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The use of rapamycin to treat vascular tumours and malformations: A single-centre experience
Soumitra Tole1, Michelle Fantauzzi2, Diana Cottingham3
1Department of Paediatrics, Division of Haematology/Oncology, Hospital for Sick Children, Toronto, Ontario.
Insights
Rapamycin is a safe and effective treatment for children with vascular tumors and malformations. Early treatment in younger children shows better response rates, suggesting timely intervention is beneficial.
Area of Science:
- Pediatric Oncology
- Vascular Biology
- Pharmacology
Background:
- Vascular tumors and malformations are rare conditions in children.
- Current treatment options have limitations.
Purpose of the Study:
- To evaluate the safety and efficacy of rapamycin in pediatric patients with vascular tumors and malformations.
Main Methods:
- Retrospective review of 42 pediatric patients treated with rapamycin.
- Efficacy assessed by symptom improvement, radiological changes, and laboratory parameters.
- Safety evaluated by adverse events and treatment discontinuation.
Main Results:
- 76% of patients showed clinical response after a median of 49 days.
- 16 out of 21 patients had reduced lesion size on imaging.
- Younger children (<4 years) demonstrated higher response rates.
- No serious infections or toxicities were reported.
Conclusions:
- Rapamycin is a safe and effective therapy for select pediatric vascular tumors and malformations.
- Early initiation of rapamycin may improve treatment outcomes in younger children.
Objectives:
To assess the safety and efficacy of rapamycin in treating children with vascular tumours and malformations.
Study Design:
We performed a retrospective review at a large tertiary care paediatric centre to assess the efficacy and safety of using rapamycin to treat vascular tumours and malformations. Response to therapy was defined by patient-reported symptom improvement, radiological reduction in size of lesions, and/or improvement of laboratory parameters.
Results:
Forty-two patients (7 with vascular tumours and 35 with vascular malformations) have been treated with rapamycin. Despite 33 of 42 patients being diagnosed in the first year of life, the median age of initiating rapamycin was 11 years. Of the 38 children treated for a minimum of 4 months, 29 (76%) exhibited a clinical response. Twenty-one patients had follow-up imaging studies and of these, 16 (76%) had radiographic decrease in lesion size. Median time to demonstration of response was 49 days. All five children with vascular tumours and all three children with vascular malformations under the age of 4 years showed a clinical response. Response rate was lower for children ≥ 4 years of age (0/2, 0% for vascular tumours; 21/28, 75% for vascular malformations). No patient experienced an infection directly related to rapamycin or discontinued rapamycin due to toxicity.
Conclusions:
Rapamycin is safe and efficacious in most children with select vascular tumours and malformations. Young children appear to respond better, suggesting that early initiation of rapamycin should be considered.
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