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Article ; Online: IL-4Rα Inhibition for Severe "Eosinophilic Gastroenteritis, Allergy, and Anaphylaxis" Syndrome due to a Gain-of-Function Variant in STAT6.

Faguer, Stanislas / Delabesse, Eric / Paul, Carle / Pasquet, Marlène

2023 Volume 44, Issue 1, Page(s) 29

MeSH term(s)	Humans ; Anaphylaxis/diagnosis ; Enteritis ; Eosinophilia/diagnosis ; Gain of Function Mutation ; STAT6 Transcription Factor/genetics ; STAT6 Transcription Factor/metabolism
Chemical Substances	STAT6 protein, human ; STAT6 Transcription Factor
Language	English
Publishing date	2023-12-22
Publishing country	Netherlands
Document type	Case Reports ; Letter
ZDB-ID	779361-3
ISSN	1573-2592 ; 0271-9142
ISSN (online)	1573-2592
ISSN	0271-9142
DOI	10.1007/s10875-023-01639-9
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Article ; Online: Artificial intelligence-based prediction models for acute myeloid leukemia using real-life data: A DATAML registry study.

Didi, Ibrahim / Alliot, Jean-Marc / Dumas, Pierre-Yves / Vergez, François / Tavitian, Suzanne / Largeaud, Laëtitia / Bidet, Audrey / Rieu, Jean-Baptiste / Luquet, Isabelle / Lechevalier, Nicolas / Delabesse, Eric / Sarry, Audrey / De Grande, Anne-Charlotte / Bérard, Emilie / Pigneux, Arnaud / Récher, Christian / Simoncini, David / Bertoli, Sarah

Leukemia research

2024 Volume 136, Page(s) 107437

Abstract: We designed artificial intelligence-based prediction models (AIPM) using 52 diagnostic variables from 3687 patients included in the DATAML registry treated with intensive chemotherapy (IC, N = 3030) or azacitidine (AZA, N = 657) for an acute myeloid ... ...

Abstract	We designed artificial intelligence-based prediction models (AIPM) using 52 diagnostic variables from 3687 patients included in the DATAML registry treated with intensive chemotherapy (IC, N = 3030) or azacitidine (AZA, N = 657) for an acute myeloid leukemia (AML). A neural network called multilayer perceptron (MLP) achieved a prediction accuracy for overall survival (OS) of 68.5% and 62.1% in the IC and AZA cohorts, respectively. The Boruta algorithm could select the most important variables for prediction without decreasing accuracy. Thirteen features were retained with this algorithm in the IC cohort: age, cytogenetic risk, white blood cells count, LDH, platelet count, albumin, MPO expression, mean corpuscular volume, CD117 expression, NPM1 mutation, AML status (de novo or secondary), multilineage dysplasia and ASXL1 mutation; and 7 variables in the AZA cohort: blood blasts, serum ferritin, CD56, LDH, hemoglobin, CD13 and disseminated intravascular coagulation (DIC). We believe that AIPM could help hematologists to deal with the huge amount of data available at diagnosis, enabling them to have an OS estimation and guide their treatment choice. Our registry-based AIPM could offer a large real-life dataset with original and exhaustive features and select a low number of diagnostic features with an equivalent accuracy of prediction, more appropriate to routine practice.
MeSH term(s)	Humans ; Antimetabolites, Antineoplastic/therapeutic use ; Artificial Intelligence ; Treatment Outcome ; Leukemia, Myeloid, Acute/diagnosis ; Leukemia, Myeloid, Acute/drug therapy ; Leukemia, Myeloid, Acute/genetics ; Azacitidine/therapeutic use ; Registries
Chemical Substances	Antimetabolites, Antineoplastic ; Azacitidine (M801H13NRU)
Language	English
Publishing date	2024-01-09
Publishing country	England
Document type	Journal Article
ZDB-ID	752396-8
ISSN	1873-5835 ; 0145-2126
ISSN (online)	1873-5835
ISSN	0145-2126
DOI	10.1016/j.leukres.2024.107437
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Zs.A 1321: Show issues

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Article ; Online: Dismal outcome of refractory or relapsing patients with myelodysplasia-related acute myeloid leukemia partially alleviated by intensive chemotherapy.

