Treatment of anaplastic histology Wilms' tumor: results from the fifth National Wilms' Tumor Study.
OBJECTIVE
An objective of the fifth National Wilms' Tumor Study (NWTS-5) was to evaluate the efficacy of treatment regimens for anaplastic histology Wilms' tumor (AH).


METHODS
Prospective single-arm studies were conducted. Patients with stage I AH were treated with vincristine and dactinomycin for 18 weeks. Patients with stages II to IV diffuse AH were treated with vincristine, doxorubicin, cyclophosphamide, and etoposide for 24 weeks plus flank/abdominal radiation.


RESULTS
A total of 2,596 patients with Wilms' tumor were enrolled onto NWTS-5, of whom 281 (10.8%) had AH. Four-year event-free survival (EFS) and overall survival (OS) estimates for assessable patients with stage I AH (n = 29) were 69.5% (95% CI, 46.9 to 84.0) and 82.6% (95% CI, 63.1 to 92.4). In comparison, 4-year EFS and OS estimates for patients with stage I favorable histology (FH; n = 473) were 92.4% (95% CI, 89.5 to 94.5) and 98.3% (95% CI, 96.4 to 99.2). Four-year EFS estimates for patients who underwent immediate nephrectomy with stages II (n = 23), III (n = 43), and IV (n = 15) diffuse AH were 82.6% (95% CI, 60.1 to 93.1), 64.7% (95% CI, 48.3 to 77.7), and 33.3% (95% CI, 12.2 to 56.4), respectively. OS was similar to EFS for these groups. There were no local recurrences among patients with stage II AH. Four-year EFS and OS estimates for patients with bilateral AH (n = 29) were 43.8% (95% CI, 24.2 to 61.8) and 55.2% (95% CI, 34.8 to 71.7), respectively.


CONCLUSIONS
The prognosis for patients with stage I AH is worse than that for patients with stage I FH. Novel treatment strategies are needed to improve outcomes for patients with AH, especially those with stage III to V disease.
Dome
Jeffrey S
JS
Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA. jeff.dome@stjude.org
Cotton
Cecilia A
CA
Perlman
Elizabeth J
EJ
Breslow
Norman E
NE
Kalapurakal
John A
JA
Ritchey
Michael L
ML
Grundy
Paul E
PE
Malogolowkin
Marcio
M
Beckwith
J Bruce
JB
Shamberger
Robert C
RC
Haase
Gerald M
GM
Coppes
Max J
MJ
Coccia
Peter
P
Kletzel
Morris
M
Weetman
Robert M
RM
Donaldson
Milton
M
Macklis
Roger M
RM
Green
Daniel M
DM
D000293:Adolescent; D000708:Anaplasia; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide; D003609:Dactinomycin / Q000008:administration & dosage; D004317:Doxorubicin / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007668:Kidney / Q000473:pathology*; D007680:Kidney Neoplasms / Q000473:pathology / Q000628:therapy*; D008297:Male; D009367:Neoplasm Staging; D009392:Nephrectomy; D011379:Prognosis; D011446:Prospective Studies; D011878:Radiotherapy; D018335:Rhabdoid Tumor / Q000473:pathology / Q000628:therapy; D018227:Sarcoma, Clear Cell / Q000473:pathology / Q000628:therapy; D016019:Survival Analysis; D014750:Vincristine / Q000008:administration & dosage; D009396:Wilms Tumor / Q000473:pathology / Q000628:therapy*
D003609:Dactinomycin; D014750:Vincristine; D005047:Etoposide; D004317:Doxorubicin; D003520:Cyclophosphamide