HWN Suggests

A blood cancer diagnosis helped me find true happiness

At some point in the future – and not even the best doctors can predict exactly when – the disease might whirr into life and start scarring my bone marrow, turning it into a barren wasteland that can no longer produce enough blood to keep me alive. I’m hopeful that science will find a fix before that time comes. There are encouraging signs on the horizon. And if not? Well, these days I try not to dwell on the future. I am here, instead, for the present. I am alive. I am alive with the spirit of Christmas.

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Featured

 New Essential Thrombocythemia Treatment Appears to Address Underlying Disease

Phase 1 study found mutant calreticulin-specific monoclonal antibody brings promising results with no dose-limiting toxicities. The monoclonal antibody INCA44989 is the first-ever treatment for essential thrombocythemia (ET) that may alter the underlying mechanism of disease for patients with mutations of calreticulin (mutCALR), according to a recent phase 1 study. The majority of patients achieved a swift and durable response.

Articles of Interest

How I treat essential thrombocythemia

Prevention of vascular events has been so far the main objective of therapy, and continues to be extremely important in the management of patients with ET. Low-dose aspirin and cytoreductive drugs can be administered to this purpose, with cytoreductive treatment being primarily given to patients at high risk of vascular complications. Currently used cytoreductive drugs include hydroxyurea, mainly used in older patients, and interferon α, primarily given to younger patients. There is a need for disease-modifying drugs that can eradicate clonal hematopoiesis and/or prevent progression to more aggressive myeloid neoplasms, especially in younger patients. In this article, we use a case-based discussion format to illustrate our approach to diagnosis and treatment of ET.

Nobody Wants Cancer. But a ‘Big C’ Label Has Surprising Upsides

The average age people are diagnosed with an MPN is 60-something. My diagnosis came at age 38. For the next 15 years, I was treated for essential thrombocythemia, a type of MPN that caused my bone marrow to produce significantly higher than normal numbers of platelets. The proliferation of platelets put me at risk for dangerous clots and ultimately led to complete blockages of two important veins of the portal system, which carries blood to the liver. The Big C label began to look like good news. Once MPNs were classified as blood cancers — including essential thrombocythemia, polycythemia vera and myelofibrosis — interest grew from research laboratories, major medical centers, and small and mega pharmaceutical companies, which now saw these rare and poorly understood conditions as an opportunity. Perhaps pieces to the MPN puzzle could shed light on more common blood cancers, like leukemia and lymphoma. And perhaps treatments for those widely studied cancers could be used to treat MPNs.

Advancements in Essential Thrombocythemia: Targeting External Proteins

ET has very few commercially available treatments today. There is only 1 FDA-approved drug for ET: anagrelide (Agrylin). However, anagrelide is often inadequate, with some prospective trials even suggesting that hydroxyurea is superior, according to Gerds. Providers will also use interferons off-label.

Assessing the Thrombotic Risk of Patients with Essential Thrombocythemia in the Genomic Era

Essential thrombocythemia (ET) is a Philadelphia-negative myeloproliferative neoplasm (MPN) characterized by clonal proliferation of the megakaryocytic lineage within the bone marrow and elevated platelet count in peripheral blood. It is a rare disease, with an incidence rate of 0.2-2.5/100,000 people per year in western countries, and it is associated with long overall survival. However, life expectancy of ET patients is shorter than in the general population,4 largely due to the occurrence of thrombotic events. Consequently, the treatment of this disorder is aimed at reducing the risk of vascular complications.

Essential thrombocythemia: 2024 update on diagnosis, risk stratification, and management

The main goal of therapy is to prevent thrombosis. In this regard, once-daily low-dose aspirin is advised for all patients and twice daily for low-risk disease. Cytoreductive therapy is advised for high-risk and optional for intermediate-risk disease. First-line cytoreductive drugs of choice are hydroxyurea and pegylated interferon-α and second-line busulfan.

Living with Essential Thrombocythemia: You've Got This!

