Peer-Reviewed Publication
Blood Adv2022;6(4):1137-1142.February 22, 2022Case Reports

Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm.

Oriana Miltiadous1,2, Kseniya Petrova-Drus3, Shipra Kaicker2, Susan Mathew4, Michael Kluk4, Julia T Geyer4, Irene Rodriguez-Sanchez5, Nancy Bouvier5, Giorgio Inghirami4, Elliot Stieglitz6, Nafa Khedoudja3, Ryma Benayed3, Michelle Richardson7, Wade Anderson7, Jamal Benhamida3, Daoqi You8, Dory Londono3, Andrew L Kung1, Susan E Prockop1, Mikhail Roshal3, Yanming Zhang3, Neerav Shukla1
1Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.
2Department of Pediatrics, Hematology & Oncology Division, New York Presbyterian Hospital, Cornell Medical Center, New York, NY.
3Department of Pathology, Memorial Sloan Kettering Cancer Center, New York, NY.
4Department of Pathology, New York Presbyterian Hospital, Cornell Medical Center, New York, NY.
5Sloan Kettering Institute, Pediatric Department, New York, NY.
6Department of Pediatrics, UCSF Benioff Children's Hospital, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA.
7Notable Labs, Foster City, CA; and.
8Marie-Josée and Henry R. Kravis Center for Molecular Oncology, Memorial Sloan Kettering Cancer Center, New York, NY.

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. Us…

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