Screening and diagnostic clinical algorithm for paroxysmal nocturnal hemoglobinuria: Expert consensus
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AbstractObjectiveParoxysmal nocturnal hemoglobinuria (PNH) is a severe, life‐threatening disorder for which early diagnosis is essential. However, given the rarity of the disease and non‐specificity of symptoms, correct diagnosis may be delayed or missed. While various hematologic guidelines note common signs and symptoms associated with PNH, international expert consensus based on real‐world clinical experience and an actionable algorithm for non‐specialists to facilitate screening and diagnosis are lacking. The objective of the study is to develop a clinically relevant, consensus‐driven screening and diagnostic algorithm on PNH for non‐specialist clinicians.MethodsAn expert advisory committee of PNH experts from North America, Europe, and Japan was convened, and a modified Delphi methodology was employed to develop an algorithm to assist non‐specialist clinicians in identifying signs/symptoms of PNH and conducting appropriate differential diagnosis. Twelve globally representative Delphi panelists with clinical expertise in PNH were identified and recruited. Panelists provided their differential diagnosis for 5 blinded case studies via 2 rounds of online questionnaires. Responses mentioned by >50% of panelists in the first round were included in the second‐round questionnaire, at which point consensus was attained if >80% of panelists agreed on an approach.ResultsConsensus was reached for 95% of screening and diagnostic decision points and 90% of tests required at decision points.ConclusionThese results facilitated development of a consensus‐based, clinically relevant algorithm, providing non‐specialist clinicians with actionable guidance on PNH screening and diagnosis.
