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Protocol Details

A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R agonist), Eltrombopag, in Patients with Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 18 Years
Max Age: N/A

Referral Letter Required


Population Exclusion(s)



Bone Marrow Fibrosis;

Recruitment Keyword(s)

Myelodysplastic Syndrome;


Myelodysplastic Syndrome (MDS)

Investigational Drug(s)

eltrombopag (Promacta)

Investigational Device(s)



Drug: Eltrombopag

Supporting Site

National Heart, Lung, and Blood Institute


- Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications.

- Eltrombopag is a drug designed to mimic the protein thrombopoietin, which causes the body to make more platelets. Eltrombopag has been able to increase platelet counts in healthy volunteers and in patients with chronic ITP (a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia), but researchers do not know if the drug can increase platelet counts in patients with MDS.


- To find out whether eltrombopag can improve platelet counts in patients with MDS.

- To determine whether eltrombopag is safe for patients with MDS.


- Patients 18 years of age and older who have consistently low blood platelet counts related to MDS that has not responded to conventional treatment.

-Platelet count 30,000/ L or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); OR hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC 500


- Treatment with eltrombopag tablets once per day for 16-20 weeks.

- Participants will be monitored closely throughout the initial treatment, with weekly blood tests and separate evaluations at the National Institutes of Health (NIH) treatment center every 4 weeks. Bone marrow biopsies may be conducted to check for abnormalities in bone marrow.

- If patients show signs of improved platelet counts after 90 days, treatment will continue with additional doses of eltrombopag.

- Patients who discontinue taking eltrombopag will be evaluated at the NIH treatment center 4 weeks after ending treatment, and again 6 months after ending treatment to check for potential side effects.

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Diagnosis of MDS, with WHO classification of refractory anemia, refractory cytopenia with unilineage dysplasia (RCUD), RARS, RCMD-RS, or RCMD.

IPSS risk scores of low, intermediate-1, or intermediate-2.

Platelet count less than or equal to 30,000/ microL or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); or hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC less than or equal to 500

Age greater than or equal to 18 years old

Treatment naive or off all other treatments for MDS (except stable dosing of filgrastim [G-CSF], erythropoietin, and transfusion support) for at least four weeks. G-CSF can be used before, during and after the protocol treatment for subjects with documented neutropenia (<500/UI) as long as they meet the criteria for other cytopenia as stated above. G-CSF must be held for 3 weeks prior to enrollment bone marrow biopsy and prior to each study assessment bone marrow biopsy, unless clinically indicated to avoid infections per PI discretion.

Adequate liver function, as evidenced by total serum bilirubin less than or equal to 1.5 times the upper limit of normal patients with Gilbert s disease are eligible, provided intermittent indirect hyperbilirubinemia, AST or ALT less than or equal to 5 times the upper limit of normal.

A serum creatinine concentration less than or equal to 2 times ULN


WHO classification of chronic myelomonocytic leukemia (CMML), RAEB-1, RAEB-2, AML

Treatment with horse or rabbit ATG or Campath within 6 months of study entry

Subjects with liver cirrhosis including subjects infected with Hepatitis B or C

Subjects with HIV

Infection not adequately responding to appropriate therapy

History of malignancy treated with chemotherapy and cytogenetic abnormalities suggestive of secondary myelodysplasia.

Moribund status or concurrent hepatic, renal, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy

Life expectancy of less than 3 months

Hypersensitivity to eltrombopag or its components

Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential

Unable to understand the investigational nature of the study or give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent per section

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Kantarjian H, Giles F, List A, Lyons R, Sekeres MA, Pierce S, Deuson R, Leveque J. The incidence and impact of thrombocytopenia in myelodysplastic syndromes. Cancer. 2007 May 1;109(9):1705-14

Houwerzijl EJ, Blom NR, van der Want JJ, Vellenga E, de Wolf JT. Megakaryocytic dysfunction in myelodysplastic syndromes and idiopathic thrombocytopenic purpura is in part due to different forms of cell death. Leukemia. 2006 Nov;20(11):1937-42. Epub 2006 Sep 7

Kalina U, Hofmann WK, Koschmieder S, Wagner S, Kauschat D, Hoelzer D, Ottmann OG. Alteration of c-mpl-mediated signal transduction in CD34(+) cells from patients with myelodysplastic syndromes. Exp Hematol. 2000 Oct;28(10):1158-63

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Principal Investigator

Referral Contact

For more information:

Neal S. Young, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CRC BG RM 3-5142
(301) 496-5093

Olga J. Rios, R.N.
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 4-5362
10 Center Drive
Bethesda, Maryland 20892
(301) 496-4462

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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