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Article ; Online: Outcome of febrile neutropenic patients treated for bacteriuria in hematology.

Delaye, Thomas / Torregrosa Diaz, José Miguel / Vallée, Maxime / Gallego Hernanz, Maria Pilar / Gyan, Emmanuel / Lanotte, Philippe / Roblot, France / Rammaert, Blandine

Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer

2023 Volume 31, Issue 2, Page(s) 102

Abstract: Introduction: Positive urine sample is a frequent finding in post-chemotherapy febrile neutropenia (FN) and can lead to prolonged antibiotic therapy. The aim of this study was to assess the outcome of bacteriuria episodes in FN patients receiving ... ...

Abstract	Introduction: Positive urine sample is a frequent finding in post-chemotherapy febrile neutropenia (FN) and can lead to prolonged antibiotic therapy. The aim of this study was to assess the outcome of bacteriuria episodes in FN patients receiving targeted antibiotic therapy. Materials and methods: A multi-centric retrospective study was conducted over a four-year period (2014-2019) on systematic urinalysis. All consecutive first bacteriuria episodes (≤ 2 bacteria with at least ≥ 10 Results: Among 97 patients, 105 bacteriuria episodes on systematic urinalysis were analyzed; 67.6% occurred in women, 41.9% in AML patients, 17.1% were bacteremic, 14.2% presented with UTS, and 61.9% were treated with short-course antibiotic treatment. One death was reported. In men, no relapse/recurrence was noted, even in the short-course antibiotic group. In women, 2.8% of episodes treated with short-course antibiotic led to relapse or recurrence. Conclusions: Relapse, recurrence, and mortality were uncommon events in FN patients experiencing bacteriuria episode, whatever the antibiotic duration. To distinguish asymptomatic bacteriuria from infection remained challenging in women. In men, systematic urinalysis at onset of FN could be useful.
MeSH term(s)	Adult ; Male ; Humans ; Female ; Bacteriuria/drug therapy ; Bacteriuria/etiology ; Bacteriuria/diagnosis ; Retrospective Studies ; Neoplasm Recurrence, Local/drug therapy ; Anti-Bacterial Agents/therapeutic use ; Fever/drug therapy ; Fever/etiology ; Hematology ; Febrile Neutropenia/drug therapy ; Urinary Tract Infections/drug therapy ; Urinary Tract Infections/etiology
Chemical Substances	Anti-Bacterial Agents
Language	English
Publishing date	2023-01-09
Publishing country	Germany
Document type	Journal Article
ZDB-ID	1134446-5
ISSN	1433-7339 ; 0941-4355
ISSN (online)	1433-7339
ISSN	0941-4355
DOI	10.1007/s00520-022-07522-4
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Zs.A 3697: Show issues

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Article ; Online: Vaccinations avant et après greffe de cellules hématopoïétiques autologues chez les patients atteints de maladies auto-immunes : propositions du groupe Maladies Auto-immunes et Thérapie Cellulaire de la SFGM-TC.

Maria, Alexandre Thibault Jacques / Campidelli, Arnaud / Castilla-Llorente, Cristina / Lansiaux, Pauline / Marjanovic, Zora / Pugnet, Grégory / Torregrosa-Diaz, Jose Miguel / Terriou, Louis / Algayres, Jean-Pierre / Urbain, Fanny / Yakoub-Agha, Ibrahim / Farge, Dominique

Bulletin du cancer

2023 Volume 110, Issue 2S, Page(s) S97–S107

Abstract: The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 12th workshop on hematopoietic stem cell transplantation clinical practices harmonization procedures on September 2021 in Lille, France. In the absence of ...

Title translation	Vaccination before and after autologous hematopoietic cell transplantation for autoimmune diseases: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (MATHEC-SFGM-TC).
Abstract	The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 12th workshop on hematopoietic stem cell transplantation clinical practices harmonization procedures on September 2021 in Lille, France. In the absence of specific national or international recommendation, the French working group for autologous stem Cell transplantation in Auto-immune Diseases (MATHEC) proposed guidances for vaccinations of patients undergoing autologous hematopoietic stem cell transplantation for autoimmune disease, including in the context of SARS-Cov-2 pandemic.
MeSH term(s)	Humans ; Hematopoietic Stem Cell Transplantation ; Bone Marrow Transplantation ; Transplantation, Autologous ; COVID-19/prevention & control ; SARS-CoV-2 ; Autoimmune Diseases/therapy ; Societies, Medical ; Vaccination ; France
Language	French
Publishing date	2023-01-17
Publishing country	France
Document type	English Abstract ; Journal Article ; Review
ZDB-ID	213270-9
ISSN	1769-6917 ; 0007-4551
ISSN (online)	1769-6917
ISSN	0007-4551
DOI	10.1016/j.bulcan.2022.11.005
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Uh III Zs.15: Show issues

