Development and Economic Evaluation of a Patient-Centered Care Model for Children With Duchenne Muscular Dystrophy: Protocol for a Quasi-Experimental Study
Notice bibliographique
Résumé
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare progressive muscular disease that primarily affects boys. A lack of comprehensive care for patients living with DMD is directly associated with a compromised quality of life (QoL) for those affected and their caregivers. This disease also has a huge economic impact on families as its treatment requires substantial direct, indirect, and informal care costs. OBJECTIVE: This study presents a protocol developed to evaluate the feasibility and efficacy of a patient-centered care (PCC) model for children with DMD. The care model was designed with the aim to empower families, improve QoL, and reduce economic burden on their families. METHODS: This study is planned as a quasi-experimental study that will enroll 70 consecutive families with boys (aged 5-15 years) with DMD visiting a tertiary care center. The study is being conducted in 2 phases (preintervention and postintervention phases, referred to as phase 1 and phase 2, respectively). During phase 1, the patients received routine care. The study is now in phase 2, with the intervention currently being administered. The intervention is based on the PCC model individualized by the intervention team. The model has a comprehensive DMD telecare component that includes teleconsultation as one of its key components to reduce in-person physician visits at the health facility. Teleconsultation is especially beneficial for late-ambulatory and nonambulatory patients. Data on economic burden are being collected for out-of-pocket expenses for both phases during in-person visits via telephone or messaging apps on a monthly basis. QoL data for patients and their primary caregivers are being collected at 3 time points (ie, time of enrollment, end of phase 1, and end of phase 2). Outcome measures are being assessed as changes in economic burden on families and changes in QoL scores. RESULTS: Participant recruitment began in July 2021. The study is ongoing and expected to be completed by March 2023. The findings based on baseline data are expected to be submitted for publication in 2023. CONCLUSIONS: This paper outlines a research proposal developed to study the impact of a PCC model for patients with DMD in low- and middle-income countries (LMICs). This study is expected to provide evidence of whether a multicomponent, patient-centric intervention could reduce economic burdens on families and improve their QoL. The results of this study could guide policy makers and health professionals in India and other LMICs to facilitate a comprehensive care program for patients living with DMD. The economic impact of a rare disease is an important consideration to formulate or evaluate any health policy or intervention related to new treatments and financial support schemes. TRIAL REGISTRATION: Clinical Trials Registry India (ICMR-NIMS) CTRI/2021/06/034274; https://www.ctri.nic.in/Clinicaltrials/pmaindet2.php?trialid=56650. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/42491.
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 | 0,048 | 0,037 |
| Méta-épidémiologie (sens strict) | 0,004 | 0,003 |
| Méta-épidémiologie (sens large) | 0,005 | 0,005 |
| Bibliométrie | 0,003 | 0,003 |
| Études des sciences et des technologies | 0,005 | 0,004 |
| Communication savante | 0,002 | 0,002 |
| Science ouverte | 0,005 | 0,003 |
| Intégrité de la recherche | 0,005 | 0,007 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,034 | 0,004 |
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 ».