Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study
Background: Gaucher disease (GD), a rare lysosomal storage disorder, is associated with considerable patient and caregiver burden. We examined the applicability of existing caregiver questionnaires and assessed the level of burden in caregivers of patients with GD. Methods: This cross-sectional, non...
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| Format: | Article |
| Language: | English |
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Elsevier
2023-09-01
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| Series: | Molecular Genetics and Metabolism Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S221442692300040X |
| _version_ | 1797737351380729856 |
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| author | Yuta Koto Aya Narita Shinichi Noto Masafumi Okada Midori Ono Terumi Baba Rieko Sagara Norio Sakai |
| author_facet | Yuta Koto Aya Narita Shinichi Noto Masafumi Okada Midori Ono Terumi Baba Rieko Sagara Norio Sakai |
| author_sort | Yuta Koto |
| collection | DOAJ |
| description | Background: Gaucher disease (GD), a rare lysosomal storage disorder, is associated with considerable patient and caregiver burden. We examined the applicability of existing caregiver questionnaires and assessed the level of burden in caregivers of patients with GD. Methods: This cross-sectional, non-interventional study was conducted in Japan. Caregivers of patients with confirmed GD (any type) were recruited (patient association group and referral) for pre-testing (May 2021) or the main survey (October–December 2021). Caregivers completed the Caregiver Impact Questionnaire (CIQ; 30 items) and Zarit Caregiver Burden Interview (ZBI; 22 items) on paper. Total CIQ and ZBI scores and subscores were determined overall and by GD type. Inter-item correlations and test-retest reliability (2 rounds, 2 weeks apart) were calculated. The relationship between caregiving duration and caregiver burden was also analyzed. Results: Nine caregivers (type 2 [GD2]: n = 6; type 3 [GD3]: n = 3) and 25 caregivers (type 1 [GD1]: n = 2; GD2: n = 17; GD3: n = 6) completed the pre-test and main survey, respectively. In the main survey, mean total CIQ score, all CIQ subscores (except emotional function), and total ZBI score were highest in caregivers of patients with GD2 compared with caregivers of patients with GD1/GD3. High test-retest reliability (Kappa >0.6) was observed for 15 CIQ items and 16 ZBI items. CIQ and ZBI scores appeared to be positively correlated with each other and negatively correlated with caregiving duration. Conclusions: The CIQ and ZBI are applicable, reliable measures to assess burden in caregivers of patients with GD in Japan. Caregiver burden was highest in caregivers of patients with GD2 and decreased with caregiving duration. |
| first_indexed | 2024-03-12T13:27:26Z |
| format | Article |
| id | doaj.art-63ae56dbca7d466f9bb2e3366d7266cd |
| institution | Directory Open Access Journal |
| issn | 2214-4269 |
| language | English |
| last_indexed | 2024-03-12T13:27:26Z |
| publishDate | 2023-09-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Genetics and Metabolism Reports |
| spelling | doaj.art-63ae56dbca7d466f9bb2e3366d7266cd2023-08-25T04:24:15ZengElsevierMolecular Genetics and Metabolism Reports2214-42692023-09-0136100994Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based studyYuta Koto0Aya Narita1Shinichi Noto2Masafumi Okada3Midori Ono4Terumi Baba5Rieko Sagara6Norio Sakai7Child Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, 2-2 Yamadaoka, Suita-shi, Osaka 565-0871, JapanDivision of Child Neurology, Institute of Neurological Science, Tottori University Faculty of Medicine, 86 Nishi-cho, Yonago-shi, Tottori 683-8503, JapanDepartment of Rehabilitation, Niigata University of Health and Welfare, 1398 Shimami-cho, Kita-ku, Niigata-shi, Niigata 950-3198, Japan; Corresponding author.Real-World Evidence Solutions & HEOR, IQVIA Solutions Japan K.K., 4-10-18 Takanawa, Minato-ku, Tokyo 108-0074, JapanJapan Medical Office, Takeda Pharmaceutical Company Limited, 2-1-1 Nihonbashi-Honcho, Chuo-ku, Tokyo 103-8688, JapanJapan Medical Office, Takeda Pharmaceutical Company Limited, 2-1-1 Nihonbashi-Honcho, Chuo-ku, Tokyo 103-8688, JapanJapan Medical Office, Takeda Pharmaceutical Company Limited, 2-1-1 Nihonbashi-Honcho, Chuo-ku, Tokyo 103-8688, JapanChild Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, 2-2 Yamadaoka, Suita-shi, Osaka 565-0871, JapanBackground: Gaucher disease (GD), a rare lysosomal storage disorder, is associated with considerable patient and caregiver burden. We examined the applicability of existing caregiver questionnaires and assessed the level of burden in caregivers of patients with GD. Methods: This cross-sectional, non-interventional study was conducted in Japan. Caregivers of patients with confirmed GD (any type) were recruited (patient association group and referral) for pre-testing (May 2021) or the main survey (October–December 2021). Caregivers completed the Caregiver Impact Questionnaire (CIQ; 30 items) and Zarit Caregiver Burden Interview (ZBI; 22 items) on paper. Total CIQ and ZBI scores and subscores were determined overall and by GD type. Inter-item correlations and test-retest reliability (2 rounds, 2 weeks apart) were calculated. The relationship between caregiving duration and caregiver burden was also analyzed. Results: Nine caregivers (type 2 [GD2]: n = 6; type 3 [GD3]: n = 3) and 25 caregivers (type 1 [GD1]: n = 2; GD2: n = 17; GD3: n = 6) completed the pre-test and main survey, respectively. In the main survey, mean total CIQ score, all CIQ subscores (except emotional function), and total ZBI score were highest in caregivers of patients with GD2 compared with caregivers of patients with GD1/GD3. High test-retest reliability (Kappa >0.6) was observed for 15 CIQ items and 16 ZBI items. CIQ and ZBI scores appeared to be positively correlated with each other and negatively correlated with caregiving duration. Conclusions: The CIQ and ZBI are applicable, reliable measures to assess burden in caregivers of patients with GD in Japan. Caregiver burden was highest in caregivers of patients with GD2 and decreased with caregiving duration.http://www.sciencedirect.com/science/article/pii/S221442692300040XCaregiver burdenGaucher diseaseJapanCaregiver Impact QuestionnaireNeuronopathic Gaucher diseaseZarit Caregiver Burden Interview |
| spellingShingle | Yuta Koto Aya Narita Shinichi Noto Masafumi Okada Midori Ono Terumi Baba Rieko Sagara Norio Sakai Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study Molecular Genetics and Metabolism Reports Caregiver burden Gaucher disease Japan Caregiver Impact Questionnaire Neuronopathic Gaucher disease Zarit Caregiver Burden Interview |
| title | Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study |
| title_full | Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study |
| title_fullStr | Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study |
| title_full_unstemmed | Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study |
| title_short | Burden of caregivers of patients with neuronopathic and non-neuronopathic Gaucher disease in Japan: A survey-based study |
| title_sort | burden of caregivers of patients with neuronopathic and non neuronopathic gaucher disease in japan a survey based study |
| topic | Caregiver burden Gaucher disease Japan Caregiver Impact Questionnaire Neuronopathic Gaucher disease Zarit Caregiver Burden Interview |
| url | http://www.sciencedirect.com/science/article/pii/S221442692300040X |
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