Leroy, Harmony / Gadaud, Noémie / Bérard, Emilie / Klein, Emilie / Luquet, Isabelle / Vial, Jean-Philippe / Rieu, Jean-Baptiste / Lechevalier, Nicolas / Tavitian, Suzanne / Leguay, Thibaut / Largeaud, Laetitia / Bidet, Audrey / Delabesse, Eric / Sarry, Audrey / de Grande, Anne-Charlotte / Récher, Christian / Pigneux, Arnaud / Bertoli, Sarah / Dumas, Pierre-Yves

Cancer medicine

2024 Volume 13, Issue 3, Page(s) e7003

Abstract: Background: Acute myeloid leukemia (AML) with myelodysplasia-related characteristics is a heterogeneous subset of AML that has been challenged throughout the history of myeloid malignancies classifications, considered to have similar outcomes as ... ...

Abstract	Background: Acute myeloid leukemia (AML) with myelodysplasia-related characteristics is a heterogeneous subset of AML that has been challenged throughout the history of myeloid malignancies classifications, considered to have similar outcomes as intermediate- or adverse-risk AML depending on the subgroup. However, little is known about the fate of these patients in refractory or relapsed situation (R/R) after first line therapy. Methods: A large series of R/R AML patients, recorded in the French DATAML registry, have received either intensive chemotherapy (ICT), azacitidine (AZA) as single agent, or best supportive care (BSC). A cohort of 183 patients (median age 63-year-old) with what was called at the time AML-MRC has been explored, and data are reported here. Results: Patient status was refractory for 93, while 90 had relapsed. Respectively, 88, 34, and 61 were included in the three treatment arms. The median OS of the whole cohort was 4.2 months (95%CI: 3.1-5.6) with a mean 1-year overall survival of 24% ± 3.2%. There was no significant survival difference between refractory and relapsed patients. The BSC group had overall a significantly worse outcome (p = 0.0001), and this remained true in both refractory (p = 0.01) and relapsed (p = 0.002) patients. Similar survivals were observed in both groups comparing ICT and AZA. Conclusions: These data, reporting about an ill-explored population, indicate the poor prognosis of this condition where both ICT and AZA can be proposed. The latter, which was demonstrated here to be a feasible option, should be added to new targeted therapies.
MeSH term(s)	Humans ; Middle Aged ; Antineoplastic Combined Chemotherapy Protocols/adverse effects ; Leukemia, Myeloid, Acute/complications ; Leukemia, Myeloid, Acute/drug therapy ; Azacitidine/therapeutic use ; Myelodysplastic Syndromes/complications ; Myelodysplastic Syndromes/drug therapy ; Recurrence
Chemical Substances	Azacitidine (M801H13NRU)
Language	English
Publishing date	2024-02-23
Publishing country	United States
Document type	Journal Article
ZDB-ID	2659751-2
ISSN	2045-7634 ; 2045-7634
ISSN (online)	2045-7634
ISSN	2045-7634
DOI	10.1002/cam4.7003
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Article ; Online: Vitamin C and D supplementation in acute myeloid leukemia.

Mouchel, Pierre Luc / Bérard, Emilie / Tavitian, Suzanne / Gadaud, Noémie / Vergez, François / Rieu, Jean Baptiste / Luquet, Isabelle / Sarry, Audrey / Huguet, Françoise / Largeaud, Laetitia / Delabesse, Eric / Huynh, Anne / Bertoli, Sarah / Récher, Christian

Blood advances

2023 Volume 7, Issue 22, Page(s) 6886–6897

Abstract: Recent studies have highlighted the role of vitamin C and D in acute myeloid leukemia (AML). In 2018, we changed our practices to add both vitamins to the supportive care for all consecutive patients with AML undergoing intensive chemotherapy. In this ... ...

Abstract	Recent studies have highlighted the role of vitamin C and D in acute myeloid leukemia (AML). In 2018, we changed our practices to add both vitamins to the supportive care for all consecutive patients with AML undergoing intensive chemotherapy. In this study, we compared the outcomes of patients treated before and after this change in practice. From 2015 to 2020, 431 patients were included, 262 of whom received no supplementation and 169 of whom received vitamin supplementation. Vitamin C and vitamin D was administered from day 10 of chemotherapy until hematologic recovery from induction and consolidation. Most patients presented at diagnosis with low levels of vitamin C and D. Upon recovery from induction, vitamin D levels among the vitamin C/D group significantly increased compared with those at diagnosis, and pretransplant levels were significantly higher in the vitamin C/D group compared with the control group (median of 33 vs 19 ng/mL; P < .0001). During induction, the rates of bacterial or fungal infection, hemorrhage, or macrophage activation syndrome were lower in the vitamin C/D group, whereas there was no difference in response rate, relapse incidence, and overall survival (OS). However, the multivariate analysis for OS showed a significant interaction between vitamin C/D and NPM1 mutation, meaning that vitamin C/D supplementation was significantly and independently associated with better OS in patients with NPM1 mutations (hazard ratio [HR], 0.52; 95% confidence interval [CI], 0.30-0.90; P = .019) compared with patients with wild-type NPM1 (HR, 1.01; 95% CI, 0.68-1.51; P = .95). In conclusion, vitamin C/D supplementation is safe and could influence the outcomes of patients with AML undergoing intensive chemotherapy.
MeSH term(s)	Humans ; Ascorbic Acid/therapeutic use ; Nucleophosmin ; Prognosis ; Mutation ; Leukemia, Myeloid, Acute/genetics ; Vitamins/therapeutic use ; Vitamin D/therapeutic use ; Dietary Supplements
Chemical Substances	Ascorbic Acid (PQ6CK8PD0R) ; Nucleophosmin (117896-08-9) ; Vitamins ; Vitamin D (1406-16-2)
Language	English
Publishing date	2023-10-30
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2023010559
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Zs.A 7153: Show issues

