Myeloablative chemotherapy with autologous stem cell transplant for desmoplastic small round cell tumor Journal Article


Authors: Forlenza, C. J.; Kushner, B. H.; Kernan, N.; Boulad, F.; Magnan, H.; Wexler, L.; Wolden, S. L.; LaQuaglia, M. P.; Modak, S.
Article Title: Myeloablative chemotherapy with autologous stem cell transplant for desmoplastic small round cell tumor
Abstract: Desmoplastic small round cell tumor (DSRCT), a rare, aggressive neoplasm, has a poor prognosis. In this prospective study, we evaluated the role of myeloablative chemotherapy, followed by autologous stem cell transplant in improving survival in DSRCT. After high-dose induction chemotherapy and surgery, 19 patients with chemoresponsive DSRCT underwent autologous stem cell transplant. Myeloablative chemotherapy consisted of carboplatin (400-700 mg/m2/day for 3 days) + thiotepa (300 mg/m2/day for 3 days) ± topotecan (2 mg/m2/day for 5 days). All patients were engrafted and there was no treatment-related mortality. Seventeen patients received radiotherapy to sites of prior or residual disease at a median of 12 weeks after transplant. Five-year event-free and overall survival were 11 ± 7% and 16 ± 8%, respectively. Two patients survive disease-free 16 and 19 years after transplant (both in complete remission before transplant). 14 patients had progression and died of disease at a median of 18 months following autologous transplant. These data do not justify the use of myeloablative chemotherapy with carboplatin plus thiotepa in patients with DSRCT. Alternative therapies should be considered for this aggressive neoplasm. © 2015 Christopher J. Forlenza et al.
Keywords: adolescent; adult; cancer chemotherapy; cancer survival; child; clinical article; event free survival; school child; treatment response; cancer surgery; overall survival; thalidomide; mortality; cancer recurrence; bevacizumab; cisplatin; doxorubicin; sunitinib; cancer growth; diarrhea; hypertension; multimodality cancer therapy; treatment duration; cancer radiotherapy; disease free survival; temozolomide; topotecan; drug megadose; prospective study; cancer palliative therapy; carboplatin; bone marrow suppression; etoposide; mucosa inflammation; vomiting; cyclophosphamide; vincristine; hematuria; ifosfamide; thiotepa; irinotecan; cancer regression; engraftment; myeloablative conditioning; minimal residual disease; dactinomycin; sepsis; hospital discharge; hyperbilirubinemia; navelbine; exatecan; hemorrhagic cystitis; hearing impairment; autologous hematopoietic stem cell transplantation; induction chemotherapy; desmoplastic small round cell tumor; human; male; female; priority journal; article
Journal Title: Sarcoma
Volume: 2015
ISSN: 1357-714X
Publisher: Hindawi Publishing Corporation  
Date Published: 2015-01-01
Start Page: 269197
Language: English
DOI: 10.1155/2015/269197
PROVIDER: scopus
PMCID: PMC4405024
PUBMED: 25945075
DOI/URL:
Notes: Export Date: 3 June 2015 -- Source: Scopus
Altmetric Score
MSK Authors
  1. Brian Kushner
    192 Kushner
  2. Nancy Kernan
    382 Kernan
  3. Farid Boulad
    255 Boulad
  4. Shakeel Modak
    160 Modak
  5. Suzanne L Wolden
    424 Wolden
  6. Leonard H Wexler
    132 Wexler
  7. Heather Magnan
    25 Magnan