Type I pleuropulmonary blastoma: a report from the International Pleuropulmonary Blastoma Registry.
OBJECTIVE
Type I pleuropulmonary blastoma (PPB) is a rare, cystic lung neoplasm in infants characterized by subtle malignant changes and a good prognosis. Recurrences after type I PPB are usually advanced type II or type III neoplasms with a poor prognosis. This article describes the first collection of type I PPB cases, analyzes outcome based on treatments of surgery or surgery plus chemotherapy, and presents type I PPB management recommendations.


METHODS
Type I PPB cases from the International PPB Registry and literature were evaluated using standard statistical methods for outcomes based on age at diagnosis, sex, thoracic side, surgical extent, length of follow-up, constitutional/familial disease, pre-existing lung cysts, intrathoracic findings, and treatments (surgery or surgery and chemotherapy).


RESULTS
Thirty-eight type I PPB cases were identified: Registry (n = 30) and literature (n = 8). Twenty children had surgery alone; eight (40%) experienced recurrence; and four died. Eighteen children had surgery and adjuvant chemotherapy; one experienced recurrence and died. All recurrences were type II or III PPB. Recurrence-free survival was higher in the surgery + chemotherapy group (P = .01); overall survival did not differ (P = .18). The improved recurrence-free survival was found only in males. Four of nine children with recurrence survived.


CONCLUSIONS
Adjuvant chemotherapy appears to benefit type I PPB patients. Benefit limited to males requires broader substantiation. Salvage after types II and III recurrence is poor (four of nine; 44%). A rigorous surveillance schedule after type I PPB diagnosis might detect early recurrence and be an acceptable alternative to adjuvant chemotherapy.
Priest
John R
JR
International Pleuropulmonary Blastoma Registry, Children's Hospitals and Clinics of Minnesota, St Paul, Minnesota 55102, USA. jprst@prodigy.net
Hill
D Ashley
DA
Williams
Gretchen M
GM
Moertel
Christopher L
CL
Messinger
Yoav
Y
Finkelstein
Marsha J
MJ
Dehner
Louis P
LP
D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D015993:Life Tables; D008175:Lung Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000175:diagnosis* / Q000188:drug therapy*; D010997:Pleural Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D011159:Population Surveillance; D018202:Pulmonary Blastoma / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery; D012042:Registries; D012189:Retrospective Studies; D016896:Treatment Outcome