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Eur J Haematol. 2018 Jul;101(1):3-11. doi: 10.1111/ejh.13059. Epub 2018 May 16.

Screening and diagnostic clinical algorithm for paroxysmal nocturnal hemoglobinuria: Expert consensus.

Author information

1
University Hospital Essen, Essen, Germany.
2
Cleveland Clinic, Cleveland, OH, USA.
3
Osaka University Graduate School of Medicine, Osaka, Japan.
4
Alexion Pharmaceuticals, Inc., New Haven, CT, USA.
5
McMaster University and the Thrombosis and Atherosclerosis Research Institute, Hamilton, ON, Canada.

Abstract

OBJECTIVE:

Paroxysmal 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.

METHODS:

An 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.

RESULTS:

Consensus was reached for 95% of screening and diagnostic decision points and 90% of tests required at decision points.

CONCLUSION:

These results facilitated development of a consensus-based, clinically relevant algorithm, providing non-specialist clinicians with actionable guidance on PNH screening and diagnosis.

KEYWORDS:

aplastic anemia and bone marrow failure; red cell disorders

PMID:
29532535
DOI:
10.1111/ejh.13059
[Indexed for MEDLINE]

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