PMID- 30290839
OWN - NLM
STAT- MEDLINE
DCOM- 20190107
LR  - 20190107
IS  - 1745-6215 (Electronic)
IS  - 1745-6215 (Linking)
VI  - 19
IP  - 1
DP  - 2018 Oct 5
TI  - Clinical significance in pediatric oncology randomized controlled treatment 
      trials: a systematic review.
PG  - 539
LID - 10.1186/s13063-018-2925-8 [doi]
LID - 539
AB  - BACKGROUND: Clinical significance in a randomized controlled trial (RCT) can be 
      determined using the minimal clinically important difference (MCID), which should 
      inform the delta value used to determine sample size. The primary objective was 
      to assess clinical significance in the pediatric oncology randomized controlled 
      trial (RCT) treatment literature by evaluating: (1) the relationship between the 
      treatment effect and the delta value as reported in the sample size calculation, 
      and (2) the concordance between statistical and clinical significance. The 
      secondary objective was to evaluate the reporting of methodological attributes 
      related to clinical significance. METHODS: RCTs of pediatric cancer treatments, 
      where a sample size calculation with a delta value was reported or could be 
      calculated, were systematically reviewed. MEDLINE, EMBASE, and the Cochrane 
      Childhood Cancer Group Specialized Register through CENTRAL were searched from 
      inception to July 2016. RESULTS: RCTs (77 overall; 11 and 66), representing 95 
      (13 and 82) randomized questions were included for non-inferiority and 
      superiority RCTs (herein, respectively). The minority (22.1% overall; 76.9 and 
      13.4%) of randomized questions reported conclusions based on clinical 
      significance, and only 4.2% (15.4 and 2.4%) explicitly based the delta value on 
      the MCID. Over half (67.4% overall; 92.3 and 63.4%) reported a confidence 
      interval or standard error for the primary outcome experimental and control 
      values and 12.6% (46.2 and 7.3%) reported the treatment effect, respectively. Of 
      the 47 randomized questions in superiority trials that reported statistically 
      non-significant findings, 25.5% were possibly clinically significant. Of the 24 
      randomized questions in superiority trials that were statistically significant, 
      only 8.3% were definitely clinically significant. CONCLUSIONS: A minority of RCTs 
      in the pediatric oncology literature reported methodological attributes related 
      to clinical significance and a notable portion of statistically insignificant 
      studies were possibly clinically significance.
FAU - Howard, A Fuchsia
AU  - Howard AF
AUID- ORCID: 0000-0001-5704-1733
AD  - School of Nursing, The University of British Columbia, T201-2211 Wesbrook Mall, 
      Vancouver, BC, V6T 2B5, Canada. fuchsia.howard@ubc.ca.
FAU - Goddard, Karen
AU  - Goddard K
AD  - Department of Radiation Oncology, BC Cancer, Vancouver, BC, V5Z 4E6, Canada.
FAU - Rassekh, Shahrad Rod
AU  - Rassekh SR
AD  - Division of Hematology/Oncology, BC Children's Hospital, Vancouver, BC, V6H 3N1, 
      Canada.
FAU - Samargandi, Osama A
AU  - Samargandi OA
AD  - Division of Plastic Surgery, QEII Health Sciences Centre, Halifax, NS, B3H 3A7, 
      Canada.
FAU - Hasan, Haroon
AU  - Hasan H
AD  - Department of Radiation Oncology, BC Cancer, Vancouver, BC, V5Z 4E6, Canada.
AD  - Epi Methods Consulting, Toronto, ON, M5V 0C4, Canada.
LA  - eng
PT  - Journal Article
PT  - Systematic Review
DEP - 20181005
PL  - England
TA  - Trials
JT  - Trials
JID - 101263253
SB  - IM
MH  - Age of Onset
MH  - Data Accuracy
MH  - Data Interpretation, Statistical
MH  - Humans
MH  - Medical Oncology/*methods/statistics & numerical data
MH  - Minimal Clinically Important Difference
MH  - Neoplasms/epidemiology/pathology/*therapy
MH  - Pediatrics/*methods/statistics & numerical data
MH  - Randomized Controlled Trials as Topic/*methods/statistics & numerical data
MH  - *Research Design/statistics & numerical data
MH  - Sample Size
MH  - Treatment Outcome
PMC - PMC6173909
OTO - NOTNLM
OT  - Clinical significance
OT  - Minimally clinically important difference
OT  - Pediatric oncology
OT  - Randomized controlled trials
COIS- ETHICS APPROVAL: This systematic review did not require ethical approval because 
      no participants were involved, nor was identifiable information collected. 
      CONSENT FOR PUBLICATION: Not applicable. COMPETING INTERESTS: The authors certify 
      that they have no affiliations with, or involvement in, any organization or 
      entity with any financial interest, or non-financial interest in the subject 
      matter or materials discussed in this manuscript. PUBLISHER'S NOTE: Springer 
      Nature remains neutral with regard to jurisdictional claims in published maps and 
      institutional affiliations.
EDAT- 2018/10/07 06:00
MHDA- 2019/01/08 06:00
PMCR- 2018/10/05
CRDT- 2018/10/07 06:00
PHST- 2018/06/04 00:00 [received]
PHST- 2018/09/19 00:00 [accepted]
PHST- 2018/10/07 06:00 [entrez]
PHST- 2018/10/07 06:00 [pubmed]
PHST- 2019/01/08 06:00 [medline]
PHST- 2018/10/05 00:00 [pmc-release]
AID - 10.1186/s13063-018-2925-8 [pii]
AID - 2925 [pii]
AID - 10.1186/s13063-018-2925-8 [doi]
PST - epublish
SO  - Trials. 2018 Oct 5;19(1):539. doi: 10.1186/s13063-018-2925-8.