Rapid VAC High Dose Melphalan for Childhood Soft Tissue Sarcomas
Author Information
Author(s): C.R. Pinkerton, J. Groot-Loonen, A. Barrett, S.T. Meller, D. Tait, S. Ashley, T.J. McElwain
Primary Institution: Children's Unit, Royal Marsden Hospital, Sutton; Department of Radiotherapy, Glasgow Institute of Radiotherapeutics and Oncology, Western Infirmary, Glasgow, UK.
Hypothesis
Can a rapid dose delivery chemotherapy protocol improve outcomes in children with malignant soft tissue sarcomas?
Conclusion
The Rapid VAC/melphalan regimen is well tolerated and shows encouraging results in standard risk patients.
Supporting Evidence
- Complete remission was achieved in 37 patients (86%).
- Actuarial survival at 5 years for all stages is 57% and event-free survival 44%.
- For patients with non-metastatic diseases, survival rates were 62% and 53% respectively.
- The treatment was well tolerated and largely administered as an out-patient.
- One toxic death occurred due to a combination of infection and possible anthracycline cardiomyopathy.
Takeaway
Doctors gave a special medicine to kids with a type of cancer to help them get better faster, and it worked pretty well.
Methodology
Forty-three children were treated with a chemotherapy protocol followed by high dose melphalan and autologous bone marrow rescue.
Limitations
The small number of high-risk patients limits conclusions regarding efficacy in these subgroups.
Participant Demographics
Patients aged 8 months to 21 years, with a median age of 5 years.
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