Patient population.
bleeding, vascular malformation, toxicity, primary malignancy, infected with HIV), infections, pain, impaired cardiac function, oozing, impairment of gastrointestinal absorption
BLEEDING, DISEASE, INFECTIONS, ADVERSE EFFECTS, CARDIAC DISEASE
To be eligible for the VASE trial, patients had to have an LM, VM, or complex slow-flow vascular malformation that was symptomatic (pain, functional limitation, decreased QoL, recurrent infections, oozing, and/or bleeding), refractory to medical treatment (pain killers, low molecular weight heparin, or compression garments), and deemed ineligible for sclerotherapy and/or surgery. Eligible patients were 3 or more months old, had adequate hepatic (bilirubin, aspartate amino transferase [ASAT], alanine amino transferase [ALAT]), medullary (neutrophils ≥1500/mm³, hemoglobin ≥8.0 mg/dL, and platelets ≥50,000/mm³) and renal function (clearance ≥70 mL/min/1.73 m²), a Karnofsky performance status of 50 or higher, and no concurrent use of cytochrome P450 3A4 enzyme inducers or inhibitors.Exclusion criteria included patients with severe concurrent and/or uncontrolled disease (impaired cardiac function or clinically important cardiac disease, impairment of gastrointestinal absorption, history of a primary malignancy within the past 5 years, immunocompromised state, such as for those infected with HIV), and pregnant or lactating women. Possible adverse effects, including that of eventual fertility problems, were discussed with all patients before enrolment. Patients could not receive surgery and/or sclerotherapy within 4 weeks prior to study entry.Malformations that involved more than 1 anatomical region (delimited as forehead, orbit, maxillary, mandibular, neck, trunk, back, abdomen, thigh, leg, foot, arm, forearm, or hand) were defined as “extensive” and those involving a muscle and/or joint and/or bone and/or internal organs as “deep.” There were 103 patients from Brussels, 12 from Paris, 10 from Amiens, 6 from Caen, and 1 from Montpellier, with 86 females (65%) and 46 males (35%). All patients were highly symptomatic and most of them (67%) had had several sessions of sclerotherapy and/or surgical resections, with continued poor QoL.At the time of analysis (October 2022), 220 patients had been enrolled. This interim analysis focuses on patients enrolled up to October 2021, who thus have had 12 or more months of follow-up after treatment onset. This also includes patients who prematurely stopped treatment due to loss of follow-up, toxicity, or other reasons.