Long-term outcomes and quality of life of high-risk neuroblastoma patients treated with a multimodal treatment including Anti-GD2 immunotherapy: a retrospective cohort study

Background: The incorporation of anti-GD2 antibodies such as ch14.18/SP2/0 into the multimodal treatment of high-risk neuroblastoma (HR-NB) patients has improved their outcomes. As studies assessing the long-term outcomes, long-term sequelae, and health-related quality of life (HRQoL) of this treatm...

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Main Authors: Flaadt, Tim (Author) , Rehm, Jonas (Author) , Simon, Thorsten (Author) , Hero, Barbara (Author) , Ladenstein, Ruth L. (Author) , Lode, Holger (Author) , Grabow, Desiree (Author) , Nolte, Sandra (Author) , Crazzolara, Roman (Author) , Greil, Johann (Author) , Ebinger, Martin (Author) , Abele, Michael (Author) , Holzer, Ursula (Author) , Döring, Michaela (Author) , Schulte, Johannes H. (Author) , Bader, Peter (Author) , Schlegel, Paul-Gerhardt (Author) , Eyrich, Matthias (Author) , Lang, Peter (Author) , Klingebiel, Thomas (Author) , Handgretinger, Rupert (Author)
Format: Article (Journal)
Language:English
Published: 5 January 2025
In: Cancers
Year: 2025, Volume: 17, Issue: 1, Pages: 1-14
ISSN:2072-6694
DOI:10.3390/cancers17010149
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.3390/cancers17010149
Verlag, kostenfrei, Volltext: https://www.mdpi.com/2072-6694/17/1/149
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Author Notes:Tim Flaadt, Jonas Rehm, Thorsten Simon, Barbara Hero, Ruth L. Ladenstein, Holger N. Lode, Desiree Grabow, Sandra Nolte, Roman Crazzolara, Johann Greil, Martin Ebinger, Michael Abele, Ursula Holzer, Michaela Döring, Johannes H. Schulte, Peter Bader, Paul-Gerhardt Schlegel, Matthias Eyrich, Peter Lang, Thomas Klingebiel and Rupert Handgretinger

MARC

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245 1 0 |a Long-term outcomes and quality of life of high-risk neuroblastoma patients treated with a multimodal treatment including Anti-GD2 immunotherapy  |b a retrospective cohort study  |c Tim Flaadt, Jonas Rehm, Thorsten Simon, Barbara Hero, Ruth L. Ladenstein, Holger N. Lode, Desiree Grabow, Sandra Nolte, Roman Crazzolara, Johann Greil, Martin Ebinger, Michael Abele, Ursula Holzer, Michaela Döring, Johannes H. Schulte, Peter Bader, Paul-Gerhardt Schlegel, Matthias Eyrich, Peter Lang, Thomas Klingebiel and Rupert Handgretinger 
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520 |a Background: The incorporation of anti-GD2 antibodies such as ch14.18/SP2/0 into the multimodal treatment of high-risk neuroblastoma (HR-NB) patients has improved their outcomes. As studies assessing the long-term outcomes, long-term sequelae, and health-related quality of life (HRQoL) of this treatment are limited, this retrospective analysis aimed to explore these. Patients and Methods: Between 1991 and 2002, 65 children received a multimodal treatment, including ch14.18/SP2/0, for primary HR-NB. All received chemotherapy according to the NB90/NB97 trial, 51 received high-dose chemotherapy, and all received ch14.18/SP2/0 treatment. We analyzed the long-term sequelae and HRQoL (EORTC QLQ-C30), and evaluated overall and event-free survival (OS/EFS). Results: Twenty-five survivors were evaluated for HRQoL and long-term effects. All reported long-term sequelae, including ototoxicity in 16/25 (64%), cardiac toxicity in 6/25 (24%), and endocrine toxicity in 19/25 (76%) patients. Chronic diarrhea was reported in 20% of female patients. Seven patients developed autoimmune diseases. HRQoL scores were better across multiple scales than those of the matched German general population. Twenty-five-year OS and EFS were 50.8% (95% confidence interval: 31-55) and 43% (30.1-55.3), with 33 (50.8%) long-term survivors. Thirty-two patients died: 28 (43.1%) because of progression/relapse and 4 (6.2%) because of secondary neoplasms. Conclusions: Multimodal treatment, including ch14.18/SP2/0, can achieve long-term survival in HR-NB patients, with a substantial proportion of survivors reporting better HRQoL compared to the general population. All patients reported long-term side effects mostly attributable to chemotherapy and radiotherapy. The relatively high prevalence of autoimmune diseases and persistent diarrhea warrants additional longitudinal research on individuals treated with anti-GD2 antibodies. 
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