Anticoagulation in antiphospholipid syndrome

Catherine Lambert Published in the journal : April 2022 Category : Hematology/Oncology

Antiphospholipid syndrome is a severe thrombotic disorder with a high risk of recurrence. For many years, the standard treatment for thrombotic complications of antiphospholipid syndrome has been anticoagulation with anti-vitamin K drugs. Over the past decade, direct oral anticoagulants have emerged as the anticoagulant treatment of choice for venous thromboembolic disease in the general population. The purpose of this article has been to review the modalities and objectives of anticoagulant therapy in antiphospholipid syndrome and to discuss the potential place of direct oral anticoagulants in this indication

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VEXAS syndrome: a new entity at the crossroads of rheumatology and hematology

Séverine Wautier*, Hélène Houssiau*, Nicole Straetmans (1) Published in the journal : April 2022 Category : Hematology/Oncology

VEXAS (Vacuoles, Enzyme E1, X-linked, Autoinflammatory, Somatic) syndrome has been recently described as a late-onset inflammatory disease caused by a somatic mutation in UBA1 in myeloid cells. It mostly affects men over 40 years of age with inflammatory symptoms (recurrent fever, dermatitis), rheumatologic disorders (arthritis, chrondritis), or hematologic abnormalities for several years. The presence of vacuoles in bone marrow myeloid cells is a characteristic (yet non-specific) sign of the disease. VEXAS syndrome may be associated with hematologic diseases, such as myelodysplasia and multiple myeloma, as well as an increased risk of thromboembolic event. The mortality is about 25-35% at 5 years. The disease is usually steroid-resistant. Studies with JAK2 inhibitors have shown promising results and are currently ongoing. Allogeneic stem cell transplantation is currently the only curative option for young, otherwise healthy patients.

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Clinical work-up of adenopathy in general medicine : when should we reassure the patient and when refer the patient for further evaluation?

Inès Dufour1, Géraldine Verstraete1, Juliette Raedemaeker1, Fabio Andreozzi1, Sarah Bailly1, Eric Van Den Neste1, Cédric Hermans1, Marie-Christiane Vekemans1 Published in the journal : March 2022 Category : Hematology/Oncology

Adenopathy investigation requires a systematic approach in order to determine the benign or malignant character of the adenopathy. It is essential to be able to recognize severity signs, which should orientate towards additional assessments, usually consisting of a biopsy. The article was aimed to help the general practitioner in the adenopathy work-up, as well as to review the main differential diagnoses in this setting.

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2021 Innovation in hematology

Marie-Christiane Vekemans, Violaine Havelange, Eric Van Den Neste, Sarah Bailly, Catherine Lambert,Nicole Straetmans, Xavier Poiré, Stefan N. Constantinescu, Cédric Hermans Published in the journal : February 2022 Category : Hematology/Oncology

The year 2021 was rich in innovations in regard to both malignant and benign hematological diseases, with the most relevant of which discussed below.

- In recent years, the prognosis of multiple myeloma has been greatly improved, yet the disease remains as yet incurable. Current strategies aim to achieve optimal disease control at diagnosis using four-drug regimens designed to delay relapse, while in relapsed patients, innovative alternatives like immunotherapy are being employed to effectively re-arm the immune system, and this represents a real hope for patients suffering from this chronic disease.

- The management of chronic myeloid leukemia is still evolving. Since 2001, tyrosine kinase inhibitors have revolutionized its prognosis, but they are also responsible for toxicities. One of the current therapeutic goals is to achieve a sufficiently deep and prolonged response, thereby enabling tyrosine kinase inhibitors to be discontinued and remission maintained.

- Myelofibrosis is a myeloproliferative neoplasm with poor prognosis. The only curative treatment available to date is peripheral stem cell allograft, which can be applied at least for some patients. New therapeutic agents are currently being investigated, which are highly promising not only on account of their efficacy on symptoms and splenomegaly, but also for their potential anti-fibrotic and reducing effect on the mutated clone. Certain agents are in clinical assays in combination with JAK2 inhibitors.

- Several recent studies have suggested that CAR-Ts were likely to emerge as the second-line treatment of choice for patients suffering from aggressive lymphomas, while secondarily reducing the number of autologous stem cell transplants.

- Patients with acute myeloid leukemia and TP53-mutated myelodysplastic syndromes still represent a challenging population to treat with low and only short-lived response rates. The advent of APR-246, which restores TP53 activity, is likely to increase the number of responders among these very high-risk patients.

- Beyond its well-known involvement in heparin thrombocytopenia, platelet factor 4 (PF4) plays a major role in the very rarely observed thrombosis with thrombocytopenia occurring in association with COVID-19 adenoviral vector vaccines. Inhibition of FXI is expected to emerge as a promising anticoagulation strategy with reduced bleeding risk, while induction of antithrombin deficiency by interfering RNA is likely to prevent bleeding in hemophilia A and B patients, either with and without inhibitors.

- Concerning the management of immune thrombocytopenic purpura, targeted therapies are currently being favored owing to their lower toxicity and individualized platelet count targets.

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What to do about hyperferritinemia?

