also indicate AML regardless of blast percentages, including specific mutations like t(8;21) and inversion(16).

Prognostic Factors for AML

  • Patient-specific factors affecting prognosis:
    • Age ≥60 is linked to reduced overall survival due to therapy tolerability issues.
    • Comorbidities and performance status impact treatment outcomes.
  • AML biology-specific factors:
    • Karyotype is the strongest prognostic factor for induction therapy responses.
    • Distinct genetic abnormalities influence overall survival and treatment sensitivity.

Complete Remission (CR) Criteria for AML

  • Absolute neutrophil count > 1 X 10^9.
  • Platelet count > 100 X 10^9.
  • Independence from transfusions.
  • Bone marrow blast percentage < 5%.
  • No Auer rod presence and absence of extramedullary disease.

Remission Induction Therapy Principles

  • Low-intensity induction therapy is suitable for older or unfit patients.
  • Focus on achieving normal hematopoiesis and improving quality of life rather than requiring complete remission.
  • Transfusion needs may decrease without achieving complete remission.

Intensive Therapy for AML

  • Younger patients often receive intensive remission induction therapy.
  • The overall response rate (ORR) can reach 43%, noting that decitabine may improve complete response rates (CR rates of 17.8% vs. 7.8%).

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