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Journal of asthmaPatient-reported outcome measures in severe asthma:an expert consensusEva Martnez-Moragn,MD,PhDa,Ignacio Antepara Ercoreca,MD,PhDb,Mara Muoz Garca,PharmD PhDc,on behalf of the PROs-SA group of expertsd,Francisco Casas Maldonado,MD PhDe,Marta Calvn Lamas,PharmD PhDf,Eusebi Chiner Vives,MD PhDg,Carlos Crespo Diz,PharmD PhDh,David Daz-Prez,NursingDi,Ibon Eguiluz Gracia,MD PhDj,Sara Garca Gil,PharmDk,Ruperto Gonzlez-Prez,MD PhDl,Alicia Habernau Mena,MD PhDm,Tamara Hermida Valverde,MDn,Snia Jornet Montaa,PharmDo,Valentn Lpez-Carrasco,NursingDp,Icar Martnez Lpez,PharmD PhDq,Vicente Merino-Bohrquez,PharmDr,lvaro Moreno-Ancillo,MDs,Ana Cristina Mnguez Cabeza,PharmDt,Emilio Monte-Boquet,PharmD PhDu,Jose Luis Revuelta-Herrero,PharmDv and Silvia Snchez-Cuellar,MD PhDwaservicio de neumologa,hospital universitario Doctor Peset,Valencia,spain;bservicio de alergologa,hospital universitario de Basurto,Bilbao,spain;cservicio de farmacia,hospital universitario ramn y Cajal,IrYCIs,madrid,spain;dnominal group participants;eservicio de neumologa,hospital universitario Clnico san Cecilio,Granada,spain;fservicio de farmacia,Complexo hospitalario universitario a Corua,a Corua,spain;gservicio de neumologa,hospital universitario san Juan de alicante,alicante,spain;hservicio de farmacia,Complejo hospitalario universitario de Pontevedra,Pontevedra,spain;iservicio de neumologa,hospital universitario nuestra seora de Candelaria,santa Cruz de tenerife,spain;jservicio de alergologa,hospital regional universitario de mlaga,mlaga,spain;kservicio de farmacia,hospital universitario de Canarias,la laguna,spain;lservicio de alergologa,hospital universitario de Canarias,la laguna,spain;mservicio de alergologa,Complejo hospitalario de mrida,Badajoz,spain;nservicio de neumologa,hospital universitario Central de asturias,oviedo,spain;oservicio de farmacia,hospital universitari Joan XXIII,tarragona,spain;pservicio de alergologa,hospital universitario la Paz,madrid,spain;qservicio de farmacia,hospital universitari son Espases,Palma de mallorca,spain;rservicio de farmacia,hospital universitario Virgen de la macarena,sevilla,spain;sservicio de alergologa,hospital General nuestra seora del Prado,talavera de la reina,spain;tservicio de farmacia,hospital universitario de araba,lava,spain;uservicio de farmacia,hospital universitario y Politcnico la fe,Valencia,spain;vservicio de farmacia,hospital General universitario Gregorio maran,madrid,spain;wservicio de neumologa,hospital universitario ramn y Cajal,madrid,spainABSTRACTObjective:The study aimed to reach a consensus on the most relevant patient-reported outcomes(PROs),the corresponding measures(PROMs),and measurement frequency during severe asthma patient follow-up.Methods:Two Delphi rounds were conducted.The questionnaire was developed based on a systematic literature review,a focus group with patients,and a nominal group with experts.It assessed PROs relevance and the appropriateness(A)and feasibility(F)of PROMs using a Likert scale(1=totally agree;9=totally disagree).The consensus was established when 75%of participants agreed(1-3)or disagreed(7-9).Results:Sixty-three professionals(25 hospital pharmacists,14 allergists,13 pulmonologists,and 11 nurses)and 5 patients answered the Delphi questionnaire.A consensus was reached on all PROs regarding their relevance.Experts agreed on the use of ACT(A:95.24%;F:95.24%),mini AQLQ(A:93.65;F:79.37%),mMRC dyspnea scale(A:85.71%;F:85.71%),TAI(A:92.06%;F:85.71%),MMAS(A:75.40%;F:82%),and the dispensing register(A:96.83%;F:92.06%).Also considered suitable were:SNOT-22(A:90.48%;F:73.80%),PSQI(A:82.54;F:63.90%),HADS(A:82.54;F:64%),WPAI(A:77.78%;F:49.20%),TSQM-9(A:79.37;F:70.50%)and knowledge of asthma questionnaire(A:77%;F:68.80%);however,their use in clinical practice was considered unfeasible.Panelists also agreed on the appropriateness of EQ-5D,which was finally included despite being considered unfeasible(A:84.13%;F:67.20%)in clinical practice.Agreement was reached on using ACT,TAI,mMRC,and a dispensing register every three months;mini-AQLQ and MMAS every six months;and EQ-5D every twelve months.Conclusion:This consensus paves the way toward patient-centered care,promoting the development of strategies supporting routine assessment of PROs in severe asthma management.2023 the author(s).Published with license by taylor&francis Group,llC.CONTACT Eva martnez-moragn head of servicio de neumologa,hospital universitario Doctor Peset,avda Gaspar aguilar 90,Valencia 46017,spain.supplemental data for this article can be accessed online at https:/doi.org/10.1080/02770903.2023.2297372.https:/doi.org/10.1080/02770903.2023.2297372this is an open access article distributed under the terms of the Creative Commons attribution license(http:/creativecommons.org/licenses/by/4.0/),which permits unre-stricted use,distribution,and reproduction in any medium,provided the original work is properly cited.the terms on which this article has been published