SCMC-KK2020: SCMC Trial on KHE With KMP (V.2020)
Study Details
Study Description
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)
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: prednison group
|
Drug: prednison
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.
|
| Experimental: prednison+sirolimus group
|
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
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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
Eligibility Criteria
Criteria
Inclusion Criteria:
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Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon
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0 - 12 years of age at the time of study entry
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Male or female
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Consent of parents (or the person having parental authority in families)
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Signed and dated written informed consent
Exclusion Criteria:
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with hematological diseases
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with other solid tumors
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with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney
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dysfunction, and cardiopulmonary insufficiency
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with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine | Shanghai | Shanghai | China | 200127 |
Sponsors and Collaborators
- Shanghai Children's Medical Center
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SCMC2020