Hematological Disorders in Children by Eiichi Ishii

Author:Eiichi Ishii
Language: eng
Format: epub
Publisher: Springer Singapore, Singapore

6.5.5.2 CY

High-dose CY (200 mg/kg) combined with CSA therapy resulted in a higher incidence of fungal infections and death compared to conventional therapy with ATG plus CSA [107]. Moderate dose CY (120 mg/kg) plus CSA therapy also showed a response rate of 41–65% [108, 109]. High-dose CY (200 mg/kg) monotherapy showed a hematological response of 79% and EFS of 64% in a pediatric or young adult cohort (22 treatment-naïve and 6 refractory/relapsed patients) [110].

6.6 Conclusions

Treatment failure, disease recurrence, and clonal evolution are the shortcomings of IST. Long-term complications and secondary malignancy remain major concerns in URD- or haplo-BMT. Sufficient evidence is lacking to recommend CBT as an up-front therapy. In the future, genomic analyses will help to select the appropriate treatment for children with AA.

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