Patient-Reported Outcome Measures (PROMs) in Pediatric Non-Malignant Hematology: A Systematic Review
Notice bibliographique
Résumé
Introduction: Patient reported outcome measures (PROMs) are questionnaires completed by patients or caregivers without interpretation by healthcare professionals. As such, they allow patient concerns about a variety of healthcare issues to be identified and addressed in an efficient and actionable manner. PROMs can be generic, with questions relevant to multiple disease groups or disease-specific, with questions targeting the symptoms, limitations, and feelings common to the disease group. This systematic review identified generic and disease-specific PROMs for monitoring symptoms and health-related quality of life (HRQoL) in 4 pediatric non-malignant hematologic disease groups: thalassemia, hemophilia, immune thrombocytopenia (ITP), and sickle cell disease (SCD). Methods: Databases (MEDLINE, Embase, HaPI, CINAHL, and PsycTESTS) were searched to identify publications that either validated or used PROMs as an outcome measure in the four disease groups. Articles were excluded when <30% of the population was pediatric (<18 years), when the study setting was inpatient, when the tool had not been validated, or when the article did not report the use of a PROM for monitoring symptoms or HRQoL. Notably, hemophilia records published prior to 2016 were not screened as a systematic review by Limperg et al. (2017) identified validated PROMs in the pediatric hemophilia population and was used to include relevant articles. Results: The search identified 1176 unique records, with 902 records remaining for title and abstract screening after removal of 274 hemophilia articles published prior to the systematic review. Including hemophilia records identified from the 2017 review, 217 articles met inclusion criteria incorporating 107 generic and 20 disease-specific PROMs. Of the generic tools, the most frequent categories identified include psychological well-being (26 tools), general quality of life (19 tools), and family impact (19 tools). The most frequently used tool was the PedsQL 4.0 Generic Core Scales (66 studies), appearing 33 times in SCD, 25 times in thalassemia, 5 times in ITP, and 3 times in hemophilia. Other commonly used generic tools include the Short Form Health Survey, Child Health Questionnaire, PROMIS Health Measures, and Child Behaviour Checklist (Table). Disease-specific tools identified in the review include the PedsQL SCD Module, Kids ITP Tool, Haemo-QoL, CHO-KLAT, and TranQol (Table). In addition, 10 studies reported on pain diaries and 9 of these studies were SCD focused, the other being hemophilia focused. Conclusion: This systematic review identified several generic and disease-specific PROMs that have been used in pediatric non-malignant hematology. Although generic tools have been used more frequently, many disease-specific tools have been validated and are available for use in the clinical environment. We are currently conducting focus groups with patients, parents, and clinicians to determine the optimal choice of tools for monitoring symptoms and HRQoL in the pediatric non-malignant clinical environment. Disclosures No relevant conflicts of interest to declare.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,013 | 0,067 |
| Méta-épidémiologie (sens strict) | 0,001 | 0,001 |
| Méta-épidémiologie (sens large) | 0,007 | 0,007 |
| Bibliométrie | 0,010 | 0,014 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,002 | 0,003 |
| Science ouverte | 0,002 | 0,002 |
| Intégrité de la recherche | 0,002 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,005 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».