First-line immunosuppressive treatment in children with aplastic anemia: rabbit antithymocyte globulin
K Pawelec1, M Salamonowicz, A Panasiuk
1Department of Pediatric, Hematology and Oncology, Medical University of Warsaw, 24 Marszalkowska St., Warsaw, 00-576, Poland, katarzyna.pawelec@litewska.edu.pl.
Insights
Rabbit antithymocyte globulin (r-ATG) and cyclosporine A offer effective first-line immunosuppressive therapy for children with acquired severe aplastic anemia (AA). This treatment shows promising remission rates and long-term survival, crucial for patients lacking a matched family donor.
Area of Science:
- Pediatric Hematology
- Immunosuppressive Therapy
- Aplastic Anemia Research
Background:
- Acquired severe aplastic anemia (AA) in children without an HLA-matched family donor necessitates effective immunosuppressive therapy.
- Rabbit antithymocyte globulin (r-ATG) combined with cyclosporine A is a primary treatment approach in such cases.
- Evaluating long-term outcomes is critical for optimizing pediatric AA management.
Purpose of the Study:
- To assess the efficacy and outcomes of first-line immunosuppressive therapy using r-ATG and cyclosporine A in pediatric patients with acquired severe aplastic anemia.
- To determine remission rates, relapse incidence, and long-term survival following this treatment regimen.
- To provide data supporting the use of r-ATG and cyclosporine A in children with AA lacking matched family donors.
Main Methods:
- A multicenter study involving 63 children diagnosed with acquired severe aplastic anemia.
- Treatment administered: rabbit antithymocyte globulin (r-ATG) and cyclosporine A as first-line therapy.
- Therapeutic effects monitored at Days 112, 180, and 360, with long-term survival analysis.
Main Results:
- Remission rates at Day 112, 180, and 360 were 44.4%, 49.2%, and 64.9% respectively.
- Complete remission was observed in 15.9% (Day 112), 23.8% (Day 180), and 38.0% (Day 360) of patients.
- Estimated 10-year overall and event-free survival rates were 67% and 57%, with a low incidence of relapse and late-onset paroxysmal nocturnal hemoglobinuria.
Conclusions:
- First-line immunosuppressive therapy with r-ATG and cyclosporine A demonstrates significant efficacy in achieving remission in children with acquired severe aplastic anemia.
- The treatment regimen offers encouraging long-term survival rates, highlighting its importance for pediatric patients without HLA-matched family donors.
- Continued monitoring for late complications such as paroxysmal nocturnal hemoglobinuria is warranted.
Abstract:
Immunosuppressive therapy is the treatment of choice in children with acquired severe aplastic anemia (AA) and no HLA-matched family donor. The paper presents results of a multicenter study of 63 children with AA treated with rabbit antithymocyte globulin (r-ATG) and cyclosporine A as the first line treatment in the years 1996-2012. Therapeutic effects were evaluated at Days 112, 180, and 360. At Day 112, remission was achieved in 28 out of the 63 patients (44.4 %), complete remission in 10 patients (15.9 %), and partial remission in 18 (28.5 %). At Day 180, 31 patients (49.2 %) were in remission including 15 cases in complete (23.8 %), and 16 cases in partial remission (25.4 %). One year after therapy onset, 34 patients (64.9 %) were in remission including 24 patients (38.0 %) in complete and 10 (15.9 %) in partial remission. Relapse occurred in 4 patients, from 8 months up to 2 years and 2 months after remission. One child, 5 years after remission, was diagnosed with paroxysmal nocturnal hemoglobinuria. The estimated 10-year overall survival rate and 10-year event-free survival rate were 67 % and 57 %, respectively.
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