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Markedly High Plasma Thrombopoietin (TPO) Level is a Predictor of Poor Response to Immunosuppressive Therapy in Children With Acquired Severe Aplastic Anemia
Author(s) -
Elmahdi Shaimaa,
Muramatsu Hideki,
Narita Atsushi,
Ismael Olfat,
Hama Asahito,
Nishio Nobuhiru,
Okuno Yusuke,
Xu Yinyan,
Wang Xinan,
Takahashi Yoshiyuki,
Kojima Seiji
Publication year - 2016
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.25820
Subject(s) - medicine , thrombopoietin , confidence interval , aplastic anemia , odds ratio , gastroenterology , multivariate analysis , anemia , bone marrow failure , immunology , bone marrow , haematopoiesis , stem cell , genetics , biology
Background Immunosuppressive therapy (IST) is commonly used for patients with acquired severe aplastic anemia (SAA). Because the clinical response rate and therapeutic outcome for individual patients to IST varies, an in vitro test that identifies potential responders would be desirable. Methods We evaluated the relationship between thrombopoietin (TPO) levels at the time of diagnosis and the response to IST at 6 months in 85 children (median age, 9.0 years; range, 1.0–15.5 years) with acquired SAA using enzyme‐linked immunosorbent assay. Thirty‐one age‐matched healthy individuals were used as controls. All patients received antithymocyte globulin and cyclosporine. Results Overall, 39 patients (45.9%) responded to IST at 6 months. TPO plasma levels were significantly higher in nonresponders than in responders (1,555.8 vs. 1,284.7 pg/ml, respectively; P = 0.031). Multivariate analysis identified the TPO levels of >1,796.7 pg/ml (TPO‐high group, 20 patients; odds ratio (OR), 8.285; 95% confidence interval (CI), 2.114–32.904; P = 0.002) as independent poor predictors of IST response at 6 months. Moreover, the TPO‐high group was associated with lower 5‐year failure‐free survival rates (30% vs. 68%, P = 0.012) compared with the TPO‐low group. Conclusion The measurement of TPO levels at diagnosis is useful for predicting the response to IST in children with SAA and may help in decision making.