Clinical efficacy of ruxolitinib monotherapy and haploidentical hematopoeitic stem cell transplantation in a child with philadelphia chromosome-like relapsed/refractory acute lymphoblastic leukemia

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2021Author
Bayram, NihanYaman, Yöntem
Özdilli, Kürşat
Telhan, Leyla
Nepesov, Serdar
Bilgen, Hülya
Elli, Murat
Behar, Sude Sema
Anak, Sema
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Bayram, N., Yaman, Y., Özdilli, K., Telhan, L., Nepesov, S., Bilgen, H. ... Anak, S. (2021). Clinical efficacy of ruxolitinib monotherapy and haploidentical hematopoeitic stem cell transplantation in a child with philadelphia chromosome-like relapsed/refractory acute lymphoblastic leukemia. Pediatric Transplantation, 25(4). https://dx.doi.org/10.1111/petr.14024Abstract
Introduction (Ph-like) ALL is a subset of leukemia which has a gene expression profile similar to Ph+disease, but without the presence of BCR-ABL1 translocation.
Case description We reported an exceptional case of a child with relapsed Ph-like ALL with IKZF1 gene deletion treated with high-dose ruxolitinib as monotherapy, after multi-agent chemotherapy. He remains in continued MRD-negative leukemia remission with full donor chimerism at 12 months post-HSCT.
Discussion The circumstance that makes our case featured is the usage of ruxolitinib as monotherapy. This report, we believe, is a pioneering report for a frequent disease with a high risk of failure for the outcome.
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Pediatric TransplantationVolume
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