12 results on '"Dalle, Jean‐Hugues"'
Search Results
2. Symptomatic osteonecrosis in French survivors of childhood and adolescent leukemia: a clinical and MRI study of LEA cohort.
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Huault, Alice, Michel, Gérard, Charon, Valérie, Chouklati, Kamal, Domenech, Carine, Chastagner, Pascal, Dalle, Jean-Hugues, Paillard, Catherine, Ducassou, Stéphane, Poirée, Marilyne, Plat, Geneviève, Tabone, Marie-Dominique, Kanold, Justyna, Baruchel, André, Berger, Claire, Pellier, Isabelle, Plantaz, Dominique, Theron, Alexandre, Mustafa, Alaa, and Auquier, Pascal
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OSTEONECROSIS ,MAGNETIC resonance imaging ,TEENAGERS ,LEUKEMIA ,ACUTE leukemia - Abstract
Osteonecrosis (ON) is a known complication of acute leukemia (AL) management, affecting 1%–10% of young patients and resulting in long-term morbidity. Widespread access to MRI over the past decade has allowed earlier detection and more accurate assessment. This study investigated clinical and MRI features of the 129 (2.5%) patients with symptomatic ON retrospectively recruited from the French LEA (Leucémies de l'Enfant et de l'Adolescent, or child and adolescent leukemias) cohort (n = 4,973). We analyzed data concerning ON risk factors, multifocal involvement, severe lesions detected by MRI, and patient quality of life (QoL). ON patients tended to be >10 years old at the time of AL diagnosis (odds ratio [OR]: 22.46; p < 10
−6 ), female (OR: 1.8; p = 0.002), or treated for relapse (OR: 1.81; p = 0.041). They more frequently suffered from other sequelae (p < 10−6 ). Most necroses involved weight-bearing joints, and they were multifocal in 69% of cases. Double-blinded review of MRIs for 39 patients identified severe lesions in 14, usually in the hips. QoL of adolescents and adults was poor and permanently impacted after onset of ON. In conclusion, age >10 at time of AL diagnosis, female sex, and relapse occurrence were risk factors for multifocal ON; MRI revealed severe ON in a third of the patients considered; and ON was associated with persistently poor QoL affecting multiple domains. Future studies should include prospective data addressing ON management and seek to identify genetic markers for targeted screening enabling early ON detection and treatment. [ABSTRACT FROM AUTHOR]- Published
- 2023
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3. Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease
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Cappelli, Barbara, Volt, Fernanda, Tozatto-Maio, Karina, Scigliuolo, Graziana Maria, Ferster, Alina, Dupont, Sophie, Simões, Belinda Pinto, Al-Seraihy, Amal, Aljurf, Mahmoud D, Almohareb, Fahad, Belendez, Cristina, Matthes, Susanne, Dhedin, Nathalie, Pondarre, Corinne, Dalle, Jean-Hugues, Bertrand, Yves, Vannier, Jean Pierre, Kuentz, Mathieu, Lutz, Patrick, Michel, Gérard, Rafii, Hanadi, Neven, Benedicte, Zecca, Marco, Bader, Peter, Cavazzana, Marina, Labopin, Myriam, Locatelli, Franco, Magnani, Alessandra, Ruggeri, Annalisa, Rocha, Vanderson, Bernaudin, Françoise, de La Fuente, Josu, Corbacioglu, Selim, Gluckman, Eliane, Eurocord, The Cellular Therapy and Immunobiology Working Party (CTIWP), The Paediatric Diseases Working Party (PDWP) of the EBMT, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique
