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Clinical Efficacy of Ruxolitinib Monotherapy and Haploidentical Hematopoeitic Stem Cell Transplantation in a Child with Philadelphia Chromosome‐like Relapsed/Refractory acute lymphoblastic leukemia
Author(s) -
Bayram Nihan,
Yaman Yöntem,
Özdilli Kürşat,
Telhan Leyla,
Nepesov Serdar,
Bilgen Hülya,
Elli Murat,
Behar Sude Sema,
Anak Sema
Publication year - 2021
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.14024
Subject(s) - medicine , ruxolitinib , philadelphia chromosome , oncology , transplantation , refractory (planetary science) , hematopoietic stem cell transplantation , chemotherapy , leukemia , acute lymphocytic leukemia , stem cell , chromosomal translocation , lymphoblastic leukemia , gene , bone marrow , physics , genetics , astrobiology , biology , myelofibrosis , biochemistry , chemistry
(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.