5-Azacitidine Monotherapy Followed by Related Haploidentical Hematopoietic Stem Cell Transplantation Achieves Durable Remission in a Pediatric Patient With Acute Undifferentiated Leukemia Refractory to High-Dose Chemotherapy

Pediatr Blood Cancer. 2016 Jun;63(6):1111-2. doi: 10.1002/pbc.25948. Epub 2016 Feb 23.

Abstract

Patients with acute leukemias of undifferentiated lineage (AUL) generally have guarded prognosis. Here, we describe the first reported pediatric patient with AUL refractory to high-dose chemotherapy who achieved clinical remission with ALL maintenance therapy and 5-azacitidine. His induction remission was followed by consolidation with reduced toxicity haploidentical hematopoietic stem cell transplant (HSCT). At 9 months post-HSCT, the patient is alive and in remission. This combination therapy of remission induction with ALL maintenance therapy and 5-azacitidine and consolidation with reduced toxicity haploidentical HSCT is novel and promising for patients who lack conventional donors and are not candidates for myeloablative therapy.

Keywords: BMT; chemotherapy; leukemia.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antimetabolites, Antineoplastic / administration & dosage*
  • Azacitidine / administration & dosage*
  • Combined Modality Therapy / methods
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Induction Chemotherapy
  • Leukemia / therapy*
  • Maintenance Chemotherapy / methods*
  • Male
  • Remission Induction
  • Transplantation, Homologous

Substances

  • Antimetabolites, Antineoplastic
  • Azacitidine