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Distinct subgroups of paroxysmal nocturnal hemoglobinuria (PNH) with cytopenia: results from South Korean National PNH Registry

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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 × 109/L; platelet count <20 × 109/L), 96 patients with PNH/aplastic anemia (AA) (at least two of the three criteria; hemoglobin ≤10 g/dL; ANC 0.5–1.5 × 109/L; platelet count 20–100 × 109/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).

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Acknowledgments

This study was presented in the form of a poster presentation at the 52nd annual meeting of the American Society of Hematology, Orlando, FL, December 4–7, 2010. This work was supported by the Korean Society of Hematology.

Conflict of interest

All authors declare that they have no conflicts of interest.

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    Authors and Affiliations

    1. Division of Hematology, Department of Internal Medicine, Severance Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea

      Jin Seok Kim

    2. Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, School of Medicine, Sungkyunkwan University, Seoul, Republic of Korea

      Jun Ho Jang

    3. Department of Internal Medicine, Seoul National University Hospital, Seoul National University College of Medicine, Seoul, Republic of Korea

      Sung-Soo Yoon

    4. Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea

      Je-Hwan Lee

    5. Department of Hematology-Oncology, Chonnam National University Hwasun Hospital, Hwasun, Jeollanamdo, Republic of Korea

      Yeo-Kyeoung Kim

    6. Department of Internal Medicine, School of Medicine, Chungnam National University Hospital, Chungnam National University, Daejeon, Republic of Korea

      Deog-Yeon Jo

    7. Division of Hematology-Oncology, Department of Internal Medicine, School of Medicine, Pusan National University Hospital, Busan, Republic of Korea

      Joo Seop Chung

    8. Department of Hematology/Oncology, Kyungpook National University Hospital, Kyungpook National University School of Medicine, Daegu, Republic of Korea

      Sang Kyun Sohn

    9. 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

      Jong Wook Lee

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    Correspondence to Jong Wook Lee.

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    Jin Seok Kim and Jun Ho Jang contributed equally to this work.

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    Kim, J.S., Jang, J.H., Yoon, SS. et al. Distinct subgroups of paroxysmal nocturnal hemoglobinuria (PNH) with cytopenia: results from South Korean National PNH Registry. Ann Hematol 95, 125–133 (2016). https://doi.org/10.1007/s00277-015-2511-z

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