allow the posting of the accepted manuscript in a repository by the author(s)or with their consent.ARTICLE HISTORYreceived 16 october 2023revised 14 December 2023accepted 16 December 2023KEYWORDSsevere asthma;patient-centered care;patient-reported outcomes;patient-reported outcome measures2E.MARTNEZ-MORAGN ETAL.IntroductionAsthma is a serious global health problem,affecting 339 million people worldwide(1).It is a chronic inflammatory disease of the airways,usually associated with bronchial hyperresponsiveness and a variable degree of airflow limitation.Its severity can vary from intermittent to severe persistent,depending on the intensity of the process and response to treatment(2,3).Therefore,disease management requires a mul-tidisciplinary and individualized approach,which must be periodically adjusted to minimize symptoms and prevent exacerbations(4).Severe asthma is characterized by intense symp-tomatology,frequent exacerbations,multiple comor-bidities,and the need for high-intensity treatment(3,57).It affects between 3%and 10%of the asth-matic population(2,8)and has a major impact on their health-related quality of life(HRQoL)(79).In fact,despite high-intensity treatment,patients with severe disease are usually refractory to therapy,and poor symptom control affects their social,working,physical and mental wellbeing(2,6,8,10).In addition,this condition is associated with higher direct and indirect medical costs compared to mild or moderate asthma(11,12).Given the significant impact of severe asthma on HRQoL,disease management has moved toward a patient-centered approach.As a result,pharmacologic and behavioral interventions have become more wide-spread,targeting disease control,assessing and treating related risk factors and comorbidities,and improving patients HRQoL(4).Additionally,recent studies have recommended using asthma-specific HRQoL measures in clinical trials for moderate-to-severe asthma,in order to assess the long-term response to treatments(7,1315).In this context,patient-reported outcomes(PROs)play an increasingly important role when assessing the impact of severe asthma healthcare interventions(1618).PROs are reported directly by the patient regarding their health condition status associated with the health care or treatment(16).Therefore,their implementation in clinical practice is essential to improve shared decision-making,symptom monitoring and management,patient satisfaction,and HRQoL(19).Patient-reported outcome measures(PROMs)are the instruments used to report PROs.These tools must be carefully defined and accurately used to cap-ture relevant patient-reported information and com-pare it with other measurements(20).PROMs can be generic or disease-specific;they assess symptoms,functional and health status,and social and psycho-logical wellbeing(8,15).Multiple PROMs have been developed and validated in the context of airway dis-eases such as severe asthma(18).However,the increasing implementation of PROMs in clinical prac-tice(16)makes it necessary to standardize their use.This project aims to reach a consensus on the most relevant PROs,PROMs,and their measurement fre-quency during severe asthma patient follow-up,taking into account both the patients and healthcare profes-sionals(HCP)perspectives,within the Spanish National Health System framework.Material and methodsThe project was led and coordinated by a scientific committee consisting of healthcare professionals who are experts in the management of severe asthma:a pulmonologist(EM),an allergologist(IA),and a hos-pital pharmacist(MM).The project comprised five phases:1)literature review;2)focus group with patients;3)first scientific committee meeting;4)two nominal groups with healthcare professionals;5)Delphi consultation with patients and healthcare professionals,and 6)final sci-entific committee meeting(Figure 1).Literature reviewTo identify PROs,PROMs,and frequency of mea-surement used during severe asthma patient follow-up,a systematic literature review according to Preferred Reporting Items for Systematic Reviews and Meta-Analysis(PRISMA)guidelines(21)was per-formed by consulting the international PubMed/Medline database.Observational studies,randomized clinical trials,and systematic reviews published in English or Spanish between 03/01/2016 and 03/01/2020 were selected and Figure 1.Project phases.JOuRNAL OF ASTHMA3reviewed.Search terms and strategy are shown in Supplementary Table S1.In addition,Clinical Practice Guidelines(CPGs)for severe asthma with information on PROs and PROMs were reviewed.Focus groups with patientsThe focus group is a variant of the group interview in which participants describe their perceptions,opin-ions,beliefs,and attitudes toward a specific topic(22).An online focus group with severe asthma patients was conducted to explore the most relevant PROs and how they were assessed during medical visits from the patients perspective.Patients were contacted and invited to participate in the focus group by the Spanish patient advocacy group(Federacin