title: 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.
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.
authors.affiliation: Division of Hematology, Oncology and Marrow and Blood Cell Transplantation, Children's Hospital at Montefiore Medical Center, Bronx, New York.|Albert Einstein College of Medicine, Bronx, New York.
authors.forename: Veronika
authors.initials: V
authors.lastname: Polishchuk
authors.affiliation: Division of Hematology, Oncology and Marrow and Blood Cell Transplantation, Children's Hospital at Montefiore Medical Center, Bronx, New York.|Albert Einstein College of Medicine, Bronx, New York.
authors.forename: Sajad
authors.initials: S
authors.lastname: Khazal
authors.affiliation: Department of Pathology, Montefiore Medical Center, Bronx, New York.
authors.forename: Giorgi
authors.initials: G
authors.lastname: Berulava
authors.affiliation: Division of Hematology, Oncology and Marrow and Blood Cell Transplantation, Children's Hospital at Montefiore Medical Center, Bronx, New York.|Albert Einstein College of Medicine, Bronx, New York.
authors.forename: Michael
authors.initials: M
authors.lastname: Roth
authors.affiliation: Division of Hematology, Oncology and Marrow and Blood Cell Transplantation, Children's Hospital at Montefiore Medical Center, Bronx, New York.|Albert Einstein College of Medicine, Bronx, New York.
authors.forename: Kris M
authors.initials: KM
authors.lastname: Mahadeo
mesh_terms: D000293:Adolescent; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage*; D001374:Azacitidine / Q000008:administration & dosage*; D003131:Combined Modality Therapy / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D060828:Induction Chemotherapy; D007938:Leukemia / Q000628:therapy*; D060046:Maintenance Chemotherapy / Q000379:methods*; D008297:Male; D012074:Remission Induction; D014184:Transplantation, Homologous
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine
keywords: BMT; chemotherapy; leukemia