Small PNH clones detected by fluorescent aerolysin predict a faster response to immunosuppressive therapy in patients with severe aplastic anaemia.

Abstract

Objectives: To clear the obscure conclusion on the prediction value of paroxysmal nocturnal haemoglobinuria (PNH) clones in severe aplastic anaemia (SAA) patients treated with immunosuppressive therapy (IST). Methods: We retrospectively analyzed 219 consecutive SAA patients treated with IST from October 2008 to October 2015 and evaluated the haematological responses to IST. Results: The presence of a PNH clone was detected in 55 (25.1%) patients prior to IST [37/88 by flow cytometry (FCM) and 18/131 by fluorescent aerolysin (FLAER)] and 27 disappeared after IST (23/37 in initial FCM group, 4/18 in initial FLAER group, p = 0.005). In patients without an initial clone, 12 (30.0%) cases in FCM and 17 (19.5%) in FLAER groups presented a PNH clone at least once after IST (p < 0.001). In patients with a pre-treatment PNH clone detected by FCM, the 3-, 6- and 12-month response rates were higher than patients without (p = 0.006; 0.002 and 0.002, respectively). And in FLAER group, the 3-month response rate was significantly higher in those with a prior clone (p = 0.017), however, the 6- and 12-month response rates showed no differences (p = 0.105, p = 0.144, respectively). By multivariate analysis, a shorter interval between diagnosis and treatment is associated with a better response and survival. Conclusions: A more reliable FLAER method allows us to draw a conclusion that PNH clone predicts a faster response but not a higher response rate to IST. Once a diagnosis is confirmed, the IST should be initiated as soon as possible.

• Authors: Xiang Ren, Xingxin Li, Jiali Huo, Yahong You, Jinbo Huang, Yingqi Shao, Meili Ge, Zhendong Huang, Jing Zhang, Min Wang, Peng Jin, Neng Nie, Yizhou Zheng
• Journal: Hematology (Amsterdam, Netherlands) (Hematology) Vol. 25 Issue 1 Pg. 348-355 (Dec 2020) ISSN: 1607-8454 [Electronic] England
• PMID: 32960154 (Publication Type: Journal Article, Video-Audio Media)

Chemical References

• Bacterial Toxins
• Pore Forming Cytotoxic Proteins
• aerolysin

Topics

• Adolescent
• Adult
• Anemia, Aplastic (blood, therapy)
• Bacterial Toxins
• Child
• Child, Preschool
• Female
• Flow Cytometry
• Follow-Up Studies
• Humans
• Immunosuppression Therapy
• Male
• Middle Aged
• Pore Forming Cytotoxic Proteins
• Severity of Illness Index