Incidence of Elevated Expression of wt1 in Primary Myelofibrosis (pmf) and Postpv-, Postet Myelofibrosis and Its Dynamics during Ruxolitinib Treatmeny

Abstract

Background. Wt1 expression is thoroughly studied in acute myeloblastic leukemia and widely used for disease response monitoring.Its role in MPN is less known. Aim. The aim of the study was to reveal the incidence of elevated Wt1 expression in PMF and secondary MF, as well as to find out clinical significance during ruxolitinib therapy. Patients and methods. 38 pts were included ( Primary Mf-31, post PV- and post ET Mf- 8, males - 12, females- 26). Wt1 expression in peripheral blood was studied by qPCR at diagnosis and during ruxolitinib treatment using Quiagen kit. Dynamics of Wt1 expression was studied in 20 pts treated by ruxolininib. Spleen size was measured in cm below costal margin. Results. Wt1 increased expression was found in 35/38 pts. Correlation of Wt1 expression and DIPSS was not found. The relation between blast cell and Wt1 expression was studied by dividing pts in 3 subgroups according to the number of blast cells in peripheral blood- 0, 1-2, >2. Wt1 expression was lowest in the 1st group, and the highest in the 3rd group((p<.05). Ruxolitinib treatment resulted in the decrease of spleen size and parallel decrease of Wt1 level( p<.05 -Fig1).Pts with transformation to acute leukemia(5) had higher level of Wt1 than before transformation. Conclusion. Wt1 expression is elevated in the majority of pts with PMF as well as in secondary MF. Correlation was found with blast level and spleen size. Wt1 could be used for monitoring efficacy of ruxolitinib therapy Disclosures Konopleva: Reata Pharmaceuticals: Equity Ownership; Abbvie: Consultancy, Research Funding; Genentech: Consultancy, Research Funding; Stemline: Consultancy, Research Funding; Eli Lilly: Research Funding; Cellectis: Research Funding; Calithera: Research Funding.

WT1 Gene Overexpression in Differential Diagnosis of Ph-Negative Myeloproliferative Disorders

“…В нашем исследовании гиперэкспрессия гена WT1 выявлялась у подавляющего большинства пациентов с первичным (ПМФ), вторичным после истинной полицитемии (пост-ИП МФ) или эссенциальной тромбоцитемии (пост-ЭТ МФ) миелофиброзом. У пациентов с МФ, получающих терапию ингибитором JAK1/2-киназ руксолитинибом, уровень экспрессии WT1 коррелировал с размером селезенки и числом бластных клеток в крови [18,19].…”