Clinical trials on Chronic myeloid leukemia (CML)

Chronic Myeloid Leukemia (CML) is a type of cancer that starts in the bone marrow and involves the overproduction of white blood cells called granulocytes. It is characterized by the presence of an abnormal chromosome known as the Philadelphia chromosome, which results from a specific genetic mutation. CML typically progresses slowly and can remain in a chronic phase for years before potentially advancing to more aggressive stages. Early symptoms may include fatigue, weight loss, and an enlarged spleen, but some patients might not show symptoms until the disease is more advanced.

Complications

  • Progression to Accelerated Phase and Blast Crisis: Without treatment, CML can evolve from the chronic phase to more aggressive forms, leading to rapid deterioration of health.
  • Anemia: Reduced production of red blood cells causes fatigue, weakness, and pale skin.
  • Infections: An abnormal increase in immature white blood cells impairs the immune system, making patients more susceptible to infections.
  • Bleeding and Bruising: Disruption in platelet production can lead to increased bleeding and bruising.
  • Bone Pain and Bone Damage: Overexpansion of bone marrow can lead to pain and structural damage to bones.
  • Splenomegaly: Enlargement of the spleen can cause abdominal pain and fullness, affecting appetite and digestion.

Treatment Methods

  • Tyrosine Kinase Inhibitors (TKIs): Drugs like imatinib, dasatinib, and nilotinib target the abnormal protein produced by the Philadelphia chromosome, effectively managing the disease in most patients.
  • Allogeneic Stem Cell Transplantation: Considered for patients who do not respond to TKIs or are in advanced stages, this treatment can potentially cure CML but involves significant risks.
  • Interferon-alpha Therapy: Used to stimulate the immune system before the widespread adoption of TKIs and in specific cases where TKIs are not suitable.
  • Chemotherapy: Applied in cases where TKIs are ineffective, especially to manage symptoms in advanced stages.

Prognosis

With the advent of TKIs, the prognosis for patients with Chronic Myeloid Leukemia has improved dramatically. Most patients treated with TKIs can expect to live normal or near-normal lifespans with well-managed symptoms. The five-year survival rate is very high when the disease is detected early and treated effectively. However, without treatment, CML can progress to more aggressive stages, significantly reducing life expectancy and quality of life. Continuous monitoring and adherence to treatment are crucial to ensure the best possible outcome and to manage any side effects or resistance to therapy.