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Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency
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| Zeitschriftentitel: | Blood Advances |
|---|---|
| Personen und Körperschaften: | , , , , , , , , , , , , , |
| In: | Blood Advances, 6, 2022, 6, S. 1844-1853 |
| Format: | E-Article |
| Sprache: | Englisch |
| veröffentlicht: |
American Society of Hematology
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| Schlagwörter: |
| author_facet |
Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. |
|---|---|
| author |
Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. |
| spellingShingle |
Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. Blood Advances Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency Hematology |
| author_sort |
al-samkari, hanny |
| spelling |
Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. 2473-9529 2473-9537 American Society of Hematology Hematology http://dx.doi.org/10.1182/bloodadvances.2021004675 <jats:title>Abstract</jats:title> <jats:p>Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Although recognition of the disease spectrum has recently expanded, data describing its impact on health-related quality of life (HRQoL) are limited. In this prospective international cohort of 254 patients (131 adults and 123 children) with PKD, we used validated measures to assess the impact of disease on HRQoL (EuroQol 5-Dimension Questionnaire, Pediatric Quality of Life Inventory Generic Core Scale version 4.0, and Functional Assessment of Cancer Therapy-Anemia) and fatigue (Patient Reported Outcomes Measurement Information System Fatigue and Pediatric Functional Assessment of Chronic Illness Therapy-Fatigue). Significant variability in HRQoL and fatigue was reported for adults and children, although individual scores were stable over a 2-year interval. Although adults who were regularly transfused reported worse HRQoL and fatigue compared with those who were not (EuroQol-visual analog scale, 58 vs 80; P = .01), this difference was not seen in children. Regularly transfused adults reported lower physical, emotional, and functional well-being and more anemia symptoms. HRQoL and fatigue significantly differed in children by genotype, with the worst scores in those with 2 severe PKLR mutations; this difference was not seen in adults. However, iron chelation was associated with significantly worse HRQoL scores in children and adults. Pulmonary hypertension was also associated with significantly worse HRQoL. Additionally, 59% of adults and 35% of children reported that their jaundice upset them, identifying this as an important symptom for consideration. Although current treatments for PKD are limited to supportive care, new therapies are in clinical trials. Understanding the impact of PKD on HRQoL is important to assess the utility of these treatments. This trial was registered at www.clinicaltrials.gov as #NCT02053480.</jats:p> Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency Blood Advances |
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| title |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_unstemmed |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_full |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_fullStr |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_full_unstemmed |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_short |
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_sort |
health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| topic |
Hematology |
| url |
http://dx.doi.org/10.1182/bloodadvances.2021004675 |
| publishDate |
2022 |
| physical |
1844-1853 |
| description |
<jats:title>Abstract</jats:title>
<jats:p>Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Although recognition of the disease spectrum has recently expanded, data describing its impact on health-related quality of life (HRQoL) are limited. In this prospective international cohort of 254 patients (131 adults and 123 children) with PKD, we used validated measures to assess the impact of disease on HRQoL (EuroQol 5-Dimension Questionnaire, Pediatric Quality of Life Inventory Generic Core Scale version 4.0, and Functional Assessment of Cancer Therapy-Anemia) and fatigue (Patient Reported Outcomes Measurement Information System Fatigue and Pediatric Functional Assessment of Chronic Illness Therapy-Fatigue). Significant variability in HRQoL and fatigue was reported for adults and children, although individual scores were stable over a 2-year interval. Although adults who were regularly transfused reported worse HRQoL and fatigue compared with those who were not (EuroQol-visual analog scale, 58 vs 80; P = .01), this difference was not seen in children. Regularly transfused adults reported lower physical, emotional, and functional well-being and more anemia symptoms. HRQoL and fatigue significantly differed in children by genotype, with the worst scores in those with 2 severe PKLR mutations; this difference was not seen in adults. However, iron chelation was associated with significantly worse HRQoL scores in children and adults. Pulmonary hypertension was also associated with significantly worse HRQoL. Additionally, 59% of adults and 35% of children reported that their jaundice upset them, identifying this as an important symptom for consideration. Although current treatments for PKD are limited to supportive care, new therapies are in clinical trials. Understanding the impact of PKD on HRQoL is important to assess the utility of these treatments. This trial was registered at www.clinicaltrials.gov as #NCT02053480.</jats:p> |
