Chronic Myeloid Leukemia (CML) Quiz

Chronic Myeloid Leukemia (CML)

Philadelphia Chromosome

• A genetic abnormality in which a piece of chromosome 22 breaks off and attaches to chromosome 9, resulting in the BCR-ABL1 fusion gene
• This abnormality leads to the production of a constitutively active tyrosine kinase, which drives the proliferation of malignant cells
• The Philadelphia chromosome is the hallmark of CML and is present in >90% of patients with the disease

Bone Marrow Transplantation (BMT)

• A curative treatment option for CML, particularly for patients in the chronic phase
• Involves replacing the patient's bone marrow with healthy bone marrow cells from a donor
• BMT can be an option for:
  • Patients with advanced disease who are resistant to tyrosine kinase inhibitors (TKIs)
  • Those with a suitable donor and who are at high risk of disease progression
  • Patients who experience a relapse after TKI treatment

Chronic Phase Management

• The goal of chronic phase management is to achieve a complete hematologic response (CHR) and a major molecular response (MMR)
• Treatment options:
  • Imatinib (first-line therapy)
  • Dasatinib or nilotinib (second-line therapy for patients who are resistant or intolerant to imatinib)
• Monitoring:
  • Regular blood counts and molecular testing to assess treatment response
  • Bone marrow biopsies to evaluate for disease progression

Blast Crisis

• A phase of CML characterized by the rapid proliferation of immature cells (blasts) in the blood and bone marrow
• Can occur in patients who are resistant to TKIs or who experience a relapse
• Treatment options:
  • Chemotherapy and/or BMT
  • Clinical trials with novel agents or combinations

Imatinib Treatment

• A TKI that targets the BCR-ABL1 fusion protein
• First-line therapy for patients in the chronic phase of CML
• Dosing: 400 mg orally once daily
• Response rates:
  • Complete hematologic response (CHR): 95-98%
  • Major molecular response (MMR): 60-80%
• Side effects:
  • Gastrointestinal disturbances
  • Fatigue
  • Muscle cramps
  • Rash

Chronic Myeloid Leukemia (CML)

Philadelphia Chromosome

• Genetic abnormality where a piece of chromosome 22 breaks off and attaches to chromosome 9, forming the BCR-ABL1 fusion gene
• Results in the production of a constitutively active tyrosine kinase, driving the proliferation of malignant cells
• Present in >90% of CML patients, making it the hallmark of the disease

Bone Marrow Transplantation (BMT)

• Curative treatment option for CML, particularly for patients in the chronic phase
• Involves replacing the patient's bone marrow with healthy donor cells
• Suitable for patients with advanced disease resistant to tyrosine kinase inhibitors, those with a suitable donor and high risk of disease progression, and those who experience relapse after TKI treatment

Chronic Phase Management

• Goal is to achieve complete hematologic response (CHR) and major molecular response (MMR)
• Treatment options include:
  • Imatinib (first-line therapy)
  • Dasatinib or nilotinib (second-line therapy for imatinib-resistant or intolerant patients)
• Monitoring involves regular blood counts and molecular testing to assess treatment response, as well as bone marrow biopsies to evaluate for disease progression

Blast Crisis

• Phase characterized by rapid proliferation of immature cells (blasts) in the blood and bone marrow
• Can occur in patients resistant to TKIs or who experience relapse
• Treatment options include chemotherapy and/or BMT, as well as clinical trials with novel agents or combinations

Imatinib Treatment

• TKI targeting the BCR-ABL1 fusion protein
• First-line therapy for patients in the chronic phase of CML
• Dosed at 400 mg orally once daily
• Response rates:
  • Complete hematologic response (CHR): 95-98%
  • Major molecular response (MMR): 60-80%
• Common side effects include gastrointestinal disturbances, fatigue, muscle cramps, and rash

Definition and Overview

• CML is a type of cancer that affects the blood and bone marrow.
• It is a myeloproliferative neoplasm, characterized by the uncontrolled growth of immature white blood cells in the bone marrow.
• CML is a slow-growing disease that can progress to a more aggressive phase if left untreated.

Causes and Risk Factors

• The exact cause of CML is unknown, but it is associated with a genetic mutation that leads to the formation of the Philadelphia chromosome.
• Risk factors include exposure to ionizing radiation, genetic predisposition, and family history of CML.

Symptoms

• Fatigue is a common symptom of CML.
• Weight loss is a common symptom of CML.
• Night sweats are a common symptom of CML.
• Bone pain is a common symptom of CML.
• Enlarged spleen is a common symptom of CML.
• Frequent infections are a common symptom of CML.
• Easy bruising or bleeding is a common symptom of CML.

Diagnosis

• Blood tests are used to detect the presence of the Philadelphia chromosome.
• Bone marrow biopsy is used to examine the bone marrow cells.
• Imaging tests (e.g., CT scans, PET scans) are used to rule out other conditions.

Treatment

• Targeted therapy (e.g., tyrosine kinase inhibitors) is used to slow the growth of cancer cells.
• Chemotherapy is used to kill cancer cells.
• Bone marrow transplantation is used to replace cancerous cells with healthy cells.
• Stem cell transplantation is used to replace cancerous cells with healthy cells.
• Treatment goals are to achieve remission and manage symptoms and improve quality of life.

Phases of CML

• CML typically progresses through three phases: chronic phase, accelerated phase, and blastic phase.
• The chronic phase is characterized by slow growth of cancer cells.
• The accelerated phase is marked by rapid growth of cancer cells.
• The blastic phase is characterized by rapid growth and aggressive behavior of cancer cells.

Prognosis

• The prognosis for CML patients has improved significantly with the development of targeted therapies.
• With proper treatment, many patients can achieve long-term remission and manage their symptoms effectively.