학술논문
Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm
Document Type
article
Author
Miltiadous, Oriana; Petrova-Drus, Kseniya; Kaicker, Shipra; Mathew, Susan; Kluk, Michael; Geyer, Julia Turbiner; Rodriguez-Sanchez, M Irene; Bouvier, Nancy; Inghirami, Giorgio GA; Stieglitz, Elliot; Nafa, Khedoudja; Benayed, Ryma; Richardson, Michelle; Anderson, Wade; Benhamida, Jamal; You, Daoqi; Londono, Dory; Kung, Andrew L; Prockop, Susan; Roshal, Mikhail; Zhang, Yanming; Shukla, Neerav Narendra
Source
Blood Advances. 6(4)
Subject
Language
Abstract
FIP1L1-RARA-a ssociated neoplasm is a very rare and aggressive disease, with only 3 previously reported cases in the literature. Here, we describe a 9-month-old boy who presented with a FIP1L1-RARA fusion-associated myelodysplastic/myeloproliferative neoplasm-like overlap syndrome, with similarities and distinct features to both acute promyelocytic leukemia and juvenile myelomonocytic leukemia. Using a combined approach of chemotherapy, differentiating agents, and allogeneic hematopoietic stem cell transplant (allo-HCT), this patient remains in remission 20 months after allo-HCT. To our knowledge, this is only the second published pediatric case involving this condition and the only case with a favorable long-term outcome. Given the aggressive disease described in the previously published case report, as well as the successful treatment course described, the combinatorial use of chemotherapy, differentiation therapy, and allo-HCT for treatment of FIP1L1-RARA fusion-associated myeloid neoplasms should be considered.