1. [A rare placental lesion: Intraparenchymal hepatic heterotopia].
Intra-placental hepatic heterotopia is a rare lesion of embryonic development, poorly understood and often discovered incidentally during histopathological analyses. The morphological aspects of this lesion, as classically described, are relatively consistent in the literature, and several etiopathogenic hypotheses have been proposed, though none is entirely satisfactory. We report the case of a placenta presenting multiple foci of hepatic heterotopia identified within the fetal vascular network. The observation concerns a patient whose pregnancy was medically terminated at 20 weeks of amenorrhea due to a fetal polymalformative syndrome, for which a post-mortem diagnosis of Noonan syndrome was established. The pathological examination revealed three placental parenchymal lesions corresponding to heterotopic hepatic tissue. To date, no risk factors have been reported in the literature, nor have any known consequences on the child's development been identified. However, Noonan syndrome is recognized as a risk factor for the development of pediatric hepatic adenomatous lesions. The association of these two lesions may be coincidental or may support the etiopathogenic hypothesis of secondary migration of a hepatocellular adenoma within the fetal vascular network. The etiopathogenic mechanisms remain debated: an aberrant migration of hepatic tissue fragments through intra-vascular embolism, the entrapment of hepatocytes by the yolk sac, or even a co-evolution of the placenta and liver from stem cells. The absence of tumor characteristics and the histological similarity with embryonic hepatic tissue support the hypothesis of hepatic cell migration within the placenta.
2. [Intestinal fibrosis in Crohn's disease : towards new therapeutic options?].
作者: Sophie Vieujean.;Émeline Bequet.;Marty Stepniak.;Marie-Alice Meuwis.;Édouard Louis.
来源: Rev Med Liege. 2025年80卷3期154-161页
Crohn's disease is a chronic inflammatory bowel disease that can lead to fibrostenotic complications. These strictures result from an imbalance between inflammation and excessive healing, leading to an abnormal accumulation of extracellular matrix and a progressive thickening of the intestinal wall. To date, no specific treatment is available to prevent or reverse intestinal fibrosis. The management of strictures primarily focuses on controlling inflammation and addressing obstructive complications through endoscopic balloon dilation, stricturoplasty, or intestinal resection. Current medical therapies, such as anti-TNF agents, can help reduce inflammation but have no direct impact on fibrosis. New therapeutic strategies are emerging, including TL1A inhibitors (duvakitug, tulisokibart), an ALK5 inhibitor (AGMB-129), and targeted AGR2 therapies (TH-009). Additionally, mesenchymal stem cells are being investigated in our hospital center in combination with endoscopic balloon dilation, aiming to assess their therapeutic potential. The development of effective anti-fibrotic therapies remains a critical medical need to improve the management of intestinal strictures and reduce the need for invasive procedures in Crohn's disease.
3. [In vitro preclinical models reproducing the respiratory epithelium: Application to the study of SARS-CoV-2 virus infection].
Highlighted by the COVID-19 pandemic, the study of respiratory infections is a global health priority. To this end, many preclinical in vitro study models have been developed to reproduce nasal, bronchial or alveolar respiratory epithelium. These models can be established from immortalised cell lines, primary culture or induced pluripotent stem cells (iPSC). They can also be constructed in various three-dimensional structures that are more or less physiological and easy to use. This synthetic review puts into perspective the advantages and limitations of these models, while highlighting their relevance for the study of the mechanisms of SARS-CoV-2 infection.
4. [A CRISPR/Cas approach to β-haemoglobinopathies].
Beta-haemoglobinopathies are severe genetic anemias caused by mutations that affect adult haemoglobin production. Many therapeutic approaches aim to reactivate the expression of the fetal hemoglobin genes. To this end, the CRISPR/Cas9 system has recently been used to genetically modify patients' hematopoietic stem/progenitor cells ex vivo and reactivate fetal hemoglobin expression in their erythroid progeny. More than 70 patients with severe β-thalassemia and sickle cell disease have been treated with the Casgevy® therapy. Most have achieved a significant improvement of clinical phenotype, with high editing efficiency in hematopoietic cells associated with normal or near normal hemoglobin levels. While the long-term safety and efficacy of this powerful approach still need to be evaluated, new strategies are being developed to further improve therapeutic outcomes, reduce potential genotoxicity and lower the costs of therapy.
