Contributions
Abstract: PB2388
Type: Publication Only
Background
Chronic condition and weakening clinical manifestations of type 1 Gaucher disease (GD1) can significantly affect patients’ quality of life (QOL). Given the extraordinary cost and degree of uncertainty in predicting the course of condition, understanding the potential benefits of ERT pertaining to improvements in psychosocial parameters and observable symptoms is important.
Aims
This study investigates survival status and QOL in GD1 patients who underwent long-term imiglucerase enzyme replacement therapy (ERT) and identifies possible relevant factors affecting QOL
Methods
Factors affecting QOL were analyzed based on clinical data, survey of living conditions, and SF-36 questionnaires of GD1 patients receiving ERT (20–40 U/kg intravenously, twice a week).
Results
Median age of onset, start of ERT, and current median age of the 22 GD1 patients (13 men, 9 women) were 6 (1–38), 26 (6–41), and 40.5 (24–52) years, respectively. Of these, 68.2% lived in developing cities, 86.4% did not receive college education, and 77.3% were with annual income <$4500. Median ERT duration was 15.75 (7–22) years. All dimensions but mental health (MH) of QOL in GD1 patients were significantly poor (P<0.05) compared with that of normal Chinese population based on SF-36 scores. History of splenectomy before treatment was an adverse factor affecting patients’ physical health (P<0.05) but not mental health (P>0.05). ERT at early ages was beneficial in increasing patients’ physical and mental health (P<0.05). Presence of splenomegaly and bone involvement despite ERT had no effect (P>0.05).
Conclusion
Compared with normal Chinese population, GD1 patients were associated with reduced QOL, which is consistent with their current living condition, poor education, and income status. History of splenectomy and age at the beginning of ERT are key factors affecting QOL.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Enzyme replacement therapy, Gaucher disease, Quality of Life
Abstract: PB2388
Type: Publication Only
Background
Chronic condition and weakening clinical manifestations of type 1 Gaucher disease (GD1) can significantly affect patients’ quality of life (QOL). Given the extraordinary cost and degree of uncertainty in predicting the course of condition, understanding the potential benefits of ERT pertaining to improvements in psychosocial parameters and observable symptoms is important.
Aims
This study investigates survival status and QOL in GD1 patients who underwent long-term imiglucerase enzyme replacement therapy (ERT) and identifies possible relevant factors affecting QOL
Methods
Factors affecting QOL were analyzed based on clinical data, survey of living conditions, and SF-36 questionnaires of GD1 patients receiving ERT (20–40 U/kg intravenously, twice a week).
Results
Median age of onset, start of ERT, and current median age of the 22 GD1 patients (13 men, 9 women) were 6 (1–38), 26 (6–41), and 40.5 (24–52) years, respectively. Of these, 68.2% lived in developing cities, 86.4% did not receive college education, and 77.3% were with annual income <$4500. Median ERT duration was 15.75 (7–22) years. All dimensions but mental health (MH) of QOL in GD1 patients were significantly poor (P<0.05) compared with that of normal Chinese population based on SF-36 scores. History of splenectomy before treatment was an adverse factor affecting patients’ physical health (P<0.05) but not mental health (P>0.05). ERT at early ages was beneficial in increasing patients’ physical and mental health (P<0.05). Presence of splenomegaly and bone involvement despite ERT had no effect (P>0.05).
Conclusion
Compared with normal Chinese population, GD1 patients were associated with reduced QOL, which is consistent with their current living condition, poor education, and income status. History of splenectomy and age at the beginning of ERT are key factors affecting QOL.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Enzyme replacement therapy, Gaucher disease, Quality of Life