Connecting with others living with ET can be an excellent source of emotional and practical support. Many hospitals, healthcare centers, or online forums offer support groups that allow you to share your experiences with individuals going through similar situations.

Low-Risk Essential Thrombocythemia: A Comprehensive Review

Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm characterized by a persistently elevated platelet count in the absence of a secondary cause. The clinical consequences of uncontrolled thrombocytosis can include both thrombosis and hemorrhage. Patients with features conferring a “high risk” of vascular events benefit from reduction of the platelet count through cytoreductive therapy. The management of patients who lack such high-risk features has until recently been less well defined, but it is now apparent that many require minimal or even no intervention.

Newly diagnosed essential thrombocythemia leading to cardiogenic shock: a case report

Essential thrombocythemia (ET) is a myeloproliferative neoplasm characterized by uninhibited platelet production. It can present with vasomotor symptoms, and less commonly, severe thrombotic events such as myocardial infarction. ST-segment elevation myocardial infarction (STEMI) secondary to the hypercoagulable state in ET is a diagnostic challenge as the complication is rare, especially outside the typical demographics affected by ET such as the female and elderly populations.

One thousand patients with essential thrombocythemia: the Mayo Clinic experience

Prognosis in ET is generally favorable with consistent risk of thrombohemorrhagic complications and disease progression into myelofibrosis (post-ET MF) or acute myeloid leukemia (AML), also known as “blast phase MPN”

The Essential Thrombocythemia in 2020: What We Know and Where We Still Have to Dig Deep

Essential Thrombocythemia (ET) is a Chronic Philadelphia-negative Myeloproliferative Neoplasm (MPN), characterized by marked thrombocytosis, thrombotic and hemorrhagic risk and constitutional symptoms. ET patients carry a low but known risk of disease evolution into other MPNs (Polycythemia Vera and Myelofibrosis) and/or Acute Leukemia.

What is essential thrombocythemia (ET)?

Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50, ET is associated with a proliferation of platelet precursors in the bone marrow and complications frequently include blood clotting and/or bleeding. Less common consequences in the later stages of ET include a transformation to myelofibrosis (marrow scarring) or acute leukemia.

Resources

4 Things to Know About a Rare Blood Cancer Diagnosis

If you’re living with polycythemia vera, myelofibrosis, or essential thrombocythemia, you may have never heard of your condition before being diagnosed—which makes sense. Collectively known as myeloproliferative neoplasms, or MPNs, these rare blood cancers are, well, very rare. About 20,000 Americans are diagnosed with an MPN each year. (For some context, in 2024, an estimated 313,510 people in the US will learn that they have breast cancer.)

MPN Research Foundation

Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50, ET is associated with a proliferation of platelet precursors in the bone marrow and complications frequently include blood clotting and/or bleeding. Less common consequences in the later stages of ET include a transformation to myelofibrosis (marrow scarring) or acute leukemia.

MPN Voice

Essential thrombocythaemia (ET) is sometimes called essential thrombocytosis or primary thrombocytosis (PT). How common is ET? ET is considered to be a rare blood cancer. The number of people diagnosed each year with ET will be between 1.5 and three cases per 100,000 and it is more common in women than men with two women diagnosed for every man.

Blood Cancer United

It is one of a related group of blood cancers known as “myeloproliferative neoplasms” (MPNs), in which cells in the bone marrow that produce blood cells develop and function abnormally. On average, individuals with ET have a normal life expectancy if they are properly monitored and treated. In a small number of patients, the disease may transform to myelofibrosis, acute myeloid leukemia (AML); or less frequently, myelodysplastic syndrome (MDS).

NORD

Over time, it became obvious that the myeloproliferative disorders are caused by genetic accidents (an internal defect) in very early blood cells (stem cells), which are then passed along to all of the progeny of that cell, even as they mature into platelets, red cells, or white blood cells (see below). In acknowledgement of this new understanding, myeloproliferative disorders have been renamed myeloproliferative neoplasms (MPN). For this reason, ET is best thought of as a chronic type of leukemia – albeit one with an overall excellent prognosis and often requiring minimal or no treatment.