No Improvement of Survival for Alveolar Rhabdomyosarcoma Patients After HLA-Matched Versus -Mismatched Allogeneic Hematopoietic Stem Cell Transplantation Compared to Standard-of-Care Therapy

Sebastian Johannes Schober, Erika Hallmen, Florian Reßle, Hendrik Gassmann, Carolin Prexler, Angela Wawer, Irene von Luettichau, Ruth Ladenstein, Bernarda Kazanowska, Gustaf Ljungman, Felix Niggli, Olli Lohi, Julia Hauer, Bernd Gruhn, Thomas Klingebiel, Peter Bader, Stefan Burdach, Peter Lang, Monika Sparber-Sauer, Ewa KoscielniakUwe Thiel

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    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.

    Original languageEnglish
    Article number878367
    Number of pages12
    JournalFrontiers in Oncology
    Publication statusPublished - 10 May 2022
    Publication typeA1 Journal article-refereed


    • allogeneic stem cell transplantation
    • alveolar rhabdomyosarcoma (RMA) stage IV
    • graft-versus-host disease
    • haploidentical
    • high-risk rhabdomyosarcoma
    • matched-pair analysis
    • transplant-related mortality

    Publication forum classification

    • Publication forum level 1

    ASJC Scopus subject areas

    • Oncology
    • Cancer Research


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