Espaola de Asociaciones de Pacientes Alrgicos y con Enfermedades Respiratorias,FENAER).During the focus group,PROs identified in the literature were presented,and various questions were asked for discussion:the impact of the disease and its treatment on their daily life,the disease-related symptomatology,and their perception of the assess-ment of PROs during medical visits.First scientific committee meetingThe first online meeting with the scientific committee was organized to present the literature review results and the focus group conclusions.The objective of this meeting was to establish the most relevant PROs and PROMs to be presented in the nominal group meet-ings with healthcare professionals.Nominal groups with healthcare professionalsThe nominal group technique is a qualitative research methodology based on a semi-structured group dis-cussion that guarantees all participants have the opportunity to express their ideas,ensuring that their participation is balanced(23).Two online multidis-ciplinary nominal group meetings with healthcare professionals were held to reach a preliminary con-sensus on the most relevant PROs,PROMs,and their frequency of measurement,for their inclusion in the Delphi consultation.Members of the nominal groups were contacted and invited to participate in the nominal group meet-ings by the coordinating team(on behalf of the sci-entific committee).They were selected based on their experience in severe asthma management,PRO measurement,implementing strategies to standardize health outcomes,and their availability and interest in the project.The PROs and PROMs selected by the scientific committee were presented to the nominal groups.A selection of PROs and PROMs was established accord-ing to their relevance for severe asthma patient follow-up and availability in the current clinical set-ting.A consensus was reached when at least 70%of the healthcare professionals agreed on the inclusion/exclusion of the PRO and PROM.Delphi consultationThe Delphi technique is a widely used consensus method implemented in research to achieve a gen-eral agreement on a particular topic,preserving participants anonymity(24).It is typically con-ducted over consecutive rounds,answered by a panel of participants with relevant expertise in the research field(24,25).The survey rounds iteratively ask the panelists to rate different statements or questions,providing controlled feedback on the previous rounds group results(26).Participants may then adjust their initial ratings based on feed-back from the overall group in several subsequent iterations(27).Two-round Delphi consultations with patients and healthcare professionals were conducted between November 2021 and January 2022.Participants were given two weeks to respond to the questionnaire for each round.In addition,three reminders were sent to non-respondents during each period.Two versions of the electronic questionnaire were developed,one for healthcare professionals and the other for patients,to make it easier for the latter to understand the wording.Furthermore,patients were not asked directly about PROMs due to their lack of knowledge on the existing PROMs and the difficulty in deciding on their suitability and feasibility of use in clinical practice.Panelists rated their responses on a nine-point scale(1=Strongly disagree;2=Disagree;3=Moderately dis-agree;4=Somewhat disagree;5=Neither agree nor disagree;6=Somewhat agree;7=Moderately agree;8=Agree;9=Strongly agree).The questionnaire of the first round consisted of four parts.1.Baseline characteristics.Sociodemographic characteristics of healthcare professionals(age,gender,specialty,region,and years of 4E.MARTNEZ-MORAGN ETAL.experience)and patients(age,gender,region,and age at diagnosis)were collected.2.PROs.Panelists(healthcare professionals and patients)were asked to rate the relevance(R)of including each PRO(classified according to the nominal groups proposal)in severe asthma patient follow-up.3.PROMs.Panelists(healthcare professionals only)were requested to rate the appropriateness(A)and feasibility(F)of the predefined PROMs for each PRO(classified according to nominal groups proposal).4.Frequency of measurement.Panelists(health-care professionals and patients)were asked to rate the appropriateness(A)and feasibility(F)of two PROMs frequencies of measurement proposals.In the case of the questionnaire for patients,the frequency was related to PROs(PROMs were not presented to them).See an example in Supplementary Material 1.In the second-round,an individualized reminder of the option that most participants had indicated accompanied the questions,and the panelists had to decide whether to maintain their assessment or to modify it.Moreover,reasons for disagreement were explored through some additional questions,and pan-elists were asked to prioritize the PROMs to be employed during severe asthma patient follow-up.PanelistsPanelists were identified and invited to participate in
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