title: High-dose chemotherapy with autologous stem cell rescue in children with nephroblastoma.
abstract: Children with Wilms tumor who have a particular risk of failure at relapse or at primary diagnosis were treated with high-dose chemotherapy (HDC) and autologous peripheral blood stem cell rescue in order to improve their probability of survival. From April 1992 to December 1998, 23 evaluable patients received HDC within the German Cooperative Wilms Tumor Studies. Nineteen were given melphalan, etoposide and carboplatin (MEC); the others received different regimens. The dose of carboplatin was adjusted according to renal function. Indications for HDC were high-risk relapse in 20 patients, bone metastases in two patients and no response in one patient. Fourteen of 23 patients are alive after a median observation time of 41 months, 11 of 14 in continuous complete remission, three in CR after relapse post HDC. The estimated survival and event-free survival for these patients are 60.9% and 48.2%. Twelve children relapsed after HDC; nine of them died within 12 months and three are surviving from 20 to 33 months after relapse. The main toxicities were hematologic, mucositis and renal (tubular dysfunction; intermittent hemodialysis in one patient). There were no toxic deaths. About half of the children suffering from Wilms tumor with very unfavorable prognostic factors survive disease-free after HDC for over 3 years. Besides hematological toxicity, mucositis and infections, renal function is at risk during HDC. With dose adjustment on glomerular filtration rate, however, no permanent renal failure was observed.
authors.affiliation: Department of Pediatric Hematology/Oncology, University of Essen, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Kremens
authors.forename: B
authors.initials: B
authors.lastname: Gruhn
authors.forename: T
authors.initials: T
authors.lastname: Klingebiel
authors.forename: C
authors.initials: C
authors.lastname: Hasan
authors.forename: H-J
authors.initials: HJ
authors.lastname: Laws
authors.forename: E
authors.initials: E
authors.lastname: Koscielniak
authors.forename: B
authors.initials: B
authors.lastname: Hero
authors.forename: B
authors.initials: B
authors.lastname: Selle
authors.forename: C
authors.initials: C
authors.lastname: Niemeyer
authors.forename: F G
authors.initials: FG
authors.lastname: Finckenstein
authors.forename: A
authors.initials: A
authors.lastname: Schulz
authors.forename: A
authors.initials: A
authors.lastname: Wawer
authors.forename: F
authors.initials: F
authors.lastname: Zintl
authors.forename: N
authors.initials: N
authors.lastname: Graf
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000031:analogs & derivatives*; D018572:Disease-Free Survival; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D005858:Germany / Q000453:epidemiology; D006402:Hematologic Diseases / Q000139:chemically induced; D006801:Humans; D007069:Ifosfamide / Q000008:administration & dosage; D007223:Infant; D007674:Kidney Diseases / Q000139:chemically induced; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000628:therapy; D015993:Life Tables; D008175:Lung Neoplasms / Q000188:drug therapy / Q000556:secondary; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D036102:Peripheral Blood Stem Cell Transplantation; D011379:Prognosis; D013280:Stomatitis / Q000139:chemically induced; D016019:Survival Analysis; D013852:Thiotepa / Q000008:administration & dosage; D014182:Transplantation, Autologous; D009396:Wilms Tumor / Q000188:drug therapy* / Q000401:mortality / Q000556:secondary / Q000628:therapy
chemical_list: D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; C003726:trofosfamide; D008558:Melphalan; D007069:Ifosfamide