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., ...Daha Fazla

Pediatric Transplantation, cilt.25, sa.4, 2021 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 25 Sayı: 4
  • Basım Tarihi: 2021
  • Doi Numarası: 10.1111/petr.14024
  • Dergi Adı: Pediatric Transplantation
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, EMBASE, MEDLINE
  • Anahtar Kelimeler: JAK inhibitors, Philadelphia&#8208, like ALL, refractory ALL, stem cell transplantation
  • İstanbul Medipol Üniversitesi Adresli: Evet

Özet

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.