title: Targeted therapeutics in treatment of children and young adults with solid tumors: an expert survey and review of the literature.
abstract: Although prognosis of children with solid tumors is steadily improving, long-term survival is not achievable in all patients, especially in patients with recurrent or refractory disease. Despite the increasing number of targeted therapeutics (TT), only very few TT have been introduced into clinical protocols. Accordingly, clinical experience concerning the efficacy and safety of these drugs is limited. This may possibly discourage oncologists from administering TT to children.We performed a comprehensive review of the literature to identify TT that may be considered for treatment of children and young adults with solid tumors. Moreover, we interviewed an expert panel of the Society for Pediatric Oncology and Hematology (GPOH) using questionnaires in a modified Delphi process in order to describe the experts' experiences in the use of these TT.Among 30 TT identified to be possibly useful in children and young adults, imatinib, bevacizumab and rapamycin were most widely used. These drugs were reported as having mostly little to no severe adverse events and seem to induce at least partial responses in a subset of patients. In addition, our study confirms and expands the present knowledge about adverse events and the potential efficacy of 5 other commonly used TT in this population.This information may be useful for oncologists when administering these TT to children and young adults with solid tumors. Controlled clinical trials are urgently needed to test their safety and efficacy.
authors.affiliation: Department of Pediatrics, Children's Cancer Research and Roman Herzog Comprehensive Cancer Center, Klinikum rechts der Isar, TU München, Munich, Germany. thomas.gruenewald@lrz.tum.de
authors.forename: T G P
authors.initials: TG
authors.lastname: Grunewald
authors.forename: N
authors.initials: N
authors.lastname: Greulich
authors.forename: U
authors.initials: U
authors.lastname: Kontny
authors.forename: M
authors.initials: M
authors.lastname: Frühwald
authors.forename: S
authors.initials: S
authors.lastname: Rutkowski
authors.forename: U
authors.initials: U
authors.lastname: Kordes
authors.forename: W
authors.initials: W
authors.lastname: Scheurlen
authors.forename: W
authors.initials: W
authors.lastname: Schmidt
authors.forename: D
authors.initials: D
authors.lastname: Stachel
authors.forename: M
authors.initials: M
authors.lastname: Metzler
authors.forename: U
authors.initials: U
authors.lastname: Mittler
authors.forename: N
authors.initials: N
authors.lastname: Graf
authors.forename: M
authors.initials: M
authors.lastname: Benesch
authors.forename: S
authors.initials: S
authors.lastname: Burdach
mesh_terms: D000293:Adolescent; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use / Q000633:toxicity; D001549:Benzamides; D000068258:Bevacizumab; D002648:Child; D003697:Delphi Technique; D006801:Humans; D000068877:Imatinib Mesylate; D058990:Molecular Targeted Therapy; D009369:Neoplasms / Q000188:drug therapy*; D010879:Piperazines / Q000627:therapeutic use / Q000633:toxicity; D011743:Pyrimidines / Q000627:therapeutic use / Q000633:toxicity; D020123:Sirolimus / Q000627:therapeutic use / Q000633:toxicity; D055815:Young Adult
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068258:Bevacizumab; D000068877:Imatinib Mesylate; D020123:Sirolimus