Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas
BackgroundPrognosis 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 hema...
| Main Authors: | , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2023-02-01
|
| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fonc.2023.1064190/full |
| _version_ | 1797901488159195136 |
|---|---|
| author | Thomas Eichholz Michaela Döring Stefano Giardino Bernd Gruhn Christian Seitz Tim Flaadt Wolfgang Schwinger Martin Ebinger Ursula Holzer Markus Mezger Heiko-Manuel Teltschik Monika Sparber-Sauer Monika Sparber-Sauer Ewa Koscielniak Ewa Koscielniak Michael Abele Rupert Handgretinger Peter Lang |
| author_facet | Thomas Eichholz Michaela Döring Stefano Giardino Bernd Gruhn Christian Seitz Tim Flaadt Wolfgang Schwinger Martin Ebinger Ursula Holzer Markus Mezger Heiko-Manuel Teltschik Monika Sparber-Sauer Monika Sparber-Sauer Ewa Koscielniak Ewa Koscielniak Michael Abele Rupert Handgretinger Peter Lang |
| author_sort | Thomas Eichholz |
| collection | DOAJ |
| description | BackgroundPrognosis 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.MethodsPatients 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.ResultsWe 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.ConclusionHaplo-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. |
| first_indexed | 2024-04-10T09:01:45Z |
| format | Article |
| id | doaj.art-ffe18b246e0349b6adb01290d8fb11c8 |
| institution | Directory Open Access Journal |
| issn | 2234-943X |
| language | English |
| last_indexed | 2024-04-10T09:01:45Z |
| publishDate | 2023-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Oncology |
| spelling | doaj.art-ffe18b246e0349b6adb01290d8fb11c82023-02-21T12:57:28ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2023-02-011310.3389/fonc.2023.10641901064190Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomasThomas Eichholz0Michaela Döring1Stefano Giardino2Bernd Gruhn3Christian Seitz4Tim Flaadt5Wolfgang Schwinger6Martin Ebinger7Ursula Holzer8Markus Mezger9Heiko-Manuel Teltschik10Monika Sparber-Sauer11Monika Sparber-Sauer12Ewa Koscielniak13Ewa Koscielniak14Michael Abele15Rupert Handgretinger16Peter Lang17University Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyHematopoietic Stem Cell Transplantation Unit, Department of Hematology and Oncology, IRCCS Istituto Giannina Gaslini, Genoa, ItalyDepartment of Pediatrics, Jena University Hospital, Jena, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyDepartment of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, AustriaUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyKlinikum der Landeshauptstadt Stuttgart gKAöR, Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Stuttgart, GermanyKlinikum der Landeshauptstadt Stuttgart gKAöR, Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Stuttgart, GermanyUniversity Tübingen, Medical Faculty, Tübingen, GermanyKlinikum der Landeshauptstadt Stuttgart gKAöR, Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Stuttgart, GermanyUniversity Tübingen, Medical Faculty, Tübingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyUniversity Children’s Hospital, Eberhard Karls University, Tuebingen, GermanyBackgroundPrognosis 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.MethodsPatients 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.ResultsWe 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.ConclusionHaplo-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.https://www.frontiersin.org/articles/10.3389/fonc.2023.1064190/fullhaploidentical hematopoietic stem cell transplantationpediatric sarcomaEwing sarcomasoft tissue sarcomarhabdomyosarcoma |
| spellingShingle | Thomas Eichholz Michaela Döring Stefano Giardino Bernd Gruhn Christian Seitz Tim Flaadt Wolfgang Schwinger Martin Ebinger Ursula Holzer Markus Mezger Heiko-Manuel Teltschik Monika Sparber-Sauer Monika Sparber-Sauer Ewa Koscielniak Ewa Koscielniak Michael Abele Rupert Handgretinger Peter Lang Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas Frontiers in Oncology haploidentical hematopoietic stem cell transplantation pediatric sarcoma Ewing sarcoma soft tissue sarcoma rhabdomyosarcoma |
| title | Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas |
| title_full | Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas |
| title_fullStr | Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas |
| title_full_unstemmed | Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas |
| title_short | Haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high-risk sarcomas |
| title_sort | haploidentical hematopoietic stem cell transplantation as individual treatment option in pediatric patients with very high risk sarcomas |
| topic | haploidentical hematopoietic stem cell transplantation pediatric sarcoma Ewing sarcoma soft tissue sarcoma rhabdomyosarcoma |
| url | https://www.frontiersin.org/articles/10.3389/fonc.2023.1064190/full |
| work_keys_str_mv | AT thomaseichholz haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT michaeladoring haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT stefanogiardino haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT berndgruhn haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT christianseitz haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT timflaadt haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT wolfgangschwinger haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT martinebinger haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT ursulaholzer haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT markusmezger haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT heikomanuelteltschik haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT monikasparbersauer haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT monikasparbersauer haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT ewakoscielniak haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT ewakoscielniak haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT michaelabele haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT ruperthandgretinger haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas AT peterlang haploidenticalhematopoieticstemcelltransplantationasindividualtreatmentoptioninpediatricpatientswithveryhighrisksarcomas |