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A longitudinal analysis of paroxysmal nocturnal haemoglobinuria-type cells in patients with bone marrow failure: Results of a prospective multi-centre study in Japan |
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| Authors: | Ken Ishiyama Yuji Yonemura Tatsuya Kawaguchi Kohei Hosokawa Chiharu Sugimori Yasutaka Ueda Hiroyuki Takamori Naoshi Obara Hideyoshi Noji Yukari Shirasugi Kiyoshi Ando Tsutomu Shichishima Haruhiko Ninomiya Shigeru Chiba Jun-ichi Nishimura Yuzuru Kanakura Shinji Nakao |
| Affiliation: | 1. Department of Hematology, Faculty of Medicine, Institute of Medical Pharmaceutical and Health Sciences, Kanazawa University, Kanazawa, Japan;2. Japan PNH Study Group, Tokyo, Japan Department of Transfusion Medicine and Cell Therapy, Kumamoto University, Kumamoto, Japan;3. Japan PNH Study Group, Tokyo, Japan Department of Medical Technology, Kumamoto Health Science University, Kumamoto, Japan;4. Japan PNH Study Group, Tokyo, Japan Department of Hematology, Ishikawa Prefectural Central Hospital, Kanazawa, Japan;5. Japan PNH Study Group, Tokyo, Japan Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Japan;6. Japan PNH Study Group, Tokyo, Japan;7. Japan PNH Study Group, Tokyo, Japan Department of Hematology, Fukushima Medical University, Fukushima, Japan;8. Japan PNH Study Group, Tokyo, Japan Department of Hematology and Oncology, Tokai University, Tokyo, Japan;9. Japan PNH Study Group, Tokyo, Japan Department of Hematology, Tsukuba University, Tsukuba, Japan |
| Abstract: | To determine the prevalence and clinical relevance of glycosylphosphatidylinositol-anchored protein-deficient (GPI[−]) cell populations (paroxysmal nocturnal haemoglobinuria [PNH]-type cells) in patients with acquired aplastic anaemia (AA) or myelodysplastic syndrome (MDS), we prospectively studied peripheral blood samples of 2402 patients (1075 with AA, 900 with MDS, 144 with PNH, and 283 with other anaemia) using a high-sensitivity flow cytometry assay in a nationwide multi-centre observational study. PNH-type cells were detected in 52.6% of AA and 13.7% of MDS patients. None of the 35 patients with refractory anaemia (RA) with ringed sideroblasts or the 86 patients with RA with excess of blasts carried PNH-type cells. Among the 317 patients possessing PNH-type granulocytes, the percentage of PNH-type granulocytes increased by ≥10% in 47 patients (14.8%), remained unchanged in 240 patients (75.7%), and decreased by ≥10% in 30 patients (9.5%) during 3 years of follow-up. PNH-type granulocyte expansion occurred more frequently (27.1%) in the 144 patients who originally carried PNH-type granulocytes ≥1% than in the 173 patients with PNH-type granulocytes <1% (4.6%). This study confirmed that PNH-type cells are undetectable in authentic clonal MDS patients, and the presence of ≥1% PNH-type granulocytes predicts a higher likelihood of PNH-type cell expansion than with <1% PNH-type granulocytes. |
| Keywords: | aplastic anaemia bone marrow failure flow cytometry myelodysplastic syndrome paroxysmal nocturnal haemoglobinuria-type cell |