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Adult ,Male ,Pediatrics ,medicine.medical_specialty ,Adolescent ,Anemia ,Graft vs Host Disease ,Disease ,Human leukocyte antigen ,Anemia, Sickle Cell ,Young Adult ,HLA Antigens ,hemic and lymphatic diseases ,Medicine ,Humans ,Sibling ,Young adult ,Child ,Online Only Articles ,Survival rate ,business.industry ,Incidence (epidemiology) ,Histocompatibility Testing ,Incidence ,Age Factors ,Hematopoietic Stem Cell Transplantation ,Infant ,Généralités ,thalassemia ,children ,adults ,Hematology ,medicine.disease ,Prognosis ,Transplantation ,Europe ,Survival Rate ,surgical procedures, operative ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Child, Preschool ,HSCT ,Female ,business ,Follow-Up Studies - Abstract
SCOPUS: le.j, info:eu-repo/semantics/published
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- 2019
4. Single-Unit versus Double-Unit Umbilical Cord Blood Transplantation in Children and Young Adults with Residual Leukemic Disease
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Bensussan, Armand, Janela, Baptiste, Thonnart, Nicolas, Bagot, Martine, Musette, Philippe, Ginhoux, Florent, Marie-Cardine, Anne, Balligand, Laura, Galambrun, Claire, Sirvent, Anne, Roux, Clémence, Pochon, Cécile, Bruno, Bénédicte, Jubert, Charlotte, Loundou, Anderson, Esmiol, Sophie, Yakoub-Agha, Ibrahim, Forcade, Edouard, Paillard, Catherine, Marie-Cardine, Aude, Plantaz, Dominique, Gandemer, Virginie, Blaise, Didier, Rialland, Fanny, Renard, Cecile, Seux, Mylène, Baumstarck, Karine, Mohty, Mohamad, Dalle, Jean-Hugues, Michel, Gérard, CCSD, Accord Elsevier, Hôpital de la Timone [CHU - APHM] (TIMONE), Aix Marseille Université (AMU), Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Service d'Hématologie [CHRU Nancy], Centre Hospitalier Régional Universitaire de Nancy (CHRU Nancy), Hôpital Jeanne de Flandre [Lille], Université de Lille, CHU Bordeaux [Bordeaux], Centre d'études et de recherche sur les services de santé et la qualité de vie (CEReSS), CHU Marseille, Lille Inflammation Research International Center - U 995 (LIRIC), Institut Pasteur de Lille, Réseau International des Instituts Pasteur (RIIP)-Réseau International des Instituts Pasteur (RIIP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Hôpital Haut-Lévêque [CHU Bordeaux], CHU Strasbourg, CHU Rouen, Normandie Université (NU), Centre Hospitalier Universitaire [Grenoble] (CHU), CHU Pontchaillou [Rennes], Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Centre de Recherche en Cancérologie de Marseille (CRCM), Aix Marseille Université (AMU)-Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Centre hospitalier universitaire de Nantes (CHU Nantes), Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS), Hospices Civils de Lyon (HCL), Assistance Publique - Hôpitaux de Marseille (APHM), Centre de Recherche Saint-Antoine (CRSA), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Hôpital Robert Debré, Université Paris Diderot - Paris 7 (UPD7), Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Institut Pasteur de Lille, Réseau International des Instituts Pasteur (RIIP)-Réseau International des Instituts Pasteur (RIIP), Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Aix Marseille Université (AMU), Centre de Recherche Saint-Antoine (CR Saint-Antoine), Sorbonne Université (SU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Institut Mondor de recherche biomédicale (IMRB), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Service de Dermatologie [Rouen], Normandie Université (NU)-Normandie Université (NU)-Hôpital Charles Nicolle [Rouen], Singapore Immunology Network (SIgN), Biomedical Sciences Institute (BMSI), Immunologie, dermatologie, oncologie, Oncodermatologie, immunologie et cellules souches cutanées (IDO (U976 / UMR_S 976)), Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Aix-Marseille Université - Faculté de médecine (AMU MED), Service d'Hématologie pédiatrique, Hôpital