Effect of Eltrombopag on Response to Immunosuppressive Therapy in Patients with Transfusion-Dependent Non-Severe Aplastic Anemia / 中国实验血液学杂志

OBJECTIVE@#To compare the efficacy of eltrombopag combined with cyclosporine A (CsA) and CsA alone in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA).@*METHODS@#The clinical data of 76 patients with treatment-naive TD-NSAA in Ningde Municipal Hospital of Ningde Normal University and Affiliated Hospital of Nantong University from December 2017 to June 2021 were retrospectively analyzed. Among them, 45 cases were treated with eltrombopag combined with CsA, and 31 patients with compatible baseline characters were treated with CsA alone. The efficacy of patients between the two groups was compared, and the factors affecting the curative effects were also analyzed.@*RESULTS@#There were significant differences in hematological response (HR) and complete response(CR) rates between the two groups at 3, 6, 12 months, and follow-up endpoint of treatment (P<0.05). With the prolongation of eltrombopag treatment time, the curative effect increased gradually, and the patients achieved more CR and HR rates by the end of the follow-up period. Simultaneously, with the increase in the maximum stable dose of eltrombopag, the HR rate increased gradually. The megakaryocyte count in eltrombopag group was higher than that in control at 6 and 12 months (P<0.05). Compared with the control group, the median time of platelet transfusion independence in eltrombopag group was more shorter (P=0.018), and the median platelets transfusion volume was lower (P=0.009). At 3, 6, 12 months after eltrombopag, the change of platelet in eltrombopag group was higher than that in the control group (P<0.05). Analysis of related factors affecting the efficacy showed that sex, age, iron overload, platelet count before treatment had no effect on the efficacy, and the median maximum stable dosage and the administration period for eltrombopag were related to the curative effect. The patients of eltrombopag group experienced adverse events of varying degrees, but the reactions were mild and mostly tolerated.@*CONCLUSION@#Eltrombopag can effectively improve the hematopoietic response and promote platelet recovery for TD-NSAA patients with relatively more residual hematopoietic cells, and it is safe and well tolerated.

The efficacy of eltrombopag plus cyclosporine A in patients with transfusion-dependent non-severe aplastic anemia: a retrospective study from single center / 中华内科杂志

The main purpose of our study was to evaluate the efficacy and safety of eltrombopag plus cyclosporine A (CsA) in transfusion-dependent non-severe aplastic anemia(TD-NSAA). The clinical characteristics of 13 TD-NSAA patients who received initial treatment of eltrombopag plus CsA from 2019 to 2021 were retrospectively analyzed. The 3-month overall hematological response (OR) rate was 12/13. Until the end of follow-up, 12 patients responded, among whom 2 patients reached complete response (CR) and 9 patients reached partial response (PR) and 1 with HR. Paroxysmal nocturnal hemoglobinuria (PNH) developed in one patient at 6 months after treatment. Five of thirteen patients reported mild adverse reactions, which were all manageable. Compared with historical data, the combination of eltrombopag with CsA is an effective regimen in patients with TD-NSAA.

Mean corpuscular volume,red blood cell volume distribution width in non-severe aplastic anemia role of early efficacy prediction / 重庆医学

Objective To evaluated the application value of mean corpuscular volume(MCV) and red blood cell volume distribu-tion width(RDW) in predicting early treatment responses of non-severe aplastic anemia(NSAA) .Methods 101 cases of patients who were newly diagnosed with non-severe aplastic anemia and were treated with Cyclosporine (CsA)combined androgen therapy . Treatment before baseline MCV ,RDW value were measured ,treatment for 3 months ,6 months test routine blood ,reticulocyte indi-cators and treatment results were statistically analyzed and receiver operating characteristic (ROC) curve was used to estimate the sensitivity and specificity of MCV ,RDW in predicting early treatment results .Results The level of MCV and RDW in non-severe aplastic anemia were significantly higher than normal reference .the responded patients had significantly higher pretreatment base-line MCV value than those non-responded .especially at 6 month ,baseline MCV were 105 .10 fl and 98 .30 fl respectively (P=0 .025) .the responded patients had significantly lower pretreatment baseline RDW value than those non-responded .especially at 3 month .baseline RDW were 14 .60% and 16 .60% respectively(P=0 .001) .The cutoff level of MCV (100 fL) and RDW (15 .55% ) for the predicting 3 and 6 month treatment responses were established based on the ROC curve ,with degree of accurancy of MCV was 62 .4% ,61 .4% and RDW 70 .3% ,63 .4% respectively .compared the treatment efficacy acuity MCV ≥100 fL/RDW <15 .55%group was significantly better than MCV < 100 fL/RDW≥15 .55% group .Multivariate analysis showed that pretreatment RDW and absolute reticulocyte value was the early prognostic factor of NSAA treatment effect .84 patients with ARC ≥ 20 × 109/L , through RDW cutoff Layered compare treatment response :RDW<15 .55% group was significantly better than the RDW≥15 .55%group(P=0 .000) .Conclusion MCV can not serve as of a significant predictor of early treatment response in non-severe aplastic a-nemia .RDW can serve as of the bone marrow failure severity indicators and a significant predictor of early treatment response in non-severe aplastic anemia .The joint reticulocyte absolute value parameters ,which can more accurately predict treatment efficacy .