Tandem High-Dose Chemotherapy and Autologous Stem Cell Transplantation for Osteosarcoma With Initial Pulmonary Metastases.
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BACKGROUND: Although the survival rates of patients with localized osteosarcoma have significantly improved, metastatic osteosarcoma still has a poor prognosis. The role of high-dose chemotherapy and autologous stem cell transplantation (HDC/ASCT) in high-risk osteosarcoma remains unclear compared to that of conventional chemotherapy, and an optimal conditioning regimen remains unestablished. We retrospectively analyzed the outcomes of tandem HDC/ASCT in patients newly diagnosed with osteosarcoma and pulmonary metastasis. METHODS: Among patients with osteosarcoma treated at Seoul National University Children's Hospital between January 2014 and May 2024, 11 underwent tandem HDC/ASCT for pulmonary metastases at diagnosis. We used melphalan, etoposide, and carboplatin as the first HDC and busulfan plus melphalan as the second. RESULTS: The median age at diagnosis was 12.2 years (range, 6.5-13.8), and the median follow-up duration was 57.4 months (range, 5.9-112.9). All the patients achieved engraftment after HDC/ASCT. The 5-year event-free and overall survival (EFS) were 41.6% and the overall survival (OS) was 68.2%, respectively. Relapse occurred in four patients (36.4%), all of whom received salvage chemotherapy. Among the four deaths (36.4%), two were due to treatment-related mortality (one veno-occlusive disease [VOD] and one therapy-related myelodysplastic syndrome) and two were due to the progression of osteosarcoma. No severe complications were observed during the initial HDC/ASCT procedure. However, during the second HDC/ASCT, four patients (36.4%) were treated for severe VOD. CONCLUSIONS: Compared to historical data, tandem HDC/ASCT could be considered a treatment option for osteosarcoma patients with pulmonary metastases. However, considering toxicity, modifications to the conditioning regimen are necessary.