Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm Journal Article
| Authors: | Miltiadous, O.; Petrova-Drus, K.; Kaicker, S.; Mathew, S.; Kluk, M.; Geyer, J. T.; Rodriguez-Sanchez, I.; Bouvier, N.; Inghirami, G.; Stieglitz, E.; Khedoudja, N.; Benayed, R.; Richardson, M.; Anderson, W.; Benhamida, J.; You, D.; Londono, D.; Kung, A. L.; Prockop, S. E.; Roshal, M.; Zhang, Y.; Shukla, N. |
| Article Title: | Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm |
| 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. © 2022 by The American Society of Hematology. |
| Keywords: | cancer chemotherapy; clinical article; case report; genetic association; genome analysis; infant; promyelocytic leukemia; gene fusion; fusion gene; genomics; allogeneic hematopoietic stem cell transplantation; childhood leukemia; leukemia remission; juvenile myelomonocytic leukemia; antileukemic agent; human; male; article; fip1l1 rara gene |
| Journal Title: | Blood Advances |
| Volume: | 6 |
| Issue: | 4 |
| ISSN: | 2473-9529 |
| Publisher: | American Society of Hematology |
| Date Published: | 2022-02-22 |
| Start Page: | 1137 |
| End Page: | 1142 |
| Language: | English |
| DOI: | 10.1182/bloodadvances.2021004966 |
| PUBMED: | 34551074 |
| PROVIDER: | scopus |
| PMCID: | PMC8864666 |
| DOI/URL: | |
| Notes: | Article -- MSK author Khedoudja Nafa's first and last names are reversed on the original publication -- Export Date: 1 April 2022 -- Funding details: National Institutes of Health, NIH, P30 CA008748 -- Funding text 1: This work was supported by the Scarlett Fund in part by the National Institutes of Health National Cancer Institute Cancer Center Support Grant (P30 CA008748). -- Source: Scopus |
Altmetric
Citation Impact
BMJ Impact Analytics
MSK Authors
Related MSK Work