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Despite the motivation of oncology patients to take part in clinical trials, only a minority of them are enrolled in clinical trials. Implementation of new practical procedures can become a barrier that withholds patients from participating in clinical trials. Treating physicians are crucial in augmenting trial accrual. The drivers that promote physicians to allocate patients for clinical trials need further assessment. We conducted two separate cross-sectional surveys, addressing patients with a haematological disease in one survey and haematologists in another survey. The patient survey was filled out by 420 patients. Significant relationships between the willingness to participate in a trial and trial knowledge (p < 0.001) and between doctor–patient relationship and participation willingness (p = 0.007) were noted. Patients above 60 years were less willing to use an electronic consent procedure vs. patients younger than 60 (p < 0.001). The physician questionnaire was completed by 42 participants of whom most (83%) were active in and (94%) motivated for clinical trials. Apart from the patient benefit and scientific interest, prestige was an equal motivator closely followed by financial remunerations. First goal was not to harm the patient. Our study confirms the high willingness of patients for trial participation and the need to rethink the structure of trial organisation. The e-consent procedure is not the method preferred by most patients above 60 years old. [ABSTRACT FROM AUTHOR]

Simple Summary: Myelodysplastic syndromes are a heterogenous group of bone-marrow cancers that prelude a possible evolution to acute myeloid leukemia. Most treatments aim at reinstalling bone marrow function and delay evolution to acute leukemia. The disease has a negative impact on health-related quality of life and amelioration is an independent treatment goal. In order to capture the impact of current treatments outside clinical trials we aimed to monitor quality of life during the course of treatment. Another key element in our study was to prospectively assess disease perception and the impact on quality of life. Not reaching our inclusion goal, we cannot make any conclusions or assumptions on our first objective. We can, however, demonstrate a clear significant relation between disease perception and health related quality of life in this patient population that may lead to a positive impact for future patients. Patients with myelodysplastic syndromes suffer from an impaired quality of life that is only partially explained by physical symptoms. In an observational study, we aimed to investigate the impact of current MDS treatments and the influence of disease perception on quality of life. Serial measurement of health-related quality of life was performed by 'the QUALMS', a validated MDS-specific patient reported outcome tool. Disease perception was evaluated by means of the Brief Illness Perception Questionnaire (B-IPQ). We prospectively collected data on 75 patients that started on a new treatment and could not demonstrate a significant change in QUALMS score or B-IPQ score during treatment. Six out of eight items evaluated in the B-IPQ correlated significantly with QUALMS score. In this small sample, no significant difference in QUALMS score was found between lower vs. higher risk MDS patients or other studied variables, e.g., targeted hemoglobin showed no correlation with QUALMS score. In daily practice attention must be paid to initial formation of disease perception as it correlates independently with health-related quality of life and does not change during treatment (clinicaltrials.gov identifier: NCT04053933). [ABSTRACT FROM AUTHOR]

Despite its considerable morbidity and mortality, paroxysmal nocturnal haemoglobinuria (PNH) is still underdiagnosed. Patients with PNH can suffer from cardiovascular, gastrointestinal, neurological or haematological symptoms and refer to several specialists. The aim of this paper is to review the diagnosis and the management of PNH patients, with the primary focus on identifying high‐risk groups. Additionally, the implementation and prognostic value of the defined high‐risk groups will be commented on and the management of PNH patients is discussed from a Belgian perspective. Finally, based on the available data, recommendations are provided. Eculizumab is a potent C5 complement inhibitor and reduces intravascular haemolysis and thrombosis in PNH patients and improves their quality of life. As thrombosis is the main cause of death in PNH patients, identifying high‐risk PNH patients in need of therapy is essential. Currently, novel complement inhibitors are in development and the first data seem promising. Another challenge in PNH is to identify new markers to assess the thrombotic risk to achieve a better risk‐based prophylactic anti‐thrombotic management. Finally, because of the low prevalence of the disease, PNH patients should be included in the prospective PNH registry, which will offer new insights on the natural course of the disease and the impact of treatment of PNH. [ABSTRACT FROM AUTHOR]

Myelodysplastic syndromes are a heterogeneous group of clonal haematological stem cell disorders. Allogeneic stem cells transplantation remains the only curative treatment but only a minority of patients are eligible for this treatment. In spite of this, it has become clear that treatment with lenalidomide and azanucleotides can lead to increased overall survival in particular subsets of patients with MDS. The relative silence on the therapeutic side is counter-balanced by major advances in the understanding of this heterogeneous disease. The introduction of high-throughput molecular techniques has resulted in the discovery that most patients harbour molecular aberrations, including pathways such as the spliceosome machinery previously not known to be involved. These newly discovered pathways will undoubtedly result in new therapeutic strategies for this difficult to treat disease. [ABSTRACT FROM AUTHOR]

