JAK2 Inhibitors RUXOLITINIB in Patients With High or Intermediate Risk Primary or Secondary Myelofibrosis Eligible for Allogeneic Stem Cell Transplantation: a Prospective Multicentric Phase II Study

Fecha de inicio: 1 de diciembre de 2012

Inclusion Criteria: Age between 18 and 69 years No comorbidity contraindicating the transplantation : Severe respiratory failure defined as dyspnea grade III or more Severe cardiac failure defined as EF < or = 30% Severe renal failure defined as creatinine clearance < 30 ml/min or dialysis Dementia or non-ability to give informed consent for the protocol Major alteration of performance status defined as ECOG > 2 Severe liver disease defined as a cirrhosis or bilirubin > 2 x ULN, or AST/ALT > 5 x ULN Primary or secondary myelofibrosis diagnosed according to WHO definition (Tefferi, et al 2007) Palpable splenomegaly or splenomegaly measured by any imagery (maximum size> 15 cm by ultrasound scan, Magnetic Resonance Imaging or computer tomography) Disease if intermediate or high risk according to published criteria and summarized as follows: At least one criterion among the following: Haemoglobin < 100 gr/L (unrelated to medication toxicity) Leucocytes < 4 G/L (unrelated to medication toxicity) or > 25 G/L Poor prognosis cytogenetics : complex karyotype, abnormalities of chromosomes 5, 7 or 17 , +8, 12p-, inv(3), 11q23 Two criteria among the following criteria : General symptoms (weight lost > 10% in less than 6 months, night swears, specific fever > 37.5°C) Peripheral blastosis > 1% observed at least twice Thrombocytopenia < 100 G/L (unrelated to treatment toxicity) Exclusion Criteria: Myelofibrosis transformed into acute leukaemia with 20% blasts of more in blood or bone marrow Previous treatment with JAK2 inhibitor Thrombopenia < 50 G/L Comorbidities contraindicating the transplantation Comorbidity score Sorror > 3 Pregnant or lactating women