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- Dokumenttyp:
- Article; Journal Article
- Autor(en):
- Schober, Sebastian Johannes; Hallmen, Erika; Reßle, Florian; Gassmann, Hendrik; Prexler, Carolin; Wawer, Angela; von Luettichau, Irene; Ladenstein, Ruth; Kazanowska, Bernarda; Ljungman, Gustaf; Niggli, Felix; Lohi, Olli; Hauer, Julia; Gruhn, Bernd; Klingebiel, Thomas; Bader, Peter; Burdach, Stefan; Lang, Peter; Sparber-Sauer, Monika; Koscielniak, Ewa; Thiel, Uwe
- Titel:
- No Improvement of Survival for Alveolar Rhabdomyosarcoma Patients After HLA-Matched Versus -Mismatched Allogeneic Hematopoietic Stem Cell Transplantation Compared to Standard-of-Care Therapy.
- Abstract:
- BACKGROUND: Patients with stage IV alveolar rhabdomyosarcoma (RMA) have a 5-year-survival rate not exceeding 30%. Here, we assess the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for these patients in comparison to standard-of-care regimens. We also compare the use of HLA-mismatched vs. HLA-matched grafts after reduced vs. myeloablative conditioning regimens, respectively. PATIENTS AND METHODS: In this retrospective analysis, we compare event-free survival (EFS), overall survival (OS), and toxicity of HLA-mismatched vs. -matched transplanted patients in uni- and multivariate analyses (total: n = 50, HLA-matched: n = 15, HLA-mismatched: n = 35). Here, the factors age at diagnosis, age at allo-HSCT, sex, Oberlin score, disease status at allo-HSCT, and HLA graft type are assessed. For 29 primarily transplanted patients, three matched non-transplanted patients per one transplanted patient were identified from the CWS registry. Outcomes were respectively compared for OS and EFS. Matching criteria included sex, age at diagnosis, favorable/unfavorable primary tumor site, and metastatic sites. RESULTS: Median EFS and OS did not differ significantly between HLA-mismatched and -matched patients. In the mismatched group, incidence of acute GvHD was 0.87 (grade III-IV: 0.14) vs. 0.80 in HLA-matched patients (grade III-IV: 0.20). Transplant-related mortality (TRM) of all patients was 0.20 and did not differ significantly between HLA-mismatched and -matched groups. A proportion of 0.58 relapsed or progressed and died of disease (HLA-mismatched: 0.66, HLA-matched: 0.53) whereas 0.18 were alive in complete remission (CR) at data collection. Multivariate and competing risk analyses confirmed CR and very good partial response (VGPR) status prior to allo-HSCT as the only decisive predictor for OS (p < 0.001). Matched-pair survival analyses of primarily transplanted patients vs. matched non-transplanted patients also identified disease status prior to allo-HSCT (CR, VGPR) as the only significant predictor for EFS. Here, OS was not affected, however. CONCLUSION: In this retrospective analysis, only a subgroup of patients with good response at allo-HSCT survived. There was no survival benefit of allo-transplanted patients compared to matched controls, suggesting the absence of a clinically relevant graft-versus-RMA effect in the current setting. The results of this analysis do not support further implementation of allo-HSCT in RMA stage IV patients.
- Zeitschriftentitel:
- Front Oncol
- Jahr:
- 2022
- Band / Volume:
- 12
- Volltext / DOI:
- doi:10.3389/fonc.2022.878367
- PubMed:
- http://view.ncbi.nlm.nih.gov/pubmed/35619911
- Print-ISSN:
- 2234-943X
- TUM Einrichtung:
- Institut für Allgemeine Pathologie und Pathologische Anatomie; Klinik und Poliklinik für Kinder- und Jugendmedizin
BibTeX
Vorkommen:
- mediaTUM GesamtbestandEinrichtungenSchools TUM School of Medicine and HealthDepartmentsClinical MedicineKlinik und Poliklinik für Kinder- und Jugendmedizin (Prof. Hauer)2022