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Eichholz, T; Döring, M; Giardino, S; Gruhn, B; Seitz, C; Flaadt, T; Schwinger, W; Ebinger, M; Holzer, U; Mezger, M; Teltschik, HM; Sparber-Sauer, M; Koscielniak, E; Abele, M; Handgretinger, R; Lang, P.
Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas.
Front Oncol. 2023; 13: 1064190
Doi: 10.3389/fonc.2023.1064190
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- Co-Autor*innen der Med Uni Graz
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Schwinger Wolfgang
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- Abstract:
- BACKGROUND: Prognosis of children with primary disseminated or metastatic relapsed sarcomas remains dismal despite intensification of conventional therapies including high-dose chemotherapy. Since haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is effective in the treatment of hematological malignancies by mediating a graft versus leukemia effect, we evaluated this approach in pediatric sarcomas as well. METHODS: Patients with bone Ewing sarcoma or soft tissue sarcoma who received haplo-HSCT as part of clinical trials using CD3+ or TCRα/β+ and CD19+ depletion respectively were evaluated regarding feasibility of treatment and survival. RESULTS: We identified 15 patients with primary disseminated disease and 14 with metastatic relapse who were transplanted from a haploidentical donor to improve prognosis. Three-year event-free survival (EFS) was 18,1% and predominantly determined by disease relapse. Survival depended on response to pre-transplant therapy (3y-EFS of patients in complete or very good partial response: 36,4%). However, no patient with metastatic relapse could be rescued. CONCLUSION: Haplo-HSCT for consolidation after conventional therapy seems to be of interest for some, but not for the majority of patients with high-risk pediatric sarcomas. Evaluation of its future use as basis for subsequent humoral or cellular immunotherapies is necessary.
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haploidentical hematopoietic stem cell transplantation
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pediatric sarcoma
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Ewing sarcoma
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soft tissue sarcoma
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rhabdomyosarcoma