Clinical Efficacy of Ruxolitinib Monotherapy and Haploidentical Hematopoeitic Stem Cell Transplantation in a Child with Philadelphia Chromosome-like Relapsed/Refractory acute lymphoblastic leukemia

BAYRAM N., YAMAN Y., ÖZDİLLİ K., TELHAN L., Nepesov S., BİLGEN H., ...More

Pediatric Transplantation, vol.25, no.4, 2021 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 25 Issue: 4
  • Publication Date: 2021
  • Doi Number: 10.1111/petr.14024
  • Journal Name: Pediatric Transplantation
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, EMBASE, MEDLINE
  • Keywords: JAK inhibitors, Philadelphia&#8208, like ALL, refractory ALL, stem cell transplantation
  • Istanbul Medipol University Affiliated: Yes

Abstract

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.