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Editorials
better response to treatment and overall survival than chil- dren with less severe SAA.9 Unfortunately, baseline lym- phocyte counts in that study were not available. We could hypothesize that similar mechanisms could explain dis- crepant results between young children and adult patients.
The data in the present study could also become a sup- portive tool to approach children and young adults with SAA who lack a matched sibling donor. The management of newly diagnosed pediatric and young adult SAA patients could be based on the following algorithm (Figure 1D): when baseline blood values are predictive of a unsat- isfactory response to IST combined with eltrombopag, and the chance of rapidly finding a suitable alternative donor exists, upfront first-line HCT with this alternative donor should be taken in serious consideration. Future research in this field should be directed at identifying fac- tors predicting long-term outcome, which would bring more precise information to adapt up-front treatment strategies.
Disclosures
No conflicts of interest to disclose.
Contributions
AT, RPL, CD and AR wrote the mansucript.
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