Chronic lymphocytic leukemia is the most common leukemia subtype, accounting for over one-third of leukemia cases in Belgium. Recent years have seen a significant expansion in the treatment range available to patients and clinicians. Today, we live in an era of targeted therapies and immunotherapies, relegating chemotherapy to very rare situations. These new treatments were shown to offer excellent prospects in terms of progression-free survival and quality of life for patients, but they have also introduced new toxicity profiles that clinicians must manage. This article sought to provide physicians with a condensed update on what they need to know about managing this disease in 2024.
Nadia Vonêche (1), Julia Riggi (2), Christine Galant (3), Martine Berlière (4), Mieke Van Bockstal (5)Published in the journal : July 2024Category : Hématologie et Oncologie médicale
The incidence of ductal carcinoma in situ (DCIS) has considerably risen since the introduction of organized mammography screening programs in the general population. Currently, all DCIS are treated surgically, though most are believed to be indolent. The difficulty in determining which lesions are at risk of becoming invasive has likely led to overtreatment for many patients as well as confusion about the actual risk of developing invasive disease or dying from breast cancer. This misperception of risk has generated significant psychological stress for patients. In order to improve their quality of life, it is crucial to better understand the development of invasive breast cancers and enhance communication between doctors and patients. Some experts are even questioning the current terminology: Should DCIS still be referred as a form of cancer?
To address these issues, new research focusing on molecular, genetic, and microenvironmental aspects of DCIS progression to invasive breast cancer must be initiated. The identification of reliable biomarkers will likely allow for the development of accurate risk prediction that are models specific to DCIS, paving the way towards therapeutic de-escalation. Additionally, this research is likely to support the creation of communication and decision-support tools with treatments being primarily focused on the patient's needs.
Pituitary adenomas, recently renamed pituitary neuroendocrine tumors (PiNETs) according the new World Health Organization classification published in 2017, are one of the two most common intracranial tumors, with an estimated prevalence of 1 in 1,100 people.
A meticulous endocrine evaluation is the first therapeutic step in their management, aimed at detecting hormonal hypersecretion and anterior hypopituitarism. High-resolution magnetic resonance imaging (MRI) of the pituitary region using a dedicated protocol is then recommended to precisely localize the tumor and determine its extra-sellar extension. Each case is discussed weekly at our multidisciplinary meeting to determine the best therapeutic option for each individual patient.
The first-line treatment is usually transnasal transsphenoidal surgery, except for microprolactinomas that are effectively managed using dopamine agonist medication like cabergoline to control the endocrinopathy. Surgical resection in expert centers allows for elevated levels of hypersecreted hormone to be normalized in 50 to 90% of cases. In the event of failure or recurrence, a second surgery or radiotherapy can be proposed. Some PiNETs, including non-functional microadenomas, only require serial MRI monitoring without treatment.
Gabriel Levy (1), Cécile Boulanger (1), Bénédicte Brichard (1), Manon Le Roux (1), Maelle de Ville de Goyet (1), An Van Damme (1)Published in the journal : July 2024Category : Hématologie et Oncologie médicale
Pediatric hemato-oncology deals with cancers occurring in patients from birth to the age of 16 years. Due to the low prevalence of these diseases, which constitute 1% of all cancers, the therapies are thus directed at rare diseases. Pediatric cancers encompass more than 60 different entities, with their frequency varying according to children’s age. In absolute numbers and across all ages, hematological malignancies are the most common, accounting for 39% of cancers in children aged 0–14 years old (27% leukemias and 12% lymphomas), followed by central nervous system tumors (27%). Although the histology of pediatric cancers may be similar to that of adult neoplasias, their biology and predisposing factors are quite distinct. Treatment of pediatric tumors, conducted exclusively in tertiary centers, is multidisciplinary, aiming at treating the pathology but also at maintaining the children within their family and social environment. With survival rates exceeding 80%, the prevention and treatment of long-term side-effects also require special focus. Encouraged by international studies, pediatric oncology specialists are now seeking to collaborate with adult oncology colleagues to treat adolescents and young adults aged 16 to 35 years, covering a group with specific pathologies, as well as precise social and therapeutical needs. Keywords Pediatric hemato-oncology, predispositon, pediatric hemato-oncology center, Belgium, long-term side-effects, adolescents, young adults
In recent years, new therapies, mainly consisting of monoclonal antibodies like daratumumab and isatuximab, have revolutionized the management of patients with multiple myeloma (MM). Despite these advances, MM remains incurable, and patients who have become refractory to the three therapeutic classes of drugs, including proteasome inhibitors, immunomodulators, and monoclonal antibodies, have a particularly poor prognosis, which highlights the need for new treatment strategies. The development of novel immunotherapeutic approaches, such as antibody-drug conjugates, bispecific antibodies, and CAR T-cells, marks a turning point for heavily pre-treated patients, while revolutionizing their future. In this article, we have reviewed the contributions of these therapies to the current treatment landscape.
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.
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.
S. Dupriez 1, A. Ferrant 1, M-Ch. Vekemans 1, B. Brichard 2, L. Michaux 3, T. Connerotte 4, E. Van Den Neste 1, Ch. Vermylen 2, L. Knoops 5, C. Graux 6, F. P. Duhoux 7, C. Lambert 1, X. Poiré 1, H. Antoine-Poirel 8Published in the journal : April 2018Category : Hématologie et Oncologie médicale
Hereditary hematological malignancies syndromes (HHMS) are still underdiagnosed. Yet, it proves essential to correctly identify these disorders in order to select the most appropriate conditioning regimen before transplantation and exclude an inherited mutation in sibling donors, while ensuring personal and familial counseling. We retrospectively reviewed 252 patients from 117 families with a personal or familial history of multiple cancers, including at least one hematological malignancy. A familial mutation was identified in eight families (GATA2, TERT, FANCA, TP53, PTCH1, BRCA1 and ATM), resulting in specific management and familial screening. This study highlights the necessity of early HHMS diagnosis through a close collaboration between hematologists and geneticists. To finish, we present recommendations to better diagnose and manage HHMS on the basis of our observations and the literature.