title: The role of paroxysmal nocturnal hemoglobinuria clones in response to immunosuppressive therapy of patients with severe aplastic anemia.
abstract: Whether paroxysmal nocturnal hemoglobinuria (PNH) clone in aplastic anemia (AA) is a prognostic factor to immunosuppressive therapy is a subject of debate. We evaluated hematological responses to immunosuppressive therapy (IST) in severe AA (SAA) patients with or without the presence of a PNH clone. In 97 SAA patients who received first-line IST between January and December 2011, 24 (24.7 %) had a PNH clone prior to treatment, with a median clone size of 7.82 % (range 1.19-45.46 %). The response rates to IST for patients with or without a PNH clone were 66.7 and 50.7 % (P < 0.172), 79.2 and 57.5 % (P < 0.057), and 79.2 and 67.1 % (P < 0.264) at 3, 6, and 12 months, respectively. Combined rate of complete and good partial responses differed between patients with or without a PNH clone: insignificantly at 3 months (41.7 vs. 21.9 %, P < 0.058), but significantly at 6 (66.7 vs. 31.5 %, P < 0.002) and 12 (75.0 vs. 46.6 %, P < 0.015) months. Multivariate analysis revealed that a pretreatment neutrophil count of >0.2 × 10(9)/L is indicative of a better response, while the presence of a PNH clone is predictive to a higher combined rate of complete and good partial responses. This study demonstrated that the presence of a PNH clone could predict a better hematological response instead of a higher response rate in patients with SAA.
authors.affiliation: Department of Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, No. 288 Nanjing Road, Heping District, Tianjin, 300020, People's Republic of China.
authors.forename: Xin
authors.initials: X
authors.lastname: Zhao
authors.forename: Li
authors.initials: L
authors.lastname: Zhang
authors.forename: Liping
authors.initials: L
authors.lastname: Jing
authors.forename: Kang
authors.initials: K
authors.lastname: Zhou
authors.forename: Yuan
authors.initials: Y
authors.lastname: Li
authors.forename: Guangxin
authors.initials: G
authors.lastname: Peng
authors.forename: Lei
authors.initials: L
authors.lastname: Ye
authors.forename: Yang
authors.initials: Y
authors.lastname: Li
authors.forename: Jianping
authors.initials: J
authors.lastname: Li
authors.forename: Huihui
authors.initials: H
authors.lastname: Fan
authors.forename: Lin
authors.initials: L
authors.lastname: Song
authors.forename: Wenrui
authors.initials: W
authors.lastname: Yang
authors.forename: Fengkui
authors.initials: F
authors.lastname: Zhang
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000097:blood* / Q000175:diagnosis / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006457:Hemoglobinuria, Paroxysmal / Q000097:blood* / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents