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Data on clinical use of ponatinib are limited. This prospective registry aimed to evaluate outcomes of ponatinib treatment in routine practice over 3 years (2016-2019) in Belgium (NCT03678454). Patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) were treated with ponatinib per current label. Fifty patients (33 CML and 17 Ph+ ALL) were enrolled. Fifty-five percent of CML and 29% of Ph+ ALL patients had received ≥3 prior tyrosine kinase inhibitors (TKIs). Reasons for starting ponatinib were intolerance (40%), relapse or refractoriness (28%) to previous TKIs, progression (16%), or T315I mutation (16%). Median follow-up was 15 months for CML and 4.5 months for Ph+ ALL patients. Best response was a major molecular response in 58% of CML and 41% of Ph+ ALL patients. Of 20 patients who started ponatinib due to intolerance to previous TKIs, 9 (64%) CML and 4 (67%) Ph+ ALL achieved a major molecular response. Three-year estimates of overall survival were 85.3% and 85.6%, respectively, in CML and Ph+ ALL patients; estimated progression-free survival was 81.6% and 48.9%. Adverse reactions were reported in 34 patients (68%); rash (26%) and dry skin (10%) were most common. Reported cardiovascular adverse reactions included vascular stenosis (3), arterial hypertension (2), chest pain (1), palpitations (1), and vascular occlusion (1). This Belgian registry confirms results from the PACE clinical trial and supports routine ponatinib use in CML and Ph+ ALL patients who are resistant or intolerant to previous TKIs or with the T315I mutation.

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Full Text, Thesis (PhD Doctorate), Doctor of Philosophy (PhD), Institute for Glycomics, Griffith Health, Human viral pathogens are a serious cause of illness worldwide, and are responsible for a significant number of infectious diseases. Recurring respiratory infections account for the greater part of the disease burden associated with viral infections in infants, while we see more and more emerging viruses affecting this population, especially in Asia. Human respiratory syncytial virus (hRSV) and Human parainhuman fluenza type-3 virus (hPIV-3) are the main aetiological agents of acute lower respiratory tract infection in in- fants worldwide. Analogously, enterovirus 71 (EV71) is a major cause of hand foot and mouth disease (HFMD) in Asia, an ordinarily benign paediatric disease that in some cases develops to a severe neurological infection. Sadly, neither vaccines nor drugs are to date available to prevent or treat infection by either of these viruses. The work presented in this Thesis is focussed on the discovery of novel inhibitors of hPIV-3, hRSV and EV71 infection, by using multidisciplinary approaches and various drug discovery strategies.

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Human viral pathogens are a serious cause of illness worldwide, and are responsible for a significant number of infectious diseases. Recurring respiratory infections account for the greater part of the disease burden associated with viral infections in infants, while we see more and more emerging viruses affecting this population, especially in Asia. Human respiratory syncytial virus (hRSV) and Human parainhuman fluenza type-3 virus (hPIV-3) are the main aetiological agents of acute lower respiratory tract infection in in- fants worldwide. Analogously, enterovirus 71 (EV71) is a major cause of hand foot and mouth disease (HFMD) in Asia, an ordinarily benign paediatric disease that in some cases develops to a severe neurological infection. Sadly, neither vaccines nor drugs are to date available to prevent or treat infection by either of these viruses. The work presented in this Thesis is focussed on the discovery of novel inhibitors of hPIV-3, hRSV and EV71 infection, by using multidisciplinary approaches and various drug discovery strategies., Thesis (PhD Doctorate), Doctor of Philosophy (PhD), Institute for Glycomics, Griffith Health, Full Text

A patient with refractory diffuse lymphoma treated for pulmonary invasive aspergillosis developed a concomitant primary cutaneous mucormycosis. The mucormycete was identified by sequencing as Mucor circinelloides. This case confirms the importance of a rapid pathogen diagnosis in immunocompromised patients and the usefulness of molecular methods for identification of rare fungal species., SCOPUS: ar.j, info:eu-repo/semantics/published

A 53-years-old man has a dysentery since two weeks. The blood test shows Coombs-positive hemolytic anemia and inflammation. Autoimmune hemolytic anemia (AIHA) is treated with corticosteroid. A colonoscopy reveals an ulcerative colitis. The evolution of the patient is complicated by a spontaneous digestive perforation treated by total proctocolectomy. After this intervention, there is a resolution of the AIHA and the patient is gradually weaned from corticosteroids. AIHA is a rare extra-intestinal manifestation of inflammatory bowel disease essentially ulcerative colitis. Identification of this cause of secondary AIHA is important for the therapeutic strategy. However treatment is nonspecific and based on low levels of evidence., SCOPUS: ar.j, info:eu-repo/semantics/published

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LETTER, SCOPUS: le.j, info:eu-repo/semantics/published

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The Children’s/Teenager’s corner is a meeting point at the hospital set up in partnership with the medical oncology teams. It is designed for children, teenagers and their respective families who come to visit a parent or a close one hospitalized for a serious illness. This location, created and suitable for children, helps to maintain the family link and communication. The activities are led by two professional psychologists who encourage personal and emotional expression through the use of tools such as games, drawing, or sharing. Entrance is free and it is open on wednesdays afternoons., SCOPUS: ar.j, info:eu-repo/semantics/published

Autologous stem cell transplantation (ASCT) consolidation remains the treatment of choice for patients with relapsed diffuse large B cell lymphoma. The impact of rituximab combined with chemotherapy in either first- or second-line therapy on the ultimate results of ASCT remains to be determined, however. This study was designed to evaluate the benefit of ASCT in patients achieving a second complete remission after salvage chemotherapy by retrospectively comparing the disease-free survival (DFS) after ASCT for each patient with the duration of the first complete remission (CR1). Between 1990 and 2005, a total of 470 patients who had undergone ASCT and reported to the European Blood and Bone Transplantation Registry with Medical Essential Data Form B information were evaluated. Of these 470 patients, 351 (74%) had not received rituximab before ASCT, and 119 (25%) had received rituximab before ASCT. The median duration of CR1 was 11 months. The median time from diagnosis to ASCT was 24 months. The BEAM protocol was the most frequently used conditioning regimen (67%). After ASCT, the 5-year overall survival was 63% (95% confidence interval, 58%-67%) and 5-year DFS was 48% (95% confidence interval, 43%-53%) for the entire patient population. Statistical analysis showed a significant increase in DFS after ASCT compared with duration of CR1 (median, 51 months versus 11 months; P < .001). This difference was also highly significant for patients with previous exposure to rituximab (median, 10 months versus not reached; P < .001) and for patients who had experienced relapse before 1 year (median, 6 months versus 47 months; P < .001). Our data indicate that ASCT can significantly increase DFS compared with the duration of CR1 in relapsed diffuse large B cell lymphoma and can alter the disease course even in patients with high-risk disease previously treated with rituximab. © 2012 American Society for Blood and Marrow Transplantation., 0, SCOPUS: ar.j, info:eu-repo/semantics/published

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