PIK3CA-Related Overgrowth Spectrum (PROS)
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1. Conform to ISSVA (2018) vascular disease diagnostic standard; 2. Gender is not limited; Less than 17 years; 3. Vascular diseases cover a wide range, involving large areas of skin on the body surface and exceeding the subcutaneous fat layer; 4. The risk of tissue and organ dysfunction is high; 5. Stable vital signs; 6. Be able to receive treatment as required; 7. Parents or legal guardians fully understand the test content, voluntarily participate in and sign the informed consent form; 8. The physical examination is qualified, and there are no serious heart, lung, liver, kidney, digestive tract, nervous system, mental disorders, metabolic abnormalities and other diseases in the past.
Exclusion criteria
Exclusion criteria: 1. Blood system diseases; 2. Autoimmune diseases; 3. Liver dysfunction; 4. Decreased renal function; 5. Suffering from serious infection and chronic infectious diseases (such as tuberculosis) in active period; 6. Allergies to macrolides or research drug components or excipients; 7. Patients who participated in any other drug clinical study three months before the start of the trial; 8. Any systemic drugs for vascular malformations were used within 4 weeks before the baseline visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Effective rate; | — |
Countries
China
Contacts
Beijing Children’s Hospital, Capital Medical University (National Center for Children’s Health, China)