title: WT1 (Wilms tumor 1) peptide immunotherapy for childhood rhabdomyosarcoma: a case report.
abstract: Immunotherapy using a Wilms tumor (WT1) peptide has been undergoing clinical trials for adulthood leukemia and solid cancer with promising results. In this study, the authors used WT1 peptide vaccination to treat a 6-year-old girl with metastatic alveolar rhabdomyosarcoma. She received weekly intradermal injection with HLA-A*2404-restricted, 9-mer WT1 peptide against residual bone disease. After 3 months her bone disease disappeared, concurrent with an increase in the frequency of WT1-specific cytotoxic T lymphocytes (CTLs). A high proportion of WT1-specific CTLs with effector or effector memory phenotype were detected in peripheral blood of this patient. She is currently still on continued WT1 peptide immunotherapy in a disease-free condition for 22 months. WT1 peptide-based immunotherapy should be a promising option for high-risk rhabdomyosarcoma in childhood.
authors.affiliation: Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Osaka, Japan. ohta@ped.med.osaka-u.ac.jp
authors.forename: Hideaki
authors.initials: H
authors.lastname: Ohta
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Hashii
authors.forename: Akihiro
authors.initials: A
authors.lastname: Yoneda
authors.forename: Sachiko
authors.initials: S
authors.lastname: Takizawa
authors.forename: Shigenori
authors.initials: S
authors.lastname: Kusuki
authors.forename: Sadao
authors.initials: S
authors.lastname: Tokimasa
authors.forename: Masahiro
authors.initials: M
authors.lastname: Fukuzawa
authors.forename: Akihiro
authors.initials: A
authors.lastname: Tsuboi
authors.forename: Ayako
authors.initials: A
authors.lastname: Murao
authors.forename: Yoshihiro
authors.initials: Y
authors.lastname: Oka
authors.forename: Yusuke
authors.initials: Y
authors.lastname: Oji
authors.forename: Katsuyuki
authors.initials: K
authors.lastname: Aozasa
authors.forename: Shin-ichi
authors.initials: S
authors.lastname: Nakatsuka
authors.forename: Haruo
authors.initials: H
authors.lastname: Sugiyama
authors.forename: Keiichi
authors.initials: K
authors.lastname: Ozono
mesh_terms: D001847:Bone Diseases / Q000188:drug therapy; D002648:Child; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007167:Immunotherapy / Q000379:methods*; D010446:Peptide Fragments / Q000627:therapeutic use*; D018232:Rhabdomyosarcoma, Alveolar / Q000188:drug therapy* / Q000473:pathology; D013602:T-Lymphocytes, Cytotoxic / Q000276:immunology; D014611:Vaccination; D025721:WT1 Proteins / Q000627:therapeutic use*
chemical_list: D010446:Peptide Fragments; D025721:WT1 Proteins