Eltrombopag Olamine in Increasing Platelet Counts in Patients Undergoing Transplant

Phase 2

Completed

Conditions: Bone Marrow Transplantation Recipient
Cord Blood Transplant Recipient
Hematopoietic Cell Transplantation Recipient

Interventions: Drug: Eltrombopag Olamine
Other: Laboratory Biomarker Analysis

Brief Summary: This phase II trial studies how well eltrombopag olamine works in increasing platelet counts in patients undergoing transplant. Eltrombopag olamine may help platelet counts and the immune system recover from blood or bone marrow transplant.

Detailed Description: PRIMARY OBJECTIVES:

I. To estimate the rate of platelet engraftment by day 60 in patients undergoing cord blood transplant (CBT) or haploidentical donor stem cell transplantation treated with eltrombopag (eltrombopag olamine).

SECONDARY OBJECTIVES:

I. To assess safety of eltrombopag in this population. II. To assess neutrophil engraftment with eltrombopag in this population. III. To characterize immune reconstitution. IV. To assess overall survival (OS). V. To assess progression free survival (PFS). VI. To assess incidence of acute graft-versus-host disease (GVHD).

OUTLINE: Patients are assigned to 1 of 2 arms.

ARM I (CORD BLOOD TRANSPLANT): Patients receive eltrombopag olamine orally (PO) once daily (QD) for 60 days beginning on day -1.

ARM II (HAPLOIDENTICAL DONOR STEM CELL TRANSPLANT): Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

After completion of study treatment, patients are followed up at 1, 2, 3, 6, and 12 months.

Recruitment & Eligibility

Inclusion Criteria

Patients undergoing a cord blood or haploidentical transplantation on any protocol or standard of care treatment plan.
Age >/= 18.
Females of child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding must be willing to use an effective contraceptive measure until 30 days after the last dose of eltrombopag. Males who have had sexual contact with female of child-bearing potential must be willing to use contraceptive techniques until 30 days after the last dose of eltrombopag.
Patient or patient's legal representative(s) is/are able to provide written informed consent to participate.

Exclusion Criteria

ALT and AST >/= 2.5 ULN.
Serum direct bilirubin >/= 1 mg/dl (except Gilbert's syndrome or hemolysis).
Patients of east Asian ancestry (Chinese, Japanese, or Korean Origin).
Calculated creatinine clearance < 30ml./min. Creatinine clearance will be calculated using the MDRD method.
Arterial or venous thrombosis in the last year except for line-related venous thrombosis more than 3 months ago.
Positive beta HCG within 7 days prior to consent in female of child-bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization or breast-feeding.

Arm && Interventions

Group	Intervention	Description
Arm I (cord blood transplant patients)	Eltrombopag Olamine	Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Arm I (cord blood transplant patients)	Laboratory Biomarker Analysis	Patients receive eltrombopag olamine PO QD for 60 days beginning on day -1.
Arm II (haploidentical donor stem cell transplant patients)	Laboratory Biomarker Analysis	Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.
Arm II (haploidentical donor stem cell transplant patients)	Eltrombopag Olamine	Patients receive eltrombopag olamine PO QD for 60 days beginning on day 5.

Primary Outcome Measures

Name	Time	Method
Number of Participants That Achieved Platelet Engraftment Greater Than 50K/ul	Up to day 60	Number of participants that achieved platelet engraftment greater than 50K/ul 60 days post transplant.

Secondary Outcome Measures

Name	Time	Method
Progression Free Survival	Up to 1 year	Number of participants without disease progression at 1 year post transplant.
Overall Survival	Up to 1 year	Number of participants that survived at one year post transplant.