Imatinib mesylate in Philadelphia chromosome-positive leukemia of childhood Journal Article


Authors: Kolb, E. A.; Pan, Q.; Ladanyi, M.; Steinherz, P. G.
Article Title: Imatinib mesylate in Philadelphia chromosome-positive leukemia of childhood
Abstract: BACKGROUND. Initial treatment for adult patients with Philadelphia chromosome-positive (Ph[+]) chronic myelogenous leukemia (CML) now includes imatinib mesylate. However, to our knowledge, there are few data regarding imatinib safety, efficacy, and response monitoring in patients age < 18 years. METHODS. In the current series, the authors report 5 consecutive patients ages 20 months to 12 years with Ph(+) leukemia who were treated with imatinib and evaluated for a response using cytogenetics, fluorescent in situ hybridization (FISH), and real-time quantitative reverse transcriptase-polymerase chain reaction (RT-PCR) on serial bone marrow aspirations. Doses of imatinib were escalated as tolerated from a starting dose of 400 mg/m2 (patients with a body surface area [BSA] < 1 m 2) or 400 mg/day (patients with a BSA > 1 m2). RESULTS. After the initiation of imatinib therapy, all 4 patients with CML were found to have no detectable Ph chromosome by cytogenetics (median of 198 days of imatinib therapy; range, 138-346 days), FISH (median of 285 days of imatinib therapy; range, 138-366 days), and real-time RT-PCR (median of 287 days of imatinib therapy; range, 224-366 days). One patient with Ph(+) acute mixed lineage leukemia achieved a morphologic disease remission with standard chemotherapy, but within 10 months had increasing Ph positivity in consecutive bone marrow aspirations. Imatinib was added to the intensive leukemia therapy, and within 26 days there were no detectable Ph(+) cells in the bone marrow. Mild thrombocytopenia was noted in two patients and transient mild hepatic toxicity was noted in one patient. CONCLUSIONS. Imatinib mesylate was found to be effective in inducing undetectable residual disease in a small cohort of pediatric patients with Ph(+) leukemia. Further studies of the use of imatinib in childhood Ph(+) malignancies are needed. © 2003 American Cancer Society.
Keywords: child; clinical article; controlled study; treatment outcome; child, preschool; survival rate; chronic myelogenous leukemia (cml); drug efficacy; drug safety; antineoplastic agents; in situ hybridization, fluorescence; imatinib; reverse transcription polymerase chain reaction; liver toxicity; bone marrow suppression; thrombocytopenia; risk factors; cytogenetics; patient monitoring; chronic myeloid leukemia; pyrimidines; childhood cancer; cancer regression; fluorescence in situ hybridization; infant; bone marrow biopsy; quantitative analysis; response; protein-tyrosine kinases; piperazines; childhood leukemia; fusion proteins, bcr-abl; philadelphia 1 chromosome; imatinib mesylate; philadelphia chromosome; leukemia, myeloid, chronic-phase; leukemia, myeloid, philadelphia-positive; pediatric patients; humans; prognosis; human; male; female; priority journal; article; philadelphia chromsome (ph)
Journal Title: Cancer
Volume: 98
Issue: 12
ISSN: 0008-543X
Publisher: Wiley Blackwell  
Date Published: 2003-12-15
Start Page: 2643
End Page: 2650
Language: English
DOI: 10.1002/cncr.11895
PUBMED: 14669284
PROVIDER: scopus
DOI/URL:
Notes: Export Date: 12 September 2014 -- Source: Scopus
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MSK Authors
  1. Marc Ladanyi
    1326 Ladanyi
  2. Peter G Steinherz
    221 Steinherz
  3. Anders E Kolb
    16 Kolb
  4. Qiulu Pan
    11 Pan