免疫抑制治疗后三个月未获血液学反应的重型/极重型再生障碍性贫血患者六个月疗效评估

OBJECTIVE: To reassess the predictors for response at 6 months in patients with severe or very severe aplastic anemia（SAA/VSAA）who failed to respond to immunosuppressive therapy（IST）at 3 months. METHODS: We retrospectively analyzed the clinical data of 173 patients with SAA/VSAA from 2017 to 2018 who received IST and were classified as nonresponders at 3 months. Univariate and multivariate logistic regression analysis were used to evaluate factors that could predict the response at 6 months. RESULTS: Univariate analysis showed that the 3-month hemoglobin（HGB）level（P＝0.017）, platelet（PLT）level（P＝0.005）, absolute reticulocyte count（ARC）（P<0.001）, trough cyclosporine concentration（CsA-C0）（P＝0.042）, soluble transferrin receptor（sTfR）level（P＝0.003）, improved value of reticulocyte count（ARC(Δ)）（P<0.001）, and improved value of soluble transferrin receptor（sTfR(Δ)）level（P<0.001）were related to the 6-month response. The results of the multivariate analysis showed that the PLT level（P＝0.020）and ARC(Δ)（P<0.001）were independent prognostic factors for response at 6 months. If the ARC(Δ) was less than 6.9×10(9)/L, the 6-month hematological response rate was low, regardless of the patient's PLT count. Survival analysis showed that both the 3-year overall survival（OS）［（80.1±3.9）％ vs（97.6±2.6）％, P＝0.002］and 3-year event-free survival（EFS）［（31.4±4.5）％ vs（86.5±5.3）％, P<0.001］of the nonresponders at 6 months were significantly lower than those of the response group. CONCLUSION: Residual hematopoietic indicators at 3 months after IST are prognostic parameters. The improved value of the reticulocyte count could reflect whether the bone marrow hematopoiesis is recovering and the degree of recovery. A second treatment could be performed sooner for patients with a very low ARC(Δ).