Myelodysplastic syndromes, what news?

Marie-Christiane Vekemans Published in the journal : July 2024 Category : Hématologie et Oncologie médicale

Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by ineffective clonal hematopoiesis responsible for cytopenia and an increased risk of transformation into acute myeloid leukemia. Although they mainly affect people over 60, their prognosis depends essentially on their cytogenetic and molecular characteristics. Two-thirds of patients are diagnosed with low-risk disease. In these patients, the aim of treatment is to improve cytopenias. New treatments are now available to improve cytopenias, particularly anemia, and several promising trials targeting the clone or inflammation are underway.

In high-risk patients, although hematopoietic stem cell transplantation is still the only curative option, hypomethylating agents remain the standard of treatment, but are unfortunately not curative. We are still waiting for the first therapeutic breakthrough that will change the outcome of these patients. Three Phase 3 trials, currently in the recruitment phase, offer such hope, combining a hypomethylating agent with venetoclax, sabatolimab or tamibarotene.

 

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Myeloproliferative neoplasia: from clinical practice... to future curative therapies?

Violaine Havelange (1,2), Stefan N. Constantinescu (1-5) Published in the journal : July 2024 Category : Hématologie et Oncologie médicale

Classic BCR::ABL1 negative myeloproliferative neoplasms (MPN) include polycythemia vera, essential thrombocythemia, and myelofibrosis. These diseases arise from the clonal proliferation of a single hematopoietic stem cell which has acquired a driver mutation. They are considered chronic diseases, manifesting by symptoms listed in the MPN-10 score, thrombosis or, more rarely, hemorrhage. These diseases can progress to myelofibrosis or blast phase, with a guarded prognosis. Diagnosis requires bone marrow biopsy. First-line treatments consist of low-dose aspirin, phlebotomies in polycythemia vera, anticoagulation, and cytoreduction using hydroxyurea in certain situations. Treatments for myelofibrosis include allogeneic stem cell transplantation for curative purposes or JAK2 inhibitors to reduce both symptoms and spleen size. Recent research advances in a better understanding of the mutated protein have led to the development of new treatments aimed at eradicating the mutated cells, including human monoclonal antibodies and bispecific antibodies. These promising targeted treatments are currently undergoing clinical trials.

 

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Hospital on-call duty: Solidarity often pays off!

Olivier S Descamps, Marie Boland, Alexandre Niset, Annaelle Doyen, Simon De Vetter, Nora El Khawand, Julie Harmant, Inès Thiebaut, Sarah Will, Rayane Laghmiche, Hugo Pierret, Victor Simon Published in the journal : April 2024 Category : Économie de la santé

This article has attempted to address a contentious issue regarding on-call duty allocation in hospitals. Let us consider a hospital scenario with 20 clinical specialist assistant candidates (MACCS), consisting of 10 juniors in their core year of general internal medicine and 10 seniors specializing in areas like cardiology, gastroenterology, pneumology, etc. These MACCS are distributed across various departments, each comprising 1 to 5 MACCS, with varying proportions of juniors and seniors. Every night or weekend, one of these doctors had to be on call to provide permanent hospital patient care that was followed by a recuperation day. A discussion is currently underway as to whether senior MACCS should participate as much as junior MACCS in on-call duty. These senior MACCS receive 12% higher remuneration than younger staff, while they assist doctors in carrying out technical examinations during the day as part of their training. Will it cost more to the institution if the 10 senior staff members take part in general on-call duty? How will their absence during recuperation affect their work and training? Would departments with more senior staff face disadvantages? How would you address these questions? Some might be rather pessimistic, but how does it actually look reality?

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Specific modifications of the soluble tau protein distinguish Alzheimer’s disease from other tauopathies

Nathalie Kyalu Ngoie Zola, Clémence Balty, Emilien Boyer, Adrian Ivanoiu, Didier Vertommen, Bernard Hanseeuw, Marc Gobert Published in the journal : April 2024 Category : Neurology

Neurodegenerative diseases are progressive, acquired brain disorders affecting a growing number of people as populations age. Clinically, these conditions can be distinguished by different symptoms, depending on the brain region affected. Biologically, these disorders are characterized by the aggregation of certain cerebral proteins, thus being called proteino- pathies. In Alzheimer’s disease, amyloid proteins aggregate without causing symptoms, though this promotes symptomatic tau protein aggregation that takes place in the mesiotemporal lobe, responsible for encoding new memory information. More rarely, in atypical Alzheimer’s disease, tauopathy can occur in other brain regions, inducing diverse symptoms. These atypical Alzheimer’s disease cannot be diagnosed clinically without biological confirmation.

Tau protein aggregation is a hallmark shared by other neurodegenerative diseases, collectively called tauopathies. Typically, tauopathy initially occurs in brain regions distinct from the mesiotemporal lobe, which occasionally resembles Alzheimer’s disease. Due to imperfect concordance between the pathology type and affected brain regions, the development of biological tools (=biomarkers) is most critical for clinical research in neurodegenerative diseases. Distinct etiological treatments may be required to cure these diseases, given that different diseases exhibit varied tau modifications leading to different aggregates upon histological analysis. Though Alzheimer’s disease can now be diagnosed in vivo based on cerebrospinal fluid analysis, this is not yet the case for other tauopathies.

