High-dose cyclophosphamide for the treatment of refractory T-cell acute lymphoblastic leukemia in children.

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.
AuthorsRachel Kobos, Neerav Shukla, Thomas Renaud, Susan E Prockop, Farid Boulad, Peter G Steinherz
JournalJournal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 36 Issue 5 Pg. e265-70 (Jul 2014) ISSN: 1536-3678 [Electronic] United States
PMID24327129 (Publication Type: Journal Article)
Chemical References
  • Antineoplastic Agents, Alkylating
  • Cyclophosphamide
  • Adolescent
  • Antineoplastic Agents, Alkylating (administration & dosage, therapeutic use)
  • Child
  • Child, Preschool
  • Cyclophosphamide (administration & dosage, therapeutic use)
  • Dose-Response Relationship, Drug
  • Drug Resistance, Neoplasm (drug effects)
  • Follow-Up Studies
  • Humans
  • Male
  • Neoplasm Recurrence, Local (drug therapy, pathology)
  • Neoplasm, Residual (drug therapy, pathology)
  • Precursor T-Cell Lymphoblastic Leukemia-Lymphoma (drug therapy, pathology)
  • Prognosis
  • Retrospective Studies
  • Survival Rate

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