Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
• Patients undergoing workup for suspected newly diagnosed sAA:
‣ Patients with severe cytopenias and a hypocellular marrow concerning for sAA
⁃ Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:
∙ Marrow Cellularity: \<25%, or 25-50% with \<30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \<500 x 10\^9/L Platelets: \<20 x 10\^9/L Absolute Reticulocyte Count: \<60 x 10\^9/L
• Patients that do not have evidence of leukemia or MDS
• Patients \< 25 years of age at time of diagnosis
• Able to tolerate emapalumab and IST (with standard institutional organ function criteria)