title: High-dose cyclophosphamide for the treatment of refractory T-cell acute lymphoblastic leukemia in children.
abstract: Despite an almost 80% overall survival rate in pediatric T-cell acute lymphoblastic leukemia (T-ALL), there is a subset of patients who are refractory to standard chemotherapy regimens and could benefit from novel treatment approaches. Over a 2-year period, we treated 5 pediatric patients with refractory T-ALL, aged 3 to 15 years, with high-dose cyclophosphamide (CY) at a dose of 2100 mg/m for 2 consecutive days either alone (n=1) or in combination with other chemotherapy agents (n=4). Four of these 5 patients had a 1.5 log decrease in disease burden. Three of the 5 patients had no evidence of minimal residual disease (MRD) after high-dose CY. One patient developed transient grade 4 transaminitis and 1 patient developed grade 3 typhlitis. All 5 patients ultimately proceeded to hematopoietic stem cell transplant when MRD levels were <0.01%. Pediatric T-ALL patients with persistent MRD after treatment with conventional chemotherapy may respond to CY at escalated dosing.
authors: Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY.
authors: Rachel
authors: R
authors: Kobos
authors: Neerav
authors: N
authors: Shukla
authors: Thomas
authors: T
authors: Renaud
authors: Susan E
authors: SE
authors: Prockop
authors: Farid
authors: F
authors: Boulad
authors: Peter G
authors: PG
authors: Steinherz
mesh_terms: D000293:Adolescent; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000627:therapeutic use; D004305:Dose-Response Relationship, Drug; D019008:Drug Resistance, Neoplasm / Q000187:drug effects*; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000473:pathology; D018365:Neoplasm, Residual / Q000188:drug therapy* / Q000473:pathology; D054218:Precursor T-Cell Lymphoblastic Leukemia-Lymphoma / Q000188:drug therapy* / Q000473:pathology; D011379:Prognosis; D012189:Retrospective Studies; D015996:Survival Rate
chemical_list: D018906:Antineoplastic Agents, Alkylating; D003520:Cyclophosphamide