Swiss Survey on current practices and opinions on clinical constellations triggering the search for PNH clones.

This national survey investigated the current practice in Switzerland by collecting participants' opinions on paroxysmal nocturnal hemoglobinuria (PNH) clone assessment and clinical practice. 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. 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. 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. 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.

Informations de copyright

Copyright © 2023 Rovó, Gavillet, Drexler, Keller, Schneider, Colucci, Beauverd, Dorland, Pollak, Schmidt, De Gottardi, Bissig, Lehmann, Duchosal and Zeerleder.

Déclaration de conflit d'intérêts

AR has received research funding from Novartis, CSL Behring, and AG Alexion, as well as honoraria from AG Alexion and Novartis. She has also received honoraria for advisory board meetings from AG Alexion, BMS, Novartis, OrPhaSwiss GmbH, and Swedish Orphan Biovitrum AG. MG has made consulting for Alexion. BD has received honoraria from AG Alexion and Novartis and has received honoraria for advisory board meetings from AG Alexion and Novartis. YB has received honoraria for advisory board meetings from AbbVie, BMS, Jazz Pharmaceuticals, and Novartis. TL has received research funding to institution from Abbvie, Celgene, and Novartis, as well as honoraria for attending advisory board meetings from BMS, Novartis, Abbvie, OrPhaSwiss, and GmbH. SZ has received speakers fees and honoraria from Sanofi, Alexion, ViroPharma, and Jazz Pharma. HD was employed by Appletree CI Group AG. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.