Double stem-cell transplant improves outcomes for children with neuroblastoma

In this 2016 press release, the American Society of Clinical Oncology shared the findings of a study of 652 children with high-risk neuroblastoma who were treated with double and single autologous haematopoetic stem cell (HSC)  transplants (stem cells from their own bone marrow).

The most significant was that at 3 years, 61.4% of patients who received a double transplant were alive and cancer-free, compared to 48.4% of those who received a single transplant. Side effects were similar between single and double transplant.

“This finding will change the way we treat children with high-risk neuroblastoma in North America, which still claims many young lives and is in urgent need of better treatments.” Julie R. Park, MD, attending physician at Seattle Children’s Hospital and professor in pediatrics at the University of Washington School of Medicine in Seattle, Washington.

“So much of the story of progress against childhood cancers has been learning to use potent therapies better. This is yet another example of how commitment to clinical research delivers potentially lifesaving results,” said Stephen P. Hunger, MD, ASCO expert in pediatric cancers. 

About Neuroblastoma

“Neuroblastoma is a tumor that begins in nerve cells outside of the brain. Although neuroblastoma is a rare cancer overall, with only 700 new diagnoses per year in the United States, it is the second most common solid tumor in children and the most common cancer in infancy.1 Neuroblastoma most often occurs in children younger than six years.

“A combination of clinical, pathologic and genetic markers is used to define neuroblastoma. Children with high-risk neuroblastoma usually receive an intensive treatment regimen, which may include surgery, chemotherapy, radiation therapy, and/or stem-cell transplantation.

“Historically, less than 50% of children with high-risk neuroblastoma live five or more years after diagnosis. A National Cancer Institute (NCI) funded phase III trial performed by the Children’s Oncology Group found that adding a second autologous stem-cell transplant (ASCT, a transplant that uses the patient’s own stem cells) to standard therapy improves outcomes for these patients.

About the Study

“The trial enrolled children newly diagnosed with high-risk neuroblastoma, who were a median age of 3.1 years. The majority of patients (88%) had Stage 4 disease and 38.2% had a tumour high-risk genetic abnormality called MYCN amplification.

“All patients received 6 cycles of a multi-agent induction chemotherapy regimen including an initial 2 cycles of high-dose cyclophosphamide/topotecan followed bya collection of stem cells from the blood to be used for subsequent transplantation. At completion of the induction therapy, patients were randomly assigned to receive a single ASCT with carboplatin-etoposide-melphalan (CEM) chemotherapy or a double (tandem) ASCT with thiotepa–cyclophosphamide prior to the first ASCT followed by a modified CEM chemotherapy prior to second ASCT. In the tandem ASCT group, patients received the two transplants in the span of 6 to 8 weeks.

“In the single ASCT group, 129 out of 179 patients were subsequently enrolled onto a trial delivering anti-GD2 (dinutuximab) plus cytokine immunotherapy after single transplant consolidation therapy. A similar proportion of patients, 121 out of 176, received this immunotherapy following a tandem transplant consolidation therapy.
The primary endpoint of the study was 3-year event-free survival (EFS) or the percentage of patients who have not had an “event” at three years after randomization. An “event” was defined as worsening or recurrence of cancer, diagnosis of a second cancer, or death from any cause.

Key Findings

“Among all patients on the study, the 3-year EFS from enrollment was 51% and 3-year overall survival (OS) was 68.3%. Among patients randomized, the 3-year EFS rate from time of randomization was significantly higher following tandem transplant (61.4%) compared to single transplant (48.4%). The 3-year overall survival rate was slightly higher in the tandem transplant group than the single transplant group (74% vs. 69.1%), but the difference was not statistically significant.

“We know that most neuroblastoma recurrences occur within two to three years from diagnosis and that patients who had not had a recurrence at three years have a better chance of long-term survival. The study was not designed to observe a difference in overall survival, as this would take many years and can not be adequately controlled for additional therapies received after an initial disease recurrence,” said Dr. Park.

The researchers will continue following patients on this study for 10 years.

Outcomes were generally better among patients who enrolled onto the immunotherapy trial that included anti-GD2 antibody plus cytokines after the transplant. Among those patients, the 3-year EFS rate was significantly higher for those who had been assigned to tandem transplant (73.2%) compared to those assigned to single transplant (55.5%). The 3-year overall survival rate was significantly higher with a tandem transplant than with a single transplant (85.6% vs. 75.8%).

The rates of severe toxicities were similar between treatment groups. Fewer treatment-related deaths occurred among patients who received a tandem transplant than among those who received a single transplant (2 vs. 8).


The study received funding from the National Institutes of Health and was performed through the Children’s Oncology Group consortium.

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Cord blood and neuroblastoma

HSCs are blood stem cells, like those found in cord blood. Unlike bone marrow stem cells, cord blood HSCs can be collected non-invasively and stored for future use. Whilst each cord blood sample only contains sufficient stem cells to treat one child, stem cells from the umbilical cord are more ‘plastic’, meaning a perfect match is not always required and transplants result in a lower incidence of graft-versus-host disease. There is also a growing body of research dedicated to “expanding” stem cells so that more than one dose may be available.

Cord blood HSCs have rarely been used to treat neuroblastoma. One success story is that of Frances Everall, “the girl who beat cancer with her own cord blood.”

The Girl Who Beat Cancer With Her Own Cord Blood

stem cell preservation

BSc (Hons) Microbiology

Chief Executive Officer | Biovault Family

Biovault Family CEO, Kate Sneddon, joined Biovault in July 2009 and became Chief Executive Officer in 2016. As health industry professional her experience includes working as a microbiologist and leader at GSK for over 10 years. Her expertise in cord blood banking has been recognised in her awards, features in Parliamentary Review and Parents Guide to Cord Blood, as well as contributions to research with UCL and others.

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