5. [Gene coexpression networks: concepts and applications].
The advent of high-throughput omics data and the generation of new algorithms provide the biologists with the opportunity to explore living processes in the context of systems biology aiming at revealing the gene interactions, the networks underlying complex cellular functions. In this article, we discuss two methods for gene network reconstruction, WGCNA (Weighted Gene Correlation Network Analysis) developed by Steve Horvath and collaborators in 2008, and MIIC (Multivariate Information-based Inductive Causation) developed by Hervé Isambert and his team in 2017 and 2024. These two methods are complementary, WGCNA generating undirected networks in which most gene-to-gene interactions are indirect, while MIIC reveals direct interactions and some causal links. We illustrate these aspects according to our own work aiming at identifying the gene interactions underlying the hematopoietic stem cell supportive activity of mesenchymal stromal cells at an early developmental stage.
6. [ISO 9001 certification: Experience of the immunology and cell therapy laboratory at Ibn Sina University Hospital, Rabat].
Medical analysis laboratories play an essential role in medical diagnosis, with their results influencing up to 70% of decisions. This means that the quality of laboratory services is a key factor in the quality of medical care. However, certification and accreditation are not yet compulsory in Morocco, and only the Guide to the Good Execution of Medical Biology Analyses (GBEA) published in 2011 governs the organisation of laboratories. The Blood Transfusion and Hemovigilance Service (STSH) at the Ibn Sina University Hospital (CHUIS) in Rabat has embarked on an ISO 9001:2015 certification process, with the aim of improving its performance and satisfying its interested parties. This process, which was launched in 2018, was carried out in two main phases. The first phase consisted of complying with current regulations, while the second phase involved an evaluation based on the ISO 9001:2015 standard. In accordance with the logic of the Deming wheel, the department developed its quality policy, set its objectives, and undertook staff training and awareness activities. The processes, along with the associated risks and opportunities, have been identified and represented. The documentation system, as well as the system for reporting and handling non-conformities, has been implemented and dematerialised. Performance and activity indicators have been defined for each process. Finally, the customer feedback system has been expanded to include all interested parties, thus allowing for an evaluation of their perceptions and the identification of areas for improvement. Despite the global pandemic of COVID-19, this work has successfully integrated ISO 9001:2015 into STSH's practices, with compliance being declared by a certification body in 2022.
7. [Medical simulation training for bone marrow harvesting from a healthy donor for an allogeneic hematopoietic cell transplant (SFGM-TC)].
作者: Tamim Alsuliman.;Leonardo Magro.;Marie Titecat.;Marie Robin.;Tarek Kanouni.;Edouard Forcade.;Charlotte Jubert.;Ibrahim Yakoub-Agha.;Catherine Faucher.
来源: Bull Cancer. 2024年
This workshop presents the recommendations of the Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC) for simulation-based training on bone marrow harvesting from healthy donors. Due to the decline in bone marrow harvests in favor of peripheral stem cells, a loss of expertise has been observed among younger hematologists. The training consists of an online theoretical component and a hands-on workshop using a mannequin at the PRESAGE simulation center at the Lille University School of Medicine. The goal is to acquire the necessary skills to safely perform bone marrow harvesting according to best practices, avoiding technical errors. Both theoretical and practical evaluations are conducted, and participants receive a training certificate upon completion.
8. [Muscle stem cells and metabolism in Duchenne muscular dystrophy, focus on AMPK].
作者: Audrey Saugues.;Anita Kneppers.;Rémi Mounier.
来源: Med Sci (Paris). 2024年40 Hors série n° 1卷60-63页
Through their myogenic activity, adult muscle stem cells (MuSCs) are crucial for the regeneration of striated skeletal muscle. Once activated, they proliferate, differentiate and then fuse to repair or form new muscle fibers (myofibers). Their progression through myogenesis requires a complex regulation involving multiple players such as metabolism, in particular via AMPK. This protein kinase regulates the self-renewal and myonuclear accretion of MuSCs after acute skeletal muscle injury or skeletal muscle contraction. However, in a context of dystrophy such as Duchenne muscular dystrophy (DMD), the regenerative capacity of MuSCs is reduced, presumably due to an increase of the proliferation that is detrimental to differentiation. We are interested here in the potential of metabolism to regulate the myogenic activity of MuSCs in DMD via AMPK.
