title: Malignant rhabdoid tumor of the kidney: significantly improved response to pre-operative treatment intensified with doxorubicin.
abstract: Case reports and in vitro testing suggest sensitivity of malignant rhabdoid tumor of the kidney (MRTK) to anthracyclines. Prospective study data supporting doxorubicin's efficacy is lacking. We compared the change of tumor volume in the kidney to upfront treatment with either actinomycin D and vincristine (AV) or doxorubicin-intensified AV (AVD) in all patients with MRTK, who had been treated from 1991-2013 in Austria, Switzerland, and Germany in the framework of three prospective Société International d'Oncologie Pédiatrique/Gesellschaft für Pädiatrische Onkologie und Hämatologie nephroblastoma studies. A total of 37 patients with MRTK received pre-operative chemotherapy (AV, n = 19; AVD, n = 18). Initial and tumor volume after pre-operative treatment was reported in all patients who received AV and 15 of 18 (83%) patients who received AVD. Mean tumor volume at diagnosis was 247 (±48) mL in the AV cohort and 345 (±47) mL in the AVD cohort. Mean volume at surgery was 249 (±46) mL and 137 (±27) mL, respectively. Relative change in tumor volume was +19 (±16)% in patients who received AV and -63 (±26)% in patients who received AVD (P < 0.001). Change in volume to AV ranged from -60 to +224%, whereas the change to AVD ranged from -9 to -92%. We provide good evidence of doxorubicin's activity in MRTK in vivo by demonstrating a significantly better response to neoadjuvant AVD compared with AV alone.
authors.affiliation: Saarland University Hospital, Pediatric Hematology and Oncology, Homburg/Saar, Germany. Electronic address: rhoikos.furtwaengler@uks.eu.
authors.forename: Rhoikos
authors.initials: R
authors.lastname: Furtwängler
authors.affiliation: Saarland University Hospital, Pediatric Hematology and Oncology, Homburg/Saar, Germany.
authors.forename: Nasinien
authors.initials: N
authors.lastname: Nourkami-Tutdibi
authors.affiliation: Pediatric Pathology, Schleswig-Holstein University Hospital, Campus Kiel, Kiel, Germany.
authors.forename: Ivo
authors.initials: I
authors.lastname: Leuschner
authors.affiliation: Pediatric Pathology, Schleswig-Holstein University Hospital, Campus Kiel, Kiel, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Vokuhl
authors.affiliation: Children's Hospital Zurich, University of Zurich, Section of Pediatric Oncology, Zurich, Switzerland.
authors.forename: Felix
authors.initials: F
authors.lastname: Niggli
authors.affiliation: St. Anna Children's Hospital, Vienna University Hospital, Vienna, Austria.
authors.forename: Leo
authors.initials: L
authors.lastname: Kager
authors.affiliation: Pediatric Hematology and Oncology, University Hospital, Tübingen, Germany.
authors.forename: Martin
authors.initials: M
authors.lastname: Ebinger
authors.affiliation: Swabian Children's Cancer Center, Children's Hospital Augsburg, Augsburg, Germany.
authors.forename: Michael C
authors.initials: MC
authors.lastname: Frühwald
authors.affiliation: Saarland University Hospital, Pediatric Hematology and Oncology, Homburg/Saar, Germany.
authors.forename: Norbert
authors.initials: N
authors.lastname: Graf
mesh_terms: D000293:Adolescent; D000328:Adult; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003609:Dactinomycin / Q000627:therapeutic use; D004317:Doxorubicin / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D011300:Preoperative Care; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D016896:Treatment Outcome; D047368:Tumor Burden; D055815:Young Adult
chemical_list: D000903:Antibiotics, Antineoplastic; D003609:Dactinomycin; D004317:Doxorubicin
keywords: Doxorubicin; childhood renal tumor; response; rhabdoid tumor; upfront chemotherapy