MPN Foundation studying MPN disease progression

Understanding how ET and other MPNs progress to more serious illnesses--myelofibrosis or acute myeloid leukemia--will be a focus of the MPN Foundation in the coming years, Dr. Raajit Rampal (Memorial Sloan Kettering), MPN expert and advisor to the foundation, said in a Zoom program February 24.

The program was also sponsored by MPN Advocacy and Education International.

Dr. Rampal outlined the many questions that still surround progression in MPN patients: Why do some patients progress and not others? Can progression be stopped? Will patients continue to need treatment if disease progression can be halted? And at what point in disease progression should any new medications be introduced to avoid what Dr. Rampal called "clinical or financial toxicity," that is the patient's ability to tolerate and afford a med that halts disease progression should one become available?

Currently, patients taking ropeginterferon, the latest generation of interferon used for some MPN patients, seem to develop fewer clots and show less progression. However, that needs more study.

The only procedure that offers a cure for an MPN is stem cell transplant (SCT). However, Dr. Rampal noted that this procedure has a 20 percent mortality rate, so it is not suggested to patients whose ET is stable and not progressing.

Kapila Viges, spokesperson for the MPN Foundation, said that its objectives will be four-fold: 

1. To come to a better definition of "progression"

2. To identify the factors that might predict progression in individual patients

3. To identify clinicians caring for MPN patients

4. To offer care guidelines for patients with regard to progression

Right now, there are some general things that can be said about disease progression in ET and other MPNs. According to Dr. Rampal, it takes 10 years or more for someone with ET to progress to another disease. 

In addition, progression from ET to acute leukemia, the most serious and largely fatal disease, is rare. Dr. Rampal said only about 2 percent of ET patients develops AML. However, he was also careful to note that statistics apply to groups, not individuals, so the trick will be to identify what happens in that 2 percent of patients who do progress to AML.

In response to questions from program viewers, Dr. Rampal said that clinicians now try to spot progression through changes in blood counts and symptoms. Fluctuations in platelet counts is normal for most ET patients. But changes in other blood counts could be a sign of progression. Drenching night sweats, weight loss, and increased fatigue might also be signs of progression, said Dr. Rampal. He encouraged patients not to be complacent about changes in symptoms, and doctors not to be complacent about periodic blood checks just because a patient has been stable for many years.

Dr. Rampal pointed to genomics tests which may give researchers clues about who is more likely to progress to a more serious illness. For instance, ET patients with the CALR gene seem to be slightly less likely to progress to a more serious disease. However, there are other mutations that may also affect progression that researchers should look at. However, studying genomes has been difficult because patients' insurance will not pay for these blood tests.

Dr. Rampal also responded to questions about young people with ET, noting that medical textbooks still say that MPNs usually occur in patients over 70. He said that an MPN at age 70 may not be a patient's biggest medical problem, but that is not true for younger patients. Much more information needs to be gathered about young patients, who will live with their MPN much longer than someone whose disease onset occurs after age 60. 

Here is more info about the MPN Research Foundation.

Be well!