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Article ; Online: VEXAS syndrome in a patient with previous spondyloarthritis with a favourable response to intravenous immunoglobulin and anti-IL17 therapy.

Magnol, Marion / Couvaras, Loukianos / Degboé, Yannick / Delabesse, Eric / Bulai-Livideanu, Cristina / Ruyssen-Witrand, Adeline / Constantin, Arnaud

Rheumatology (Oxford, England)

2021 Volume 60, Issue 9, Page(s) e314–e315

MeSH term(s)	Antibodies, Monoclonal, Humanized/administration & dosage ; Antibodies, Monoclonal, Humanized/immunology ; Hereditary Autoinflammatory Diseases/drug therapy ; Hereditary Autoinflammatory Diseases/immunology ; Humans ; Immunoglobulins, Intravenous/administration & dosage ; Immunoglobulins, Intravenous/immunology ; Interleukin-17/antagonists & inhibitors ; Interleukin-17/immunology ; Male ; Middle Aged ; Spondylarthritis/complications ; Spondylarthritis/immunology
Chemical Substances	Antibodies, Monoclonal, Humanized ; Immunoglobulins, Intravenous ; Interleukin-17 ; secukinumab (DLG4EML025)
Language	English
Publishing date	2021-03-10
Publishing country	England
Document type	Case Reports ; Journal Article
ZDB-ID	1464822-2
ISSN	1462-0332 ; 1462-0324
ISSN (online)	1462-0332
ISSN	1462-0324
DOI	10.1093/rheumatology/keab211
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Zs.B 240: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 2021: Bestellungen von Artikeln über das Online-Bestellformular ab Jg. 2022: Lesesaal (EG)
Zs.MO 497: Show issues

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Article ; Online: Subsequent development of histiocytic sarcoma and follicular lymphoma: cytogenetics and next-generation sequencing analyses provide evidence for transdifferentiation of early common lymphoid precursor-a case report and review of literature.

Péricart, Sarah / Waysse, Charlotte / Siegfried, Aurore / Struski, Stephanie / Delabesse, Eric / Laurent, Camille / Evrard, Solène

Virchows Archiv : an international journal of pathology

2019 Volume 476, Issue 4, Page(s) 609–614

Abstract: Histiocytic sarcoma (HS) is a rare aggressive hematologic neoplasm that can be associated with low-grade B cell lymphoma. The development of both neoplasms is currently being considered a transdifferentiation mechanism but remains elusive. We report the ... ...

Abstract	Histiocytic sarcoma (HS) is a rare aggressive hematologic neoplasm that can be associated with low-grade B cell lymphoma. The development of both neoplasms is currently being considered a transdifferentiation mechanism but remains elusive. We report the case of a 65-year-old patient with synchronous development of peritoneal/abdominal HS and grade 1-2 follicular lymphoma (FL). Cytogenetic analysis and targeted next-generation sequencing of both FL and HS tumors identified common genomic alterations such as IGH-BCL2 rearrangement, CREBBP and KMT2D, and aberrations of chromosomes 9q and 19q. However, only the HS tumor had a KRAS mutation while the lymph node involved by FL harbored a TNFAIP3 mutation and both tumors also showed distinct chromosomal alterations. This report strengthens the hypothesis of a common lymphoid progenitor which accumulates genetic alterations leading to two different hematologic malignant diseases with significantly distinct prognoses.
MeSH term(s)	Aged ; Cell Transdifferentiation/physiology ; DNA-Binding Proteins/genetics ; Histiocytic Sarcoma/diagnosis ; Histiocytic Sarcoma/pathology ; Humans ; Lymph Nodes/pathology ; Lymphoma, B-Cell/pathology ; Lymphoma, Follicular/pathology ; Male ; Neoplasm Proteins/genetics ; Prognosis
Chemical Substances	DNA-Binding Proteins ; KMT2D protein, human ; Neoplasm Proteins
Language	English
Publishing date	2019-12-05
Publishing country	Germany
Document type	Case Reports ; Journal Article ; Review
ZDB-ID	1184867-4
ISSN	1432-2307 ; 0945-6317
ISSN (online)	1432-2307
ISSN	0945-6317
DOI	10.1007/s00428-019-02691-w
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Article ; Online: Targeted High-throughput Sequencing for Hematological Malignancies: A GBMHM Survey of Practice and Cost Evaluation in France.