de la Timone, Marseille, Assistance Publique - Hôpitaux de Marseille (APHM)- Hôpital de la Timone [CHU - APHM] (TIMONE), Hôpital Pasteur [Nice] (CHU), Institut d'Histoire de la Pensée Classique (IHPC), Centre National de la Recherche Scientifique (CNRS)-Université Jean Monnet [Saint-Étienne] (UJM)-Université Blaise Pascal - Clermont-Ferrand 2 (UBP)-Université Lumière - Lyon 2 (UL2)-École normale supérieure - Lyon (ENS Lyon), Dynamique Musculaire et Métabolisme (DMEM), Institut National de la Recherche Agronomique (INRA)-Université de Montpellier (UM), Unités d'Activité Médicale [Lille] (UAM), Hôpital Claude Huriez [Lille], CHU Lille-CHU Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Composantes innées de la réponse immunitaire et différenciation (CIRID), Université Bordeaux Segalen - Bordeaux 2-Centre National de la Recherche Scientifique (CNRS), CIC - Clermont Ferrand, Institut National de la Santé et de la Recherche Médicale (INSERM), Hôpital Charles Nicolle [Rouen], Service Hématologie Infantile, CHU Grenoble, Hématogoie pédiatrique, hôpital Sud, Service d’Hématologie [Institut Paoli Calmettes, Marseille], Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Cryopic, Stress Cellulaire, Université de la Méditerranée - Aix-Marseille 2-Institut National de la Santé et de la Recherche Médicale (INSERM), Centre de Recherche en Cancérologie Nantes-Angers (CRCNA), Centre Hospitalier Universitaire d'Angers (CHU Angers), PRES Université Nantes Angers Le Mans (UNAM)-PRES Université Nantes Angers Le Mans (UNAM)-Hôtel-Dieu de Nantes-Institut National de la Santé et de la Recherche Médicale (INSERM)-Hôpital Laennec-Centre National de la Recherche Scientifique (CNRS)-Faculté de Médecine d'Angers-Centre hospitalier universitaire de Nantes (CHU Nantes), Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris], Service d'Immuno-hématologie pédiatrique [Hôpital Robert Debré, Paris], Hôpital Robert Debré-Hôpital Robert Debré, Franche-Comté Électronique Mécanique, Thermique et Optique - Sciences et Technologies (UMR 6174) (FEMTO-ST), Université de Technologie de Belfort-Montbeliard (UTBM)-Ecole Nationale Supérieure de Mécanique et des Microtechniques (ENSMM)-Université de Franche-Comté (UFC), Université Bourgogne Franche-Comté [COMUE] (UBFC)-Université Bourgogne Franche-Comté [COMUE] (UBFC)-Centre National de la Recherche Scientifique (CNRS), Hôpital Claude Huriez-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Hôpital Sud, Centre de Recherche en Cancérologie / Nantes - Angers (CRCNA), Centre hospitalier universitaire de Nantes (CHU Nantes)-Faculté de Médecine d'Angers-Centre Hospitalier Universitaire d'Angers (CHU Angers), PRES Université Nantes Angers Le Mans (UNAM)-PRES Université Nantes Angers Le Mans (UNAM)-Centre National de la Recherche Scientifique (CNRS)-Hôpital Laennec-Institut National de la Santé et de la Recherche Médicale (INSERM)-Hôtel-Dieu de Nantes, Université de Franche-Comté (UFC)-Centre National de la Recherche Scientifique (CNRS)-Ecole Nationale Supérieure de Mécanique et des Microtechniques (ENSMM)-Université de Technologie de Belfort-Montbeliard (UTBM), Hôpital Charles Nicolle [Rouen]-CHU Rouen, Normandie Université (NU)-Normandie Université (NU), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Diderot - Paris 7 (UPD7), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Rouen Normandie (UNIROUEN), and École normale supérieure - Lyon (ENS Lyon)-Université Lumière - Lyon 2 (UL2)-Université Blaise Pascal - Clermont-Ferrand 2 (UBP)-Université Jean Monnet [Saint-Étienne] (UJM)-Centre National de la Recherche Scientifique (CNRS)
