RESUMO
This national survey investigated the current practice in Switzerland by collecting participants' opinions on paroxysmal nocturnal hemoglobinuria (PNH) clone assessment and clinical practice. Aim: This study aimed to investigate clinical indications prompting PNH clones' assessment and physician's accessibility of a flow cytometry facility, and also to understand clinical attitudes on the follow-up (FU) of patients with PNH clones. Methods: The survey includes 16 multiple-choice questions related to PNH and targets physicians with a definite level of experience in the topic using two screener questions. Opinion on clinical management was collected using hypothetical clinical situations. Each participant had the option of being contacted to further discuss the survey results. This was an online survey, and 264 physicians were contacted through email once a week for 5 weeks from September 2020. Results: In total, 64 physicians (24.2%) from 23 institutions participated (81.3% hematologists and 67.2% from university hospitals). All had access to flow cytometry for PNH clone testing, with 76.6% having access within their own institution. The main reasons to assess for PNH clones were unexplained thrombosis and/or hemolysis, and/or aplastic anemia (AA). Patients in FU for PNH clones were more likely to be aplastic anemia (AA) and symptomatic PNH. In total, 61% of the participants investigated PNH clones repetitively during FU in AA/myelodysplastic syndromes patients, even when there was no PNH clone found at diagnosis, and 75% of the participants tested at least once a year during FU. Opinions related to clinical management were scattered. Conclusion: The need to adhere to guidelines for the assessment, interpretation, and reporting of PNH clones emerges as the most important finding, as well as consensus for the management of less well-defined clinical situations. Even though there are several international guidelines, clear information addressing specific topics such as the type of anticoagulant to use and its duration, as well as the indication for treatment with complement inhibitors in some borderline situations are needed. The analysis and the discussion of this survey provide the basis for understanding the unmet needs of PNH clone assessment and clinical practice in Switzerland.
RESUMO
BACKGROUND: Fecal microbiota transplantation (FMT) represents a new therapeutic option that has been studied in two randomized-controlled trials in ulcerative colitis patients. Our study aimed to identify patients' views on the use of this novel therapeutic approach. METHODS: Using an anonymous questionnaire, we obtained data from 574 inflammatory bowel disease (IBD) patients on their knowledge and willingness to undergo FMT. RESULTS: A large proportion of IBD patients (53.5%) are unaware that FMT is a therapeutic option in Clostridium difficile infection and potentially IBD. More responders preferred FMT (31.5%) to a study with a new medication (28.9%), although the difference was not significant (P=0.37), and the preferred way of transplantation was colonoscopy (49.7%). In all, 38.3% preferred a family member as a donor, but there was fear about the procedure (41.5% mentioned fear of infectious diseases, 26.5% expressed disgust). The knowledge of successful FMT treatment in other patients was important for 82.2% of responders and for 50.7%, a discussion with a specialist would likely change their opinion about FMT. CONCLUSION: FMT represents a therapeutic procedure that is of interest for IBD patients. As FMT has been receiving increasing interest as an alternative treatment in IBD and more studies on FMT in IBD are being carried out, it is important to learn about the knowledge, attitude, and preferences of patients to provide better education to patients on this topic. However, there are reservations because of the fact that data on the benefits of FMT in IBD are controversial and several limitations exist on the use of FMT in IBD.