Targeted therapeutics in treatment of children and young adults with solid tumors: an expert survey and review of the literature.
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Targeted therapeutics in treatment of children and young adults with solid tumors: an expert survey and review of the literature. / Grunewald, T G P; Greulich, N; Kontny, U; Frühwald, M; Rutkowski, Stefan; Kordes, Uwe; Scheurlen, W; Schmidt, W; Stachel, D; Metzler, M; Mittler, U; Graf, N; Benesch, M; Burdach, S.
In: KLIN PADIATR, Vol. 224, No. 3, 3, 2012, p. 124-131.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Targeted therapeutics in treatment of children and young adults with solid tumors: an expert survey and review of the literature.
AU - Grunewald, T G P
AU - Greulich, N
AU - Kontny, U
AU - Frühwald, M
AU - Rutkowski, Stefan
AU - Kordes, Uwe
AU - Scheurlen, W
AU - Schmidt, W
AU - Stachel, D
AU - Metzler, M
AU - Mittler, U
AU - Graf, N
AU - Benesch, M
AU - Burdach, S
PY - 2012
Y1 - 2012
N2 - 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.
AB - 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.
KW - Humans
KW - Adolescent
KW - Young Adult
KW - Child
KW - Delphi Technique
KW - Neoplasms/drug therapy
KW - Molecular Targeted Therapy
KW - Antibodies, Monoclonal, Humanized/therapeutic use/toxicity
KW - Piperazines/therapeutic use/toxicity
KW - Pyrimidines/therapeutic use/toxicity
KW - Sirolimus/therapeutic use/toxicity
KW - Humans
KW - Adolescent
KW - Young Adult
KW - Child
KW - Delphi Technique
KW - Neoplasms/drug therapy
KW - Molecular Targeted Therapy
KW - Antibodies, Monoclonal, Humanized/therapeutic use/toxicity
KW - Piperazines/therapeutic use/toxicity
KW - Pyrimidines/therapeutic use/toxicity
KW - Sirolimus/therapeutic use/toxicity
M3 - SCORING: Journal article
VL - 224
SP - 124
EP - 131
JO - KLIN PADIATR
JF - KLIN PADIATR
SN - 0300-8630
IS - 3
M1 - 3
ER -
