Abstract
The Wilms tumor gene WT1 is a useful marker of clonal hematopoiesis and it has been shown to be a good marker of residual disease and it reflects the response to therapy. Although myelofibrosis is characterized by mutations of JAK2 and calreticulin (CALR), these mutations are not useful to monitor response to therapy. In this study we demonstrated that in patients affected by myelofibrosis WT1 correlates with the International Prognostic Scoring System (IPSS) score at diagnosis. Furthermore WT1 is a good marker of response to JAK2 inhibitors especially for patients without blasts and for patients who develop anemia or thrombocytopenia not for progression but as therapy related toxicity. Finally, WT1 transcript reduction can mirror a benefit of therapy on the disease burden. This study demonstrated that WT1 is a good marker for monitoring the response to therapy in patients affected by myelofibrosis.
Highlights
Myelofibrosis (MF) is a clonal stem cell disorder characterized by bone marrow fibrosis, extramedullary hematopoiesis, and an increased risk of transformation to acute leukemia
Based on the available data, JAK2V617F cannot be considered a molecular marker of residual disease or drug response
To establish the role of WT1 as marker of disease, we followed patients during disease progression and found a progressive and significant increase of WT1 that started a median of 3 months before leukemic transformation when the blood parameters were stable. (Fig. 1C and D)
Summary
Myelofibrosis (MF) is a clonal stem cell disorder characterized by bone marrow fibrosis, extramedullary hematopoiesis, and an increased risk of transformation to acute leukemia. It was shown in phases II and III clinical trials that ruxolitinib is effective in the reduction of spleen size and disease-related symptoms. More recently in a phase III study, a reduction in allele burden from baseline was observed and correlated with ruxolitinib treatment response, in particular with reduction in symptoms and spleen volume.
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