Cerebral histological abnormalities have enabled the classification of tauopathies based on the observation of abnormal phosphorylated 3R or 4R tau protein aggregates (3R vs. 4R denomination corresponding to the type of tau protein isoform). In Alzheimer’s disease, all isoforms do aggregate, whereas in other tauopathies only one isoform type does. Nevertheless, the distinction of tauopathies based on the sole measurement of tau isoforms in cerebrospinal fluid remains elusive.

The article focuses on a biochemical study of the tau protein and its post-translational modifications. It paves the way for confirming the diagnosis of non-Alzheimer’s tauopathies during the patient’s lifetime, thereby establishing a biological diagnosis. This advancement provides a better clinical understanding of these diseases, their evolution, and prognosis, both for patients and their families. In terms of research, this approach should enable the inclusion of patients into therapeutic trials at an early disease stage. This breakthrough could also provide insights into the exact role of the tau protein, linking it with genetic advancements in these diseases, for diagnosis at a pre-clinical stage. Through this study, tauopathies are entering a new era, transitioning from a post-mortem clinical-histological classification to an in vivo clinical-biological classification.

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Obesity: multi and interdisciplinarity

Pauline Gérard, Audrey Goffaux Published in the journal : April 2024 Category : RESUMES asbl (Réseau Multidisciplinaire d’Échange Scientifique)

This article summarizes the most relevant messages shared at the annual symposium of RESUMES asbl (Réseau Multidisciplinaire d'Échange Scientifique) on obesity. This theme was addressed through 10 disciplines, including pediatrics, neurology, internal medicine, dietetics, etc. In Belgium, 15.9% of adults are obese, which means that every healthcare professional is regularly confronted with the consequences of obesity. This article attempts to review obesity in its multiple dimensions and in a practical way.

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Drug Shortage Management in Diabetology

Régis P Radermecker Published in the journal : April 2024 Category : 18e Congrès UCL d’Endocrino-Diabétologie - Mars 2024

Drug shortages are becoming increasingly common. Diabetology is also highly affected. The reasons for these shortages are numerous and complex, and can occur at every stage of the drug supply chain. In addition to the extra costs, these shortages cause stress for patients and could lead to errors with harmful consequences.

Several ideas are currently being explored.

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New obesity drugs: a paradigm shift?

Jean-Paul Thissen Published in the journal : April 2024 Category : 18e Congrès UCL d’Endocrino-Diabétologie - Mars 2024

GLP-1 receptor agonists (GLP-1RAs) are the most extensively studied drug class for obesity treatment. These molecules induce significant weight loss and an improvement regarding comorbidities associated with excess weight. Weight loss results almost exclusively from caloric intake reduction. GLP-1RAs are well tolerated and have a satisfactory safety profile. Molecules currently under development aim to enhance weight loss, notably by targeting other receptors, and to improve patient compliance. Their use raises many questions (cost and possible reimbursement, duration of use, very long-term safety, place in relation to dietary management and bariatric surgery, etc.). Even though all these questions must be addressed, a paradigm shift is taking shape in the management of obesity, after decades of failure.

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Treatment guidelines for Type 2 diabetes

Michel P. Hermans Published in the journal : April 2024 Category : 18e Congrès UCL d’Endocrino-Diabétologie - Mars 2024

The new joint guidelines from the ADA-EASD and the EAS put emphasis on personalizing the choice of the preferential use of molecules from the GLP1-RA and SGLT2-i classes. They have demonstrated cardio-renal benefits beyond their hypoglycemic and weight-lowering effects in Type 2 diabetes (T2DM) patients with proven cardiovascular (CV) damage or high cardio-renal risk. For insulin treated T2DM patients who have benefited from continuous interstitial glucose measurement, reimbursed since 2023, well-defined glucometric targets have been proposed in addition to the usual HbA1c measurement. In addition, the 10-year CV risk can be assessed in T2DM patients undergoing primary cardio-renal prevention, using the recently introduced European SCORE2-Diabetes calculator.

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Hepatic steatosis: what the endocrinologist must know

Nicolas Lanthier Published in the journal : April 2024 Category : 18e Congrès UCL d’Endocrino-Diabétologie - Mars 2024

Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common condition in patients with metabolic syndrome and, a fortiori, type 2 diabetes. Simple tools are available to define disease severity, which should be assessed in routine practice.

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Transplantation, chronic inflammatory bowel disease, and hepatic steatosis: highlights in 2023

Géraldine Dahlqvist, Olivier Dewit, Nicolas Lanthier, Peter Stärkel Published in the journal : February 2024 Category : Hépato-gastroentérologie

The year 2023 marked numerous developments and advances in the field of hepato-gastroenterology. The deleterious impact of frailty and sarcopenia on the prognosis of patients with cirrhosis, listed for transplantation, is clear. A care pathway that covers pre-transplant preparation and rehabilitation for up to two years after liver transplantation has been developed. It includes a full nutritional and functional assessment of patients and offers enhanced dietetic, physiotherapeutic, and psychosocial care. For inflammatory bowel diseases, two new drugs have emerged: risankizumab for Crohn's disease and upadacitinib for ulcerative colitis. Their benefits and significance relative to existing treatments are presented. Finally, a standardized nomenclature for steatotic liver disease (SLD) has been published and accepted by the scientific communities. The two main SLDs are alcohol-related liver disease (ALD) and metabolic dysfunction-associated steatotic liver disease (MASLD). Patients suffering from both diseases are classified as MetALD. This article reviews the diagnostic criteria for these common conditions.

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