Clinical outcomes of pediatric patients with severe idiopathic aplastic anemia in King Chulalongkorn Memorial Hospital
Keywords:
severe aplastic anemia, immunosuppressive therapy, Stem cell transplantationAbstract
Background: Idiopathic severe aplastic anemia (SAA) is a rare disorder among children. The modality of treatment is matched sibling, allogeneic stem cell transplantation or immunosuppressive among patients who do not have a matched donor as first line therapy. The responses to the treatment vary depending on the reports.
Objectives: The study aimed to evaluate the outcomes of children with idiopathic SAA in our institution.
Methods: A retrospective chart review included all patients aged 1 to 18 years, diagnosed with SAA between
June 1, 2004 and June 30, 2019, who had been treated at King Chulalongkorn Memorial Hospital.
Results: Thirty pediatric patients (22 males and eight females) median age 6.4 years were enrolled. Twenty-three patients (76%) received ATG plus cyclosporine, while 3 patients (10%) received androgen plus low dose prednisolone as a first treatment. Eight patients, (34.7%) receiving ATG plus cyclosporine, achieved a response at 3 months. Among unresponsive patients, 8 patients (53.3%) received androgen plus low dose prednisolone while the remaining 7 patients received 2nd ATG plus cyclosporine. Five-year overall survival was 72.9% (95%CI: 0.49 – 0.86) in the ATG plus cyclosporine group. Four patients underwent matched sibling, allogeneic stem cell transplantation while 3 patients survived without the disease. The 5-year overall survival of all patients was 69.4% (95%CI: 0.49 – 0.82), and the median follow-up totaled 4.3 years.
Conclusion: This study demonstrated that treatment of SAA using immunosuppressive therapy, consisting of ATG plus cyclosporine, had acceptable outcomes. Matched sibling, allogeneic stem cell transplantation is considered a first-line treatment with superior outcomes compared with immunosuppressive therapy. However, the limitation is donor availability.
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