Home » Rare diseases » Acute myeloid leukemia is not a ‘one-size-fits-all’ diagnosis

Dr Kelvin Tan

General Manager, Jazz Pharmaceuticals, UK & Ireland

Acute myeloid leukemia (AML) is an aggressive blood cancer that can quickly become life-threatening if left untreated. It is a complex disease that is not a one-size-fits-all diagnosis.

There are multiple sub-types of AML and it is important to be able to correctly identify and understand them. This insight can give patients and their healthcare professionals a clearer picture of why the disease occurred and what treatment options might be appropriate.

For example, high-risk AML (which comprises nearly a third of all AML diagnoses) is associated with a high risk of the leukemia coming back. Sadly, patients with high-risk AML have few treatment options and some of the lowest survival rates compared to people with other forms of leukemia. This is why early diagnosis and intervention is so important.

AML treatment landscape is evolving

Here is the good news – the treatment landscape for AML is evolving rapidly, a number of new medicines have entered the market in the United States and the European Union, where previously there were limited treatment options.

Today, early identification of high-risk AML can be key to getting patients the most appropriate treatment. A medical history and physical examination provide a physician with important information about risk factors and symptoms and signs of disease.

Cells from the blood and bone marrow are taken and reviewed to detect further abnormalities. This is done using blood taken from a patient’s arm and bone marrow from the hip bone. Further tests are performed on a patient’s DNA to identify which specific abnormalities exist. This level of detail allows for more tailored treatment approaches for AML patients.

Up to one in 20 people will live with a rare disease

Looking beyond AML, building awareness of rare diseases is vital because as many as one in 20 people will live with a rare disease at some point in their life. With increased awareness around these conditions there is also an increased likelihood of early diagnosis, resulting in earlier treatment intervention when possible and research for treatment advancements.

In AML, we’ve seen increased awareness lead to additional treatment options and an understanding among physicians of the needs for early diagnosis and tailored treatment, and on this Rare Disease Day, we hope the same for other rare diseases as well.

In the healthcare industry, we know there is always more work to do, which is why, at Jazz, we’ve never lost sight of the integrity, intellectual curiosity and commitment to continuous evolution that has defined our culture from the beginning and why we continue to raise awareness for rare diseases, like AML.

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