Busulphan-melphalan as a myeloablative therapy (MAT) for high-risk neuroblastoma: Results from the HR-NBL1/SIOPEN trial.

Ladenstein, Ruth; Pötschger, Ulrike; Luksch, Roberto; Brock, Pénélope; Castel, Victoria; Yaniv, Isaac; Papadakis, Vassilios; Laureys, Geneviève; Malis, J; Balwierz, Walentyna; Kogner, Per; Schroeder, H.; Lacerda, Ana Forjaz de; Popović, M.; Bician, P.; Garami, Miklós; Trahair, Toby N.; Pearson, A; Couanet, D.

doi:10.1200/jco.2011.29.15_suppl.2

Cited by 32 publications

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“…In this study, HDC/SCR was performed using carboplatin/etoposide/cyclophosphamide followed in rapid sequence by thiotepa/cyclophosphamide. Alternative consolidation regimens in HDC/SCR studies include melphalan (as a single agent) [2], busulphan/melphalan [17] and carboplatin/etoposide/melphalan (CEM) [14]. A recent and compelling report from the ENSG suggests that, in a randomized comparison of patients who achieved a good response with a platinum-intensive induction regimen, busulphan/melphalan is associated with an improved event-free survival compared with CEM [17].…”

Section: Discussionmentioning

confidence: 99%

Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: A Pediatric Oncology Group study: A Report from the Children's Oncology Group

Granger

Grupp

Kletzel

et al. 2012

Pediatric Blood & Cancer

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Background The Pediatric Oncology Group performed a pilot study to assess the feasibility of tandem high dose chemotherapy with stem cell rescue (HDC/SCR). We report here the results of this single arm trial of induction chemotherapy, local control measures (surgery and local radiation), and tandem HDC/SCR. Procedure Patients with high risk neuroblastoma underwent five cycles of induction chemotherapy and resection of primary tumors. PBSC were collected after Course 3 without exvivo manipulation. Myeloablative chemotherapy was performed in rapid sequence after induction chemotherapy and surgery. The ability of patients to complete both cycles of HDC/SCR was a primary endpoint. Transplant-related toxicity, progression-free survival (PFS) and overall survival (OS) were recorded. Results A total of 33 patients were enrolled. Twenty-two patients completed at least one HDC/SCR procedure and 17 patients completed both. Only one patient had insufficient stem cells collected for both transplants. There was one transplant-related death; engraftment was rapid and toxicity was as expected. The PFS of the 33 patients treated on this study is 24.2%±7.5% and OS is 36.4%±8.4% at 5 years. For patients who received at least one transplant PFS is 36.4%±11.0% and OS is 45.5%±11.2% at 5 years. Conclusions The treatment of high risk neuroblastoma with tandem HDC/SCR is feasible in terms of transplant-related mortality and the ability to collect adequate PBSC for 2 transplants. The outcomes from this intensified treatment have been used to design a Children's Oncology Group Phase III study testing the efficacy of tandem HDC/SCR.

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Section: Discussionmentioning

confidence: 99%

Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: A Pediatric Oncology Group study: A Report from the Children's Oncology Group

Granger

Grupp

Kletzel

et al. 2012

Pediatric Blood & Cancer

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“…These were the findings of a phase III trial (HR-NBL1) [1] of the European SIOP Neuroblastoma (SIOP-EN) Group involving 598 randomized children (median age 3 years), presented by Ruth Ladenstein (St Anna Children's Hospital and Research Institute, Vienna, Austria), the lead author. This is a welcome step forward for this very difficult-to-treat disease.…”

Section: High-risk Neuroblastomamentioning

confidence: 96%

“…The positive results with the use of BuMel [1] and the encouraging toxicity profile of mAb ch14.18 [2] suggest that we can significantly improve the outcomes of these patients with hardto-treat neuroblastomas.…”

Section: High-risk Neuroblastomamentioning

confidence: 99%

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47th Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago, IL, USA, 3–7 June 2011

Wilde

2011

Pediatric Drugs

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There was much excitement at the 47th Annual Meeting of the American Society of Clinical Oncology (ASCO) and respect for patients and families who participated in the trials that were presented. The theme of the conference was patients, pathways, and progress. An estimated 35 000-40 000 oncology professionals attended.We now have a wealth of knowledge about the biology and pathology of tumors and tumor resistance, and a better understanding of the immune system and the cancer molecular pathways and targets. An exponential number of new drugs targeted to specific cancer pathway targets are becoming available or are in development. In addition, and importantly, we have increased knowledge of molecular gene expression profiling (which can aid determination of tumor aggressiveness and patient selection), predictive and prognostic biomarkers, and the necessary companion diagnostics now often needed for approval of many drugs. The future will see a multitude of tools for individualization of treatment, early detection of primary cancer, monitoring for relapse, and surveillance of specific patients at high risk of cancer development. The overriding view was that although we have come such a long way we are really only at the beginning of what will be known as 'The Genomic Era' of oncology.As for all areas of medicine, oncology data for the pediatric population are lagging behind that for adults. However, it was very reassuring that two of the six plenary session presentations were related to pediatric oncology, reinforcing the importance of and progress in this field. Furthermore, Dr Michael P. Link (the Lydia J. Lee Professor in Pediatric Cancer, Stanford University School of Medicine, Stanford, CA, USA) has been elected as the ASCO President for the 2011-12 term. Although he will not be representing pediatric oncology in his role as ASCO president, his background further represents the importance that pediatric oncology has made to advances in oncology.The relative dearth of information and lack of progress in diagnosing and treating adolescents and young adults with cancer has been acknowledged and addressed by ASCO launching the Focus Under Forty Ô online education programme (http://university.asco.org/focusunder40). This programme aims to help physicians better care for patients with cancer aged 15-39 years.Some of the more notable developments in pediatric oncology presented at ASCO 2011, including the two plenary presentations mentioned above, are discussed here. Neuroblastoma High-Risk NeuroblastomaThat current practice should now be in favour of busulphan plus melphalan (BuMel) as the preferred myeloablative therapy in children with high-risk neuroblastoma was the consensus view of several pediatric oncology key opinion leaders. This is a welcome step forward for this very difficult-to-treat disease. Event-free survival (49% vs 33%) and overall survival (60% vs 48%) rates after 3 years in children with high-risk neuroblastoma were significantly higher with the combination myeloablative regimen BuMel than with a...

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“…Recently the SIOPEN group has been testing the combination of busulfan and melphalan for autologous SCT, comparing this conditioning regimen to CEM. Although these data have not yet been published, they were presented in a plenary session by Dr. Ruth Ladenstein at ASCO this year(20). Although only about a third of enrolled patients actually underwent the randomization, the study reports a statistically significant difference in outcome between the Bu/Mel and CEM arms, with 3 year EFS in the Bu/Mel being 48% compared to 33% with CEM.…”

Section: Bu/mel Vs Cemmentioning

confidence: 99%

Neuroblastoma: Issues in Transplantation

Grupp

Asgharzadeh

Yanik

2012

Biology of Blood and Marrow Transplantation

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Busulphan-melphalan as a myeloablative therapy (MAT) for high-risk neuroblastoma: Results from the HR-NBL1/SIOPEN trial.

Cited by 32 publications

References 0 publications

Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: A Pediatric Oncology Group study: A Report from the Children's Oncology Group

Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: A Pediatric Oncology Group study: A Report from the Children's Oncology Group

47th Annual Meeting of the American Society of Clinical Oncology (ASCO), Chicago, IL, USA, 3–7 June 2011

Neuroblastoma: Issues in Transplantation

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