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Influence of Iron Overload on Immunosuppressive Therapy in Children with Severe Aplastic Anemia
Katarzyna Pawelec1, Małgorzata Salamonowicz, Anna Panasiuk
1Department of Pediatric Hematology and Oncology, Medical University of Warsaw, 24 Marszalkowska St, 00-576, Warsaw, Poland, katarzyna.pawelec@litewska.edu.pl.
Insights
Elevated serum ferritin in children with severe aplastic anemia (AA) negatively impacts treatment response and increases relapse risk. Iron overload affects event-free survival, highlighting the need for careful monitoring during immunosuppressive therapy.
Area of Science:
- Pediatric Hematology
- Oncology
- Immunosuppressive Therapy
Background:
- Children with severe aplastic anemia (AA) often require frequent red blood cell transfusions.
- Transfusions can lead to iron overload, indicated by high serum ferritin levels.
- The impact of iron overload on treatment outcomes in pediatric AA is not fully understood.
Purpose of the Study:
- To retrospectively analyze the influence of elevated serum ferritin on survival, relapse risk, and treatment response in children with AA undergoing immunosuppressive therapy.
Main Methods:
- Retrospective analysis of 38 children with AA treated with a standard protocol.
- Patients categorized into three groups based on serum ferritin levels (<285 ng/mL, 286-1,000 ng/mL, >1,000 ng/mL).
- Kaplan-Meier plots used for survival analysis; treatment response assessed at specific time points.
Main Results:
- Overall survival did not differ significantly across ferritin groups.
- Event-free survival was significantly shorter in patients with ferritin >1,000 ng/mL (p=0.03).
- Time to relapse was significantly shorter in the high ferritin group (p=0.02); treatment response differed at days 84 and 112 (p=0.03, p<0.0001).
Conclusions:
- Elevated serum ferritin is associated with poorer event-free survival and increased relapse rates in pediatric AA.
- Iron overload negatively impacts treatment efficacy and outcomes in children with AA.
- Monitoring and managing iron levels may be crucial for improving treatment responses in pediatric aplastic anemia.
Abstract:
Children with severe aplastic anemia (AA) require multiple transfusions of the red blood cells during the immunosuppressive therapy. This leads to iron overload and manifests as elevated levels of ferritin in blood. The aim of this study was a retrospective analysis of the influence of the elevated serum ferritin on the overall survival, event-free survival, the risk of relapse, and response to treatment in children with AA during immunosuppressive therapy. We analyzed 38 children with AA (19 girls, 19 boys, aged 2-17 years) treated according to the obligatory protocol for AA in Poland. The response rate was assessed on days 84, 112, and 360. Patients were divided into three groups: group I consisted of children with ferritin below 285 ng/mL (6 children), group II with ferritin between 286 and 1,000 ng/mL (13 children), and group III ferritin>1,000 ng/mL (19 children). Kaplan-Meier plot was used to estimate the overall survival and event-free survival. We found the overall survival did not differ between the three groups. Event-free survival was significantly shorter (p=0.03) in patients with ferritin levels>1,000 ng/mL compared with the groups with ferritin bellow 1,000 ng/mL. The time to relapse was significantly shorter in group III than in the other two groups (p=0.02). We also found the differences in the treatment response at day 84 (p=0.03) and day 112 (p<0.0001) of immunosuppressive therapy. These findings confirm a negative influence of iron overload in children with AA on the effect of treatment and the risk of relapse.
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