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


Authors: Kobos, R.; Shukla, N.; Renaud, T.; Prockop, S. E.; Boulad, F.; Steinherz, P. G.
Article 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. Copyright © 2014 by Lippincott Williams & Wilkins.
Keywords: adolescent; child; clinical article; preschool child; school child; treatment response; antibiotic agent; human cell; prednisone; drug tolerability; neutropenia; cancer combination chemotherapy; monotherapy; treatment duration; cancer patient; cytarabine; methotrexate; drug megadose; infection; multiple cycle treatment; cyclophosphamide; dexamethasone; vincristine; kidney failure; hematopoietic stem cell transplantation; continuous infusion; oncology; retrospective study; acute lymphoblastic leukemia; drug dose escalation; disease severity; minimal residual disease; cardiotoxicity; daunorubicin; glucocorticoid; ondansetron; clostridium difficile infection; aminotransferase; mesna; recombinant granulocyte colony stimulating factor; childhood leukemia; t cell leukemia; hemorrhagic cystitis; inappropriate vasopressin secretion; alemtuzumab; infusion fluid; typhlitis; hypertransaminasemia; nelarabine; antileukemic agent; asparaginase macrogol; neutropenic enterocolitis; high-dose cyclophosphamide; human; male; priority journal; article; pediatric t-all
Journal Title: Journal of Pediatric Hematology/Oncology
Volume: 36
Issue: 5
ISSN: 1077-4114
Publisher: Lippincott Williams & Wilkins  
Date Published: 2014-07-01
Start Page: e265
End Page: e270
Language: English
DOI: 10.1097/mph.0000000000000080
PROVIDER: scopus
PUBMED: 24327129
DOI/URL:
Notes: Export Date: 1 August 2014 -- CODEN: JPHOF -- Source: Scopus
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MSK Authors
  1. Farid Boulad
    329 Boulad
  2. Susan E Prockop
    262 Prockop
  3. Peter G Steinherz
    221 Steinherz
  4. Thomas Merriam Renaud
    18 Renaud
  5. Rachel Kobos
    75 Kobos
  6. Neerav Shukla
    159 Shukla