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Antithymocyte globulin and cyclosporine in children with aplastic anemia: a developing country experience
Rajni Sharma1, Jagdish Chandra, Sunita Sharma
1Division of Pediatric Hematology and Oncology, Department of Pediatrics, Kalawati Saran Children's Hospital, Lady Hardinge Medical College, New Delhi, India. drrajnisharma@yahoo.com
Insights
Immunosuppressive therapy (IST) using antithymocyte globulin (ATG) and cyclosporine is a viable treatment for pediatric aplastic anemia in resource-limited settings. Long-term follow-up shows sustained survival and transfusion independence in responders.
Area of Science:
- Pediatric Hematology
- Immunosuppressive Therapy
- Aplastic Anemia Research
Background:
- Bone marrow transplant is often infeasible in developing countries for aplastic anemia.
- Immunosuppressive therapy (IST) serves as a critical alternative treatment.
- This study evaluates long-term outcomes of IST in children with aplastic anemia.
Purpose of the Study:
- To assess the long-term efficacy and safety of IST in pediatric aplastic anemia.
- To provide data on treatment outcomes in a developing country context.
- To evaluate the role of antithymocyte globulin (ATG) and cyclosporine in managing aplastic anemia.
Main Methods:
- Retrospective review of pediatric aplastic anemia cases treated with IST from 2001-2009.
- Analysis of patient data including disease severity, treatment response, and survival.
- Focus on outcomes in very severe and severe aplastic anemia.
Main Results:
- 28 patients analyzed; 57% overall response rate after IST, including a second ATG course.
- Median follow-up for responders was 40 months.
- Children with >4 years follow-up (n=7) showed sustained survival and transfusion independence.
Conclusions:
- IST with ATG and cyclosporine is a feasible and effective alternative for pediatric aplastic anemia in developing nations.
- The treatment offers good long-term outcomes, including survival and quality of life.
- This approach addresses resource limitations hindering bone marrow transplantation.
Background:
Human leukocyte antigen-matched bone marrow transplant in the treatment of aplastic anemia is generally not feasible in developing countries due to lack of resources and expertise and immunosuppressive therapy (IST) has been used as an alternative. This study aims to report the long-term outcome of children with aplastic anemia treated with IST [antithymocyte globulin (ATG) and cyclosporine] in our hospital.
Procedure:
Case files of children with aplastic anemia who received IST from January 2001 to November 2009 were reviewed.
Results:
Thirty-five patients with aplastic anemia (14 very severe aplastic anemia; 21 severe aplastic anemia) were given IST. Seven patients expired within 3 months of therapy and were excluded. The analysis was done in 28 patients (24 male and 4 female; 12 very severe aplastic anemia and 16 severe aplastic anemia). The median age was 10 years (range, 5 to 12 y). Ten patients achieved partial response and 4 patients complete response at 1 year with overall response rate of 50%. Three nonresponders received a second course of ATG after 12 months out of which 2 responded. Hence, overall response including second course was 16 (57%). Three patients relapsed after a median interval of 23 months. The median duration of follow-up of 16 responders was 40 months (range, 15 to 119 mo). In the patients with long-term follow-up for >4 years (n = 7), all were surviving and independent of transfusions.
Conclusions:
In a developing country setting, IST with ATG and cyclosporine seems to be a good alternative treatment for aplastic anemia in children.
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