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Adult ,Male ,medicine.medical_specialty ,[SDV.IMM] Life Sciences [q-bio]/Immunology ,[SDV]Life Sciences [q-bio] ,Cord blood unit ,Disease ,law.invention ,Young Adult ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,law ,Internal medicine ,hemic and lymphatic diseases ,medicine ,Humans ,Prospective Studies ,Young adult ,Children ,ComputingMilieux_MISCELLANEOUS ,[SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/Pediatrics ,Transplantation ,Acute leukemia ,business.industry ,Minimal residual disease ,Stem cell transplantation ,[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology ,Hematology ,3. Good health ,body regions ,[SDV] Life Sciences [q-bio] ,Leukemia, Myeloid, Acute ,030220 oncology & carcinogenesis ,Double-Unit Umbilical Cord Blood Transplantation ,Cord blood ,[SDV.IMM]Life Sciences [q-bio]/Immunology ,Female ,Cord Blood Stem Cell Transplantation ,business ,030215 immunology ,Young adults - Abstract
International audience; We previously reported in a French prospective randomized study that transplantation of 2 unrelated cord blood (UCB) units instead of 1 unit does not decrease the risk of transplantation failure but may enhance alloreactivity. Here we evaluated the influence of pretransplantation minimal residual disease (MRD) on leukemia relapse and survival after single- versus double-UCB transplantation (UCBT). Among 137 children and young adults who underwent UCBT in this randomized study, 115 had available data on MRD assessment done immediately before initiation of the pretransplantation conditioning regimen. MRD was considered positive at a level of ≥10-4, which was the case of 43 out of 115 patients. Overall, the mean 3-year survival probability was 69.1 ± 4.4%, and it was not significantly influenced by the MRD level: 70.7 ± 5.4% in MRD-negative (
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- 2019
5. L'allogreffe pour leucémie aiguë dans la population pédiatrique en 2019.
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Dalle, Jean-Hugues
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STEM cell transplantation , *LEUKEMIA in children , *ACUTE leukemia , *TEENAGERS , *MEDICAL care - Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is a combination of replacement medicine (rather than surgery) and immunotherapy. Whatever the transplanted stem cell source (i.e. bone marrow, peripheral blood stem cells or cord blood stem cells), the graft provide both new hematopoiesis (erythrocyte, white blood cell and platelet-production) and new "immunopoiesis". This new infused immunologic system is able to participate in the underlying malignant disease control in association with previous chemotherapy and/or radiotherapy by providing graft-versus-leukelia effect. The main steps of HSCT are 1. Administration of a conditioning regimen, mainly myelo-ablative and then toxic in the frame of acute leukemia in children and adolescents; 2. Management of prolonged and severe neutropenia and of chemotherapy-related toxicities; 3. Management of possible immunological conflict between competent T-cells from the graft and recipient tissues (namely graft versus host disease); 4. Relapse occurrence monitoring and management; 5. Management of HSCT related late-effects and sequelae. [ABSTRACT FROM AUTHOR]
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- 2019
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6. Low Body Mass Index Is Associated with Increased Risk of Acute GVHD after Umbilical Cord Blood Transplantation in Children and Young Adults with Acute Leukemia: A Study on Behalf of Eurocord and the EBMT Pediatric Disease Working Party.