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Article ; Online: Efficacy of antibiotic short course for bloodstream infections in acute myeloid leukemia patients with febrile neutropenia: A retrospective comparative study.

Metais, Agathe / Torregrosa Diaz, Jose Miguel / Gallego Hernanz, Maria Pilar / Pichon, Maxime / Desmier, Deborah / Roblot, France / Rammaert, Blandine

The Journal of infection

2021 Volume 84, Issue 1, Page(s) 1–7

Abstract: Objectives: There is no specific recommendation about antimicrobial treatment length for documented infections in chemotherapy induced febrile neutropenia (FN). Practices have changed along time in our center regarding length of antibiotic treatment. ... ...

Abstract	Objectives: There is no specific recommendation about antimicrobial treatment length for documented infections in chemotherapy induced febrile neutropenia (FN). Practices have changed along time in our center regarding length of antibiotic treatment. The aim of this study was to compare long versus short antibiotic course for bloodstream infection (BSI) treatment in acute myeloid leukemia (AML) patients during FN. Methods: This monocentric retrospective comparative study included all consecutive BSI episodes among AML patients with FN for 3 years (2017-2019). Episodes were classified regarding the length of antibiotic treatment, considered as short course if the treatment lasted ≤ 7 days, except for nonfermenting bacteria and Staphylococcus aureus or lugdunensis for which the threshold was ≤ 10 days and ≤ 14 days, respectively. The primary outcome was the number of BSI relapses in both groups within 30 days of antibiotic discontinuation. Results: Among 71 AML patients, 104 BSI episodes were included; 48 (46%) received short course treatment. Only 8 (7.6%) BSI episodes relapsed within 30 days of antibiotic discontinuation, 5 having received short course treatment. No association was found between risk of relapse and short course of antibiotic treatment (p = 0.37). The only risk factor significantly associated with BSI relapse was neutropenia duration (p = 0.005). Conclusion: Antibiotic short course seemed as effective as prolonged treatment for BSI in AML patients during FN, with very few relapses at day 30. These encouraging findings should be confirmed through prospective studies.
MeSH term(s)	Anti-Bacterial Agents/adverse effects ; Bacteremia/drug therapy ; Febrile Neutropenia/complications ; Febrile Neutropenia/drug therapy ; Humans ; Leukemia, Myeloid, Acute/complications ; Leukemia, Myeloid, Acute/drug therapy ; Prospective Studies ; Retrospective Studies ; Sepsis/drug therapy
Chemical Substances	Anti-Bacterial Agents
Language	English
Publishing date	2021-10-29
Publishing country	England
Document type	Journal Article
ZDB-ID	424417-5
ISSN	1532-2742 ; 0163-4453
ISSN (online)	1532-2742
ISSN	0163-4453
DOI	10.1016/j.jinf.2021.10.017
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Zs.A 1501: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Late Onset of Chronic Granulomatous Disease Revealed by Paecilomyces lilacinus Cutaneous Infection.

Lemaigre, Clément / Suarez, Felipe / Martellosio, Jean-Philippe / Barbarin, Cindy / Brunet, Kévin / Chomel, Jean Claude / Hainaut, Ewa / Rammaert, Blandine / Roblot, France / Torregrosa-Diaz, José Miguel

Journal of clinical immunology

2021 Volume 42, Issue 1, Page(s) 60–63

Abstract: Chronic granulomatous disease (CGD) is an inherited immunodeficiency due to defective leukocyte NADPH responsible for recurrent infections and aberrant inflammation. Mutations in the CYBB gene are responsible for the X-linked CGD and account for ... ...

