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Outcome of children with relapsed or refractory neuroblastoma: A meta‐analysis of ITCC/SIOPEN European phase II clinical trials
Author(s) -
Moreno Lucas,
Rubie Herve,
Varo Amalia,
Deley Marie Cecile,
Amoroso Loredana,
Chevance Aurelie,
Garaventa Alberto,
Gambart Marion,
Bautista Francisco,
ValteauCouanet Dominique,
Geoerger Birgit,
Vassal Gilles,
Paoletti Xavier,
Pearson Andrew D. J.
Publication year - 2017
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26192
Subject(s) - medicine , refractory (planetary science) , topotecan , neuroblastoma , vincristine , clinical trial , interquartile range , progression free survival , temozolomide , dacarbazine , oncology , surgery , chemotherapy , cyclophosphamide , physics , astrobiology , biology , genetics , cell culture
Background Few randomized trials have been conducted in children with relapsed/refractory neuroblastoma and data about outcomes including progression‐free survival (PFS) in these patients are scarce. Procedure A meta‐analysis of three phase II studies of children with relapsed/refractory neuroblastoma conducted in Europe (temozolomide, topotecan–vincristine–doxorubicin and topotecan–temozolomide) was performed. Individual patient data with extended follow‐up were collected from the trial databases after publication to describe trial outcomes (response rate, clinical benefit ratio, duration of treatment, PFS, and overall survival [OS]). Characteristics of subjects with relapsed/refractory neuroblastoma were compared. Results Data from 71 children and adolescents with relapsed/refractory neuroblastoma were collected. Response definitions were not homogeneous in the three trials. Patients were on study for a median of 3.5 months (interquartile range [IQR] 1.9–6.2). Of those, 35.2% achieved a complete or partial response, 26.3% experienced a response after more than two cycles, and 23.9% received more than six cycles. Median PFS from study entry for all, refractory, and relapsed patients was 6.4 ± 1.0, 12.5 ± 6.8, and 5.7 ± 1.0 months, respectively ( P = 0.006). Median OS from study entry for all, refractory, and relapsed patients was 16.1 ± 4.3, 27.9 ± 20.2, and 11.0 ± 1.6 months, respectively ( P = 0.03). Conclusions Baseline data for response rate, clinical benefit ratio, duration of treatment, PFS, and OS were provided. Two subpopulations (relapsed/refractory) were clearly distinct and should be included in the interpretation of all trials. These results should help informing the design of forthcoming studies in relapsed/refractory neuroblastoma.