Efficacy and safety of immunosuppressive therapy with or without eltrombopag in pediatric patients with acquired aplastic anemia: A Chinese retrospective study.
Pediatr Hematol Oncol
; 38(7): 633-646, 2021 Oct.
Article
em En
| MEDLINE
| ID: mdl-33724146
ABSTRACT
To determine the efficacy and safety of eltrombopag (E-PAG) combined with intensive immunosuppressive therapy (IST) for the treatment of pediatric patients with severe aplastic anemia (SAA). A total of 57 pediatric patients with newly diagnosed severe aplastic anemia were enrolled in this study. Thirty nine patients were treated with IST alone, consisting of porcine anti-human thymocyte globulin (30 mg/kg/day × 5 days) and cyclosporine A (CsA) (treated for 2 years, with a trough concentration maintained at 200-250 ng/mL), and 18 patients were treated with IST + E-PAG (12.5-50 mg/day, maintained for 6 months). We found no statistical difference between the response rates at 3 months for the two groups (CR 12.8% vs. 22.2% p > 0.05, ORR 56.4% vs. 77.7% p > 0.05). However, we found a statistical difference between the response rates at 6 months for the two groups (CR 17.9% vs. 50% p < 0.05, ORR 69.2% vs. 94.4% p < 0.05). The main side-effect during treatment with E-PAG was having a slightly to moderately elevated bilirubin level, which was temporary and controllable, accounting for approximately 66.6% (12/18) of patients in the IST + E-PAG group vs. 20.5% (8/39) of those in the IST group (p < 0.05). IST + E-PAG therapy appears to be more effective than IST alone for the treatment of pediatric SAA, with good tolerability and compliance. This approach deserves further exploration.
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Base de dados:
MEDLINE
Assunto principal:
Pirazóis
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Benzoatos
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Hidrazinas
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Imunossupressores
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Anemia Aplástica
Tipo de estudo:
Observational_studies
Limite:
Animals
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Child
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Humans
País como assunto:
Asia
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article