Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
Conditions
Brief summary
a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)
Interventions
DRUGprednison and Sirolimus
If the intravenous prednisolone 4 mg / kg / d (one time in the morning and one time in the evening, 2mg / kg body weight each time) is effective, it will be gradually reduced to 2mg / kg / D (one time in the morning and one time in the evening, 1mg / kg body weight each time), gradually converted to oral prednisone of equal dose, and the hormone will be removed within 4-6 weeks. At the same time, the dosage of sirolimus oral liquid is 0.8 mg / m2 twice a day, with an interval of 12 hours, maintaining the blood concentration of 8-15 ng / ml. if there is no intolerable side effect, the treatment will last for 6 months
DRUGprednison
Prednison is taken at a dose of 4mg/kg/d. If the hormone treatment is effective, it will be gradually reduced to 2mg / kg / d (one time in the morning and one time in the evening, 1 mg / kg body weight each time), and repeated after 2 months of continuous treatment and 1 month of drug withdrawal.
Sponsors
Shanghai Children's Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 12 Years
Healthy volunteers
No
Inclusion criteria
* Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon * 0 - 12 years of age at the time of study entry * Male or female * Consent of parents (or the person having parental authority in families) * Signed and dated written informed consent
Exclusion criteria
* with hematological diseases * with other solid tumors * with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney * dysfunction, and cardiopulmonary insufficiency * with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| response to treatment | 6 months after taking the drug | Complete Response： platelets counts is greater than 100×10\^9/L. significant volume reduction is greater than 80%. Fibrinogen levels at 2-4g/L. The surface skin of the tumor is lighter or the tumor is softer significantly. Partial Response: platelets counts is greater than 40×10\^9/L. significant volume reduction is greater than 50%. Fibrinogen levels at less than 50% reduction from baseline. The surface skin of the tumor and palpation of the tumor have no change or less change. No Response: platelets counts is less than 40×10\^9/L. significant volume reduction is less than 50% or the tumor is bigger. Fibrinogen levels at grater then 50% reduction from baseline. The surface skin of the tumor is darker or the tumor is harder. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| side effect rate | 6 months after taking the drug | Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms |
Countries
China
Contacts
Primary ContactSong Gu
Outcome results
None listed