Episode 43. Essential Thrombocythemia with Dr. Raajit Rampal

Blood Cancer Talks

In this episode, we discuss the diagnosis and management of Essential Thrombocythemia with Dr. Raajit Rampal. Here are the shownotes:
1. IPSET (revised) system, risk categories are defined by the presence of a prior thrombosis, age, and JAK2 mutation status:

· Very low risk: No prior thrombosis, age ≤60 years, JAK2-unmutated

· Low risk: No prior thrombosis, age ≤60 years, JAK2-mutated

· Intermediate risk: No thrombosis, age >60 years, JAK2-unmutated

· High risk: History of thrombosis (any age/genotype), or age >60 years with JAK2 mutation

https://ashpublications.org/blood/article/120/26/5128/30914/Development-and-validation-of-an-International

2. The MIPSS-ET provides points for:

· Age > 60 years (3 points)

· Adverse mutation (SF3B1/SRSF2/U2AF1/TP53) (2 points)

· Male sex (1 point)

· White blood cell count ≥11 × 10^9/L (1 point)

· https://onlinelibrary.wiley.com/doi/abs/10.1111/bjh.16380?sid=nlm%3Apubmed

3. Aspirin

o Is there a benefit of twice-daily aspirin dosing, especially in high-risk or JAK2-mutant disease?

https://ashpublications.org/blood/article/136/2/171/454293/A-randomized-double-blind-trial-of-3-aspirin

4. Cytoreduction in High-risk ET

Hydroxyurea: https://www.nejm.org/doi/full/10.1056/NEJM199504273321704

Pegylated interferon alfa

MPD-RC 112 Mascarenhas J et al. A randomized phase 3 trial of interferon- α vs hydroxyurea in polycythemia vera and essential thrombocythemia. Blood . 2022)

https://ashpublications.org/blood/article/139/19/2931/483404/A-randomized-phase-3-trial-of-interferon-vs

Anagrelide in ET

https://ashpublications.org/blood/article/121/10/1720/31186/Anagrelide-compared-with-hydroxyurea-in-WHO

5. Ruxolitinib

In a randomized study phase 2 study (MAJIC-ET), ruxolitinib treatment did not deliver better rates of hematological response than best-available therapy, although symptoms did improve.

· MAJIC-ET study which is a randomized phase 2 trial

· ET patients resistant/intolerant to HU were randomized to receive RUX or standard treatment (HU 71%, anagrelide 48%, IFN 40%). The primary study outcome was CR, defined by normal platelet and white cell counts and normal spleen size.

· At 1 year, response was achieved in 46% of patients in the RUX group and in 44% in the standard arm, without statistically significant difference between the two groups.

· In addition, no difference was observed in the rates of thrombosis, hemorrhage, and disease transformation after 2 years of follow-up.

· MRs were also uncommon. RUX was superior, however, in symptom control.

https://ashpublications.org/blood/article/130/17/1889/36509/Ruxolitinib-vs-best-available-therapy-for-ET