Géraldine Verstraete Published in the journal : January 2022 Category : Hematology/Oncology

Regularly dosed upon routine biological work-ups, ferritin is revealed increased in a certain proportion of the population. At times, detection of hyperferritinemia leads to exhaustive and useless work-up, often including HFE genotyping. Although hemochromatosis is the most common genetic disease, this condition is not the primary cause of hyperferritinemia. In Western countries, metabolic syndrome and consumption of alcoholic beverages are the first etiologies to be considered. In general, a complete diagnostic approach, including complete medical history, clinical examination, and biology with a complete martial assessment, allows for identifying, without any specialized consultation required.

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Thrombosis and vaccines: a new challenge of the COVID-19 pandemic

Cédric Hermans, Michel Goldman Published in the journal : April 2021 Category : Hematology/Oncology

Venous thromboses at unusual locations, such as cerebral and visceral sites, along with thrombocytopenia have been detected in patients who received COVID-19 vaccines using adenovirus vectors. The concomitant presence of antibodies targeting platelet factor 4 is suggestive of an autoimmune process associated with heparin thrombocytopenia. These thromboses, although very rare, significantly impact the vaccination strategy.

This article seeks to review the available data on this topic. Accordingly, the observations that led to its identification, its hypothetical mechanisms, the diagnostic approach, as well as the therapeutic strategies to implement are presented in a concise and practical manner.

This article should help any health professional to better understand this rare vaccination-related complication and answer the patients' many questions.

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Management of relapsed/refractory diffuse large B-cell lymphomas in 2020 in Belgium. A 1-year experience with tisa-cel (Kymriah®), the first anti-CD19 CAR-T cell to be reimbursed

Sarah Bailly, Xavier Poiré, Nicole Straetmans, Violaine Havelange, Marie-Christiane Vekemans, Eric Van Den Neste Published in the journal : February 2021 Category : Hematology/Oncology

Recently, the management of relapsed/refractory diffuse large B-cell lymphoma has been revolutionized by the introduction of of anti-CD19 CAR-T cells. These treatments have been available in Belgium since mid-2019.

What to remember after one year of experience?

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2020 innovations in the field of hematopoietic stem cell transplantation

Raphaël Lattenist, MD, Xavier Poiré, MD, PhD Published in the journal : February 2021 Category : Hematology/Oncology

The 2020 annual meeting of the American Society of Hematology (ASH) provided us with several contributions to the field of hematopoietic stem cell transplantation (HSCT). Post-transplant cyclophosphamide (PTCy) occupied a prominent place on the stage. This modality of graft-versus-host disease (GvHD) prophylaxis, which was initially developed in the haploidentical HSCT setting, was associated with major benefits so that its use tended to spread across other donor types. PTCy appears to be particularly invested with the benefit of improving outcomes in HSCT from mismatched unrelated donors (MMUD). This appears to be of great usefulness for patients from underserved ethnicities that lack a matched donor. Likewise, management of chronic graft-versus-host disease (cGvHD) had its share of improvement with the result presentation of the REACH3 study. Notably, in this study, ruxolitinib was compared to the best available therapy. The approach of reduced intensity conditioning (RIC) for myeloid malignancies similarly received some attention with a boost to supporting its use for higher-risk myelodysplastic syndrome (MDS), including the elderly. Finally, we present a Phase 1 study, which was designed to improve its efficacy in high-risk myeloid malignancies by adding venetoclax .

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From immunotherapy, the new therapeutic pillar, to allogeneic hematopoietic stem cell transplantation

Xavier Poiré, Nicole Straetmans, Sarah Bailly, Marie-Christiane Vekemans Published in the journal : February 2020 Category : Hematology/Oncology

In this article focused on innovations in onco-hematology are highlighted the successes of immunotherapy, as well as current challenges of allogeneic hematopoietic stem cell transplantation.

The treatment of hematological malignancies has traditionally been dominated by chemotherapy or radiotherapy. The discovery of the cytogenetic and molecular bases of these diseases has paved the way for targeted therapies; more recently, the field of immunotherapy has emerged with the development of CAR-Ts, BiTEs, immunoconjugates, and checkpoint inhibitors. These innovations have enabled us to better understand the complex interactions between a patient's immune system and potential tumor cells. However, the idea of exploiting the patient's immune system in an attempt to control his disease is not a recent concept, since allogeneic haematopoietic stem cell transplantation is based on the principle of using graft’s T lymphocytes to eliminate the recipient's tumor cells.

To date, allogeneic hematopoietic stem cell transplantation remains a mainstay of therapeutic weapons in hematology. Moreover, there is a great deal of research being done to improve outcomes. In order to reduce the risk of relapse without increasing toxicity, conditioning regimens with reduced toxicity are becoming a new standard. Graft-versus-host disease would be better prevented by administering post-transplant cyclophosphamide. Finally, numerous post-transplant relapses are the consequence of immune escape. A better understanding of their mechanisms likely opens the way to new therapeutic strategies.

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Side effects of multiple myeloma treatments

Sarah Bailly, Sarah Amat, Catherine Percy, Thomas Gérard, Laurane Lallemand, Aline Munting, Marie-Christiane Vekemans Published in the journal : January 2020 Category : Hematology/Oncology

The prolonged use of new drugs such as proteasome inhibitors, immunomodulators, and monoclonal antibodies in the treatment of multiple myeloma is associated with adverse side effects that represent a daily challenge for physicians. We propose a series of recommendations based on clinical cases.

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