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Updated: Jun 27, 2026

Quantification of the Immunosuppressant Tacrolimus on Dried Blood Spots Using LC-MS/MS
Published on: November 8, 2015
Complete response to tacrolimus in a child with severe aplastic anemia resistant to cyclosporin A
Christine Macartney1, Marshall Freilich, Isaac Odame
1Marrow Failure and Myelodysplasia Program, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children, and The University of Toronto, Toronto, Ontario, Canada.
Insights
Tacrolimus offers a potential treatment for pediatric aplastic anemia (AA). In a severe AA case, switching from cyclosporin A to tacrolimus resolved gingival hyperplasia and induced complete AA remission.
Area of Science:
- Pediatric Hematology
- Immunosuppressive Therapy
- Gingival Health
Background:
- Aplastic anemia (AA) is a rare pediatric condition.
- Standard treatment involves immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporin A.
- Alternative therapies with comparable efficacy are limited.
Observation:
- A child with severe aplastic anemia (SAA) experienced severe gingival hyperplasia due to cyclosporin A.
- This hyperplasia was unresponsive to dental interventions.
- Switching IST to tacrolimus led to significant improvement in gingival hyperplasia.
Findings:
- The pediatric patient achieved a complete response of severe aplastic anemia within months of initiating tacrolimus.
- Tacrolimus effectively managed the cyclosporin A-induced gingival hyperplasia.
Implications:
- Tacrolimus may represent a viable alternative IST for pediatric severe aplastic anemia.
- This approach could mitigate side effects associated with traditional therapies.
- Further research into tacrolimus for pediatric AA is warranted.
Abstract:
Aplastic anemia (AA) is a rare disorder in children, usually treated with immunosuppressive therapy (IST) including antithymocyte globulin (ATG) and cyclosporin A. There are no current widely used alternative therapies with comparable efficacy. We describe a child with severe aplastic anemia (SAA), who developed severe gingival hyperplasia secondary to cyclosporin A, unresponsive to intensive dental intervention. When IST was changed to tacrolimus there was a significant improvement in the gingival hyperplasia, but equally important, he achieved complete response of his AA within several months. The use of tacrolimus in children with AA may be a potential modality of treatment.
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