Darlington, Meryl / Sujobert, Pierre / Kosmider, Olivier / Luque Paz, Damien / Kaltenbach, Sophie / Figeac, Martin / Hayette, Sandrine / Mezaour, Nadia / Coquerelle, Séverine / Alary, Anne-Sophie / Bidet, Audrey / Le Bris, Yannick / Delabesse, Eric / Davi, Frédéric / Preudhomme, Claude / Durand-Zaleski, Isabelle / Macintyre, Elizabeth

HemaSphere

2023 Volume 7, Issue 9, Page(s) e943

Abstract: The objective of this study was to assess the clinical impact and financial costs of next-generation sequencing (NGS) in 5 categories of pediatric and adult hematological cancers. NGS prescriptions were prospectively collected from 26 laboratories, with ... ...

Abstract	The objective of this study was to assess the clinical impact and financial costs of next-generation sequencing (NGS) in 5 categories of pediatric and adult hematological cancers. NGS prescriptions were prospectively collected from 26 laboratories, with varied technical and reporting practice (all or only significant targets). Impact was defined by the identification of (1) an actionable mutation, (2) a mutation with prognostic and/or theranostic value, and/or (3) a mutation allowing nosological refinement, reported by local investigators. A microcosting study was undertaken in 4 laboratories, identifying the types and volumes of resources required for each procedural step. Individual index prescriptions for 3961 patients were available for impact analysis on the management of myeloid disorders (two thirds) and, mainly mature B, lymphoid disorders (one third). NGS results were considered to impact the management for 73.4% of prescriptions: useful for evaluation of prognostic risk in 34.9% and necessary for treatment adaptation (actionable) in 19.6%, but having no immediate individual therapeutic impact in 18.9%. The average overall cost per sample was 191 € for the restricted mature lymphoid amplicon panel. Capture panel costs varied from 369 € to 513 €. Unit costs varied from 0.5 € to 5.7 € per kb sequenced, from 3.6 € to 11.3 € per target gene/hot-spot sequenced and from 4.3 € to 73.8 € per target gene/hot-spot reported. Comparable costs for the Amplicon panels were 5-8 € per kb and 10.5-14.7 € per target gene/hot-spot sequenced and reported, demonstrating comparable costs with greater informativity/flexibility for capture strategies. Sustainable funding of precision medicine requires a transparent discussion of its impact on care pathways and its financial aspects.
Language	English
Publishing date	2023-08-24
Publishing country	United States
Document type	Journal Article
ISSN	2572-9241
ISSN (online)	2572-9241
DOI	10.1097/HS9.0000000000000943
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Article ; Online: Sorafenib plus all-trans retinoic acid for AML patients with FLT3-ITD and NPM1 mutations.

Guenounou, Sarah / Delabesse, Eric / Récher, Christian

European journal of haematology

2014 Volume 93, Issue 6, Page(s) 533–536

Abstract: Knowledge of the molecular basis of acute myeloid leukaemia has increased considerably in the past few years, and therapies targeting specific molecular defects of this disease are intensively investigated. Patients with both NPM1 and FLT3-ITD mutations ... ...

