Expert Consensus of Multidisciplinary Diagnosis and Treatment for Paroxysmal Nocturnal Hemoglobinuria(2024)

Miao Chen; Chen Yang; Ziwei Liu; Wei Cao; Bo Zhang; Xin Liu; Jingnan LI; Wei Liu; Jie Pan; Jian Wang; Yuehong Zheng; Yuexin Chen; Fangda LI; Shunda DU; Cong Ning; Limeng Chen; Cai Yue; Jun NI; Min Peng; Xiaoxiao Guo; Tao Wang; Hongjun LI; Rongrong LI; Tong WU; Bing Han; Shuyang Zhang; Multidiscipline Collaboration Group ON Rare Disease AT Peking Union Medical College Hospital

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Expert Consensus of Multidisciplinary Diagnosis and Treatment for Paroxysmal Nocturnal Hemoglobinuria(2024)

VernacularTitle:阵发性睡眠性血红蛋白尿症多学科诊疗专家共识(2024)
Author: Miao CHEN ¹ ; Chen YANG ¹ ; Ziwei LIU ¹ ; Wei CAO ² ; Bo ZHANG ³ ; Xin LIU ³ ; Jingnan LI ⁴ ; Wei LIU ⁵ ; Jie PAN ⁶ ; Jian WANG ⁶ ; Yuehong ZHENG ⁷ ; Yuexin CHEN ⁷ ; Fangda LI ⁷ ; Shunda DU ⁸ ; Cong NING ⁸ ; Limeng CHEN ⁹ ; Cai YUE ⁹ ; Jun NI ^{1
,

10} ; Min PENG ^{1
,

11} ; Xiaoxiao GUO ^{1
,

2
,

12} ; Tao WANG ^{1
,

3
,

13} ; Hongjun LI ^{1
,

4
,

14} ; Rongrong LI ^{1
,

5
,

15} ; Tong WU ^{1
,

6
,

16} ; Bing HAN ¹ ; Shuyang ZHANG ^{1
,

2
,

12} ; MULTIDISCIPLINE COLLABORATION GROUP ON RARE DISEASE AT PEKING UNION MEDICAL COLLEGE HOSPITAL
Author Information

1. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
2. Department of Infectious Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
3. Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
4. Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
5. Department of Radiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
6. Department of Radiointervention, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
7. Department of Vascular Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
8. Department of Liver Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
9. Department of Nephrology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
10. Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
11. Department of Respiratory and Critical Medicine, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
12. Department of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
13. Department of Dermatology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
14. Department of Urology Surgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
15. Department of Nutrition, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
16. Department of Hematopoietic Stem Cell Transplantation, Beijing GoBroad Boren Hospital, Beijing 100070, China
Publication Type:Journal Article
Keywords: paroxysmal nocturnal hemoglobinuria; multidisciplinary treatment; multiple system involvement; consensus
From: Medical Journal of Peking Union Medical College Hospital 2024;15(5):1011-1028
CountryChina
Language:Chinese
Abstract:
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disease caused by abnormal expression of glycosylphosphatidylinositol (GPI) on the cell membrane due to mutations in the phosphatidylinositol glycan class A(PIGA) gene. It is commonly characterized by intravascular hemolysis, repeated thrombosis, and bone marrow failure, as well as multiple systemic involvement symptoms such as renal dysfunction, pulmonary hypertension, swallowing difficulties, chest pain, abdominal pain, and erectile dysfunction. Due to the rarity of PNH and its strong heterogeneity in clinical manifestations, multidisciplinary collaboration is often required for diagnosis and treatment. Peking Union Medical College Hospital, relying on the rare disease diagnosis and treatment platform, has invited multidisciplinary clinical experts to form a unified opinion on the diagnosis and treatment of PNH, and formulated the Expert Consensus of Multidisciplinary Diagnosis and Treatment for Paroxysmal Nocturnal Hemoglobinuria (2024), with the hope of promoting the standardization of PNH diagnosis and treatment.