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.

Zusammenfassung

Ziele Die Lebensqualität (QoL) bei Patienten mit Mukopolysaccharidose (MPS) ist direkt mit körperlich-psychologischem Wohlbefinden sowie familiären und umweltbedingten Faktoren assoziiert. Die vorliegende Studie zielt darauf ab, die Faktoren zu untersuchen, die die QoL von sowohl MPS-Patienten als auch ihren Betreuungspersonen beeinflussen.

Methode Die soziodemographischen Charakteristika, klinischen Befunde und besonderen Bedürfnisse von 37 Patienten mit MPS wurden abgefragt. Darunter wurden 28 pädiatrische Patienten mit der KINDL-Elternskala auf ihre QoL hin evaluiert. Die Lebensqualitätsbewertung der Weltgesundheitsorganisation (WHO-QoL-BREF) wurde verwendet, um die QoL von 37 Betreuungspersonen von Patienten zu bewerten.

Ergebnisse Die Patienten mit MPS Typ III hatten unter den anderen Typen den niedrigsten Medianwert im Bereich des Selbstwertgefühls. Die Patienten, die eine Enzymersatztherapie machen, hatten hohe Werte im Bereich Freundschaften.Im physisch-psychologischen/emotionalen Bereich waren die Werte der Patienten mit Schlafstörungen und ihrer Eltern niedriger als bei denen ohne. Patienten mit Appetit-/Essproblemen hatten niedrige Werte im Bereich Selbstbewusstsein und ihre Betreuungspersonen hatten niedrige Werte im Bereich soziale Beziehungen. Die Werte im psychologischen/emotionalen Bereich von Patienten, die Sonderpädagogik benötigten, und ihrer Eltern waren niedriger als die derjenigen, die keine brauchten. Die Gesamtergebnisse und KINDL-Krankheitsbereichsergebnisse waren bei denjenigen vermindert, die eine psychiatrische medikamentöse Behandlung erhielten.

Schlussfolgerung In dieser Studie wurde die Lebensqualität von MPS-Patienten und deren Eltern bewertet. Schlafstörungen, Appetitprobleme, Sonderpädagogik und psychiatrische Behandlungen beeinträchtigten die Lebensqualität.

Keywords

quality of life - mucopolysaccharidosis - KINDL - WHO-QoL-BREF

Schlüsselwörter

Lebensqualität - Mukopolysaccharidose - KINDL - WHO-QoL-BREF

Publication History

Article published online:
10 December 2024

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