Child, Veno-occlusive Disease
Conditions
Brief summary
Veno-occlusive disease (VOD) after hematopoietic stem cell transplantation (HSCT) remains the major complication. VOD occurs in 11-31% of pediatric HSCT and the mortality reaches up to 50%. Prostaglandin E1 (PGE1) have been reported to prevent and relieve the severity of VOD by Gluckman et al.. Lipo-PGE1 is a transporter of PGE1, which is superior in concentrating PGE1 and acts for prolonged time. Because of the prolonged effective time, lipo-PGE1 acts comparable effects by 1/4\ 1/8 of PGE1 dose. Empirically, pediatric HSCT centers adopt lipo-PGE1 in dose of 1 mcg/kg/day (0.042 mcg/kg/hr), which is 1/7 of the dose recommended by Gluckman, et al. This prospective study will investigate the concentration of lipo-PGE1 with preventive lipo-PGE1 with empirical dose (1 mcg/kg/day).
Detailed description
Primary Objective: To evaluate the pharmacokinetics of lipo-PGE1. Secondary objective: To evaluate the relation between lipo-PGE1 concentration and VOD occurrence, severity. Inclusion criteria 1. Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin. * high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation * contraindication of heparin : low platelet count, bleeding tendency, allergy 2. Patients (or one of parents if patients age \< 19) should sign informed consent. Exclusion criteria 1. Patient with heart failure. 2. Patient with bleeding (intracranial hemorrhage, gastrointestinal tract bleeding, hemoptysis). 3. History of hypersensitivity reaction as shock to lipo-PGE1. 4. Psychiatric disorder that would preclude compliance. 5. If the clinician decides that there is a condition improper for the clinical study.
Interventions
DRUGlipoprostaglandin E1
Blood sampling before administration (baseline), between 1 hour to 72 hours from administration (plateau 1), 72 hours after engraftment (plateau 2), when the clinician determine to do blood sampling because of change of kidney/liver function change (optional 1), 72 hours from the change of lipoprostaglandin E1 concentration change (optional 2)
Sponsors
Seoul National University Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin. * high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation * contraindication of heparin : low platelet count, bleeding tendency, allergy 2. Patients (or one of parents if patients age \< 19) should sign informed consent.
Exclusion criteria
1. Patient with heart failure. 2. Patient with bleeding (intracranial hemorrhage, gastrointestinal tract bleeding, hemoptysis). 3. History of hypersensitivity reaction as shock to lipo-PGE1. 4. Psychiatric disorder that would preclude compliance. 5. If the clinician decides that there is a condition improper for the clinical study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| pharmacokinetics of lipo-PGE1 (Lipo-PGE1 level (B, P1, P2, O1, O2)) | baseline (pre-dose), 1 to 72 hours from starting continuous infusion of lipo-PGE1 | Lipo-PGE1 drug level of time points (B, P1, P2, O1, O2) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of VOD after transplantation | until 3 months after transplantation | cumulative incidence of VOD until 3 months after transplantation |
Countries
South Korea
Outcome results
None listed