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ATG-Fresenius S combined with cyclosporine a: an effective immunosuppressive therapy for children with aplastic

Cheng-Juan Luo1, Yi-Jin Gao, Jing-Yan Tang

  • 1*Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University †Children's Hospital, Shanghai Medical College, Fudan University, Shanghai, China.

Journal of Pediatric Hematology/Oncology
|May 9, 2014
PubMed

Insights

Antithymocyte globulin (ATG)-Fresenius S plus cyclosporine effectively treats severe aplastic anemia in children. This immunosuppressive therapy shows a 55.6% response rate and a 74.7% 5-year survival rate in pediatric patients.

Area of Science:

  • Pediatric Hematology
  • Immunosuppressive Therapy
  • Aplastic Anemia Research

Background:

  • Severe aplastic anemia (SAA) is a rare but serious condition.
  • Standard immunosuppressive therapy (IST) for SAA includes antithymocyte globulin (ATG) and cyclosporine.
  • Limited data exists on the efficacy of ATG-Fresenius S specifically in pediatric SAA.

Purpose of the Study:

  • To evaluate the efficacy and safety of ATG-Fresenius S combined with cyclosporine as a first-line treatment for pediatric severe aplastic anemia.
  • To determine response rates, survival outcomes, and long-term complications in children receiving this IST regimen.

Main Methods:

  • A retrospective, 2-center study involving 124 pediatric patients with acquired severe aplastic anemia.
  • Patients received a 5-day course of ATG-Fresenius S (5 mg/kg/day) plus cyclosporine.
  • Outcomes assessed included response rate, time to response, relapse, clonal evolution, and overall survival.

Main Results:

  • The overall response rate to ATG-Fresenius S plus cyclosporine was 55.6%.
  • Median time to response was 6 months, with a median follow-up of 29 months.
  • The 5-year overall survival rate was 74.7%, significantly higher in responders (100%) than non-responders (70.7%).
  • Relapse and clonal evolution rates were low (3.2% and 0.8%, respectively).
  • Mortality was primarily due to sepsis (13.7% overall).

Conclusions:

  • First-line treatment with ATG-Fresenius S plus cyclosporine is effective in children with severe aplastic anemia.
  • This regimen offers a viable treatment option, particularly where other ATG products are less accessible.
  • Improved survival rates underscore the importance of timely and effective immunosuppressive therapy in pediatric SAA.

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