Abstract	Chronic granulomatous disease (CGD) is an inherited immunodeficiency due to defective leukocyte NADPH responsible for recurrent infections and aberrant inflammation. Mutations in the CYBB gene are responsible for the X-linked CGD and account for approximately 70% of the cases. CGD is diagnosed during childhood in males. Female carriers may have biased X-inactivation and may present with clinical manifestations depending on the level of residual NADPH oxidase activity. We report the case of a previously asymptomatic female carrier who was diagnosed at age 67 with a skin infection with the rare fungus Paecilomyces lilacinus as the first manifestation of CGD. Dihydrorhodamine 123 (DHR) activity was below 10%. Next-generation sequencing (NGS) revealed mutations in DNMT3A, ASXL1, and STAG2 suggesting that clonal hematopoiesis could be responsible for a progressive loss of NADPH oxidase activity and the late onset of X-linked CGD in this patient. Long-term follow-up of asymptomatic carrier women seems to be essential after 50 years old.
MeSH term(s)	Aged ; Granulomatous Disease, Chronic/diagnosis ; Granulomatous Disease, Chronic/genetics ; Humans ; Hypocreales ; Middle Aged ; NADPH Oxidases/genetics ; X Chromosome Inactivation
Chemical Substances	NADPH Oxidases (EC 1.6.3.-)
Language	English
Publishing date	2021-10-01
Publishing country	Netherlands
Document type	Case Reports ; Journal Article
ZDB-ID	779361-3
ISSN	1573-2592 ; 0271-9142
ISSN (online)	1573-2592
ISSN	0271-9142
DOI	10.1007/s10875-021-01140-1
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Zs.A 1640: Show issues

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Article ; Online: CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study.

The Lancet. Haematology

2023 Volume 10, Issue 7, Page(s) e521–e529

Abstract: Background: CPX-351, an encapsulated form of cytarabine and daunorubicin, has shown greater efficacy than the classic 3 + 7 treatment administration in secondary acute myeloid leukaemia. Given that higher-risk myelodysplastic syndrome and chronic ... ...

Abstract	Background: CPX-351, an encapsulated form of cytarabine and daunorubicin, has shown greater efficacy than the classic 3 + 7 treatment administration in secondary acute myeloid leukaemia. Given that higher-risk myelodysplastic syndrome and chronic myelomonocytic leukaemia share similarities with secondary acute myeloid leukaemia, we aimed to investigate the safety and efficacy of CPX-351 in this context. Methods: This investigator-initiated two-cohort phase 2 trial was conducted by the Groupe Francophone des Myélodysplasies, with 12 participating centres in France. It comprised cohort A (reported here and completed), which included patients in first-line treatment, and cohort B, which was stopped for lack of inclusion (ie, not enough patients met the inclusion criteria), for patients with hypomethylating agent failure that is not reported here. Cohort A enrolled patients with newly diagnosed higher-risk myelodysplastic syndrome or chronic myelomonocytic leukaemia (aged 18-70 years old) with an Eastern Cooperative Oncology Group performance status of 0-1. Intravenous CPX-351 (100 mg/m Findings: Between April 29, 2020, and Feb 10, 2021, 21 (68%) male and ten (32%) female patients were enrolled. 27 (87%) of 31 patients responded (95% CI 70-96). 16 (52%) of the 31 patients received at least one consolidation cycle. 30 (97%) of the 31 patients included were initially considered eligible for allogeneic HSCT and 29 (94%) of the 31 patients had the procedure. Median follow-up was 16·1 months (IQR 8·3-18·1). The most common grade 3-4 adverse events were pulmonary (eight [26%] of 31 patients) and cardiovascular (six [19%] of 31 patients). There were 14 serious adverse events (mainly hospitalisation for infection [n=5] and only one was treatment-related) and no treatment-related death. Interpretation: CPX-351 appears to be active and safe in patients with higher-risk myelodysplastic syndrome and chronic myelomonocytic leukaemia, allowing bridging to allogenic HSCT in most patients. Funding: Jazz Pharmaceuticals.
MeSH term(s)	Humans ; Male ; Female ; Adolescent ; Young Adult ; Adult ; Middle Aged ; Aged ; Leukemia, Myelomonocytic, Chronic/drug therapy ; Cytarabine ; Daunorubicin/therapeutic use ; Leukemia, Myeloid, Acute/drug therapy ; Myelodysplastic Syndromes/drug therapy ; Myelodysplastic Syndromes/etiology ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use
Chemical Substances	CPX-351 ; Cytarabine (04079A1RDZ) ; Daunorubicin (ZS7284E0ZP)
Language	English
Publishing date	2023-05-25
Publishing country	England
Document type	Multicenter Study ; Clinical Trial, Phase II ; Journal Article
ISSN	2352-3026
ISSN (online)	2352-3026
DOI	10.1016/S2352-3026(23)00090-X
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Article ; Online: Long-term molecular remission in a patient with acute myeloid leukemia harboring a new NUP98-LEDGF rearrangement.