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Paviglianiti, Annalisa, Dalle, Jean Hugues, Ayas, Mouhab, Boelens, Jan Jaap, Volt, Fernanda, Iori, Anna Paola, de Souza, Mair Pedro, Diaz, Miguel Angel, Michel, Gerard, Locatelli, Franco, Jubert, Charlotte, Yakoub-Agha, Ibrahim, Bittencourt, Henrique, Bertrand, Yves, Kenzey, Chantal, Tozatto Maio, Karina, Hayashi, Hiromi, Rocha, Vanderson, Bader, Peter, and Gluckman, Eliane
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BODY mass index , *GRAFT versus host disease , *HEMATOPOIETIC stem cell transplantation , *UMBILICAL cord , *LEUKEMIA - Abstract
Body mass index (BMI) may influence outcomes after allogeneic hematopoietic stem cell transplantation (HSCT). However, the impact of BMI on survival in children undergoing HSCT is not well defined, with conflicting results being reported on this issue. We analyzed 855 patients age 2 to 20 years with diagnosis of acute leukemia who underwent umbilical cord blood transplantation (UCBT) from 1990 to 2015. Patients were classified according to BMI as normal (fifth to 85th percentile), underweight (less than fifth percentile), overweight (85th to 95th percentile), and obese (>95th percentile) using growth charts for age and sex. All patients received single-unit UCBT after a myeloablative conditioning regimen. Diagnosis was acute lymphoblastic leukemia in 68% of the patients. Sixty-one percent of patients (n = 523) were in the normal BMI category, 11% (n = 96) were underweight, 16% (n = 137) overweight, and 12% (n = 99) obese. The cumulative incidence of grade II to IV acute graft-versus-host disease (aGVHD) was 35% (32% to 38%). According to pretransplantation BMI, aGVHD was 46% (33% to 59%) for underweight, 34% (31% to 42%) for normal, 36% (18% to 38%) for overweight, and 27% (15% to 37%) for obese ( P = .04). In multivariate analysis, a BMI less than the fifth percentile was associated with higher incidence of acute grade II to IV GVHD compared with normal-BMI patients (hazard ratio, 1.61; 95% confidence interval, 1.15 to 2.26; P = .006). Our results show that being underweight at the time of transplantation is associated with an increased risk of aGVHD, highlighting the importance of nutritional status before UCBT. [ABSTRACT FROM AUTHOR]
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- 2018
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7. Outcome of relapse after allogeneic HSCT in children with ALL enrolled in the ALL- SCT 2003/2007 trial.
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Kuhlen, Michaela, Willasch, Andre M., Dalle, Jean‐Hugues, Wachowiak, Jacek, Yaniv, Isaac, Ifversen, Marianne, Sedlacek, Petr, Guengoer, Tayfun, Lang, Peter, Bader, Peter, Sufliarska, Sabina, Balduzzi, Adriana, Strahm, Brigitte, von Luettichau, Irene, Hoell, Jessica I., Borkhardt, Arndt, Klingebiel, Thomas, Schrappe, Martin, von Stackelberg, Arend, and Glogova, Evgenia
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LYMPHOBLASTIC leukemia in children ,CANCER relapse ,HEMATOPOIETIC stem cell transplantation ,SALVAGE therapy ,CANCER treatment ,DISEASE progression ,LEUKEMIA treatment ,THERAPEUTICS - Abstract
Relapse remains the major cause of treatment failure in children with high-risk acute lymphoblastic leukaemia ( ALL) undergoing allogeneic haematopoietic stem-cell transplantation (allo- SCT). Prognosis is considered dismal but data on risk factors and outcome are lacking from prospective studies. We analysed 242 children with recurrence of ALL after first allo- SCT enrolled in the Berlin-Frankfurt-Munster ( BFM) ALL- SCT- BFM 2003 and ALL- SCT- BFM international 2007 studies. Median time from allo- SCT to relapse was 7·7 months; median follow-up from relapse after allo- SCT until last follow-up was 3·4 years. The 3-year event-free survival ( EFS) was 15% and overall survival ( OS) was 20%. The main cause of death was disease progression or relapse (86·5%). The majority of children (48%) received salvage therapy without second allo- SCT, 26% of the children underwent a second allo- SCT and 25% received palliative treatment only. In multivariate analyses, age, site of relapse, time to relapse and type of salvage therapy were identified as significant prognostic factors for OS and EFS, whereas factors associated with first SCT were not statistically significant. Combined approaches incorporating novel immunotherapeutic treatment options and second allo- SCT hold promise to improve outcome in children with post allo- SCT relapse. [ABSTRACT FROM AUTHOR]
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- 2018
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8. Relationship between cytomegalovirus ( CMV) reactivation, CMV-driven immunity, overall immune recovery and graft-versus-leukaemia effect in children.
