Analysis of platelets by flow cytometry in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Araten, David J.; Boxer, Daniel; Zamechek, Leah; Sherman, Erik; Nardi, Michael A.

doi:10.1016/j.bcmd.2019.102372

Cited by 3 publications

(1 citation statement)

References 34 publications

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“…The hypercoagulable state in PNH is multifactorial. The contribution of platelets derived from the mutant stem cell clone susceptible to complement-mediated activation plays an important role in the pathogenesis of thrombophilia in this situation ( 62 ); however, clinical data demonstrating a benefit of anti-aggregation in PNH are lacking, and in line with this, none of the participants considered aspirin as a valid therapeutic option.…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2023

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

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Section: Discussionmentioning

confidence: 99%