Gallego Hernanz, Maria Pilar / Torregrosa Diaz, José Miguel / Sorel, Nathalie / Bobin, Arthur / Dindinaud, Elodie / Bouyer, Sabrina / Desmier, Deborah / Brizard, Françoise / Leleu, Xavier / Maillard, Natacha / Chomel, Jean-Claude

Cancer medicine

2019 Volume 8, Issue 4, Page(s) 1765–1770

Abstract: A large variety of molecular rearrangements of the NUP98 gene have been described in the past decades (n = 72), involving fusion partners coding for different transcription factors, chromatin modifying enzymes, as well as various cytosolic proteins. Here, ...

Abstract	A large variety of molecular rearrangements of the NUP98 gene have been described in the past decades (n = 72), involving fusion partners coding for different transcription factors, chromatin modifying enzymes, as well as various cytosolic proteins. Here, we report the case of an AML-M2 patient with a variant NUP98-LEDGF/PSIP1 gene fusion (N9-L10). In this patient, three different NUP98-LEDGF fusion mRNAs were characterized due to alternative splicing in LEDGF exon 11. Targeted high-throughput sequencing revealed the presence of IDH1, SRSF2, and WT1 additional pathogenic mutations. To improve the therapeutic monitoring, quantification of NUP98-LEDGF mRNA by real-time PCR was developed. Because of poor response to conventional chemotherapy, allogeneic stem cell transplantation was performed, followed by 20 cycles of azacitidine-based preemptive treatment of relapse. More than 31 months after diagnosis, corresponding to 25 months post SCT and 4 months after the last cycle of azacytidine, the patient is in complete molecular remission (undetectable NUP98-LEDGF mRNA transcripts). This study highlights the considerable variability in breakpoint location within both NUP98 and LEDGF, associated with alternative splicing affecting LEDGF. It also emphasizes the need to fully characterize the breakpoints within the two genes and the identification of all fusion mRNAs, particularly for the development of a molecular monitoring assay. All these data seem critical for the optimal management of NUP98-LEDGF + hematological malignancies commonly associated with a poor prognosis.
MeSH term(s)	Adaptor Proteins, Signal Transducing/genetics ; Antineoplastic Combined Chemotherapy Protocols/therapeutic use ; Follow-Up Studies ; Gene Fusion ; Gene Rearrangement ; Humans ; Karyotype ; Leukemia, Myeloid, Acute/drug therapy ; Leukemia, Myeloid, Acute/genetics ; Male ; Middle Aged ; Mutation ; Neoplasm Proteins/genetics ; Nuclear Pore Complex Proteins/genetics ; Remission Induction ; Transcription Factors/genetics
Chemical Substances	Adaptor Proteins, Signal Transducing ; Neoplasm Proteins ; Nuclear Pore Complex Proteins ; Nup98 protein, human ; PSIP1 protein, human ; Transcription Factors
Language	English
Publishing date	2019-03-07
Publishing country	United States
Document type	Case Reports ; Journal Article
ISSN	2045-7634
ISSN (online)	2045-7634
DOI	10.1002/cam4.2051
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Article ; Online: Efficacy and safety of mammalian target of rapamycin inhibitors in systemic mastocytosis: A nationwide French pilot study.

