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No Improvement of S...
No Improvement of Survival for Alveolar Rhabdomyosarcoma Patients After HLA-Matched Versus -Mismatched Allogeneic Hematopoietic Stem Cell Transplantation Compared to Standard-of-Care Therapy
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- Schober, Sebastian Johannes (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Hallmen, Erika (författare)
- Olga Hosp, Stuttgart Canc Ctr, Zent Kinder, Klinikum Landeshauptstadt Stuttgart gKAoR,Padiatri, Stuttgart, Germany.
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- Ressle, Florian (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Gassmann, Hendrik (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Prexler, Carolin (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Wawer, Angela (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- von Luettichau, Irene (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Ladenstein, Ruth (författare)
- Med Univ, St Anna Childrens Hosp, Childrens Canc Res Inst, Dept Pediat, Vienna, Austria.
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- Kazanowska, Bernarda (författare)
- Wroclaw Med Univ, Dept Pediat Hematol Oncol, BMT, Wroclaw, Poland.
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- Ljungman, Gustaf, 1958- (författare)
- Uppsala universitet,Barnonkologisk forskning - särskilt fokus på komplikationer
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- Niggli, Felix (författare)
- Univ Hosp Zurich, Pediat Hematol & Oncol, Fac Med & Hlth Technol, Tampere Ctr Child Adolescent & Maternal Hlth Res, Zurich, Switzerland.
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Lohi, Olli (författare)
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- Hauer, Julia (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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- Gruhn, Bernd (författare)
- Jena Univ Hosp, Dept Pediat, Jena, Germany.
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- Klingebiel, Thomas (författare)
- Universitatsklinikum Frankfurt, Dept Pediat Hematol & Oncol, Frankfurt, Germany.
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- Bader, Peter (författare)
- Universitatsklinikum Frankfurt, Dept Pediat Hematol & Oncol, Frankfurt, Germany.
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- Burdach, Stefan (författare)
- Tech Univ Munich, Inst Pathol, Sch Med, Munich, Germany.
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- Lang, Peter (författare)
- Universitatsklinikum Tubingen, Dept Pediat Hematol & Oncol, Tubingen, Germany.
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- Sparber-Sauer, Monika (författare)
- Olga Hosp, Stuttgart Canc Ctr, Zent Kinder, Klinikum Landeshauptstadt Stuttgart gKAoR,Padiatri, Stuttgart, Germany.;Med Fak Univ Tubingen, Tubingen, Germany.
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- Koscielniak, Ewa (författare)
- Olga Hosp, Stuttgart Canc Ctr, Zent Kinder, Klinikum Landeshauptstadt Stuttgart gKAoR,Padiatri, Stuttgart, Germany.;Med Fak Univ Tubingen, Tubingen, Germany.
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- Thiel, Uwe (författare)
- Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany.
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Tech Univ Munich, Childrens Canc Res Ctr, Sch Med, Dept Pediat, Munich, Germany Olga Hosp, Stuttgart Canc Ctr, Zent Kinder, Klinikum Landeshauptstadt Stuttgart gKAoR,Padiatri, Stuttgart, Germany. (creator_code:org_t)
- 2022-05-10
- 2022
- Engelska.
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Ingår i: Frontiers in Oncology. - : Frontiers Media S.A.. - 2234-943X. ; 12
- Relaterad länk:
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https://doi.org/10.3...
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https://uu.diva-port... (primary) (Raw object)
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https://urn.kb.se/re...
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https://doi.org/10.3...
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Abstract
Ämnesord
Stäng
- BackgroundPatients 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 MethodsIn 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. ResultsMedian 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. ConclusionIn 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Cancer och onkologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Cancer and Oncology (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Nyckelord
- allogeneic stem cell transplantation
- haploidentical
- high-risk rhabdomyosarcoma
- alveolar rhabdomyosarcoma (RMA) stage IV
- matched-pair analysis
- graft-versus-host disease
- transplant-related mortality
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Schober, Sebasti ...
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Hallmen, Erika
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Ressle, Florian
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Gassmann, Hendri ...
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Prexler, Carolin
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Wawer, Angela
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von Luettichau, ...
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Ladenstein, Ruth
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Kazanowska, Bern ...
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Ljungman, Gustaf ...
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Niggli, Felix
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Lohi, Olli
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Hauer, Julia
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Gruhn, Bernd
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Klingebiel, Thom ...
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Bader, Peter
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Burdach, Stefan
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Lang, Peter
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Sparber-Sauer, M ...
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Koscielniak, Ewa
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Thiel, Uwe
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