Quality of Life and Related Factors in Patients Diagnosed with Mucopolysaccharidosis and Their Caregivers.

Abstract

Objectives: Quality of life (QoL) is directly associated with physical-psychological well-being, family and environmental factors in patients with Mucopolysaccharidosis (MPS). The present study aims to investigate the factors affecting QoL in both MPS patients and their caregivers.

Methods: The sociodemographic characteristics, clinical findings, and special needs of 37 patients with MPS were questioned, among which 28 pediatric patients were evaluated for QoL using the KINDL-parents scale. The World Health Organization Quality of Life Assessment (WHO-QoL-BREF) was used to assess the QoL of 37 caregivers of patients.

Results: The patients with MPS type III had the lowest median score in the self-esteem domain among the other types. The patients undergoing enzyme replacement therapy scored high in the friends domain, while the physical-psychological/emotional domain scores of the patients with sleep disturbance and their parents were lower than those without. Patients with appetite/eating problems scored low in the self-esteem domain and their caregivers scored low in the social relationship domain. The psychological/emotional domain scores of the patients who needed special education and their parents were lower than those who did not. Total scores and KINDL disease domain scores were decreased in those receiving psychiatric medical treatment.

Conclusions: In this study, the aim was to simultaneously assess the quality of life of MPS patients and their parents. Sleep disturbances, appetite problems, special education, and psychiatric treatment negatively impacted QoL.