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| description | <jats:title>Abstract</jats:title> <jats:p>Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Although recognition of the disease spectrum has recently expanded, data describing its impact on health-related quality of life (HRQoL) are limited. In this prospective international cohort of 254 patients (131 adults and 123 children) with PKD, we used validated measures to assess the impact of disease on HRQoL (EuroQol 5-Dimension Questionnaire, Pediatric Quality of Life Inventory Generic Core Scale version 4.0, and Functional Assessment of Cancer Therapy-Anemia) and fatigue (Patient Reported Outcomes Measurement Information System Fatigue and Pediatric Functional Assessment of Chronic Illness Therapy-Fatigue). Significant variability in HRQoL and fatigue was reported for adults and children, although individual scores were stable over a 2-year interval. Although adults who were regularly transfused reported worse HRQoL and fatigue compared with those who were not (EuroQol-visual analog scale, 58 vs 80; P = .01), this difference was not seen in children. Regularly transfused adults reported lower physical, emotional, and functional well-being and more anemia symptoms. HRQoL and fatigue significantly differed in children by genotype, with the worst scores in those with 2 severe PKLR mutations; this difference was not seen in adults. However, iron chelation was associated with significantly worse HRQoL scores in children and adults. Pulmonary hypertension was also associated with significantly worse HRQoL. Additionally, 59% of adults and 35% of children reported that their jaundice upset them, identifying this as an important symptom for consideration. Although current treatments for PKD are limited to supportive care, new therapies are in clinical trials. Understanding the impact of PKD on HRQoL is important to assess the utility of these treatments. This trial was registered at www.clinicaltrials.gov as #NCT02053480.</jats:p> |
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| spelling | Al-Samkari, Hanny van Beers, Eduard J. Morton, D. Holmes Eber, Stefan W. Chonat, Satheesh Kuo, Kevin H. M. Kollmar, Nina Wang, Heng Breakey, Vicky R. Sheth, Sujit Sharma, Mukta Forbes, Peter W. Klaassen, Robert J. Grace, Rachael F. 2473-9529 2473-9537 American Society of Hematology Hematology http://dx.doi.org/10.1182/bloodadvances.2021004675 <jats:title>Abstract</jats:title> <jats:p>Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Although recognition of the disease spectrum has recently expanded, data describing its impact on health-related quality of life (HRQoL) are limited. In this prospective international cohort of 254 patients (131 adults and 123 children) with PKD, we used validated measures to assess the impact of disease on HRQoL (EuroQol 5-Dimension Questionnaire, Pediatric Quality of Life Inventory Generic Core Scale version 4.0, and Functional Assessment of Cancer Therapy-Anemia) and fatigue (Patient Reported Outcomes Measurement Information System Fatigue and Pediatric Functional Assessment of Chronic Illness Therapy-Fatigue). Significant variability in HRQoL and fatigue was reported for adults and children, although individual scores were stable over a 2-year interval. Although adults who were regularly transfused reported worse HRQoL and fatigue compared with those who were not (EuroQol-visual analog scale, 58 vs 80; P = .01), this difference was not seen in children. Regularly transfused adults reported lower physical, emotional, and functional well-being and more anemia symptoms. HRQoL and fatigue significantly differed in children by genotype, with the worst scores in those with 2 severe PKLR mutations; this difference was not seen in adults. However, iron chelation was associated with significantly worse HRQoL scores in children and adults. Pulmonary hypertension was also associated with significantly worse HRQoL. Additionally, 59% of adults and 35% of children reported that their jaundice upset them, identifying this as an important symptom for consideration. Although current treatments for PKD are limited to supportive care, new therapies are in clinical trials. Understanding the impact of PKD on HRQoL is important to assess the utility of these treatments. This trial was registered at www.clinicaltrials.gov as #NCT02053480.</jats:p> Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency Blood Advances |
| spellingShingle | Al-Samkari, Hanny, van Beers, Eduard J., Morton, D. Holmes, Eber, Stefan W., Chonat, Satheesh, Kuo, Kevin H. M., Kollmar, Nina, Wang, Heng, Breakey, Vicky R., Sheth, Sujit, Sharma, Mukta, Forbes, Peter W., Klaassen, Robert J., Grace, Rachael F., Blood Advances, Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency, Hematology |
| title | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_full | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_fullStr | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_full_unstemmed | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_short | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_sort | health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| title_unstemmed | Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency |
| topic | Hematology |
| url | http://dx.doi.org/10.1182/bloodadvances.2021004675 |