Haematologica (Aug 2014)

Peripheral blood lymphocyte telomere length as a predictor of response to immunosuppressive therapy in childhood aplastic anemia

  • Hirotoshi Sakaguchi,
  • Nobuhiro Nishio,
  • Asahito Hama,
  • Nozomu Kawashima,
  • Xinan Wang,
  • Atsushi Narita,
  • Sayoko Doisaki,
  • Yinyan Xu,
  • Hideki Muramatsu,
  • Nao Yoshida,
  • Yoshiyuki Takahashi,
  • Kazuko Kudo,
  • Hiroshi Moritake,
  • Kazuhiro Nakamura,
  • Ryoji Kobayashi,
  • Etsuro Ito,
  • Hiromasa Yabe,
  • Shouichi Ohga,
  • Akira Ohara,
  • Seiji Kojima

DOI
https://doi.org/10.3324/haematol.2013.091165
Journal volume & issue
Vol. 99, no. 8

Abstract

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Predicting the response to immunosuppressive therapy could provide useful information to help the clinician define treatment strategies for patients with aplastic anemia. In our current study, we evaluated the relationship between telomere length of lymphocytes at diagnosis and the response to immunosuppressive therapy in 64 children with aplastic anemia, using flow fluorescence in situ hybridization. Median age of patients was ten years (range 1.5–16.2 years). Severity of the disease was classified as very severe in 23, severe in 21, and moderate in 20 patients. All patients were enrolled in multicenter studies using antithymocyte globulin and cyclosporine. The response rate to immunosuppressive therapy at six months was 52% (33 of 64). The probability of 5-year failure-free survival and overall survival were 56% (95% confidence interval (CI): 41–69%) and 97% (95%CI: 87–99%), respectively. Median telomere length in responders was −0.4 standard deviation (SD) (−2.7 to +3.0 SD) and −1.5 SD (−4.0 to +1.6 (SD)) in non-responders (P