SCMC Trial on KHE With KMP (V.2020)

Study Overview
=================
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)

Official Title
-----------------
A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance

Conditions
-----------------
Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)

Intervention / Treatment
-----------------
* Drug: prednison and Sirolimus
* Drug: prednison


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: prednison group<br> | Drug: prednison<br>* 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.<br>|
| Experimental: prednison+sirolimus group<br> | Drug: prednison and Sirolimus<br>* 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<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| response to treatment | 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. | 6 months after taking the drug | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| side effect rate | Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms | 6 months after taking the drug |