Abstract	Knowledge of the molecular basis of acute myeloid leukaemia has increased considerably in the past few years, and therapies targeting specific molecular defects of this disease are intensively investigated. Patients with both NPM1 and FLT3-ITD mutations encompass 20% of cytogenetically normal AML. The multikinase and FLT3 inhibitor, sorafenib, has shown some efficacy in patients with relapsed FLT3-ITD(+) AML. In addition, it is suggested that all-trans retinoic acid (ATRA) used in combination with chemotherapy has shown to improve outcome of patients harbouring NPM1 mutations. We report here the clinical course of three patients with refractory or relapsed FLT3-ITD(+) /NPM1(+) AML who achieved significant response upon sorafenib and ATRA combination.
MeSH term(s)	Adolescent ; Adult ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Fatal Outcome ; Female ; Humans ; Leukemia, Myeloid, Acute/diagnosis ; Leukemia, Myeloid, Acute/drug therapy ; Leukemia, Myeloid, Acute/genetics ; Male ; Middle Aged ; Mutation ; Niacinamide/administration & dosage ; Niacinamide/analogs & derivatives ; Nuclear Proteins/genetics ; Phenylurea Compounds/administration & dosage ; Treatment Outcome ; Tretinoin/administration & dosage ; Young Adult ; fms-Like Tyrosine Kinase 3/genetics
Chemical Substances	Nuclear Proteins ; Phenylurea Compounds ; nucleophosmin (117896-08-9) ; Niacinamide (25X51I8RD4) ; Tretinoin (5688UTC01R) ; sorafenib (9ZOQ3TZI87) ; fms-Like Tyrosine Kinase 3 (EC 2.7.10.1)
Language	English
Publishing date	2014-12
Publishing country	England
Document type	Case Reports ; Journal Article
ZDB-ID	392482-8
ISSN	1600-0609 ; 0902-4441
ISSN (online)	1600-0609
ISSN	0902-4441
DOI	10.1111/ejh.12334
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Article ; Online: Genomic landscape of hyperleukocytic acute myeloid leukemia.

Largeaud, Laetitia / Bertoli, Sarah / Bérard, Emilie / Tavitian, Suzanne / Picard, Muriel / Dufrechou, Stéphanie / Prade, Naïs / Vergez, François / Rieu, Jean Baptiste / Luquet, Isabelle / Sarry, Audrey / Huguet, Françoise / Ruiz, Jean / De Mas, Véronique / Delabesse, Eric / Récher, Christian

Blood cancer journal

2022 Volume 12, Issue 1, Page(s) 4

MeSH term(s)	Genomics ; Humans ; Leukemia, Myeloid, Acute/complications ; Leukemia, Myeloid, Acute/genetics ; Leukocyte Count ; Leukocytosis/complications ; Leukocytosis/genetics ; Mutation ; Retrospective Studies
Language	English
Publishing date	2022-01-05
Publishing country	United States
Document type	Letter ; Research Support, Non-U.S. Gov't
ZDB-ID	2600560-8
ISSN	2044-5385 ; 2044-5385
ISSN (online)	2044-5385
ISSN	2044-5385
DOI	10.1038/s41408-021-00601-5
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Article ; Online: Azacitidine, intensive chemotherapy or best supportive care in relapsed or refractory acute myeloid leukemia, a DATAML registry study.

Gadaud, Noémie / Leroy, Harmony / Bérard, Emilie / Tavitian, Suzanne / Leguay, Thibaut / Dimicoli-Salazar, Sophie / Rieu, Jean-Baptiste / Luquet, Isabelle / Largeaud, Laetitia / Bidet, Audrey / Delabesse, Eric / Klein, Emilie / Sarry, Audrey / de Grande, Anne-Charlotte / Bories, Pierre / Pigneux, Arnaud / Récher, Christian / Dumas, Pierre-Yves / Bertoli, Sarah

Leukemia & lymphoma

2022 Volume 63, Issue 6, Page(s) 1398–1406

Abstract: We analyzed 526 consecutive acute myeloid leukemia patients refractory to or relapsing after chemotherapy. 270 patients received intensive salvage chemotherapy (IC), 97 azacitidine (AZA) and 159 best supportive care (BSC). Complete response was obtained ... ...

Abstract	We analyzed 526 consecutive acute myeloid leukemia patients refractory to or relapsing after chemotherapy. 270 patients received intensive salvage chemotherapy (IC), 97 azacitidine (AZA) and 159 best supportive care (BSC). Complete response was obtained in 37/19/0% (
MeSH term(s)	Antimetabolites, Antineoplastic/therapeutic use ; Azacitidine/therapeutic use ; Chronic Disease ; Humans ; Leukemia, Myeloid, Acute/drug therapy ; Leukemia, Myeloid, Acute/genetics ; Registries ; Treatment Outcome
Chemical Substances	Antimetabolites, Antineoplastic ; Azacitidine (M801H13NRU)
Language	English
Publishing date	2022-01-04
Publishing country	United States
Document type	Journal Article
ZDB-ID	1042374-6
ISSN	1029-2403 ; 1042-8194
ISSN (online)	1029-2403
ISSN	1042-8194
DOI	10.1080/10428194.2021.2022140
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