Dr. Ayesha Warsi

Blast phase disease is a form of acute leukemia defined by at least 20% of blasts in the peripheral blood or bone marrow, and management of these cases will be guided by a malignant hematologist.

Time for a medicine minute.

The COMFORT 1 and 2 trials controlled myelofibrosis study with oral JAK inhibitor treatment published in 2012 were landmark randomized control trials that revolutionized the treatment of myelofibrosis.

These studies demonstrated the superiority of ruxolitinib over placebo and best available therapy in reducing spleen size and improving myelofibrosis-related symptoms.

These led to the approval of ruxolitinib, a JAK inhibitor, as the first targeted therapy for myelofibrosis, targeting the overactive JAK-STAT pathway implicated in primary myelofibrosis pathophysiology.

It has since given rise to a second generation of JAK inhibitors, including fedrotinib.

Prior to JAK inhibitors, treatment options were limited to symptom management, hydroxyurea, and stem cell transplant.

More recently, as of 2025, a novel JAK inhibitor, mamelotinib, is also approved in Canada for management of splenomegaly and myelofibrosis-related symptoms in patients with intermediate to high-risk disease who have moderate to severe anemia defined by a hemoglobin less than 100 grams per liter.

Thank you for listening to today's episode entitled When the Bone Marrow Breaks Down Primary Myelofibrosis.

This episode was written by Ayesha Warsi, a PGY-3 internal medicine resident at the University of Toronto, and reviewed by Dr. Zachary Lederman, hematology staff, Dr. Clarissa Skoropski, hematology fellow, and Dr. Ryan Luther, GIM staff.

The Internet Work series was created and executively produced by Alison Lai alongside executive producers Zahra Murali and Laia Karanopoulos.

This podcast was produced and recorded by Annie Wu.

Theme song was created by Lakshman Visanthamoen.

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The Internet Work releases its podcast every two weeks on Sundays.

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