Blood advances · 2022
Successful treatment and integrated genomic analysis of an infant with FIP1L1-RARA fusion-associated myeloid neoplasm.
Клинический случайСтатьяПоддержка NIH: внешняя программаПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
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Miltiadous Oriana, Petrova-Drus Kseniya, Kaicker Shipra, Mathew Susan, Kluk Michael, Geyer Julia T, Rodriguez-Sanchez Irene, Bouvier Nancy, Inghirami Giorgio, Stieglitz Elliot, Khedoudja Nafa, Benayed Ryma, Richardson Michelle, Anderson Wade, Benhamida Jamal, You Daoqi, Londono Dory, Kung Andrew L, Prockop Susan E, Roshal Mikhail, Zhang Yanming, Shukla Neerav
Аннотация
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.