a study of eltrombopag in patients with cmml and thrombocytopenia

Conditions: CMML; Thrombocytopenia

Summary: treatment of patients with chronic myelomonocytic leukemia (cmml) and thrombocytopenia.

all eligible patients will be treated with eltrombopag for a minimum of twelve weeks and a maximum of 24 months.

Eligibility criteria:
inclusion criteria: * age 18 years or older * chronic myelomonocytic leukemia (cmml) according to who criteria: * stable excess in blood monocytes \> 1 g/l * lack of bcr-abl rearrangement (or philadelphia chromosome) * bone marrow blast cells \< 20% * dysplasia of at least one lineage or clonality marker or blood monocytosis during more than 3 months w/o other explanation * platelet counts \< 50 g/l on two successive blood counts in the 2 weeks preceding inclusion * either of d1 or d2 criteria: * lack of features of advanced disease if white blood cell count (wbc) \< 13 g/l: international prognostic scoring system (ipss) low or intermediate-1 if wbc ≥ 13 g/l: no more than one of the following criteria: * clonal cytogenetic abnormality other than t(5;12) (q33; p13) * absolute neutrophil count (anc) \> 16 g/l * anemia (hb \< 100 g/l) * extramedullary localization (documented cutaneous, pleural or pericardial effusion, etc...) or d2- features of advanced disease if wbc \< 13 g/l: ipss intermediate-2 or high if wbc ≥ 13 g/l: two or more of the following criteria: * clonal cytogenetic abnormality other than t(5;12) (q33; p13) * anc \> 16 g/l * anemia (hb \< 100 g/l) * extramedullary localization (documented cutaneous, pleural or pericardial effusion, etc...) and having resisted (progression or stable disease without hematological improvement according to international working group (iwg) 2006 criteria) or relapsed after a treatment with a hypomethylating agent (azacitidine or decitabine for a minimum of 6 cycles) * blast cells ≤ 5% in the bone marrow * performance status 0-2 on the eastern cooperative oncology group (ecog) scale * serum creatinin \< 2 times the upper limit of normal (uln) * alanine transaminase (alt) and aspartate transaminase (ast) \< 3 uln, total bilirubin \< 1.5 uln (except gilbert syndrome) * adequate contraception if relevant * signed informed consent exclusion criteria: * cmml with t(5 ;12) or platelet-derived growth factor beta receptor (pdgfbetar) rearrangement * acute blastic transformation of cmml with bone marrow blast cells \> 20% * bone marrow blast cells \> 5% * patients eligible for allogeneic bone marrow transplantation with an identified donor * intensive chemotherapy given less than 3 months before inclusion * pregnant or breastfeeding * hepatitis c infection * splenomegaly \> 16 cm by ultrasound or ct scan (not applicable in patients without palpable splenomegaly) * significant (grade ii-iv) myelofibrosis (bone marrow trephine if bone marrow aspirate with poor cellularity, or features of myelofibrosis on the peripheral blood smear (teardrop erythrocytes) * clinically relevant thromboembolic risk factor which, in the investigator's opinion, is such that the benefit/risk ratio becomes unfavourable if platelet counts increase * liver cirrhosis (child-pugh score ≥ 5) * prior cancer (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years) * serious concomitant systemic disorder, including active bacterial, fungal or viral infection that, in the opinion of the investigator, would compromise the safety of the patient and/or his/her ability to complete the study. * hypersensitivity to eltrombopag

Keywords: chronic myelomonocytic leukemia thrombocytopenia eltrombopag