title: Desmoplastic small round-cell tumor: prolonged progression-free survival with aggressive multimodality therapy.
abstract: OBJECTIVE
  To test intensive alkylator-based therapy in desmoplastic small round-cell tumor (DSRCT).
  
  
  METHODS
  Patients received the P6 protocol, which has seven courses of chemotherapy. Courses 1, 2, 3, and 6 included cyclophosphamide 4,200 mg/m2, doxorubicin 75 mg/m2, and vincristine (HD-CAV). Courses 4, 5, and 7 consisted of ifosfamide 9 g/m2 and etoposide 500 mg/m2 for previously untreated patients, or ifosfamide 12 g/m2 and etoposide 1,000 mg/m2 for previously treated patients. Courses started after neutrophil counts reached 500/microL and platelet counts reached 100,000/microL. Tumor resection was attempted. Post-P6 treatment options included radiotherapy and a myeloablative regimen of thiotepa (900 mg/m2) plus carboplatin (1,500 mg/m2), with stem-cell rescue.
  
  
  RESULTS
  Ten previously untreated and two previously treated patients have completed therapy. The male-to-female ratio was 11:1. Ages were 7 to 22 years (median, 14). The largest masses were infradiaphragmatic (n = 11) or intrathoracic (n = 1). Other findings included serosal implants (n = 11), regional lymph node invasion (n = 8), ascites or pleural effusion (n = 7), and metastases to liver (n = 5), lungs (n = 4), distant lymph nodes (n = 3), spleen (n = 2), and skeleton (n = 2). Tumors uniformly responded to HD-CAV, but there were no complete pathologic responses. One patient died at 1 month from tumor-related Budd-Chiari syndrome. Of seven patients who achieved a complete remission (CR), five remain in CR 9, 12, 13, 33, and 38 months from the start of P6, one patient died of infection at 12 months (autopsy-confirmed CR), and one patient relapsed 4 months off therapy. Of four patients who achieved a partial remission (PR), one remains progression-free at 34 months and three developed progressive disease. Five patients received local radiotherapy: three were not assessable for response, but in two patients, antitumor effect was evident. Four patients received thiotepa/carboplatin: two were in CR and remain so, and two patients had measurable disease that did not respond.
  
  
  CONCLUSIONS
  For control of DSRCT, our experience supports intensive use of HD-CAV, aggressive surgery to resect visible disease, radiotherapy to high-risk sites, and myeloablative chemotherapy with stem-cell rescue in selected cases.
authors.affiliation: Department of Pathology, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
authors.forename: B H
authors.initials: BH
authors.lastname: Kushner
authors.forename: M P
authors.initials: MP
authors.lastname: LaQuaglia
authors.forename: N
authors.initials: N
authors.lastname: Wollner
authors.forename: P A
authors.initials: PA
authors.lastname: Meyers
authors.forename: K L
authors.initials: KL
authors.lastname: Lindsley
authors.forename: F
authors.initials: F
authors.lastname: Ghavimi
authors.forename: T E
authors.initials: TE
authors.lastname: Merchant
authors.forename: F
authors.initials: F
authors.lastname: Boulad
authors.forename: N K
authors.initials: NK
authors.lastname: Cheung
authors.forename: M A
authors.initials: MA
authors.lastname: Bonilla
authors.forename: G
authors.initials: G
authors.lastname: Crouch
authors.forename: J F
authors.initials: JF
authors.lastname: Kelleher
authors.forename: P G
authors.initials: PG
authors.lastname: Steinherz
authors.forename: W L
authors.initials: WL
authors.lastname: Gerald
mesh_terms: D000008:Abdominal Neoplasms / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000627:therapeutic use; D004317:Doxorubicin / Q000627:therapeutic use; D005047:Etoposide / Q000627:therapeutic use; D005260:Female; D006801:Humans; D007069:Ifosfamide / Q000627:therapeutic use; D008297:Male; D011446:Prospective Studies; D016019:Survival Analysis; D014750:Vincristine / Q000627:therapeutic use
chemical_list: D014750:Vincristine; D005047:Etoposide; D004317:Doxorubicin; D003520:Cyclophosphamide; D007069:Ifosfamide