title: Distinct subgroups of paroxysmal nocturnal hemoglobinuria (PNH) with cytopenia: results from South Korean National PNH Registry.
abstract: We retrospectively assessed the clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria (PNH) according to severity of cytopenia. A total of 282 patients with hematological parameters assessed at the time of diagnosis of PNH were included. There were 24 patients with PNH/severe aplastic anemia (SAA) (at least two of the three criteria; hemoglobin ≤8 g/dL; absolute neutrophil count (ANC) <0.5 × 10(9)/L; platelet count <20 × 10(9)/L), 96 patients with PNH/aplastic anemia (AA) (at least two of the three criteria; hemoglobin ≤10 g/dL; ANC 0.5-1.5 × 10(9)/L; platelet count 20-100 × 10(9)/L), and 162 classic PNH patients. Compared with the classic PNH subgroup, the PNH/SAA subgroup had a significantly lower median granulocyte PNH clone size (26.7 vs. 51.0 %, P = 0.021) and lower incidence of lactate dehydrogenase ≥1.5 times the upper limit of normal (52.9 vs. 80.0 %, P = 0.049). The incidence of thromboembolism was similar in both subgroups. Overall survival was significantly lower in the PNH/SAA subgroup than in the classic PNH subgroup (P = 0.033). Our findings suggest that identification of patients with PNH/SAA at the time of diagnosis is important because of different clinical manifestations and poorer outcome compared with patients with classic PNH (clinicaltrials.gov identifier: #NCT01224483).
authors.affiliation: Division of Hematology, Department of Internal Medicine, Severance Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea.
authors.forename: Jin Seok
authors.initials: JS
authors.lastname: Kim
authors.affiliation: Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, School of Medicine, Sungkyunkwan University, Seoul, Republic of Korea.
authors.forename: Jun Ho
authors.initials: JH
authors.lastname: Jang
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, Seoul National University College of Medicine, Seoul, Republic of Korea.
authors.forename: Sung-Soo
authors.initials: SS
authors.lastname: Yoon
authors.affiliation: Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
authors.forename: Je-Hwan
authors.initials: JH
authors.lastname: Lee
authors.affiliation: Department of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Jeollanamdo, Republic of Korea.
authors.forename: Yeo-Kyeoung
authors.initials: YK
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, School of Medicine, Chungnam National University Hospital, Chungnam National University, Daejeon, Republic of Korea.
authors.forename: Deog-Yeon
authors.initials: DY
authors.lastname: Jo
authors.affiliation: Division of Hematology-Oncology, Department of Internal Medicine, School of Medicine, Pusan National University Hospital, Busan, Republic of Korea.
authors.forename: Joo Seop
authors.initials: JS
authors.lastname: Chung
authors.affiliation: Department of Hematology/Oncology, Kyungpook National University Hospital, Kyungpook National University School of Medicine, Daegu, Republic of Korea.
authors.forename: Sang Kyun
authors.initials: SK
authors.lastname: Sohn
authors.affiliation: Division of Hematology, Department of Internal Medicine, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, 222 Banpo-daero, Seocho-gu, Seoul, 137-701, Republic of Korea. jwlee@catholic.ac.kr.
authors.forename: Jong Wook
authors.initials: JW
authors.lastname: Lee
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000145:classification* / Q000175:diagnosis* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D010198:Pancytopenia / Q000145:classification* / Q000175:diagnosis* / Q000401:mortality; D012042:Registries; D056910:Republic of Korea / Q000453:epidemiology; D012189:Retrospective Studies; D015996:Survival Rate / Q000639:trends; D055815:Young Adult
keywords: Aplastic anemia; Cytopenia; Paroxysmal nocturnal hemoglobinuria; Thromboembolism