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

Alicia Rovó; Mathilde Gavillet; Beatrice Drexler; Peter Keller; Jenny Sarah Schneider; Giuseppe Colucci; Yan Beauverd; Hendrika Anette van Dorland; Matthias Pollak; Adrian Schmidt; Andrea De Gottardi; Marina Bissig; Thomas Lehmann; Michel A Duchosal; Sacha Zeerleder

doi:10.3389/fmed.2023.1200431

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

Front Med (Lausanne). 2023 Jul 26:10:1200431. doi: 10.3389/fmed.2023.1200431. eCollection 2023.

Authors

Alicia Rovó¹, Mathilde Gavillet^{2

3}, Beatrice Drexler⁴, Peter Keller⁵, Jenny Sarah Schneider¹, Giuseppe Colucci⁶, Yan Beauverd⁷, Hendrika Anette van Dorland⁸, Matthias Pollak¹, Adrian Schmidt⁹, Andrea De Gottardi¹⁰, Marina Bissig¹¹, Thomas Lehmann¹², Michel A Duchosal², Sacha Zeerleder¹³

Affiliations

¹ Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
² Service and Central Laboratory of Hematology, Department of Oncology, Lausanne University Hospital (CHUV), Lausanne, Switzerland.
³ Department of Laboratory Medicine and Pathology, Lausanne University Hospital (CHUV), Lausanne, Switzerland.
⁴ Division of Hematology, University Hospital Basel, Basel, Switzerland.
⁵ Hospital Langenthal, Langenthal, Switzerland.
⁶ Clinica Sant'Anna, Lugano, Switzerland.
⁷ Division of Hematology, Geneva University Hospitals and Faculty of Medicine, Geneva, Switzerland.
⁸ Appletree CI Group AG, Winterthur, Switzerland.
⁹ Department of Internal Medicine, Clinic of Medical Oncology and Hematology, Municipal Hospital Zurich Triemli, Zürich, Switzerland.
¹⁰ Servizio di Gastroenterología e Epatologia, Ente Ospedaliero Cantonale, Università della Svizzera Italiana, Lugano, Switzerland.
¹¹ Department of Medical Oncology and Hematology, University Hospital of Zürich, Zürich, Switzerland.
¹² Kantonsspital St. Gallen, Clinic for Medical Oncology and Hematology, St. Gallen, Switzerland.
¹³ Department of Hematology, Kantonsspital Luzern, Lucerne and University of Bern, Bern, Switzerland.

Abstract

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.

Keywords: PNH clone; aplastic anemia; bone marrow failure (BMF); paroxysmal nocturnal hemoglobinuria (PNH); survey.

Grants and funding

This study was supported by a grant from Alexion Pharmaceuticals, Inc. Alexion had no say in the survey design and no member of Alexion took part in the survey. Alexion provided a courtesy scientific accuracy review; however, the authors retain control and final authority of publication content and decisions, including the choice of journal. Alcimed received a fee from the University Hospital of Bern to launch the survey. The medical writing support was funded by Alexion, AstraZeneca Rare Disease.