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Jeljeli, Mohamed, Guérin‐El Khourouj, Valérie, Porcher, Raphael, Fahd, Mony, Leveillé, Sandrine, Yakouben, Karima, Ouachée‐Chardin, Marie, LeGoff, Jerome, Cordeiro, Debora Jorge, Pédron, Beatrice, Baruchel, Andre, Dalle, Jean‐Hugues, and Sterkers, Ghislaine
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CYTOMEGALOVIRUSES ,IMMUNITY ,GRAFT versus host disease ,LEUKEMIA in children ,HEMATOPOIETIC stem cells ,FOLLOW-up studies (Medicine) - Abstract
The interplay between immune recovery, cytomegalovirus ( CMV)-reactivation, CMV-driven immunity and graft-versus-leukaemia effect ( GVL) was analysed in 108 children (median age: 8 years) who underwent haematopoietic-stem cell transplantation ( HSCT) for acute leukaemia. Follow-up was 2 years unless death or relapse occurred. CMV-polymerase chain reaction ( PCR) was programmed weekly until month +3 post- HSCT. Immunomonitoring consisted of sequential lymphocyte subset enumerations and analyses of T-cell proliferative and γ-interferon responses to CMV and to adenovirus. In the 108 recipients, the 2-year relapse rate ( RR) was 25% (median time to onset 4·5 months; range: 24 d-17 months). CMV reactivation occurrence was 31% (median time to onset 26 d). Donor/recipient CMV serostatus did not influence RR. Among the 89 recipients disease-free after day +120, i) early CMV-reactivation before day +30 was more frequent ( P = 0·01) in the relapse recipient group opposed to the non-relapse group. ii) CD8
+ / CD28− and CD4+ CD45 RA− T-cell expansions induced by CMV did not influence RR, iii) Recovery of anti- CMV and also anti-adenovirus immunity and of naïve CD4+ T-cells was faster in the non-relapse group ( P = 0·008; 0·009 and 0·002 respectively). In contrast to adult acute myeloid leukaemia, CMV reactivation was associated with increased RR in this paediatric series. Accelerated overall immune recovery rather than CMV-driven immunity had a favourable impact on RR. [ABSTRACT FROM AUTHOR]- Published
- 2014
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9. Hematopoietic stem cell transplantation in SCD.
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Dalle, Jean-Hugues
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HEMATOPOIETIC stem cell transplantation , *STEM cell transplantation , *VASCULAR diseases , *CORD blood transplantation - Abstract
Abstract: Hematopoietic stem cell transplantation (HSCT) is the one and only curative therapy available for patient with severe sickle cell disease (SCD). Until today, several hundreds of patients have undergone geno-identical HSCT. More than 200 patients were transplanted in France. The first indication was cerebral vasculopathy. Among both malignant and non-malignant diseases treated with HSCT, the success rate obtained in SCD patients appears as the best one. From the year 2000, more than 95% of transplanted patients survived the HSCT procedure and more than 90% are completely cured and experience a very satisfying health condition post-transplantation. However, the current standard procedure includes a myeloablative conditioning regimen for warranting engraftment. Such regime is linked to severe long-term side effects such as hypofertility. Due to the excellent obtained results, we have to think about a possible widening of indications, a decrease of conditioning intensity and toxicity, and about HSCT from alternative stem cell sources, such as mismatch family donor, unrelated volunteer donor or unrelated cord blood. [Copyright &y& Elsevier]
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- 2013
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10. Quantitative and Qualitative CD4 T Cell Immune Responses Related to Adenovirus DNAemia in Hematopoietic Stem Cell Transplantation
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Guérin-El Khourouj, Valérie, Dalle, Jean-Hugues, Pédron, Béatrice, Yakouben, Karima, Bensoussan, Danièle, Cordeiro, Débora Jorge, Peltier, Lucas, Ouachée-Chardin, Marie, Baruchel, André, and Sterkers, Ghislaine
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T cell receptors , *IMMUNOREGULATION , *ADENOVIRUS diseases , *HEMATOPOIETIC stem cells , *STEM cell transplantation , *CORD blood , *CYTOKINES , *CHILDHOOD cancer , *CANCER chemotherapy - Abstract
