We saw Doctor Halpern at Fred Hutchinson Cancer Center today to (1) have more blood tests and (2) discuss potential medical trials R might participate in. It’s always good to see the doctor. I read a lot about this disease and follow a super helpful Facebook group, all of which provide me with information and answers but also generate questions that rattle around inside my brain.

R continues to gain weight (almost back to his original weight after losing a bunch unintentionally). It’s a known side effect of taking Jakafi. Dr Halpern felt (palpated) R’s spleen, and while it may have shrunk a little, it’s still much too large. He will go in for an MRI soon for a more exact measurement. He will also need a new bone marrow biopsy (not pleasant), scheduled for February 26th.

R’s blood work shows more disease progression–some of his blood indicators are stable but still out of normal range, while others are worsening. One significant change is the percentage of “blasts” in his blood. Blasts are a form of immature white blood cells. Healthy people have zero blasts. R’s blood work shows that 4% of his blood has blasts–a real concern as it tells the doctor that his bone marrow is growing more fibrous. White blood cells help fight infection, so if the white cells produced by the marrow are immature in the form of blasts, the patient can’t fight infection. The doctor thinks R’s probably gone from a “pre-fibrotic stage” of the disease to an intermediate level of the disease (“intermediate 1” by some measures and “intermediate 2” by others). However, only a bone marrow biopsy can confirm that. We’ll find out after the biopsy on February 26^th.

I asked her to explain what his gene mutations mean in terms of prognosis. R has the JAK2 and ASXL1 mutations and does not have the CALR mutation. This combination increases his risk of early mortality without a stem cell transplant. She confirmed he WILL need a transplant. If he were seventy-five-plus years old, he would not be eligible because it’s too dangerous of a procedure with lots of associated risks that could have lasting negative health implications or lead to death. It’s also the only “curative” treatment for Myelofibrosis. A double-edged sword, indeed. She told us that when he does have a transplant, he won’t be “back to normal” for 1-2 years if the procedure goes well. His immune system will be severely compromised as his body tries to “accept” the donor cells, thereby replacing the cancerous ones. Some patients do remarkably well after the transplant, and others, not so much. It’s a gamble, and that’s why they tend to want to postpone it until the benefits outweigh the risks.

​There are two medical trials he might be a candidate for. One is for a drug called Imetelstat (requires weekly IV infusions), and the other is called INCB057643 (taken orally). The doctor has to see if the studies have available spaces and if R qualifies for them. In the meantime, we have reams of paper to read with information about both.

Despite the above, which probably sounds pretty bad, R feels good! He still has some leg cramps and nighttime overheating, plus his spleen causes pressure inside his abdomen, but he’s skiing this winter. She told him to take it easy, though, because a serious fall could cause a spleen rupture. He assures me he’s slowing down a bit from the daring-do skier he’s always been. He’s skiing this winter! That’s a good sign, for sure.

Imetelstat Study

UCB057643 Study