Cystic fibrosis: The era of CFTR modulators

Olivier Lebecque (1), Teresinha Leal (2), Patrick Lebecque (3) Published in the journal : February 2019 Category : Pneumology

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules aimed at improving CFTR function by specifically targeting the different classes of CFTR mutations. Recent Phase II studies of triple therapy, including new generation correctors, have demonstrated spectacular improvements in forced expiratory volume in 1s (FEV1), likely to translate into improved quality of life and increased life expectancy. Within the next 5 years, a highly effective CFTR modulator therapy will probably be approved for most cystic fibrosis patients, including those carrying at least one copy of the F508del mutation (88% of Belgian patients). Patients with well-preserved lungs will benefit most from these treatments.

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Improved patients’ prognosis by using innovative treatments at earlier stages

Jean-Francois Baurain, Jean-Pascal Machiels, François Duhoux Published in the journal : February 2019 Category : Oncologie

There has been no revolution in the oncology field in 2018. We have, however, been able to further confirm the major benefits of immunotherapy for patients with metastatic cancers. Anti-PD1 antibodies have now become the standard of treatment for head and neck cancers, bladder cancers, melanoma, lung carcinomas, kidney cancers, and cutaneous spinocellular carcinomas. Based on mature survival data, we’ll likely be able to cure numerous metastatic cancer patients. Amongst targeted therapies, PARP inhibitors will possibly be more effective in terms of increased progression-free survival. These inhibitors block one of the two DNA repair mechanisms and are active in ovarian cancer patients responding to platinum salt-based chemotherapy. At present, we are witnessing another prognosis improvement in our patients, as a consequence of using these agents at earlier stages and preventing relapse after curative surgery in patients at high-risk of relapse. At the King Albert II Cancer Institute, Cliniques universitaires Saint-Luc, Brussels, these treatments are now available, as are other new treatments still being investigated, which may become the standard of care in the future.

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Innovations in onco-hematology in 2018

Violaine Havelange (1), Xavier Poire (1), Jean-Philippe Defour (2), Stefan N Constantinescu (3), Pascale Saussoy (2), Eric Van Den Neste (1), Sarah Bailly (1) Published in the journal : February 2019 Category : Onco-Hématologie

In 2018, major developments have been achieved in the diagnostic and therapeutic fields of CAR-T cell immunotherapy, next-generation sequencing (NGS), and targeted therapies. Several studies have demonstrated the benefits of CAR-T cells in refractory or relapsing diffuse large B-cell lymphomas. NGS is a major breakthrough in the diagnosis and management of patients with myeloid neoplasia and represents an important prognostic tool that will probably replace many other conventional factors in the future. Finally, several targeted therapies reviewed in this article are about to revolutionize the management of acute myeloblastic leukemia.

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Innovations in Nephrolgy in 2018

Eric Goffin (1), Arnaud Devresse (1), Jean-François Baurain (2), Isabelle Tromme (3), Michel Mourad (4), Nada Kanaan (1) Published in the journal : February 2019 Category : Nephrology

This article reviews innovations in kidney transplantation, particularly in terms of cutaneous carcinomas and ABO incompatibility. Kidney transplant recipients are at risk of developing basal cell or squamous cell carcinomas due to immunosuppression induced by anti-rejection therapy. A multidisciplinary approach to these skin lesions is required to optimize the therapeutic management, thereby improving patient prognosis. Kidney transplantation from ABO-incompatible living donor patients was introduced in 1982 and successfully taken up in our institution last year. It is rendered possible due to a strategy combining desensitization of the recipient and reinforced immunosuppression. Survival of patients and grafts appears to be equivalent to that observed in ABO compatible transplant patients.

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What challenges for the management of rare diseases? Current situation in Europe and Belgium

Nathalie Lannoy (1), Marie-Françoise Vincent (2), Fabienne Lohest (2), Cédric Hermans (1) Published in the journal : February 2019 Category : Rare Diseases

In Europe, a disease is defined as rare when its prevalence is less than 1 in 2,000. The management of patients with rare diseases refers to the complex and heterogeneous status of some 6,000 to 8,000 diseases, 80% of which are of genetic origin. Rare diseases mostly affect children. Due to the large phenotypic heterogeneity, rarity and unusual nature of rare diseases, the affected patients regularly encounter a lack of knowledge regarding their disease, which is most often associated with diagnostic wavering until an accurate diagnosis is made and lack of treatment.

