lymphatic malformations
Conditions
Interventions
Model-informed dosing regimen group:Calculation of individualized dosing regimen based on a PopPK model combined with TDM
TDM-guided dosing regimen group:Calculation of individualized dosing regimen based on TDM
Retrospective data analysis – ‘Target Therapeutic Window’ group:none
Retrospective data-based PopPK model group:None
Sponsors
Beijing Children's Hospital, Capital Medical University, Beijing, China
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: Part I: We retrospectively collected clinical data from patients with iLM who received SRL treatment at our hospital between January 2017 and June 2023. Inclusion criteria: 1.Age <18 years, no restriction on sex; 2.Treated with SRL; Part II: Inclusion Criteria 1.Age <18 years, no restriction on sex; 2.Treated with SRL; Part III: Inclusion criteria: 1.The subject or legal guardian voluntarily signed informed consent and was able to complete the study according to protocol requirements; 2.Age <18 years, no restriction on sex; 3.Treated with SRL; 4.Diagnosed with intractable lymphatic malformation, specifically including: Capillary–lymphatic–venous malformation (CLVM);Venous–lymphatic malformation (VLM);Macrocystic lymphatic malformation (Ma-LM);Microcystic lymphatic malformation (Mi-LM);Mixed lymphatic malformation (Mx-LM). Tumor involvement included the skull base (mastoid, jugular foramen, jugular bulb, pterygopalatine fossa), and/or pharyngeal region (nasopharynx, oropharynx, hypopharynx, parapharyngeal space), and/or trachea, and/or esophagus, and/or carotid sheath, and/or bone (clavicle, skull, vertebrae, sternum), and/or mediastinum; and/or with complications such as coagulopathy, chronic pain, recurrent cellulitis, impaired cardiac function, local ulceration; or cases deemed by clinicians to have poor prognosis and in which conventional treatments were ineffective.
Exclusion criteria
Exclusion criteria: Part I: Exclusion Criteria 1.Patients who did not undergo SRL blood concentration monitoring during treatment; 2.Patients who did not undergo other auxiliary examinations for the assessment of adverse reactions or therapeutic efficacy during treatment, such as imaging or laboratory tests; 3.Subjects deemed unsuitable for participation in this study by the investigators for any other reasons. Part II: Exclusion Criteria Same as Part I. Part III: Exclusion Criteria 1.Subjects deemed unsuitable for participation in this study by the investigators, for example: Tumor compressing the airway leading to =grade 2 airway obstruction without the possibility of establishing a safe airway temporarily; Patients with lymphedematous diseases such as Milroy disease, Meige lymphedema, Hennekam syndrome, Njolstad syndrome, Aagenaes syndrome, or Fabry disease; Patients unable to undergo contrast-enhanced MRI examination; Patients currently receiving other immunosuppressants, patients with immunodeficiency, or those with hepatic/renal failure; Patients with gastrointestinal diseases causing impaired absorption or metabolism of SRL; Patients with uncontrolled or severe infections; Patients with poor general health status; Participants who had received contraindicated medications with SRL within the past 15 days; Patients requiring concomitant use of drugs that interfere with CYP3A4 and/or P-glycoprotein activity, or drugs such as cisapride or metoclopramide; Patients allergic to peanuts, soybeans, or SRL; Patients concurrently enrolled in other clinical research projects; Patients with abnormal ophthalmologic findings; Patients with growth retardation or other abnormalities; Patients unable to complete the study or adhere to scheduled follow-up due to personal reasons. 2.Patients who did not undergo SRL blood concentration monitoring during treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| blood drug concentration;percentage change in lymphatic malformation lesion volume; | — |
Countries
China
Contacts
Public ContactXiaoling Wang
Beijing Children's Hospital, Capital Medical University, Beijing, China
Outcome results
None listed