Ruxolitinib Therapy Followed by Reduced Intensity Conditioning for Hematopoietic Cell Transplantation for Myelofibrosis – Myeloproliferative Disorders Research Consortium 114 study

Myelofibrosis (MF) is a chronic haematological malignancy characterized by variable degrees of anemia and thrombocytopenia, marrow fibrosis, extramedullary hematopoiesis, splenomegaly, cytokine driven inflammatory symptoms, and an increased risk of transformation to acute myeloid leukemia (AML) and reduced life expectancy. Dysregulation of the JAK-STAT pathway is a hallmark of MF irrespective of phenotypic driver mutations in JAK2, CALR and MPL genes. The use of JAK 1/2 inhibitors (JAKi) in MF patients have led to control of disease related symptoms, resulting in approval of ruxolitinib.

Source: Biology of Blood and Marrow Transplantation - September 8, 2018 Category: Hematology Authors: Vikas Gupta, Heidi E. Kosiorek, Adam Mead, Rebecca B Klisovic, John P. Galvin, Dmitriy Berenzon, Abdulraheem Yacoub, Auro Viswabandya, Ruben A. Mesa, Judith Goldberg, Leah Price, Mohamed E Salama, Rona Singer Weinberg, Raajit Rampal, Noushin Farnoud, Amyl Source Type: research