FIBRAPLO - Haplo-identical transplantation in patients with myleofibrosis

Prior treatment

•  Contraindications or any circumstance that precludes the use of the drugs involved in the protocol (especially Thiotepa and Fludarabine)

Other illness

•  Contraindications to treosulfan o Hypersensitivity to the active substance o Active non-controlled infectious disease o Fanconi anaemia and other DNA breakage repair disorders o Administration of live vaccine
•  Cardiac failure with EF < or = 50% currently or in the past (even if corrected after treatment)

Lab values

•  Renal failure with creatininemia > 130 µmol/L or clearance < 50ml/min
•  Biological significant liver abnormalities; ASAT or ALAT> 2 x normal range, bilirubin > 1,5 x normal range

Other

•  Myelofibrosis transformed into acute leukemia
•  Pregnant woman or breastfeeding
•  Poor performance status with ECOG 3 or more
•  Respiratory function altered with vital capacity < 70% or forced expired volume < 70%
•  HLA matched donor available
•  Tutorship or curatorship
•  Unwilling or unable to comply with the protocol

•  Patients aged between 18 and 70 years
•  Primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia Vera proven by marrow biopsy
• The myelofibrosis should combine at least 2 of the following criteria: o constitutional symptoms: weight loss > 10% in one year, fever (without infection), recurrent muscle, bone or join pains, extreme fatigue o anemia with hemoglobin < 10 gr/dL or red blood cell transfusion requirement o thrombocytopenia < 100 G/L o peripheral blast count > 1% at least found 2 times o white blood cell count > 25 G/L (before a cytoreductive treatment) o Karyotype: +8, -7/7q-, i(17q), -5, 5q-, 12p-, inv(3), 11q23
•  Performance status according to ECOG at 0, 1 or 2
•  With health insurance coverage
•  Having signed a written informed consent
•  Women agreed to take nomegestrol acetate as contraception during and up to 6 months after treatment by treosulfan
•  Men agreed not to conceive child during and up to 6 months after treatment by treosulfan