In order to draw attention on and address this issue, the European Commission has been developing initiatives aiming to ensure the development of concrete measures for the patients and their families across its member states for more than two decades.

Responding to this call, Belgium is committed to improving the management of rare diseases and has thus developed a national plan. Multidisciplinary diagnostic structures were created and supported, partnerships with European reference networks were established, and registers were set up in order to centrally and uniformly collect patient data. This article offers an overview of the characteristics of rare diseases, as well as an inventory of existing measures in both Europe and Belgium.

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News on infectious diseases in 2018: What the general practitioner should know

Jean-Cyr Yombi Published in the journal : February 2019 Category : Infectiologie

In various fields of medicine, several innovations and novelties were introduced in 2018, and infectiology does not escape the rule (1). In this article, we have presented the relevant news and novelties that the GP should be aware of.

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2018 innovations in orthopedic surgery and traumatology

Th. Schubert (1), M. Van Cauter (1), O. Barbier (1), D. Mathieu, D. Morcillo, L. Thoreau, S. Vandergugten, N.Pireau, A. Sirbu, V. Druez, N. Irda, L. Kaminski, K. Tribak, D. Putineanu, J-E. Dubuc, X. Libouton, E. Thienpont, J-C. Yombi, X. Geets (2),et al. Published in the journal : February 2019 Category : Chirurgie orthopédique et traumatologie

Arthroplasty has become the standard approach for hip replacement and has even been described as “The operation of the century” in The Lancet. The drive to optimize care has led to many improvements, particularly regarding minimally invasive surgery, thereby enabling faster functional recovery. Thanks to these advances, it is now possible to consider performing a bilateral arthroplasty, when required, in the same operating session, without impacting the final result of both arthroplasties and at a lower cost. The concept of arthroplasty also extends to the structures involved in the movements of the over- and underlying joints, notably with arthroplasties able to restore the pronation-supination movements of the forearm.

The discovery of a soft tissue mass is often trivialized. Yet, when its volume exceeds 5 centimeters and it is located under the fascia, a malignant tumor must be considered, and multidisciplinary care is required. While surgery has long been regarded as the standard therapeutic option, it is now well established that the co-management with radiation therapists offers a better chance of success, especially if it can precede surgery. Strategies combining state-of-the-art radiotherapy techniques to achieve skin saving and surgical management techniques with a very conservative approach to soft tissue management and negative pressure wound therapy significantly improve the tumor control.

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European Society of Cardiology and European Society of Hypertension joint guidelines for the management of arterial hypertension: key messages

Agnès Pasquet, Alexandre Persu Published in the journal : February 2019 Category : Cardiology

The European Society of Cardiology and European Society of Hypertension have published new guidelines regarding management of hypertension. This paper summarizes the key messages of these guidelines.

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The innovative technology of virtual reality

Damien Henrard, Fabienne Roelants Published in the journal : February 2019 Category : Anesthesiology

The role of the anesthesiologist is to care for the patient throughout the perioperative period and to ensure his/her well-being notably through anxiolysis and effective pain management. In addition to conventional drug approaches, an innovative technique is slowly proving its worth in the medical world: virtual reality. While already well known to the general public for its recreational benefits, virtual reality will probably become in the near future an essential tool in the management of pain and anxiety in a wide variety of painful medical procedures. This technology has already been successfully studied in both adult and pediatric patients in a range of potentially painful medical procedures, such as the treatment of wounds in seriously burned patients, dental procedures and routine medical procedures, and has now started to make its way into anesthesiology...

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History of the youngest medical specialty : (Part 4 of 4 parts)

Yves Pirson Published in the journal : January 2019 Category : Ama Contacts

Nephrology as a full-blown specialty was born in the middle of th 20th century with the advent of kidney replacement therapy. As regards knowledge of kidney diseases, decisive steps forward were introduction of percutaneous kidney biopsy (with examination by immunofluorescence staining and electron microscopy), as well as investigation of individual nephron function by micropuncture and more recently molecular biology techniques. As regards therapy, nephrology benefited the tremendous development of phamacology with the advent of diuretics, hypotensive agents and immunosuppressive drugs. But the revolution of the time was actually the invention of hemodialysis and peritoneal dialysis as well as the first success of kidney transplantation, allowing a rapidly growing number of patients worldwide to survive renal insufficiency for decades. Access to renal replacement therapy gave rise to novel ethical issues, which has been the foundation of medical bioethics.

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