Journal
BLOOD ADVANCES
Volume 6, Issue 4, Pages 1137-1142Publisher
ELSEVIER
DOI: 10.1182/bloodadvances.2021004966
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Funding
- Scarlett Fund
- National Institutes of Health National Cancer Institute Cancer Center Support Grant [P30 CA008748]
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FIP1L1-RARA-associated neoplasm is a rare and aggressive disease. This study presents a pediatric case with successful long-term outcome using a combination of chemotherapy, differentiating agents, and allogeneic hematopoietic stem cell transplant.
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.
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