Clinical trials in children commonly go uncompleted or unpublished

Currently, the only popular trend in science is to publish only those results that look as a breakthrough or display statistically significant data. Obsession with positive outcome leads to discontinuation and non-publishing the all other data which don’t meet the requirements.  As a result researchers and public did not know mistakes, efforts and drilling details of non-positive studies, so they have no opportunity to review this data, refine the research hypothesis and technical performance. This leads to waste of time and funding money.

The very recent study by Natalie Pica, MD, PhD, and Florence Bourgeois MD, MPH from the Department of Pediatrics at Harvard Medical School and Boston Children’s Hospital, both in Massachusetts has again confirmed the problem. The researchers carried out a retrospective, cross-sectional study of childhood randomized controlled trials and published their findings in Pediatrics (DOI: 10.1542/peds.2016-0223). They collected information from all trials that were registered from 2008 to 2010, then searched scientific publications based on the trials. They also verified final status of the selected trials (completed or discontinued) by the end of 2012. If researchers found no publication, they contacted investigators and sponsors associated with trials to clarify the issue.

The main findings were:

  1. 19% of 559 trials were discontinued early representing approximately 8369 children. The most common reason for discontinuation – difficulty with patient accrual (37%).
  2. 30 % of the 455 completed trials were not published, representing 69 165 children participants.
  3. Only 42 unpublished trials posted results on
  4. Trials were less likely to be dropped if they were funded by industry.
  5. Trials funded by industry were more than twice as likely to result in nonpublication and a longer mean time to publication when compared with trials sponsored by academia.

Researchers concluded that “withdrawal and nonpublication were common, resulting in thousands of children exposed to interventions that did not lead to informative or published findings. Trial funding source was an important determinant of these outcomes, with both academic and industry sponsors contributing to inefficiencies.” (Pica N & Bourgeois F, 2016, e 20160223)

Pica N & Bourgeois F, Discontinuation and Nonpublication of Randomized Clinical Trials Conducted in Children. PEDIATRICS V 138(3) 2016:e 20160223 Access to the study can be found here:



Neuroblastoma summary

Neuroblastoma is a childhood cancer caused by the abnormal growth and development of non-mature nerve cells, called neuroblasts [1]. The disease commonly affects children age 5 years or younger. Approximately 50% of children have tumours that have spread at diagnosis [1]. The main challenge in treating neuroblastoma is to stop tumour spread and resistance to multiple drugs. Despite major advances in available therapies, children with drug resistant and/or recurrent neuroblastoma have a dismal outlook with 5 year survival rates of less than 20% [2-4]. Therefore, this cancer needs more research and funding as well as people awareness of these needs.


  1. Davidoff, A. M. Neuroblastoma. Semin. Pediatr. Surg. 21, 2–14 (2012).
  2. Gatta, G. et al. Childhood cancer survival in Europe 1999-2007: Results of EUROCARE-5-a population-based study. Lancet Oncol. 15, 35–47 (2014)
  3. Peinemann, F., Tushabe, D. A., van Dalen, E. C. & Berthold, F. Rapid COJEC versus standard induction therapies for high-risk neuroblastoma. The Cochrane database of systematic reviews 5, CD010774 (2015).
  4. Peinemann, F., van Dalen, E. C., Tushabe, D. A. & Berthold, F. Retinoic acid post consolidation therapy for high-risk neuroblastoma patients treated with autologous hematopoietic stem cell transplantation. Cochrane database Syst. Rev. 1, CD010685 (2015)


Hi readers!

This blog is about neuroblastoma biology, its research challenges, and people and media perception of this disease. I am researcher and active supporter of science communication.  I hope you will find interesting to read my blog and ask questions. Your questions would help me to cover topics which I have not heard of or may not plan to cover yet.