Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH).

Anemia, Aplastic / therapy Hemoglobinuria, Paroxysmal / therapy Humans Patient Reported Outcome Measures Quality of Life Surveys and Questionnaires
Aplastic anemia Paroxysmal nocturnal hemoglobinuria Patient-reported outcome Quality of life Symptom

Journal

Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602

Informations de publication

Date de publication:
17 09 2020
Historique:
received: 10 03 2020
accepted: 07 09 2020
entrez: 18 9 2020
pubmed: 19 9 2020
medline: 19 5 2021
Statut: epublish

Résumé

The introduction of new therapy modalities has significantly improved the outcome of aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. However, relatively little is known about the exact disease burden of AA/PNH since standardized assessments of symptoms including health-related quality of life (HRQoL) are frequently missing or inadequately designed for this rare patient group. We aimed to develop AA/PNH-specific questionnaires for self-reporting of symptoms, which could be included in electronic platforms for data collection and patient care. By scoping review, we extracted any reported symptoms in AA/PNH and their prevalence from the literature (Phase I). Consensus rounds with patients and medical experts were conducted to identify core symptoms reported in the literature and to add missing items (Phase II). Ultimately, AA/PNH-specific patient-reported outcome (PRO) questionnaires including the selected measures were designed (Phase III). AA symptoms from 62 and PNH symptoms from 45 observational studies were extracted from the literature. Twenty-four patients and seven medical experts identified 11 core symptoms including HRQoL issues after three consensus rounds. Significant differences in the symptom ranking of patients versus medical experts could be observed. Therefore, patient- as well as expert-centered PRO questionnaires in AA and PNH were created following the concepts of validated instruments. The development of symptom self-reporting questionnaires for AA and PNH was feasible and the disease-specific PRO questionnaires can now be validated within a web-based workflow in a subsequent feasibility study.

Sections du résumé

BACKGROUND
The introduction of new therapy modalities has significantly improved the outcome of aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) patients. However, relatively little is known about the exact disease burden of AA/PNH since standardized assessments of symptoms including health-related quality of life (HRQoL) are frequently missing or inadequately designed for this rare patient group. We aimed to develop AA/PNH-specific questionnaires for self-reporting of symptoms, which could be included in electronic platforms for data collection and patient care.
METHODS
By scoping review, we extracted any reported symptoms in AA/PNH and their prevalence from the literature (Phase I). Consensus rounds with patients and medical experts were conducted to identify core symptoms reported in the literature and to add missing items (Phase II). Ultimately, AA/PNH-specific patient-reported outcome (PRO) questionnaires including the selected measures were designed (Phase III).
RESULTS
AA symptoms from 62 and PNH symptoms from 45 observational studies were extracted from the literature. Twenty-four patients and seven medical experts identified 11 core symptoms including HRQoL issues after three consensus rounds. Significant differences in the symptom ranking of patients versus medical experts could be observed. Therefore, patient- as well as expert-centered PRO questionnaires in AA and PNH were created following the concepts of validated instruments.
CONCLUSION
The development of symptom self-reporting questionnaires for AA and PNH was feasible and the disease-specific PRO questionnaires can now be validated within a web-based workflow in a subsequent feasibility study.

Identifiants

DOI: 10.1186/s13023-020-01532-3 PMID: 32943103 PMC: PMC7495826
pubmed: 32943103
doi: 10.1186/s13023-020-01532-3
pii: 10.1186/s13023-020-01532-3
pmc: PMC7495826
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

249

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Auteurs

Kimmo Weisshaar (K)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Hannah Ewald (H)

University Medical Library, University of Basel, 4051, Basel, Switzerland.
Basel Institute for Clinical Epidemiology and Biostatistics, Department of Clinical Research, University Hospital Basel, 4031, Basel, Switzerland.

Jörg Halter (J)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Sabine Gerull (S)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Sandra Schönfeld (S)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Yuliya Senft (Y)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Maria Martinez (M)

Department of Diagnostic Hematology, University of Basel, 4051, Basel, Switzerland.

Anne Leuppi-Taegtmeyer (A)

Department of Clinical Pharmacology and Toxicology, University and University Hospital Basel, 4051, Basel, Switzerland.

Nina Khanna (N)

Infectious Diseases and Hospital Epidemiology, University Hospital Basel, 4031, Basel, Switzerland.

Birgit Maier (B)

Department of Psychosomatic Medicine, University Hospital Basel, 4031, Basel, Switzerland.

Antonio Risitano (A)

Hematology, Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy.
Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, Netherlands.

Regis Peffault de Latour (R)

Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, Netherlands.
French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France.

Andre Tichelli (A)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Jakob Passweg (J)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland.

Beatrice Drexler (B)

Division of Hematology, University Hospital Basel, 4031, Basel, Switzerland. Beatrice.Drexler@usb.ch.
ORCID: 0000-0002-7046-0825

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Classifications MeSH

questionsmedicales.fr › Hémopathies et maladies lymphatiques › Hémopathies › Maladies de la moelle osseuse › Aplasies médullaires › Anémie aplasique › Development of a patient-reported outcome...
questionsmedicales.fr › Hémopathies et maladies lymphatiques › Hémopathies › Maladies de la moelle osseuse › Syndromes myélodysplasiques › Hémoglobinurie paroxystique › Development of a patient-reported outcome...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Development of a patient-reported outcome...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Collecte de données › Enquêtes et questionnaires › Enquêtes sur les soins de santé › Mesures des résultats rapportés par les patients › Development of a patient-reported outcome...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Mesures épidémiologiques › Démographie › État de santé › Qualité de vie › Development of a patient-reported outcome...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Collecte de données › Enquêtes et questionnaires › Development of a patient-reported outcome...