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Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.

Publication ,  Journal Article
Rogers, ZR; Nakano, TA; Olson, TS; Bertuch, AA; Wang, W; Gillio, A; Coates, TD; Chawla, A; Castillo, P; Kurre, P; Gamper, C; Bennett, CM ...
Published in: Haematologica
October 2019

Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and outcomes for 314 children treated from 2002 to 2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin (hATG) plus cyclosporine (CyA) with a median 61 months follow up. Following hATG/CyA, 71.2% (95%CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response achieved in pediatric patients was high, with 59.8% (95%CI: 53.7,65.8) complete response and 68.2% (95%CI: 62.2,73.8) achieving at least a very good partial response with a platelet count ≥50×109L. At five years post-hATG/CyA, overall survival was 93% (95%CI: 89,96), but event-free survival without subsequent treatment was only 64% (95%CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis after a median 25.2 months (range: 4.3-71 months) post treatment. Myelodysplastic syndrome or leukemia developed in 6 of 314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95%CI: 0.08,0.47; P=0.0003). This study highlights the need for improved therapies to achieve sustained high-quality remission for children with severe aplastic anemia.

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Published In

Haematologica

DOI

EISSN

1592-8721

Publication Date

October 2019

Volume

104

Issue

10

Start / End Page

1974 / 1983

Location

Italy

Related Subject Headings

  • United States
  • Retrospective Studies
  • Male
  • Infant
  • Immunosuppression Therapy
  • Immunology
  • Humans
  • Follow-Up Studies
  • Female
  • Cyclosporine
 

Citation

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Rogers, Z. R., Nakano, T. A., Olson, T. S., Bertuch, A. A., Wang, W., Gillio, A., … Shimamura, A. (2019). Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica, 104(10), 1974–1983. https://doi.org/10.3324/haematol.2018.206540
Rogers, Zora R., Taizo A. Nakano, Timothy S. Olson, Alison A. Bertuch, Winfred Wang, Alfred Gillio, Thomas D. Coates, et al. “Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.Haematologica 104, no. 10 (October 2019): 1974–83. https://doi.org/10.3324/haematol.2018.206540.
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica. 2019 Oct;104(10):1974–83.
Rogers, Zora R., et al. “Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.Haematologica, vol. 104, no. 10, Oct. 2019, pp. 1974–83. Pubmed, doi:10.3324/haematol.2018.206540.
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, Coates TD, Chawla A, Castillo P, Kurre P, Gamper C, Bennett CM, Joshi S, Geddis AE, Boklan J, Nalepa G, Rothman JA, Huang JN, Kupfer GM, Cada M, Glader B, Walkovich KJ, Thompson AA, Hanna R, Vlachos A, Malsch M, Weller EA, Williams DA, Shimamura A. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica. 2019 Oct;104(10):1974–1983.

Published In

Haematologica

DOI

EISSN

1592-8721

Publication Date

October 2019

Volume

104

Issue

10

Start / End Page

1974 / 1983

Location

Italy

Related Subject Headings

  • United States
  • Retrospective Studies
  • Male
  • Infant
  • Immunosuppression Therapy
  • Immunology
  • Humans
  • Follow-Up Studies
  • Female
  • Cyclosporine