Table 8.

Recommended Surveillance: Targeted Off-label Therapy for TEK-Related Venous Malformations

TreatmentEvaluationFrequency
Sirolimus 1, 2 Eval by vascular anomalies specialist
  • Based on the VASE study (see Therapies Under Investigation), persons should be evaluated every mo for 1st 3 mos to adjust daily dosage, & then every 3 mos for duration of treatment.
  • The optimal treatment duration is at treating clinician's discretion. After 2 yrs of treatment, frequency of consultation is at treating clinician's discretion.
Eval for dose modification in case of adverse events
Laboratory tests:
  • Hemogram (Hgb, leukocytes, platelets)
  • Thyroid function tests (TSH, T4) to exclude reversible causes of fatigue
  • Cholesterol level
  • Fasting blood glucose level
  • Renal function tests
  • Liver function tests

Hgb = hemoglobin; T4 = thyroxine; TSH = thyroid-stimulating hormone

1.

The decision to treat VM using sirolimus should be initiated by a vascular anomalies expert.

2.

Sirolimus has not been approved by the FDA or EMA for treatment of VM, and thus despite its efficacy remains an off-label therapy. A large prospective Phase III trial (VASE) studying the efficacy of sirolimus in VM is ongoing (see Therapies Under Investigation).

From: TEK-Related Venous Malformations

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