The nature of adenovirus (AdV)-specific T cells that could best predict the capacity of immunocompromised host to fight AdV is unclear. To this aim, 47 pediatric patients were enrolled for at least 3 months either at allogeneic bone marrow transplantation (BMT) (23 genoidentical, 18 unrelated of which 9 were 10/10 and 9 were 9/10 HLA-matched) or at unrelated cord blood transplantation (n = 6). Enumeration of AdV-specific CD4 T cells secreting cytokines (flow cytometry) and proliferative responses to AdV (3HT-incorporation) were compared to AdV-DNAemia. A total of 44/47 patients did not evidence AdV-DNAemia. Thirty-two of 44 (73%) developed CD4-mediated interferon-gamma (IFN-γ) responses to AdV (median 0.36 CD4/μL of blood) since the first month post-HSCT (n = 11: 8 genoidentical and 3 unrelated) or the third month (n = 21 additional patients). At 3 months, both incidence and level intensities of AdV-specific CD4 appeared similar in genoidentical and unrelated BMT (70% and 80%; 0.36 and 0.21 CD4/μL, respectively) and not statistically different from age-matched controls (76%; 1.35 CD4/μL), whereas cord blood transplanted patients exhibited similar incidence but higher level intensities (67%; 1.49 CD4/μL). Polyfunctional (IL2 + IFN-γ) and proliferative responses appeared later, after the third month. Three of 4 9/10 HLA-matched unrelated HSCT that did not develop immunity to AdV presented chemotherapy-resistant AdV-DNAemia at 3 to 5 months post-hematopoietic stem cell transplantation (HSCT). Two were successfully treated with AdV-specific CTL infusion. Monitoring, since month 1 post-HSCT, of IFN-γ-secreting AdV-specific CD4 appears suitable for early detection of at-risk patients especially in 9/10 HLA-matched unrelated HSCT and preferable to monitoring of more delayed IL2- and proliferative responses. [ABSTRACT FROM AUTHOR]
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- 2011
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11. Efficacy of fractionated gemtuzumab ozogamicin combined with cytarabine in advanced childhood myeloid leukaemia.
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Brethon, Benoit, Yakouben, Karima, Oudot, Caroline, Boutard, Patrick, Bruno, Bénédicte, Jérome, Cécile, Nelken, Brigitte, de Lumley, Lionel, Bertrand, Yves, Dalle, Jean-Hugues, Chevret, Sylvie, Leblanc, Thierry, and Baruchel, Andr
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ACUTE myeloid leukemia treatment ,ANTINEOPLASTIC agents ,DRUG efficacy ,JUVENILE diseases ,STEM cell transplantation - Abstract
Gemtuzumab ozogamicin (GO) monotherapy is reported to yield a 20–30% response rate in advanced acute myeloid leukaemia (AML). This study examined the efficacy and tolerability of GO combined with cytarabine (GOCYT) in children with refractory/relapsed CD33
+ AML. Seventeen children received GO 3 mg/m2 on days 1, 4 and 7 plus cytarabine 100 mg/m2 /d for 7 d on a compassionate-use basis. Seven patients then received GO-based consolidation. At the outset of GOCYT, two patients were refractory; eight patients were in refractory first relapse; six patients had relapsed after stem cell transplantation (SCT); and one patient [del(5q) therapy-related AML (t-AML)] had not yet been treated. Mean follow-up was 17 months (8–33 months). Ten responses were obtained after GOCYT induction, including complete remission (CR) or CR without complete recovery of platelets (CRp) in six patients (35%). The responses improved in three children who received GOCYT consolidation, increasing the CR + CRp rate to 53%. SCT was subsequently performed in eight responders. Grade 3–4 adverse events consisted of haematological disorders ( n = 17, 100%) and documented infections ( n = 5, 29%). No cases of sinusoidal obstructive syndrome occurred. Three patients were alive at the cut-off date for this analysis, all of whom had responded to GOCYT. GOCYT combination therapy yielded a high response rate (53%) and showed acceptable toxicity in heavily pretreated children with refractory/relapsed AML. These results warrant a larger prospective study. [ABSTRACT FROM AUTHOR]- Published
- 2008
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12. Discontinuation of Imatinib in Children with Chronic Myeloid Leukemia: A Study from the International Registry of Childhood CML.
