Academic Journal
NGS-based stratification refines the risk stratification in T-ALL and identifies a very-high-risk subgroup of patients
| العنوان: | NGS-based stratification refines the risk stratification in T-ALL and identifies a very-high-risk subgroup of patients |
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| المؤلفون: | Simonin, Mathieu, Vasseur, Loïc, Lengliné, Etienne, Lhermitte, Ludovic, Cabannes-Hamy, Aurélie, Balsat, Marie, Schmidt, Aline, Dourthe, Marie-Emilie, Touzart, Aurore, Graux, Carlos, Grardel, Nathalie, Cayuela, Jean-Michel, Arnoux, Isabelle, Gandemer, Virginie, Huguet, Françoise, Ducassou, Stéphane, Lhéritier, Véronique, Chalandon, Yves, Ifrah, Norbert, Dombret, Hervé, Macintyre, Elizabeth, Petit, Arnaud, Rousselot, Philippe, Lambert, Jérôme, Baruchel, André, Boissel, Nicolas, Asnafi, Vahid |
| المساهمون: | Hôpital Necker - Enfants Malades AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Institut Necker Enfants-Malades (INEM - UM 111 (UMR 8253 / U1151)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), CHU Trousseau APHP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Hopital Saint-Louis AP-HP (AP-HP), Epidemiology and Clinical Statistics for Tumor, Respiratory, and Resuscitation, Centre for Research in Epidemiology and Statistics, Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Institut de Recherche Saint-Louis - Hématologie Immunologie Oncologie (Département de recherche de l’UFR de médecine, ex- Institut Universitaire Hématologie-IUH) (IRSL), Université Paris Cité (UPCité), Centre Hospitalier de Versailles André Mignot (CHV), Centre Hospitalier Lyon Sud CHU - HCL (CHLS), Hospices Civils de Lyon (HCL), Centre Hospitalier Universitaire d'Angers (CHU Angers), PRES Université Nantes Angers Le Mans (UNAM), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes-Angers (CRCI2NA), Université d'Angers (UA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Nantes Université - UFR de Médecine et des Techniques Médicales (Nantes Univ - UFR MEDECINE), Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ), AP-HP Hôpital universitaire Robert-Debré Paris, Université Catholique de Louvain = Catholic University of Louvain (UCL), Hôpital Claude Huriez Lille, Centre Hospitalier Régional Universitaire CHU Lille (CHRU Lille), Laboratoire d'hématologie biologique Hôpital de la Timone - Hôpital Nord - APHM, Aix Marseille Université (AMU)-Assistance Publique - Hôpitaux de Marseille (APHM)-Hôpital de la Timone CHU - APHM (TIMONE), Service de médecine de l'enfant et de l'adolescent CHU Rennes, Centre Hospitalier Universitaire de Rennes CHU Rennes = Rennes University Hospital Pontchaillou, Institut de Génétique et Développement de Rennes (IGDR), Université de Rennes (UR)-Centre National de la Recherche Scientifique (CNRS)-Structure Fédérative de Recherche en Biologie et Santé de Rennes (Biosit : Biologie - Santé - Innovation Technologique), Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037), Université Toulouse III - Paul Sabatier (UT3), Université de Toulouse (UT)-Université de Toulouse (UT)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Institut National de la Santé et de la Recherche Médicale (INSERM), Service Hématologie - IUCT-Oncopole CHU Toulouse, Pôle Biologie CHU Toulouse, Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Pôle IUCT CHU Toulouse, Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Centre Hospitalier Universitaire de Bordeaux (CHU Bordeaux), Geneva University Hospitals and Geneva University, Swiss Group for Clinical Cancer Research (SAKK), Service de biostatistiques et information médicale Saint-Louis, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal Paris, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris Cité (UPCité), Service d'Hématologie Biologique Hôpital Robert Debré, Paris, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP) |
| المصدر: | ISSN: 0006-4971. |
| بيانات النشر: | CCSD American Society of Hematology |
| سنة النشر: | 2024 |
| المجموعة: | Aix-Marseille Université: HAL |
| مصطلحات موضوعية: | MESH: Adolescent, MESH: Adult, MESH: Young Adult, MESH: F-Box-WD Repeat-Containing Protein 7, MESH: High-Throughput Nucleotide Sequencing, MESH: Mutation, MESH: Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, MESH: Prognosis, MESH: Receptor, Notch1, MESH: Risk Assessment, MESH: Clinical Trials as Topic, MESH: Aged, MESH: Child, Preschool, MESH: Female, MESH: Humans, MESH: Infant, MESH: Male, MESH: Middle Aged, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology |
| الوصف: | International audience ; We previously reported a better outcome in adult and pediatric T-cell acute lymphoblastic leukemia (T-ALL) harboring NOTCH1 and/or FBXW7 mutations without alterations of K-N-RAS and PTEN genes. Availability of high-throughput next-generation sequencing (NGS) strategies led us to refine the outcome prediction in T-ALL. Targeted whole-exome sequencing of 72 T-ALL–related oncogenes was performed in 198 adults with T-ALLs in first remission from the GRAALL-2003/2005 protocols and 242 pediatric patients with T-ALLs from the FRALLE2000T. This approach enabled the identification of, to our knowledge, the first NGS-based classifier in T-ALL, categorizing low-risk patients as those with N/F, PHF6, or EP300 mutations, excluding N-K-RAS, PI3K pathway (PTEN, PIK3CA, and PIK3R1), TP53, DNMT3A, IDH1/2, and IKZF1 alterations, with a 5-year cumulative incidence of relapse (CIR) estimated at 21%. Conversely, the remaining patients were classified as high risk, exhibiting a 5-year CIR estimated at 47%. We externally validated this stratification in the pediatric cohort. NGS-based classifier was highly prognostic independently of minimal residual disease (MRD) and white blood cell (WBC) counts, in both adult and pediatric cohorts. Integration of the NGS-based classifier into a comprehensive risk-stratification model, including WBC count at diagnosis and MRD at the end of induction, enabled the identification of an adverse-risk subgroup (25%) with a 5-year CIR estimated at 51%, and a favorable-risk group (32%) with a 5-year CIR estimated at 12%. NGS-based stratification combined with WBC and MRD sharpens the prognostic classification in T-ALL and identifies a new subgroup of patients who may benefit from innovative therapeutic approaches. The GRAALL-2003/2005 studies were registered at www.ClinicalTrials.gov as #NCT00222027 and #NCT00327678. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| Relation: | info:eu-repo/semantics/altIdentifier/pmid/38848537; PUBMED: 38848537 |
| DOI: | 10.1182/blood.2023023754 |
| الاتاحة: | https://nantes-universite.hal.science/hal-04752342 https://doi.org/10.1182/blood.2023023754 |
| Rights: | http://creativecommons.org/licenses/by-nc/ |
| رقم الانضمام: | edsbas.C531926D |
| قاعدة البيانات: | BASE |
| DOI: | 10.1182/blood.2023023754 |
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