Abstract: A 20-year old man with ulcerative colitis (UC) and primary sclerosing cholangitis (PSC) was diagnosed with a rectal non-Hodgkin''s lymphoma (NHL) at surveillance endoscopy while being in remission on infliximab therapy. Further staging identified a diffuse large B-cell NHL, EBV negative restricted to the rectal submucosa (stage IA). Until now, there has not been any evidence of an increased risk of NHL in patients with UC nor of an increased risk of lymphoproliferative disorders in IBD patients. Hence, the role of concomitant PSC in the pathogenesis of intestinal NHL is unclear. However, IBD patients treated with purine analogues and with anti-TNF are at risk of NHL, especially hepatosplenic T-cell lymphoma. The management of this particular young patient is further complicated by the possibility of a future colectomy due to intractable disease which compromises the use of radiotherapy for this localized disease. [Copyright &y& Elsevier]

Background. Toxoplasmosis is an often fatal opportunistic infection following allogeneic hematopoietic stem cell transplantation and is largely due to deferred diagnosis. In addition, breakthrough infections occur during prophylaxis with trimethoprim-sulfamethoxazole. Methods. From November 2001 onwards, we routinely monitored all stem cell transplant recipients who were seropositive for Toxoplasma gondii and/or who received a transplant from a donor who was seropositive for T. gondii reactivation by polymerase chain reaction of peripheral blood samples. The aim of this study was to evaluate the incidence of and the risk factors for Toxoplasma reactivation in this population not receiving specific prophylaxis. We also studied the feasibility of a preemptive treatment approach based on this routine monitoring. Results. We report a toxoplasmosis incidence of 8.7% (18 of 208 patients). Twelve patients (5.8%) had a T. gondii infection at diagnosis; 6 patients (2.9%) had Toxoplasma disease, including cerebral toxoplasmosis (n=5) and cardiopulmonary toxoplasmosis (n=1). We identified myeloablative conditioning and conditioning with high-dose total body irradiation (10-12 Gy) as risk factors for T. gondii reactivation, whereas patients with a seropositive donor were less likely to experience reactivation. Patients with T. gondii disease had a significantly higher number of transcripts in blood than did patients with a T. gondii infection. Finally, with a strategy of routine monitoring and preemptive treatment with clindamycin-pyrimethamine, we only had 3 Toxoplasma-related deaths among our patients, which is a much lower rate than that reported in the literature. Conclusions. Systematic monitoring with polymerase chain reaction is a good means to detect T. gondii reactivation and could reduce T. gondii-related mortality among hematopoietic stem cell transplant recipients. [ABSTRACT FROM AUTHOR]

Abstract: We studied the immune compartment in patients with myelodysplastic syndromes. We show increased surface expression of activation markers (HLA-DR+, CD57+, CD28−, CD62L−) on T lymphocytes in blood and bone marrow (n =131). T cell activation was not restricted to any relevant clinical subgroup (FAB, IPSS, cytogenetics) and did not correlate with blood counts or need for treatment. In vitro clonogenic growth of marrow mononuclear cells (n =18) was not influenced by T cells expressing these markers. In addition, using X-chromosome inactivation analysis (n =12) we demonstrate clonal involvement of NK and B cells in half of these patients. We conclude that although activated T lymphocytes can be found in MDS, their role in disease pathogenesis remains unclear in the majority of patients. [Copyright &y& Elsevier]

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. [ABSTRACT FROM AUTHOR]

Background. Prompt diagnosis of invasive pulmonary aspergillosis (IPA) remains a challenge. Galactomannan (GM) detection in bronchoalveolar lavage (BAL) fluid by the Platelia enzyme immunoassay aims to further improve upon the test's utility by applying it directly to specimens from the target organ. Methods. A retrospective analysis of the Platelia assay was performed on BAL samples from 99 evaluable highrisk hematology patients, including 58 with proven or probable IPA. Results. BAL GM demonstrated an improved sensitivity profile (91.3% with an optical density [OD] index cutoff of ≥1.0) in comparison with culture and microscopy (50% and 53.3%, respectively). The diagnostic accuracy as given by the area under the receiver operating characteristic curve was 0.93 (95% confidence interval, 0.88-0.99); further decreasing the OD index cutoff to ≥0.5 compromised specificity more than it improved sensitivity. Estimates of the positive and negative predictive value of the Platelia assay on BAL samples (OD index, ≥1.0) were 76% and 96%, respectively. The mean BAL GM OD index was not different in neutropenic versus nonneutropenic case patients (3.9 and 4.5, respectively; P = .3); however, a trend toward decreased sensitivity in patients receiving mold-active prophylaxis was noted. Conclusion. BAL GM is a valuable adjunctive diagnostic tool to other conventional microbiologic and radiologic studies. [ABSTRACT FROM AUTHOR]