Moraly, Josquin / Rossignol, Julien / Rouzaud, Claire / Gabas, Thomas / Bouktit, Hassiba / Lhermitte, Ludovic / Canioni, Danielle / Fraitag, Sylvie / Bruneau, Julie / Barete, Stéphane / Suarez, Felipe / Ballul, Thomas / Meni, Cécile / Polivka, Laura / Terriou, Louis / Launay, David / Bouillet, Laurence / Gaudy-Marqueste, Caroline / Gousseff, Marie /

Le Mouel, Edwige / Neel, Antoine / Ranta, Dana / Jaussaud, Roland / Guilpain, Philippe / Frenzel, Laurent / Agopian, Julie / Dubreuil, Patrice / Greco, Céline / Dimicoli-Salazar, Sophie / Heiblig, Mael / Gourguechon, Clément / Tournilhac, Olivier / Javier, Rose-Marie / Castelain, Florence / Cabrera, Quentin / Gourin, Marie Pierre / Wierzbicka-Hainaut, Ewa / Torregrosa-Diaz, Jose Miguel / Bulai, Cristina / Lavigne, Christian / Hoarau, Cyrille / Arock, Michel / Damaj, Gandhi / Lortholary, Olivier / Hermine, Olivier

American journal of hematology

2024 Volume 99, Issue 6, Page(s) 1095–1102

Abstract: Systemic mastocytosis (SM) corresponds to a rare and heterogeneous spectrum of diseases characterized by the accumulation of atypical mast cells (MCs). Advanced mastocytosis (Adv-SM) is associated with poor survival; in contrast, patients with non- ... ...

Abstract	Systemic mastocytosis (SM) corresponds to a rare and heterogeneous spectrum of diseases characterized by the accumulation of atypical mast cells (MCs). Advanced mastocytosis (Adv-SM) is associated with poor survival; in contrast, patients with non-advanced SM (non-Adv-SM) usually have a normal life expectancy but may experience poor quality of life. Despite recent therapeutic progress including tyrosine kinase inhibitors, new treatment options are needed for refractory and/or intolerant patients with both severely symptomatic and Adv-SM. In vitro, the mTOR pathway is activated in MCs from patients bearing the KIT D816V mutation. Furthermore, rapamycin induces the apoptosis of KIT D816V MCs selectively. In this nationwide study, we report the outcomes of patients diagnosed with SM and treated with a mammalian target of rapamycin inhibitor (imTOR) within the French National Reference Center for mastocytosis (CEREMAST). All patients registered were relapsing, treatment-refractory, or ineligible for other cytoreductive therapy. Non-Adv-SM patients received imTOR as a monotherapy (rapamycin/everolimus), and Adv-SM patients received imTOR as a monotherapy or in combination with cytarabine. The objective response rate (ORR) in non-Adv-SM was 60% (partial response in 40% and major response in 20%), including reductions in skin involvement, mediator release symptoms, and serum tryptase. In the Adv-SM group, the ORR was 20% (including one major response and one partial response, both in patients with a KIT D816V mutation), which enabled a successful bridge to allogeneic stem cell transplantation in one patient. Our results suggest that imTOR treatment has potential benefits in patients with SM harboring a KIT D816V mutation.
MeSH term(s)	Humans ; Mastocytosis, Systemic/drug therapy ; Pilot Projects ; Female ; Male ; Middle Aged ; Adult ; France ; Aged ; Sirolimus/therapeutic use ; Sirolimus/adverse effects ; MTOR Inhibitors/therapeutic use ; Proto-Oncogene Proteins c-kit/genetics ; Proto-Oncogene Proteins c-kit/antagonists & inhibitors ; Everolimus/therapeutic use ; Everolimus/adverse effects ; Treatment Outcome ; TOR Serine-Threonine Kinases/antagonists & inhibitors ; Aged, 80 and over
Language	English
Publishing date	2024-04-06
Publishing country	United States
Document type	Journal Article
ZDB-ID	196767-8
ISSN	1096-8652 ; 0361-8609
ISSN (online)	1096-8652
ISSN	0361-8609
DOI	10.1002/ajh.27323
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Zs.A 1251: Show issues

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Article ; Online: Indications de l’autogreffe de cellules hématopoïétiques dans la Maladie de Crohn : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire.

