Patient-Reported Characteristics of Pernicious Anaemia:A first step to initiate James Lind Alliance Priority Setting Partnership Driven Research

Author:

Thain AlfieORCID,Visser PetraORCID,Hart KathrynORCID,Nexo EbbaORCID,McCaddon AndrewORCID,Hannibal LucianaORCID,Wolffenbuttel Bruce HRORCID,Green RalphORCID,Ward NicolaORCID,Seage Catherine HeidiORCID,Owen JulianORCID,Burchell Katrina,Dib Marie-JoeORCID,Ahmadi Kourosh RORCID

Abstract

AbstractObjectivePernicious anaemia (PA) is characterised by vitamin B12 deficiency due to autoimmune-mediated loss of gastric parietal cells and intrinsic factor. The Pernicious Anaemia Society (PAS) identified 10 research priorities for PA through a James-Lind Alliance Priority Setting Partnership (JLA-PSP). This study aimed to survey PAS members to identify and characterise a cohort of patients to form a PA research repository.MethodsAn online survey was designed using SurveyMonkey, comprising 21 questions on diagnosis, comorbidities, family history, and management. The survey was sent to 3,482 PAS members (April-September 2022) via the PAS website and email.ResultsAtotal of 1,191 PAS members completed the survey. Among individuals with a probable (n=471) or suspected PA (n=500) diagnosis, 84% were UK-based, and 81% were female, with an age range of 23-93 years. Diagnosis was predominantly based on low serum B12 (50%), positive intrinsic factor (38%), and/or parietal cell autoantibodies (15%). Diagnostic delays were common, with 37% waiting ≥3 years for a diagnosis. Nearly half had one or more other autoimmune diseases. One-third reported having at least 2 and up to 7 family members with PA or other autoimmune diseases. Vitamin B12 treatment frequency varied widely, ranging from daily to 3-monthly injections.ConclusionThis study highlights gaps in current diagnostic and management approaches for PA, paving the way for future work in line with the JLA-PSP research priorities. By characterising a cohort of PA patients and compiling baseline data, we provide a foundation for research to develop more effective diagnostic and management strategies.

Publisher

Cold Spring Harbor Laboratory

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