9. [Role of the transcription factor PAX3 during myogenesis: from the embryo to the adult stage].
作者: Virginia Zoglio.;Joana Esteves de Lima.;Frédéric Relaix.
来源: Med Sci (Paris). 2024年40 Hors série n° 1卷56-59页
PAX3 plays a crucial role in embryonic myogenesis, controlling the specification, migration, proliferation, and differentiation of muscle progenitor cells to ensure normal skeletal muscle development in the embryo. However, PAX3 potential role in a context of muscle homeostasis and regeneration remains poorly investigated. The adult muscle stem cells, known as satellite cells (SCs) exhibit heterogeneity in Pax3 expression in various muscles throughout the body and display a bimodal response to environmental stress exposure. To explore the role of PAX3 in the context of tissue damage, we performed regeneration studies, which unveiled a functional heterogeneity of the SCs populations depending on Pax3 expression. Together, this project aims to decipher cell-type specific dysregulations linked to tissue damage and identify PAX3 downstream gene regulatory networks that can lead to specific SC behavior, thus potentially providing novel strategies for muscle disease preventive therapies.
10. [The contribution of cerebral organoids to the understanding and treatment of rare genetic diseases with neurodevelopmental disorders].
作者: Fatima El It.;Laurence Faivre.;Christel Thauvin-Robinet.;Antonio Vitobello.;Laurence Duplomb.
来源: Med Sci (Paris). 2024年40卷8-9期643-652页
Rare genetic diseases with neurodevelopmental disorders (NDDs) encompass several heterogeneous conditions (autism spectrum disorder (ASD), intellectual disability (ID), attention deficit hyperactivity disorder (ADHD), specific learning disorder (SLD), among others). Currently, few treatments are available for these patients. The difficulty in accessing human brain samples and the discrepancies between human and animal models highlight the need for new research approaches. One promising approach is the use of the cerebral organoids. These 3D, self-organized structures, generated from induced pluripotent stem cells (iPSCs), enable the reproduction of the stages of human brain development, from the proliferation of neural stem cells to their differentiation into neurons, oligodentrocytes, and astrocytes. Cerebral organoids hold great promise in understanding brain development and in the search for treatments.
12. High-Frequency Hearing Loss Amongst Smart Mobile Phone Users: A Case-Control Study.
作者: Indira Jha.;Md Kabir Alam.;Chandan Kumar.;Niska Sinha.;Tarun Kumar.
来源: Ann Afr Med. 2024年23卷4期684-687页
In past 20 years, there is increase in mobile phone users from 12.4 million to about 5.6 billion i.e 70 % of the world's population.[1] Electromagnetic radiations emitted from mobile phone damages inner ear, cochlea and outer hair cells of inner ear and auditory pathway (AP).[2].
13. New Insight in Using of Mesenchyme Stem Cell Conditioning Medium for the Impaired Muscle related Biomarkers: In vivo Study with Rat Model.
作者: Ronald Winardi Kartika.;Veronika Maria Sidharta.;Tena Djuartina.;Cynthia Retna Sartika.;Kris Herawan Timotius.
来源: Ann Afr Med. 2024年23卷4期674-679页
This study aimed to investigate the effects of Umbilical Cord Mesencymal Stem Cell Conditioning Medium (UC MSC-CM) administration on body weight recovery and the level of four molecular biomarkers, namely Superoxide Dismutase (SOD), vascular Endothelial Growth Factor (VEGF), C-Reactive Protein (CRP), and myostatin.
14. [Clinical and transversal competencies of advanced practice nurses (APNs) within a cellular therapy team (SFGM-TC)].
作者: Thomas Jezequel.;Rezarta Ajazi Hub.;Caroline Bompoint.;Céline Chauvel.;Elodie Colonnese.;Coralie Corbineau.;Claire Desmedt.;Céline Kicki.;Sandra Lomazzi.;Anne Sirvent.;Solène Evard.;Karine Roux.;Léonardo Magro.;Fabienne Colledani.