Puyade, Mathieu / Treton, Xavier / Alric, Laurent / Badoglio, Manuela / Castilla Llorente, Cristina / Fotsing, Ginette / Marjanovic, Zora / Rovira, Montserrat / Terriou, Louis / Torregrosa Díaz Jose, Miguel / Tran-Minh, My-Linh / Allez, Matthieu / Pugnet, Grégory / Laharie, David / Yakoub-Agha, Ibrahim / Farge, Dominique

Bulletin du cancer

2020 Volume 107, Issue 12S, Page(s) S140–S150

Abstract: Crohn's Disease (CD) is an auto-inflammatory disease, which may involve the entire gastro-intestinal tract. CD is diagnosed on several clinical, biological, endoscopic and histological criteria. First line therapy is based on oral or iv steroids. In case ...

Title translation	Crohn's disease and autologous hemapoietic cell transplantation: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).
Abstract	Crohn's Disease (CD) is an auto-inflammatory disease, which may involve the entire gastro-intestinal tract. CD is diagnosed on several clinical, biological, endoscopic and histological criteria. First line therapy is based on oral or iv steroids. In case of steroids dependence or resistance, several types of immunosuppressive or immunomodulating therapies are available: classical antimetabolites (thiopurines or methotrexate) or monoclonal antibodies against TNFα, against interleukin 12/23 or against integrin. Nonetheless, Crohn's disease may remain active despite the use of several lines of therapy. In such cases, autologous hematopoietic cell transplantation (AHCT) is an effective therapeutic option in highly selected CD patients with specific criteria. The MATHEC-SFGM-TC Good Clinical Practice Guidelines (GCPG) were developed by a multidisciplinary group of experts including gastroenterologists, hematologists and members of the reference center for stem cell therapy in auto-immune diseases (MATHEC), including members of the French groupe d'étude thérapeutique des affections inflammatoires du tube digestif(GETAID) under the auspices of the French speaking Society of bone marrow transplantation and cellular therapy (SFGM-TC). The aim of the present guidelines is to define the eligibility criteria for CD patients when candidates to AHCT, the procedures for mobilization of hematopoietic stem cell (HSC), conditioning regimen and standardized follow-up after AHCT including monitoring of gastroenterological treatments during AHCT and thereafter throughout all follow-up.
MeSH term(s)	Contraindications, Procedure ; Crohn Disease/immunology ; Crohn Disease/therapy ; Hematopoietic Stem Cell Mobilization ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Immunosuppressive Agents/therapeutic use ; Mesenchymal Stem Cell Transplantation ; Patient Selection ; Societies, Medical ; Transplantation Conditioning ; Transplantation, Homologous
Chemical Substances	Immunosuppressive Agents
Language	French
Publishing date	2020-10-14
Publishing country	France
Document type	Journal Article ; Practice Guideline ; Review
ZDB-ID	213270-9
ISSN	1769-6917 ; 0007-4551
ISSN (online)	1769-6917
ISSN	0007-4551
DOI	10.1016/j.bulcan.2020.08.009
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Article ; Online: Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network.

Itzykson, Raphael / Santini, Valeria / Thepot, Sylvain / Ades, Lionel / Chaffaut, Cendrine / Giagounidis, Aristoteles / Morabito, Margot / Droin, Nathalie / Lübbert, Michael / Sapena, Rosa / Nimubona, Stanislas / Goasguen, Jean / Wattel, Eric / Zini, Gina / Torregrosa Diaz, Jose Miguel / Germing, Ulrich / Pelizzari, Anna Maria / Park, Sophie / Jaekel, Nadja /

Metzgeroth, Georgia / Onida, Francesco / Navarro, Robert / Patriarca, Andrea / Stamatoullas, Aspasia / Götze, Katharina / Puttrich, Martin / Mossuto, Sandra / Solary, Eric / Gloaguen, Silke / Chevret, Sylvie / Chermat, Fatiha / Platzbecker, Uwe / Fenaux, Pierre

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

2022 Volume 41, Issue 10, Page(s) 1888–1897

Abstract: Purpose: Hydroxyurea (HY) is a reference treatment of advanced myeloproliferative neoplasms. We conducted a randomized phase III trial comparing decitabine (DAC) and HY in advanced myeloproliferative chronic myelomonocytic leukemias (CMML).: Patients ... ...

