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Open Access 01-12-2024 | Paroxysmal Nocturnal Hemoglobinuria | Research

Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China

Authors: Huaxin Yu, Shengnan Duan, Pei Wang, Rong Fu, Zixuan Lv, Yuchi Yu, Pu Miao, Junwei Shi, Niekun Zhuang, Huiying Hu, Ni Yuan, Sijia Che

Published in: Orphanet Journal of Rare Diseases | Issue 1/2024

Abstract

Background

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder, leading to various complications and impairments in patients’ health-related quality of life (HRQOL). Limited research has been conducted to evaluate the HRQOL of Chinese patients with PNH. Understanding the HRQOL in this specific population is crucial for providing effective healthcare interventions and improving patient’ health outcomes. This study aimed to assess HRQOL of Chinese patients with PNH, and identify key determinants.

Methods

A cross-sectional study was conducted during 2022 to recruit patients with PNH in China. The study population was recruited from PNH China, one of the largest public welfare PNH patient mutual aid organization in China. Data were collected via an online questionnaire including the EQ-5D-5L (5L), and social-demographic and clinical characteristics. Descriptive statistics were employed to summarize the characteristics of the participants and their HRQOL. Multiple linear and logistic regression analyses were adopted to explore key factors affecting HRQOL.

Results

A total of 329 valid questionnaires were collected. The mean (SD) age of the patients was 35.3 (10.0) years, with 52.3% of them being male. The patients reported more problems in Anxiety/Depression (81.5%) and Pain/Discomfort (69.9%) dimensions compared to the other three 5L dimensions. The mean (SD) of 5L health utility score (HUS) and EQ-VAS score were 0.76 (0.21) and 62.61 (19.20), respectively. According to multiple linear regression, initial symptoms (i.e., Anemia [fatigue, tachycardia, shortness of breath, headache] and back pain) and complication of thrombosis were significant influencing factors affecting 5L HUS. Total personal income of the past year, initial symptom of hemoglobinuria and complication of thrombosis were significantly influencing factors of VAS score. Social-demographic and clinical characteristics, such as gender, income, and thrombosis, were also found to be significantly related to certain 5L health problems as well.

Conclusion

Our study manifested the HRQOL of PNH patients in China was markedly compromised, especially in two mental-health related dimensions, and revealed several socio-demographic and clinical factors of their HRQOL. These findings could be used as empirical evidence for enhancing the HRQOL of PNH patients in China.

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Title: Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China
Authors: Huaxin Yu
Shengnan Duan
Pei Wang
Rong Fu
Zixuan Lv
Yuchi Yu
Pu Miao
Junwei Shi
Niekun Zhuang
Huiying Hu
Ni Yuan
Sijia Che
Publication date: 01-12-2024
Publisher: BioMed Central
Keywords: Paroxysmal Nocturnal Hemoglobinuria
Thrombosis
Published in: Orphanet Journal of Rare Diseases / Issue 1/2024
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-024-03178-x

2024 ASCO Annual Meeting

Springer Medicine

Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China

Abstract

Background

Methods

Results

Conclusion

2024 ASCO Annual Meeting

Springer Medicine

Abstract

Background

Methods

Results

Conclusion

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