Tandem High-Dose Chemotherapy and Autologous Stem Cell Transplantation for Osteosarcoma With Initial Pulmonary...

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.

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.

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.

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.