Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation

moshe yeshurun

Status and phase: Unknown; Phase 2

Conditions: Pediatric Patients Undergoing Allogeneic Cord Blood Transplantation.

Treatments: Drug: eltrombopag

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01940562

0195-13

Details and patient eligibility

About

Platelet recovery is significantly hampered following umbilical cord blood transplantation (UCB). Thus, after UCB transplantations, patients are platelet transfusion-dependent for prolong periods of time.

Eltrombopag is a thrombopoietin-receptor agonist that initiates thrombopoietin-receptor signaling and thereby induces proliferation and maturation of megakaryocytes.

We will evaluate the safety and efficacy of eltrombopag for enhancing platelet engraftment in pediatric patients undergoing cord blood transplantation.

Enrollment: 20 estimated patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patient ≥ 1 year and < 18 years old.
2. Patients receiving unmanipulated single or double UCB allogeneic grafts.
3. Malignant and non malignant indications for transplantation.
4. Myeloablative and reduced intensity conditioning regimens.
5. Patients must meet all other pre-transplantation criteria of the transplantation center including acceptable tests of heart, liver, kidney, and lung function (standard screening for transplantation per PI, and co-investigators).
6. Written informed must be obtained from the patient's guardian, and accompanying informed assent from the patient for children over 6 years old.
7. Able to comply with study protocol.

Exclusion criteria

1. Indications for transplantation

   1. Patients with primary myelofibrosis.
   2. French-American-British classification (FAB)M7 acute myeloid leukemia. Acute leukemia secondary to a myeloproliferative neoplasm.
   3. Patients with persistent acute leukemia (>5% bone marrow blasts) at the time of transplantation.

2. Patients with prior thromboembolic event. Patients with previous catheter related thrombosis will be eligible if more than 3 months elapsed.

3. Hypersensitivity to eltrombopag.

4. Liver enzymes abnormalities:

Alanine aminotransferase (ALT) levels > 3 times the upper limit of normal (ULN) or serum bilirubin > 1.5 ULN (unless due to Gilbert's syndrome or hemolytic bilirubin).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Eltrombopag

Experimental group

Description:

Pediatric patients undergoing allogeneic UCB transplantation will receive eltrombopag from day +1 until platelet count has exceeded 50,000/microliter for 14 consecutive days without platelet transfusion. Starting doses are 100 mg/d for children \>40 kg body weight (BW), 50 mg/d for children 20-40 kg BW, and 2 mg/kg/d for children \<20 kg BW. If unsupported platelet count has not reached the threshold of 20,000/microliter, doses will be escalated every 2 weeks up to maximal doses of 200 mg/d for children ≥ 40 kg BW, 150 mg/d for children 20-40 kg BW and 3.5 mg/kg for children \<20 kg BW.

Treatment:

Drug: eltrombopag