Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

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    Associazione Qol-one
Updated on 23 January 2021
platelet count
stem cell transplantation
myeloid leukemia
myelodysplastic syndromes
granulocyte colony stimulating factor
cell transplantation
bone marrow procedure
colony stimulating factor
blood transfusion
platelet transfusion
autoimmune thrombocytopenia


Myelodysplastic syndromes (MDS) prevail in older age and are characterized by ineffective erythropoiesis and peripheral cytopenias. Supportive therapy is the main therapeutic option for most patients. Quality of Life (QoL) is mainly deteriorated by anemia and by the limitations associated with thrombocytopenia, neutropenia and transfusion dependence. The only available treatment for severe thrombocytopenia, in the presence of bleeding, is platelet transfusion.

Eltrombopag is an orally bioavailable agonist of the thrombopoietin receptor. In adult patients with chronic immune thrombocytopenia (ITP), Eltrombopag rapidly increases platelet counts and significantly reduces bleeding episodes during treatment. Eltrombopag is well tolerated. In 2007, Eltrombopag has received the Orphan Drug Designation for the treatment of ITP (EMEA/OD/031/07), and in 2008 the Food and Drug Association approved Eltrombopag for the treatment of ITP refractory or resistant. It has been shown that in patients affected by MDS and by acute myeloid leukemia, Eltrombopag neither increases the proliferation, nor the clonogenic growth capacity of bone marrow blasts. Furthermore, Eltrombopag induces an increase in the megakaryocytic differentiation and in the formation of normal megakaryocytic colonies. These results provide the rationale for pursuing further research on Eltrombopag for the treatment of thrombocytopenia in case of MDS.

The study is open to adult patients with myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk (Index Prognostic Score System).

Severe thrombocytopenia associated with MDS may lead to death from hemorrhage, even in low prognostic risk patients. The benefit of platelet transfusion is short-termed. Patients become refractory in the long term. The availability of a treatment that induces the increase of platelet count is extremely important, either in terms of quality of life, and in overall survival.

Condition Bone marrow disorder, Preleukemia, MYELODYSPLASTIC SYNDROME, Thrombocytopenia, Myelodysplastic Syndromes (MDS), Thrombocytopenia and Thrombocytopenia Prevention, myelodysplastic syndromes, myelodysplastic syndrome (mds)
Treatment Placebo, Eltrombopag/Revolade
Clinical Study IdentifierNCT02912208
SponsorAssociazione Qol-one
Last Modified on23 January 2021


Yes No Not Sure

Inclusion Criteria

Adult subjects (18 years of age or older) with low or intermediate-1 IPSS risk MDS and stable disease
Subjects must have a platelet count taken within the 4 weeks prior to randomization that is <30 Gi/L
Subjects must be ineligible or relapsed or refractory to receive other treatment options (such as azacitidine or lenalidomide) and must be ineligible to receive intensive chemotherapy or autologous/allogeneic stem cell transplantation
Subjects must have platelet count and platelet transfusion data available over a period of 8 weeks prior to randomization
During the 2 months prior to randomization, subjects must have a baseline Bone Marrow examination which includes cytomorphology and cytogenetics. Histopathology should be performed
Erythropoiesis-stimulating agents (ESAs) in anemic subjects or granulocyte colonystimulating factor (G-CSF) in subjects with severe neutropenia and recurrent infections are allowed during the study as per accepted standards. Subjects who enter the study on ESAs or G-CSF should continue at the same dose schedule until the optimal dose of study medication has been established
ECOG (Eastern Cooperative Oncology Group) Performance Status 0-3
Subject is able to understand and comply with protocol requirements and instructions
Subject has signed and dated informed consent
Adequate baseline organ function defined by the criteria below
total bilirubin (except for Gilbert's Syndrome) 1.5 x Upper Limit Normal
Alanine aminotransferase and Aspartate aminotransferase 3 x Upper Limit Normal
creatinine 2 x Upper Limit Normal albumin must not be below the lower limit of
normal by more than 20%
\. Subject is practicing an acceptable method of contraception. Female
subjects (or female partners of male subjects) must either be of non-
childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal
ligation or post-menopausal >1 year), or of childbearing potential and use of
an highly effective method of contraception from 2 weeks prior to
administration of study medication, throughout the study, and 28 days after
completion or premature discontinuation from the study

Exclusion Criteria

MDS with intermediate-2 or high IPSS risk
History of treatment for cancer other than MDS with systemic chemotherapy and/or radiotherapy within the last 2 years
History of treatment with romiplostim or other Thrombopoietin receptor agonists
Pre-existing cardiovascular disease (including congestive heart failure, New York Heart Association Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. persistent atrial fibrillation), or subjects with a QTc >450 msec (QTc >480 msec for subjects with Bundle Branch Block)
BM fibrosis that leads to an inability to aspirate marrow for assessment
Peripheral monocytosis > 1000/uL prior to Day 1 of study medication
Leukocytosis >=25,000/uL prior to Day 1 of study medication
Female subjects who are nursing or pregnant (positive serum or urine Beta-human chorionic gonadotropin [B-hCG] pregnancy test) at screening or pre-dose on Day 1
Current alcohol or drug abuse
Treatment with an Investigational Product within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication
Active and uncontrolled infections
Subjects infected with Hepatitis B, C or Human Immunodeficiency Virus (HIV)
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