High-dose chemotherapy with autologous stem cell rescue in children with nephroblastoma

• Published in: Bone marrow transplantation (Basingstoke) Vol. 30; no. 12; pp. 893 - 898
• Main Authors: KREMENS, B, GRUHN, B, SCHULZ, A, WAWER, A, ZINTL, F, GRAF, N, KLINGEBIEL, T, HASAN, C, LAWS, H.-J, KOSCIELNIAK, E, HERO, B, SELLE, B, NIEMEYER, C, FINCKENSTEIN, F. G
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.12.2002
• Subjects: Antineoplastic agents; Antineoplastic Combined Chemotherapy Protocols - administration & dosage; Antineoplastic Combined Chemotherapy Protocols - adverse effects; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Autografts; Biological and medical sciences; Bone marrow; Carboplatin; Carboplatin - administration & dosage; Carboplatin - adverse effects; Chemotherapy; Child; Child, Preschool; Children; Combined Modality Therapy; Combined treatments (chemotherapy of immunotherapy associated with an other treatment); Cyclophosphamide - administration & dosage; Cyclophosphamide - analogs & derivatives; Disease-Free Survival; Dosage; Etoposide; Etoposide - administration & dosage; Etoposide - adverse effects; Female; Germany - epidemiology; Glomerular filtration rate; Hematologic Diseases - chemically induced; Hematopoietic stem cells; Hemodialysis; Humans; Ifosfamide - administration & dosage; Infant; Kidney Diseases - chemically induced; Kidney Neoplasms - drug therapy; Kidney Neoplasms - mortality; Kidney Neoplasms - therapy; Kidneys; Life Tables; Lung Neoplasms - drug therapy; Lung Neoplasms - secondary; Male; Medical prognosis; Medical sciences; Melphalan; Melphalan - administration & dosage; Melphalan - adverse effects; Metastases; Mucositis; Nephrology. Urinary tract diseases; Patients; Peripheral blood; Peripheral Blood Stem Cell Transplantation; Pharmacology. Drug treatments; Prognosis; Remission; Renal failure; Renal function; Risk; Stem cell transplantation; Stem cells; Stomatitis - chemically induced; Survival; Survival Analysis; Thiotepa - administration & dosage; Toxicity; Transplantation, Autologous; Tumors; Tumors of the urinary system; Wilms Tumor - drug therapy; Wilms Tumor - mortality; Wilms Tumor - secondary; Wilms Tumor - therapy; Germany; Human; Kidney disease; Antineoplastic agent; Relapse; Urinary system disease; Toxicity; Stem cell; Adjuvant treatment; Hematopoietic cell; Malignant tumor; Survival; Blood; Autograft; Chemotherapy; Treatment; Wilms tumor; Graft; Evolution; autologous bone marrow transplantation; High dose; Child; high-dose chemotherapy
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/sj.bmt.1703771

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.