Contributions
Abstract: PS1444
Type: Poster Presentation
Presentation during EHA23: On Saturday, June 16, 2018 from 17:30 - 19:00
Location: Poster area
Background
Pyruvate kinase deficiency (PKD) is the most common enzymopathy of anaerobic glycolysis in erythrocytes. PKD is a rare autosomal recessive disease with a prevalence of 1 in 20.000 people causing a hereditary non- spherocytic hemolytic anemia (Zanella et al 2005). There are no published studies concerning health-related quality of life (HRQoL) in patients with PKD.
Aims
This study describes HRQoL in children with PKD in Germany.
Methods
16 pediatric patients with compound heterozygous or homozygous PKD who participated in Germany in the PKD Natural History Study were included in the analysis. The German version of the PedsQL 4.0 (Pediatric Quality of Life) questionnaire was used to assess HRQoL. The patients were divided into 3 groups according to age in years: 2-4 (n=8), 5-7 (n=2), 8-18 (n=6). 63% of the patients (n=10) had severe anemia (Hb< 8 g/dl), 4 % (n=1) moderate anemia (Hb 8-10 g/dl) and 33% (n=5) mild anemia (Hb 10-12 g/dl). The questionnaire was answered by all children ages≥5 years and by parents of children of all ages. The answers were transformed into a score from 0 to 100 (with 100 representing the best HRQoL) and were compared using the Mann-Whitney Test. PedsQL≥81 is considered an indicator of a good HRQoL , 61-80 intermediate HRQoL and ≤60 poor HRQoL (Beverung et al 2015).
Results
The mean parental PedsQL scores of all 16 children were 79.10 (range: 0-100) in the physical- and 79.81 (range: 21.15 -100) in the psychosocial health summary score.
The mean PedsQL scores of children≥5 years were 89.45 (range: 78.13 -100) in the physical - and 84.10 (range: 63.33 -100) in the psychosocial health summary score. These were not significantly different in comparison to the mean scores given by their parents (83.59, range: 46.86-100 in the physical, p=0.42- and 81.02, range: 43.33-93.33 in the psychosocial health summary score, p=0.13), with the mean scores suggesting a good HRQoL. 63 % (n=5) of the children in this group had mild- and 37% (n=3) severe anemia.
The mean PedsQL scores reported by the parents of children <5 years were not significantly different than parental report of children ≥ 5 years (74.60, range: 59.3-100, versus 83.59, range: 46.86-100 in the physical, p=0.28 - and 78.60, range: 21.15-100, versus 81.02, range: 43.33-93.33 in the psychosocial health summary score, p= 0.33). The mean parental PedsQL scores of children <5 years are consistent with an intermediate HRQoL. 88% (n=7) of children < 5 years had severe- and 12% (n=1) moderate anemia.
Conclusion
This is the first study concerning HRQoL in pediatric patients with PKD. Using the PedsQL 4.0, mean child reported scores are consistent with a good HRQoL and parental scores are consistent with an intermediate HRQoL. Parents of children≥5 years give higher scores than parents of children <5 years. Given the severity of anemia in the children in this study, the PedsQL measure may not sufficiently detect the symptoms and impacts of children with PKD. Future studies of disease specific HRQoL measures may be helpful in detecting the impact of this rare anemia in children.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Anemia, Children, Quality of Life
Abstract: PS1444
Type: Poster Presentation
Presentation during EHA23: On Saturday, June 16, 2018 from 17:30 - 19:00
Location: Poster area
Background
Pyruvate kinase deficiency (PKD) is the most common enzymopathy of anaerobic glycolysis in erythrocytes. PKD is a rare autosomal recessive disease with a prevalence of 1 in 20.000 people causing a hereditary non- spherocytic hemolytic anemia (Zanella et al 2005). There are no published studies concerning health-related quality of life (HRQoL) in patients with PKD.
Aims
This study describes HRQoL in children with PKD in Germany.
Methods
16 pediatric patients with compound heterozygous or homozygous PKD who participated in Germany in the PKD Natural History Study were included in the analysis. The German version of the PedsQL 4.0 (Pediatric Quality of Life) questionnaire was used to assess HRQoL. The patients were divided into 3 groups according to age in years: 2-4 (n=8), 5-7 (n=2), 8-18 (n=6). 63% of the patients (n=10) had severe anemia (Hb< 8 g/dl), 4 % (n=1) moderate anemia (Hb 8-10 g/dl) and 33% (n=5) mild anemia (Hb 10-12 g/dl). The questionnaire was answered by all children ages≥5 years and by parents of children of all ages. The answers were transformed into a score from 0 to 100 (with 100 representing the best HRQoL) and were compared using the Mann-Whitney Test. PedsQL≥81 is considered an indicator of a good HRQoL , 61-80 intermediate HRQoL and ≤60 poor HRQoL (Beverung et al 2015).
Results
The mean parental PedsQL scores of all 16 children were 79.10 (range: 0-100) in the physical- and 79.81 (range: 21.15 -100) in the psychosocial health summary score.
The mean PedsQL scores of children≥5 years were 89.45 (range: 78.13 -100) in the physical - and 84.10 (range: 63.33 -100) in the psychosocial health summary score. These were not significantly different in comparison to the mean scores given by their parents (83.59, range: 46.86-100 in the physical, p=0.42- and 81.02, range: 43.33-93.33 in the psychosocial health summary score, p=0.13), with the mean scores suggesting a good HRQoL. 63 % (n=5) of the children in this group had mild- and 37% (n=3) severe anemia.
The mean PedsQL scores reported by the parents of children <5 years were not significantly different than parental report of children ≥ 5 years (74.60, range: 59.3-100, versus 83.59, range: 46.86-100 in the physical, p=0.28 - and 78.60, range: 21.15-100, versus 81.02, range: 43.33-93.33 in the psychosocial health summary score, p= 0.33). The mean parental PedsQL scores of children <5 years are consistent with an intermediate HRQoL. 88% (n=7) of children < 5 years had severe- and 12% (n=1) moderate anemia.
Conclusion
This is the first study concerning HRQoL in pediatric patients with PKD. Using the PedsQL 4.0, mean child reported scores are consistent with a good HRQoL and parental scores are consistent with an intermediate HRQoL. Parents of children≥5 years give higher scores than parents of children <5 years. Given the severity of anemia in the children in this study, the PedsQL measure may not sufficiently detect the symptoms and impacts of children with PKD. Future studies of disease specific HRQoL measures may be helpful in detecting the impact of this rare anemia in children.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Anemia, Children, Quality of Life