来源: Bull Cancer. 2025年112卷1S期S92-S102页
The advanced practice nurse (APN) has been introduced in France, following the 2016 health law and implementing decrees published in 2018. In this context, the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) has already issued guidelines regarding the allocation of APNs' new clinical competences and their collaboration with physicians. It is now providing new recommendations on the transversal activities that can be fulfilled by APNs, such as research, leadership, training and teaching. Additionally, the guidelines outline how APNs can cooperate with other professionals in departments of haematology and cellular therapy, including nurses, coordinators and health managers.
15. [Prerequisite and organisation of health-care pathways for Cell and Gene therapies, using Mesenchymal Stromal Cells (MSC) or Chimeric Antigen Receptor (CAR) T cells, in patients with autoimmune systemic diseases].
作者: Christina Castilla-Llorente.;Agnès Bonnin.;Pauline Lansiaux.;Jean-Jacques Tudesq.;Clément Beuvon.;Jean-Roch Fabreguettes.;Yves-Marie Pers.;Grégory Pugnet.;Alexandre Thibault Jacques Maria.;Mathieu Puyade.;Fanny Urbain.;Louis Terriou.;Vincent Poindron.;Marie Jachiet.;Carlotta Cacciatore.;Alain Lescoat.;Pedro Henrique Prata.;Ingrid Munia.;Isabelle Madelaine.;Catherine Thieblemont.;Karin Tarte.;Ibrahim Yakoub-Agha.;Leonardo Magro.;Dominique Farge.;Zora Marjanovic.
来源: Bull Cancer. 2025年112卷1S期S36-S53页
First-line treatments of autoimmune systemic diseases (ARD) are based on the use of various types of immunosuppressive or immunomodulatory drugs, either alone or in association, according to standardized reference protocols. Prolonged use of these drugs in severe or refractory ARD is associated with high morbidity and increased mortality. Innovative cell therapies represent a new promising approach for patients with ARDs, with the recent clinical use of: a) mesenchymal stromal cells (MSCs), based on their immunomodulatory, antifibrotic and pro-angiogenic properties and b) Chimeric Antigen Receptors (CAR) T cell therapies T lymphocytes, where genetically modified expression of a chimeric antigen receptor (CAR-T cells). Therapeutic use of MSC or CAR-T cells, remains indications of exception in patients with severe ARDs resistant to prior standard therapies with new prerequisite and organisation of health-care pathways as compared to traditional drugs, not only for the Cell and Gene Therapy (CGT) product definition and delivery process, but also for the patient clinical management before and after administration of the CGT product. The aim of this workshop under the auspices of the French Speaking Society of Bone Marrow and Cell transplantation (SFGM-TC) working group on autoimmune diseases (MATHEC) is to describe: a) the prerequisite for French hospitals to set-up the specific health-care pathways for MSC or CART therapy in ARDs patients, in accordance with regulatory and safety needs to perform academic or industry sponsored clinical trials, and b) the care-pathway for ARD patients treated with CGT, highlighting the importance of working in tandem between the ARD and the CAR-T cell specialist all along the indication, procedures and follow-up of ARDs. Patient safety considerations are central to guidance on patient selection to be validated collectively at the multidisciplinary team meeting (MDTM) based on recent (less than 3 months) thorough patient evaluation. MSC and CAR-T procedural aspects and follow-up are then carried out within appropriately experienced and SFGM-TC accredited centres in close collaboration with the ADs specialist.
16. [Recto-colic graft-versus-host disease (GVH). Diagnostic and prognostic criteria in a cohort of patients from Amiens university hospital].
作者: Benjamin Ducloux-Lebon.;Delphine Lebon.;Jean-René Tesson.;Mathurin Fumery.;Jean-Pierre Marolleau.;Denis Chatelain.
来源: Ann Pathol. 2025年45卷3期205-213页
Recto-colic graft-versus-host disease (GVHD) is a frequent and serious complication of hematopoietic stem cell allogeneic transplantation, which is sometimes difficult to diagnose. The aim of our study was to identify histological diagnostic and prognostic criteria for recto-colic GVH.
17. [Acquired severe aplastic anemia in emerging countries: Management from allogeneic hematopoietic cell transplantation indication until post-transplant follow-up SFGM-TC].
作者: Nabil Yafour.;Mohamed Amine Bekadja.;Ibtissam El Bejjaj.;Jean El-Cheikh.;Maria El Kababri.;Léonardo Magro.;Fati Hamzy.