Abstract	Purpose: Hydroxyurea (HY) is a reference treatment of advanced myeloproliferative neoplasms. We conducted a randomized phase III trial comparing decitabine (DAC) and HY in advanced myeloproliferative chronic myelomonocytic leukemias (CMML). Patients and methods: Newly diagnosed myeloproliferative CMML patients with advanced disease were randomly assigned 1:1 to intravenous DAC (20 mg/m Results: One-hundred seventy patients received DAC (n = 84) or HY (n = 86). Median age was 72 and 74 years, and median WBC count 32.5 × 10 Conclusion: Compared with HY, frontline treatment with DAC did not improve EFS in patients with advanced myeloproliferative CMML (ClinicalTrials.gov identifier: NCT02214407).
MeSH term(s)	Humans ; Aged ; Leukemia, Myelomonocytic, Chronic/drug therapy ; Leukemia, Myelomonocytic, Chronic/diagnosis ; Decitabine ; Hydroxyurea/adverse effects ; Leukemia, Myelomonocytic, Acute/drug therapy ; Proportional Hazards Models
Chemical Substances	Decitabine (776B62CQ27) ; Hydroxyurea (X6Q56QN5QC)
Language	English
Publishing date	2022-12-01
Publishing country	United States
Document type	Randomized Controlled Trial ; Clinical Trial, Phase III ; Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	604914-x
ISSN	1527-7755 ; 0732-183X
ISSN (online)	1527-7755
ISSN	0732-183X
DOI	10.1200/JCO.22.00437
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Zs.A 1847: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Indications de l’autogreffe de cellules souches hématopoïétiques dans la polyradiculonévrite inflammatoire démyélinisante chronique : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC).

Puyade, Mathieu / Labeyrie, Céline / Badoglio, Manuela / Cintas, Pascal / Guenounou, Sarah / Lansiaux, Pauline / Marjanovic, Zora / Nicolas, Guillaume / Pomies, Amélie / Terriou, Louis / Torregrosa Diaz, Jose-Miguel / Baron, Clément / Castilla Llorente, Cristina / Yakoub-Agha, Ibrahim / Farge, Dominique

Bulletin du cancer

2019 Volume 107, Issue 1S, Page(s) S104–S113

Abstract: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is a chronic autoimmune disease involving the peripheral nervous system, characterized by focal and segmental demyelination accounting for neurological deficit. CIDP diagnosis is based on several ... ...

Title translation	Indication of autologous stem cell transplantation in chronic inflammatory demyelinating polyneuropathy: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).
Abstract	Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is a chronic autoimmune disease involving the peripheral nervous system, characterized by focal and segmental demyelination accounting for neurological deficit. CIDP diagnosis is based on several criteria and requires the presence of specific clinical symptoms and of demyelinating criteria on the electroneuromyogram (ENMG) or of additional supportive criteria (spinal fluid examination with dissociation between albumin level and cellular abnormalities, nervous abnormalities on MRI or other minor abnormalities on ENMG, demyelinating features on nerve biopsy or patient improvement under so-called first-line therapy with immunodulator treatment). After failure of two successive first line immunomodulating drug therapies (corticosteroids, immunomodulating immunoglobulins, or plasma exchange), several options can be considered as second line therapies. The efficacy of autologous hematopoietic cell transplantation (AHCT) has been shown in CIDP patients. The aim of these recommendations established by a working group of experts from the "Société française de greffe de moelle osseuse et thérapie cellulaire (SFGM-TC)", the group "maladies auto-immunes et thérapie cellulaire (MATHEC)" and the "filière de santé maladies rares neuromusculaire (FILNEMUS)" is to specify the eligibility criteria for AHCT in CIPD patients, to describe the mobilization and the conditioning regimen for the AHCT procedure, as well as the patient standardized post-transplant follow-up and the management of neurological treatment throughout the all procedure.
MeSH term(s)	Hematopoietic Stem Cell Mobilization/standards ; Hematopoietic Stem Cell Transplantation/methods ; Hematopoietic Stem Cell Transplantation/standards ; Humans ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/diagnosis ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/immunology ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/therapy ; Transplantation Conditioning ; Transplantation, Autologous
Language	French
Publishing date	2019-12-07
Publishing country	France
Document type	Journal Article ; Practice Guideline ; Review
ZDB-ID	213270-9
ISSN	1769-6917 ; 0007-4551
ISSN (online)	1769-6917
ISSN	0007-4551
DOI	10.1016/j.bulcan.2019.11.002
Database	MEDical Literature Analysis and Retrieval System OnLINE

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