One of the 652 children who participated in the trial was Katie Belle of Seattle, who was 3½ years old in 2009, when doctors found a baseball-sized tumor in her abdomen that turned out to be high-risk neuroblastoma. This is known as a stem cell transplant (SCT). Three-year event-free survival — meaning survival without relapse, progressive disease, or a second malignancy — was significantly better in the double-transplant group (62 vs. 48 percent). Bone Marrow Transplant 2016; 51:227. Lancet Oncol. Diller was the study’s senior investigator. All Rights Reserved. However, the results of a Phase III trial presented on Sunday in the Plenary Session of the American Society of Clinical Oncology Annual Meeting (3–7th June 2016, IL, USA) have indicated that the addition of a second autologous stem cell transplant (ASCT), utilizing a patient’s own stem cells, could improve survival outcomes. Radiation therapy followed all transplants. Before the COG trial, the standard treatment for high-risk neuroblastoma was a single-transplant approach that involved high-dose chemotherapy to destroy as many cancer cells as possible, followed by an autologous (self-donating) blood stem cell transplant. Zhuang SH, et al. “We don’t know what the other long-term complications will be,” she said. Children with a median age of 3.1 years who had been newly diagnosed with high-risk neuroblastoma were enrolled onto the trial. Some children might have 2 stem cell transplants, called tandem stem cell transplants. Privacy Policy| He can be reached at kushnerb@mskcc.org. 1999;doi:10.1056/NEJM199910143411601. The new approach in the recent COG trial was to double-up that treatment, with the hopes of increasing the percentage of patients who would benefit from this intense therapy. 2005;doi:10.1002/pbc.20219. 2014;doi:10.1200/JCO.2013.53.8009. or, by Nancy Fliesler, Children's Hospital Boston. These cells are later returned, helping the child weather the toxic effects of the chemo.

Jennifer worries about her future. Apart from any fair dealing for the purpose of private study or research, no Pinto NR, et al. Building off this finding, Park and colleagues reported outcomes of children enrolled on the randomized phase 3 ANBL0532 trial, in which children with high-risk neuroblastoma were randomly assigned to one or two consolidative myeloablative treatments with autologous stem cell rescue. Customize your JAMA Network experience by selecting one or more topics from the list below. All individuals received six cycles of multi-agent induction chemotherapy regimen, including an initial two cycles of high-dose cyclophosphamide/topotecan followed by stem cell collection from the blood to be used for subsequent transplantation. By continuing to use our site, or clicking "Continue," you are agreeing to our, 2020 American Medical Association. 2019;doi:10.1001/jama.2019.11642. Get weekly and/or daily updates delivered to your inbox. Over the course of a decade, Lisa Diller, MD and her colleagues tested and refined the double-transplant treatment. “Through continued research, we keep moving the needle and improving survival,” Julie reflected. Bagatelli R and Irwin MS. JAMA. 2018;doi:10.1001/jamaoncol.2018.4005. The researchers also note that overall survival was not statistically significantly different between the tandem- and single-transplant groups, and that the benefit of tandem transplant might not extend to patients treated with different chemotherapy regimens before transplant. About 800 children are diagnosed with neuroblastoma each year in the United States.

In a multicenter randomized trial led by investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Seattle Children’s Hospital, the double transplant enabled children to survive longer without new or recurrent disease over three years of follow-up. Moroz V, et al. So there might be patients who don't benefit from tandem transplant that were underrepresented in the study. Three years after treatment for neuroblastoma, 61.4% of patients who received the dual autologous stem-cell transplants (ASCT) were alive without cancer compared with 48.4% of … If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. The team presented initial results of the trial at the American Society of Clinical Oncology annual meeting in 2016. has remained low: mortality in high-risk neuroblastoma can be as high as 50 2005;doi:10.1016/S1470-2045(05)70291-6. Yalcin B, et al. First, to make the approach feasible, they developed a new technique for harvesting circulating blood stem cells from very small children.

2013;doi:10.1002/14651858.CD006301.pub3. Cochrane Database Syst Rev. Since then, tandem transplant has been considered the standard of care for ongoing and future Children’s Oncology Group Trials. Berthold F, et al. The researchers also note that overall survival was not statistically significantly different between the tandem- and single-transplant groups, and that the benefit of tandem transplant might not extend to patients treated with different chemotherapy regimens before transplant. In sequential studies at Memorial Sloan Kettering Cancer Center, transplant did not improve OS, whereas anti-GD2 murine antibody 3F8 plus GM-CSF did. Neuroblastoma, a malignancy of the sympathetic nervous system that arises in cells derived from the neural crest, predominantly affects young children.

Diller, who is Dana-Farber/Boston Children’s chief medical officer, notes that neuroblastoma therapy is a challenging ordeal for children. Over the course of a decade, Lisa Diller, MD and her colleagues tested and refined the double-transplant treatment. These stem cells are collected in a process called apheresis. Acute complications, such as sinusoidal obstructive syndrome and microangiopathy, may increase risk for relapse by preventing timely implementation of local radiotherapy and immunotherapy. Nearly half of the original cohort ultimately wasn’t randomized to double versus single transplant. In the trial, one group of patients received the standard treatment (chemotherapy and a stem cell transplant followed by another round of chemotherapy and a second, or tandem, stem cell transplant).

