A Phase 1 interventional study of prednison and Sirolimus and prednison in Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP), sponsored by Shanghai Children's Medical Center. Status unknown at 1 site in China. Open to participants aged Up to 12 Years. Per ClinicalTrials.gov, last updated 2020-06-09.
Sponsored by Shanghai Children's Medical Center · Phase 1, Interventional, and Treatment
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)
162 studies on the registry are indexed under Sarcoma, Kaposi; 29 are open to participants now.
This study's planned enrollment of 20 is below the median of 32 across 114 interventional studies indexed under Sarcoma, Kaposi.
Browse Sarcoma, Kaposi studies →Shanghai Children's Medical Center is the lead sponsor of 37 studies on the registry; 14 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Drug: prednison
Drug: prednison 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
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.
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.
Time frame: 6 months after taking the drug
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
Time frame: 6 months after taking the drug
This study is status unknown, as verified in Jun 2020. You cannot join it, but the record below documents what was studied.
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Shanghai Children's Medical Center