Aim: To develop a patient-reported outcome measure (PROM) assessing upper limb function related to activities of daily living (ADL) that cannot be observed in a clinical setting, specifically for patients with Duchenne muscular dystrophy (DMD) across a wide age range, applicable in the different stages of the disease. Method: The developmental process was based on US Food and Drug Administration guidelines. This included item generation from a systematic review of existing tools and expert opinion on task difficulty and relevance, involving individuals with DMD. Cultural aspects affecting ADL were taken into consideration to make this tool applicable to the broad DMD community. Items were selected in relation to a conceptual framework reflecting disease progression covering the full range of upper limb function across different ADL domains. Results: After pilot testing and iterative Rasch analyses, redundant or clinically irrelevant items were removed. The final questionnaire consists of 32 items covering four domains of ADL (food, self-care, household and environment, leisure and communication). Test–retest reliability was excellent. Interpretation: A DMD-specific upper limb PROM was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self-reported natural history of DMD and assess the efficacy of interventions.

Klingels, K., Mayhew, A. G., Mazzone, E. S., Duong, T., Decostre, V., Werlauff, U., Vroom, E., Mercuri, E. M., Goemans, N. M., Eagle, M., De Groot, I., Main, M., Messina, S., Campion, G., Servais, L., Van Den Hauwe, M., Es, E., Pane, M., Buccella, F., Kuijer, J., Ceradini, F., Henricson, E., Mcdonald, C., Florence, J., Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM, <<DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY>>, 2017; 59 (2): 224-231. [doi:10.1111/dmcn.13277] [https://hdl.handle.net/10807/260283]

Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM

Mazzone, Elena Stacy;Mercuri, Eugenio Maria;Pane, Marika;
2017

Abstract

Aim: To develop a patient-reported outcome measure (PROM) assessing upper limb function related to activities of daily living (ADL) that cannot be observed in a clinical setting, specifically for patients with Duchenne muscular dystrophy (DMD) across a wide age range, applicable in the different stages of the disease. Method: The developmental process was based on US Food and Drug Administration guidelines. This included item generation from a systematic review of existing tools and expert opinion on task difficulty and relevance, involving individuals with DMD. Cultural aspects affecting ADL were taken into consideration to make this tool applicable to the broad DMD community. Items were selected in relation to a conceptual framework reflecting disease progression covering the full range of upper limb function across different ADL domains. Results: After pilot testing and iterative Rasch analyses, redundant or clinically irrelevant items were removed. The final questionnaire consists of 32 items covering four domains of ADL (food, self-care, household and environment, leisure and communication). Test–retest reliability was excellent. Interpretation: A DMD-specific upper limb PROM was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self-reported natural history of DMD and assess the efficacy of interventions.
2017
Inglese
Klingels, K., Mayhew, A. G., Mazzone, E. S., Duong, T., Decostre, V., Werlauff, U., Vroom, E., Mercuri, E. M., Goemans, N. M., Eagle, M., De Groot, I., Main, M., Messina, S., Campion, G., Servais, L., Van Den Hauwe, M., Es, E., Pane, M., Buccella, F., Kuijer, J., Ceradini, F., Henricson, E., Mcdonald, C., Florence, J., Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM, <<DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY>>, 2017; 59 (2): 224-231. [doi:10.1111/dmcn.13277] [https://hdl.handle.net/10807/260283]
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/260283
Citazioni
  • ???jsp.display-item.citation.pmc??? 15
  • Scopus 32
  • ???jsp.display-item.citation.isi??? 31
social impact