scholarly journals Hematopoietic Stem Cell Transplantation versus Immunosuppressive Therapy in Patients with Acquired Severe Aplastic Anemia: A Cost-Effectiveness Analysis

Author(s):  
Meng-Xue Zhang ◽  
Qian Wang ◽  
Xiao-Qin Wang

Abstract BackgroundControversy remains regarding which therapy to initially select for severe aplastic anemia (SAA) patients aged 35-50-years-old. This analysis of cost-effectiveness using a Markov model compared immunosuppressive therapy (IST) with hematopoietic stem cell transplantation (HSCT) in age-stratified patients with SAA.MethodsIn younger patients (aged 18-35-years-old), HSCT yielded 22.67 quality-adjusted life years (QALYs), compared with 12.21 QALYs for IST therapy, offering an expected benefit with HSCT of 10.46 QALY.ResultsThe HSCT strategy dominated in younger patients, though it was $146,970 more expensive than IST and the ICER of HSCT to IST was $14,054.19/QALY, which was less than the willingness to pay (WTP) value of $25,397.57/QALY. The IST strategy dominated in older patients because it was $72,009 less expensive than HSCT and yielded 3.24 QALYs more than HSCT. The model was vigorous in the sensitivity analyses of the key variables tested through the plausible ranges that were acquired from costing sources and previously-published literature.ConclusionsThe preferred induction strategy for younger patients with SAA appears to be HSCT and the preferred strategy for older patients is IST, which minimizes cost while maximizing QALYs.

2021 ◽  
Vol 11 ◽  
Author(s):  
Limin Liu ◽  
Xin Zhao ◽  
Miao Miao ◽  
Yanming Zhang ◽  
Wenjing Jiao ◽  
...  

Background and AimsThis study aimed at comparing the efficacy and safety of severe aplastic anemia (SAA) cases that had met the criteria for SAA at the time of diagnosis (group A) with SAA that had progressed from non-SAA (NSAA) (group B), both undergoing first-line immunosuppressive therapy (IST). Additionally, group B was compared with SAA that had progressed from NSAA and who had been treated by allogeneic hematopoietic stem cell transplantation (allo-HSCT) (group C).MethodsWe retrospectively compared 608 consecutive patients in group A (n = 232), group B (n = 229) and group C (n = 147) between June 2002 and December 2019. Six months after treatment, the rate of overall response and the fraction of patients who had achieved normal blood values, treatment-related mortality (TRM), secondary clonal disease, 5-year overall survival (OS) and failure-free survival (FFS) were indirectly compared between group A and group B, group B and group C.ResultsSix months after treatment, the rate of overall response and the fraction of patients who had achieved normal blood values in group A was higher than in group B (65.24% vs. 40.54%, P < 0.0001; 23.33% vs. 2.25%, P < 0.0001); the same was true for group C (92.50% vs. 2.25%, P < 0.0001). The rate of relapse in group B was higher than in group C (P < 0.0001), but there were no differences in TRM and secondary clonal disease (P > 0.05). There were no differences in estimated 5-year OS between groups A and B (83.8% ± 2.6% vs. 85.8% ± 2.6%, P = 0.837), or between B and C (85.8% ± 2.6% vs. 77.9% ± 3.4%, P = 0.051). The estimated 5-year FFS in groups A and C was higher than for group B (57.1% ± 3.3% vs. 39.7% ± 3.4%, P < 0.001; 76.7% ± 3.5% vs. 39.7% ± 3.4%, P < 0.0001).ConclusionThese results indicate that IST is less effective in SAA progressing from non-SAA but allo-HSCT can improve outcomes.


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