Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation
- Conditions
- Pediatric Patients Undergoing Allogeneic Cord Blood Transplantation.
- Interventions
- Registration Number
- NCT01940562
- Lead Sponsor
- moshe yeshurun
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 20
- Patient ≥ 1 year and < 18 years old.
- Patients receiving unmanipulated single or double UCB allogeneic grafts.
- Malignant and non malignant indications for transplantation.
- Myeloablative and reduced intensity conditioning regimens.
- 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).
- Written informed must be obtained from the patient's guardian, and accompanying informed assent from the patient for children over 6 years old.
- Able to comply with study protocol.
-
Indications for transplantation
- Patients with primary myelofibrosis.
- French-American-British classification (FAB)M7 acute myeloid leukemia. Acute leukemia secondary to a myeloproliferative neoplasm.
- Patients with persistent acute leukemia (>5% bone marrow blasts) at the time of transplantation.
-
Patients with prior thromboembolic event. Patients with previous catheter related thrombosis will be eligible if more than 3 months elapsed.
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Hypersensitivity to eltrombopag.
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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).
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Eltrombopag eltrombopag 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.
- Primary Outcome Measures
Name Time Method Platelet engraftment rate up to 50 days post transplantation, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter. 50 days
- Secondary Outcome Measures
Name Time Method Time to partial platelet engraftment, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter. 180 days Time to sustained complete platelet engraftment defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter. 180 days Complete platelet engraftment rates up to 50 days post transplantation defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter. 50 days Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter. 60 days
Trial Locations
- Locations (2)
Hadassah hospital
🇮🇱Jerusalem, Israel
Schneider Children's Medical Center of Israel
🇮🇱Petah Tikva, Israel