Extensive data suggest tandem transplant is not helpful for high-risk neuroblastoma. She has suffered side effects—including loss of kidney function and hearing loss—but now enjoys a good quality of life. The results demonstrate that in the context of the induction and postconsolidation therapies administered, even in the era of precision medicine, tandem autologous stem cell transplant is a more effective consolidation treatment than single autologous stem cell transplant for most patients with high-risk neuroblastoma. 2010;doi:10.1200/JCO.2009.27.3524. Accessibility Statement, Tandem vs Single Autologous Stem Cell Transplant and Event-Free Survival in High-Risk Neuroblastoma, Julie R. Park, MD; Susan G. Kreissman, MD; Wendy B. London, PhD; Arlene Naranjo, PhD; Susan Lerner Cohn, MD; Michael D. Hogarty, MD; Sheena C. Tenney, MSPH; Daphne Haas-Kogan, MD; Peter John Shaw, MA, MB, BS; Jacqueline M. Kraveka, DO; Stephen S. Roberts, MD; James Duncan Geiger, MD; John J. Doski, MD; Stephan D. Voss, MD, PhD; John M. Maris, MD; Stephan A. Grupp, MD, PhD; Lisa Diller, MD. Emily Coughlin was one of the patients in the trial. Tandem high-dose chemotherapy with thiotepa and busulfan-melphalan and autologous stem cell transplantation in very high-risk neuroblastoma patients. Patients in the tandem transplant group had superior 3-year EFS outcomes (61.6% vs 48.4%; P = .006). Pasqualini C, Dufour C, Goma G, et al. The stem cell transplant is needed because the intense chemotherapy also destroys blood-forming stem cells in the patient’s bone marrow. Despite these highly encouraging outcomes, caution is warranted before declaring tandem myeloablative transplants as the standard of care for all patients with high-risk neuroblastoma. Neuroblastoma is marked by wide clinical and biological heterogeneity.

Matthay KK, et al. Extensive data suggest tandem transplant is not helpful for high-risk neuroblastoma. sign up for alerts, and more, to access your subscriptions, sign up for alerts, and more, to download free article PDFs, sign up for alerts, customize your interests, and more, to make a comment, download free article PDFs, sign up for alerts and more, Archives of Neurology & Psychiatry (1919-1959), JAMAevidence: The Rational Clinical Examination, JAMAevidence: Users' Guides to Medical Literature, FDA Approval and Regulation of Pharmaceuticals, 1983-2018, Global Burden of Skin Diseases, 1990-2017, Health Care Spending in the US and Other High-Income Countries, Life Expectancy and Mortality Rates in the United States, 1959-2017, Medical Marketing in the United States, 1997-2016, Practices to Foster Physician Presence and Connection With Patients in the Clinical Encounter, US Burden of Cardiovascular Disease, 1990-2016, Waste in the US Health Care System: Estimated Costs and Potential for Savings, Register for email alerts with links to free full-text articles. Overall, neuroblastoma accounts for 10 to 12 In the only reported randomized trial involving tandem transplant, comparison was … This site uses cookies to assist with navigation, analyse your use of our services, and provide content from third parties. Our syndication services page shows you how. Dana-Farber/Boston Children's researchers pioneered the idea of tandem stem cell transplants for neuroblastoma in 1994. Although she is generally in good health now, Katie did experience side effects from the treatment, including sterility and thyroid dysfunction. to download free article PDFs, A phase III randomized clinical trial of tandem myeloablative autologous stem cell transplant using peripheral blood stem cell as consolidation therapy for high risk neuroblastoma: A Children’s Oncology Group study. Their promising results led the national Children’s Oncology Group to open a randomized trial in 2007 (ANBL0532) to definitively compare two transplants, given roughly six weeks apart, versus one. Children who received post-consolidation immunotherapy with dinutuximab demonstrated further improvements in 3-year EFS (73.3% for tandem vs. 54.7% for single; P = .004) and OS (84% for tandem vs. 73.4% for single; P = .04). percent of all child cancer deaths. Lancet Oncol. Floating in fluid deep in the brain are small, little-understood fronds of tissue. The information you enter will appear in your e-mail message and is not retained by Medical Xpress in any form. She was in the intensive care unit twice during the first transplant; luckily, the second one was a little easier. Within this subgroup, patients receiving two stem cell transplants versus just one also had better three-year event-free survival (73 vs. 55 percent).
Dana-Farber/Boston Children’s researchers pioneered the idea of tandem stem cell transplants for neuroblastoma in 1994. Healio.com. First, to make the approach feasible, they developed a new technique for harvesting circulating blood stem cells from very small children. One hundred and twenty nine out of 179 patients in the single ASCT group and 121 out of 176 in the tandem transplant group also subsequently received anti-GD2 (dinutuximab) plus cytokine immunotherapy. After completing this phase, 355 patients were eligible for and consented to enter the next phase — which randomly assigned them to receive either two autologous stem cell transplants or just one. So there might be patients who don’t benefit from tandem transplant that were underrepresented in the study. Treatment includes intensive chemotherapy, aggressive surgeries, double transplant, post-transplant radiation, and immunotherapy. Outcomes were also slightly better in individuals who had received anti-GD2 antibody plus cytokines after the transplant. 2012;doi:10.1200/JCO.2011.41.3807.
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