Optimizing diagnostic methods and stem cell transplantation outcomes in pediatric bone marrow failure: a 50-year single center experience

Conclusion: Unbiased protocolized in-depth diagnostic strategies are crucial to increase the frequency of identifiable causes within the heterogeneous group of pediatric BMF. A comprehensive approach to identify the cause of BMF can prevent treatment delay and be useful to tailor treatment and follow-up protocols.What is Known:• Irreversible BMF in pediatric patients can be caused by a wide spectrum of underlying diseases including (pre)malignant disease, IBMFS and AA. Identifying the exact underlying cause of BMF is crucial for tailored therapy, however often challenging and time-consuming.• Frontline allogeneic HSCT is offered to most pediatric patients with severe BMF as curative treatment.What is New:• Protocolized unbiased diagnostics, short time to treatment (<  3 months) and maximal supportive care until curative treatment can prevent complications with a negative effect on survival such as infection and bleeding.• Personalized follow-up protocols for IBMFS patients are essential to prevent a second decline in survival due to long-term treatment toxicity and extra-hematological disease complications.
Source: European Journal of Pediatrics - Category: Pediatrics Source Type: research