来源: Bull Cancer. 2025年112卷1S期S10-S23页
Management of acquired aplastic anemia (AA) in emerging countries depends on the means of prognostic stratification, treatment and logistics available. During the 13th annual harmonization workshop of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines for allogeneic hematopoietic cell transplantation (Allo-HCT) in this disease. In terms of practice, the conclusions are as follows; The use of anti-tymocyte globuline (ATG) is mainly from rabbit and very little from horse. Access to bone marrow graft, total body irradiation, and the international unrelated donor registries is limited, which justifies the use of peripheral blood stem cells, chemotherapy-based conditioning, and related alternative donor. The workshop recommends matched sibling allo-HCT in all patients aged less than 40 years with acquired severe or very severe AA. For patients aged over than 40 years, or who lack an HLA-identical donor, treatment with the combination of cyclosporin, horse ATG, eltrombopag or cyclosporine, eltrombopag is recommended. If horse ATG and eltrombopag are not available, matched sibling allo-HCT may be indicated as first-line therapy in patients aged between 40-60 years, and good performance status. Although, in patients who have failed immunosuppressive treatments and thrombopoietin agonists, and in the absence of HLA-matched donor, a haplo-identical allo-HCT with modified Baltimore conditioning is recommended.
19. [Pre-, per- and post-allogeneic haematopoietic stem cell transplant rehabilitation (SFGM-TC)].
作者: Virgile Pinelli.;Laure Christophe.;Nathalie Cheron.;Sarah Morin.;Lila Gilis.;Candy Heuze.;Dominique Clerc-Renaud.;Laurence Morotti.;Benoit Vilhet.;Sandra Bissardon.;Leonardo Magro.
来源: Bull Cancer. 2025年112卷1S期S2-S9页
Allogeneic transplantation of haematopoietic stem cells is still the only curative treatment for certain haematological malignancies. This treatment can be responsible for a number of side-effects, leading to multiple and interdependent physical and psychological deficiencies that affect patients' quality of life and social participation, and can be experienced as a handicap, sometimes for several years after the transplant. For several years now, the integration of post-transplant rehabilitation pathways has been becoming more widespread, and initiatives to provide multidisciplinary care at an increasingly early stage are being studied. The aim of this early management is to improve the patient's overall functional state before, during and after the transplant, in order to limit the impact of the treatment and ensure the quickest possible return to a life that is as satisfying as possible. The international literature and the experiments carried out throughout the French-speaking world describe heterogeneous practices. Based on this literature and experience, the aim of this study is to issue homogenous recommendations for good clinical practice and to identify areas for further research into pre-transplant, per-transplant and post-transplant rehabilitation of haematopoietic stem cells.
20. [Preventive and therapeutic strategies for relapse after hematopoietic stem cell transplant for pediatric AML (SFGM-TC)].
作者: Cécile Renard.;Alizee Corbel.;Catherine Paillard.;Cécile Pochon.;Pascale Schneider.;Nicolas Simon.;Nimrod Buchbinder.;Mony Fahd.;Ibrahim Yakoub-Agha.;Charlotte Calvo.
来源: Bull Cancer. 2025年112卷1S期S135-S145页
Treatment of pediatric high-risk acute myeloid leukemia (AML), defined either on molecular or cytogenetic features, relies on bone marrow transplant after cytologic remission. However, relapse remains the first post-transplant cause of mortality. In this 13th session of practice harmonization of the francophone society of bone marrow transplantation and cellular therapy (SFGM-TC), our group worked on recommendations regarding the management of post-transplant relapse in AML pediatric patients based on international literature, national survey and expert opinion. Overall, immunomodulation strategy relying on both measurable residual disease (MRD) and chimerism evaluation should be used for high-risk AML. In very high-risk (VHR) AML with a 5-year overall survival ≤30 %, a post-transplant maintenance should be proposed using either hypomethylating agents, combined with DLI whenever possible, or FLT3 tyrosine kinase inhibitors if this target is present on leukemia cells. In the pre-emptive or early relapse settings (< 6 months post-transplant), treatments combining DLI, Azacytidine and Venetoclax should be considered. Access to phase I/II trails for targeted therapies (menin, IDH or JAK inhibitors) should be discussed in each patient according to the underlying molecular abnormalities of the disease.
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