- Author
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Millot, Frédéric, Suttorp, Meinolf, Ragot, Stéphanie, Leverger, Guy, Dalle, Jean-Hugues, Thomas, Caroline, Cheikh, Nathalie, Nelken, Brigitte, Poirée, Marilyne, Plat, Geneviève, Versluys, Birgitta, Lausen, Birgitte, and Borisevich, Marina
- Subjects
STATISTICS ,CONFIDENCE intervals ,CHRONIC myeloid leukemia ,DESCRIPTIVE statistics ,IMATINIB - Abstract
Simple Summary: About 50% of adults with chronic myeloid leukemia (CML) in sustained deep molecular response (DMR) to tyrosine kinase inhibitors (TKI) could discontinue the treatment permanently without molecular relapse. Recommendations regarding discontinuation apply only for adults because childhood CML is a very rare disease and represents a separate entity. The aim of our retrospective study was to assess within the International Registry of Childhood CML, the rate of children remaining in molecular response after discontinuation of imatinib in a context of DMR defined as BCR-ABL1/ABL1 < 0.01% (MR
4 ) for at least two years. Eighteen patients less than 18 years old at diagnosis of CML exhibiting a sustained DMR followed by imatinib discontinuation were identified. After discontinuation, the molecular free remission rate was 61%, 56% and 56% at 6, 12 and 36 months, respectively. Our findings represent the basis of recommendation regarding discontinuation for physicians involved in the pediatric CML field. Within the International Registry of Childhood Chronic Myeloid Leukemia (CML), we identified 18 patients less than 18 years old at diagnosis of CML who were in the chronic phase and exhibiting a sustained deep molecular response (DMR) to imatinib defined as BCR-ABL1/ABL1 < 0.01% (MR4 ) for at least two years followed by discontinuation of imatinib. Before discontinuation, the median duration of imatinib was 73.2 months (range, 32–109) and the median duration of MR4 was 46.2 months (range, 23.9–98.6). Seven patients experienced loss of major molecular response (MMR) 4.1 months (range, 1.9–6.4) after stopping and so restarted imatinib. The median molecular follow-up after discontinuation was 51 months (range, 6–100) for the nine patients without molecular relapse. The molecular free remission rate was 61% (95% CI, 38–83%), 56% (95% CI, 33–79%) and 56% (95% CI, 33–79%) at 6, 12 and 36 months, respectively. Six of the seven children who experienced molecular relapse after discontinuation regained DMR (median, 4.7 months; range, 2.5–18) after restarting imatinib. No withdrawal syndrome was observed. In univariate analysis, age, sex, Sokal and ELTS scores, imatinib treatment and DMR durations before discontinuation had no influence on treatment free remission. These data suggest that imatinib can be safely discontinued in children with sustained MR4 for at least two years. [ABSTRACT FROM AUTHOR]- Published
- 2021
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