Abstract
Cytogenetic abnormalities are a major risk factor for relapse after hematopoietic stem cell transplantation (HSCT) for myelodysplastic syndrome (MDS). We aimed to evaluate the value of the five-group cytogenetic classification according to the revised International Prognostic Scoring System (R-IPSS) for predicting the outcome after HSCT in pediatric patients with MDS. We retrospectively analyzed the Japanese registration data of 242 pediatric patients with MDS. According to the R-IPSS classification, 112 (45.5%) patients had good, 55 (22.7%) had intermediate, 64 (26.4%) had poor, and 11 (4.6%) had very poor cytogenetics. The 5-year overall survival (5yOS) was 72%, 69%, 59%, and 30% in the good, intermediate, poor, and very poor cytogenetic subgroups (p = 0.026), respectively. The very good, good, and intermediate subgroups were grouped into a “standard” subgroup and reclassified into three subgroups (standard, poor, and very poor). Patients with very poor risk had worse 5yOS (hazard ratio 2.17, 95% confidence interval (CI) 1.02–4.61; p = 0.04) and a much higher 5yCIR (hazard ratio 2.52, 95% CI 1.05–6.04; p = 0.04) than those of patients in the standard group in the multivariate analysis, indicating that very poor risk cytogenetic characteristics independently predicted worse outcome after HSCT in pediatric patients with MDS.
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We thank all medical and co-medical staff who participated in the treatment of pediatric patients with MDS in Japan.
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SY, MK, KW, SI, DH, YA, and NY designed the study, SY performed the statistical analysis, SY, and NY wrote the paper, and MN, AH, MS, TK, FI, HY, YT, YK, YH, and AM were the principal investigators at the centers recruiting the highest number of patients for the study. All authors reviewed and approved the final version of the paper.
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Yamamoto, S., Kato, M., Watanabe, K. et al. Prognostic value of the revised International Prognostic Scoring System five-group cytogenetic abnormality classification for the outcome prediction of hematopoietic stem cell transplantation in pediatric myelodysplastic syndrome. Bone Marrow Transplant 56, 3016–3023 (2021). https://doi.org/10.1038/s41409-021-01446-z
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DOI: https://doi.org/10.1038/s41409-021-01446-z