Distinct clinical characteristics of paroxysmal nocturnal hemoglobinuria in patients in Southern Taiwan: A multicenter investigation

Hui Ching Wang, Ching Yuan Kuo, I-Ting Liu, Tsai-Yun Chen, Yu Hsiang Chang, Shyh Jer Lin, Shih Feng Cho, Yi Chang Liu, Ta Chih Liu, Sheng Fung Lin, Chao Sung Chang

Research output: Contribution to journalArticle

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Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare acquired disorder. The aim of this study was to investigate the demographics, clinical manifestations, and outcomes of PNH patients in southern Taiwan. Data on PNH patients diagnosed over a 30-year period (1985–2015) were retrospectively collected from four tertiary medical centers in southern Taiwan. Blood samples were collected for hematologic panel testing and flow cytometry detection of PNH clones. Radiologic studies were performed to assess the frequency of complications. Twenty-four patients were enrolled in this study. The median duration of disease in the study participants was 10.8 years. The median granulocyte PNH clone size was 92.5% (range, 1.3%–99.8%), and the median lactate dehydrogenase (LDH) level was 2920.2 ± 1462.0 IU/L. The incidence of thromboembolism and impaired renal function was 16.7% and 29.2%, respectively. The primary treatment strategies included steroids (79.2%), androgens (42.0%), eculizumab (33.3%), immunosuppressants (16.7%), and anticoagulants (4.2%). In eight patients treated with eculizumab, there was a marked reduction in the LDH levels of 14.89-fold–1.63-fold that of the upper limit of normal; seven patients exhibited decreased transfusion requirements. Twenty-one patients were alive with regular follow-up at the time of publication. Our study demonstrates that PNH patients in southern Taiwan may exhibit different clinical characteristics and outcomes relative to patients in other countries. There was a trend toward a greater PNH granulocyte clone size, which may lead to more hemolysis. In our study, the percentage of patients with impaired renal function, but not the percentage of patients with thrombotic events, was higher than values reported worldwide and in the observational cross-sectional International PNH Registry. More large-scale studies with comprehensive data on the clinical response to different treatments are needed.

Original languageEnglish
Pages (from-to)405-410
Number of pages6
JournalKaohsiung Journal of Medical Sciences
Volume33
Issue number8
DOIs
Publication statusPublished - 2017 Aug 1

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Paroxysmal Hemoglobinuria
Taiwan
Clone Cells
L-Lactate Dehydrogenase
Granulocytes
Kidney
Thromboembolism
Immunosuppressive Agents
Hemolysis
Anticoagulants
Androgens
Registries
Publications
Flow Cytometry
Steroids
Demography

All Science Journal Classification (ASJC) codes

  • Medicine(all)

Cite this

Wang, Hui Ching ; Kuo, Ching Yuan ; Liu, I-Ting ; Chen, Tsai-Yun ; Chang, Yu Hsiang ; Lin, Shyh Jer ; Cho, Shih Feng ; Liu, Yi Chang ; Liu, Ta Chih ; Lin, Sheng Fung ; Chang, Chao Sung. / Distinct clinical characteristics of paroxysmal nocturnal hemoglobinuria in patients in Southern Taiwan : A multicenter investigation. In: Kaohsiung Journal of Medical Sciences. 2017 ; Vol. 33, No. 8. pp. 405-410.
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abstract = "Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare acquired disorder. The aim of this study was to investigate the demographics, clinical manifestations, and outcomes of PNH patients in southern Taiwan. Data on PNH patients diagnosed over a 30-year period (1985–2015) were retrospectively collected from four tertiary medical centers in southern Taiwan. Blood samples were collected for hematologic panel testing and flow cytometry detection of PNH clones. Radiologic studies were performed to assess the frequency of complications. Twenty-four patients were enrolled in this study. The median duration of disease in the study participants was 10.8 years. The median granulocyte PNH clone size was 92.5{\%} (range, 1.3{\%}–99.8{\%}), and the median lactate dehydrogenase (LDH) level was 2920.2 ± 1462.0 IU/L. The incidence of thromboembolism and impaired renal function was 16.7{\%} and 29.2{\%}, respectively. The primary treatment strategies included steroids (79.2{\%}), androgens (42.0{\%}), eculizumab (33.3{\%}), immunosuppressants (16.7{\%}), and anticoagulants (4.2{\%}). In eight patients treated with eculizumab, there was a marked reduction in the LDH levels of 14.89-fold–1.63-fold that of the upper limit of normal; seven patients exhibited decreased transfusion requirements. Twenty-one patients were alive with regular follow-up at the time of publication. Our study demonstrates that PNH patients in southern Taiwan may exhibit different clinical characteristics and outcomes relative to patients in other countries. There was a trend toward a greater PNH granulocyte clone size, which may lead to more hemolysis. In our study, the percentage of patients with impaired renal function, but not the percentage of patients with thrombotic events, was higher than values reported worldwide and in the observational cross-sectional International PNH Registry. More large-scale studies with comprehensive data on the clinical response to different treatments are needed.",
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Distinct clinical characteristics of paroxysmal nocturnal hemoglobinuria in patients in Southern Taiwan : A multicenter investigation. / Wang, Hui Ching; Kuo, Ching Yuan; Liu, I-Ting; Chen, Tsai-Yun; Chang, Yu Hsiang; Lin, Shyh Jer; Cho, Shih Feng; Liu, Yi Chang; Liu, Ta Chih; Lin, Sheng Fung; Chang, Chao Sung.

In: Kaohsiung Journal of Medical Sciences, Vol. 33, No. 8, 01.08.2017, p. 405-410.

Research output: Contribution to journalArticle

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T1 - Distinct clinical characteristics of paroxysmal nocturnal hemoglobinuria in patients in Southern Taiwan

T2 - A multicenter investigation

AU - Wang, Hui Ching

AU - Kuo, Ching Yuan

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AU - Chen, Tsai-Yun

AU - Chang, Yu Hsiang

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AU - Cho, Shih Feng

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AU - Chang, Chao Sung

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