Myelofibrosis

Question 1

Myelofibrosis

Answer 1

Uncontrolled proliferation of megakeryocytes

increased production of fibroblast growth factor
increasd extracellular matrix
marrow fibrosis

decreased production of white cells and red cells
(platelets can be decreased or increased)

PMF occurs mainly in middle aged and older adults. The median age at presentation is 67 years

Question 2

clinical presentation of myelofibrosis

Answer 2

Constitutional symptoms
Spleen >10 cm below left costal margin
low Hemoglobin
low white cell count

Question 3

splenomegaly in myelofibrosis

Answer 3

Symptoms due to splenic disease often figure prominently in PMF. Patients may note a dragging or heavy sensation in the left upper abdomen, and the spleen may compress the patient’s stomach, leading to early satiety. Severe left upper quadrant pain, with or without left shoulder pain,

Question 4

gene mutations in myelofibrosis

Answer 4

●JAK2 mutation – 60 to 65 percent
●CALR mutation – 20 to 25 percent
(calriticulin)

Question 5

diagnosis of myelofibrosis

Answer 5

bone marrow biopsy:

Megakaryocytic proliferation and atypia, with reticulin and/or collagen fibrosis

Demonstration of a JAK2, CALR, or MPL mutation or another clonal marker

Question 6

treatment of myelofibrosis

Answer 6

●PMF is a heterogeneous disease with some patients surviving more than a decade with supportive care alone.
●With the exception of allogeneic hematopoietic cell transplantation (HCT), most cases of PMF cannot be cured by current treatment options.
●The main goals of therapy for most patients with PMF are to control symptoms and to improve quality of life, which includes minimizing the toxicity of therapy.
●There is no evidence that the treatment of asymptomatic patients improves long-term survival.

Question 7

aysmptomatic treatment of myelofibrosis

Answer 7

aspirin if increased platelets
blood transfusions for anaemia

follow expectantly

Question 8

symptomatic treatment of myelofibrosis

Answer 8

low risk patients should get hydroxyurea

high risk patients should consider allogenic stem cell transplant

if not for SCT and debilitating constitutional symptoms or severely symptomatic splenomegaly not participating in a clinical trial, we offer treatment with the JAK2 inhibitor ruxolitinib

Question 9

ruxolitinib

Answer 9

jak 2 inhibitor

used to treat high risk myelofibrosis