PMID- 28581685
OWN - NLM
STAT- MEDLINE
DCOM- 20170927
LR  - 20200523
IS  - 1097-0142 (Electronic)
IS  - 0008-543X (Print)
IS  - 0008-543X (Linking)
VI  - 123
IP  - 19
DP  - 2017 Oct 1
TI  - Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new 
      trial endpoint?
PG  - 3799-3806
LID - 10.1002/cncr.30782 [doi]
AB  - BACKGROUND: Pediatric participants on phase 1 or phase 2 clinical trials for 
      incurable cancer are at risk of experiencing toxicities (adverse events [AEs]) 
      related to trial participation. Multiple AEs are subjective; thus, the real 
      impact of trial treatment cannot be known unless patient subjective reports are 
      solicited. METHODS: The authors assessed the feasibility and acceptability of 
      soliciting symptom, function, and quality of life (QOL) reports from participants 
      aged 8 to 18 years who were enrolled on phase 1/2 clinical trials at 4 cancer 
      centers during the first course of chemotherapy. The authors also assessed the 
      reliability and validity of 6 self-report Patient-Reported Outcomes Measurement 
      Information System (PROMIS) pediatric measures and 4 open-ended interview 
      questions at 2 time points (at the time of trial enrollment [T1] and 3 to 4 weeks 
      later [T2]). RESULTS: The enrollment rate of 75.9% (20 participants) exceeded the 
      feasibility criterion, and missingness of measures by person, measure, and items 
      at T1 and T2 were lower than the acceptability criteria. New QOL themes were 
      limited to the impact of treatment on families and being away from home, family, 
      and friends for treatment. All but one measure at T1 met the reliability 
      criterion and all measures did so at T2. Validity support was limited however 
      because as theorized, mobility decreased and fatigue increased as AEs increased. 
      CONCLUSIONS: Soliciting and documenting symptom, function, and QOL reports from 
      patients aged 8 to 18 years who are enrolled on a phase 1/2 clinical trial is 
      feasible and acceptable to participants, particularly when embedded in trials. 
      Reliable and valid findings can result, making patient self-reported outcomes a 
      possible new trial endpoint. Cancer 2017;123:3799-3806. (c) 2017 American Cancer 
      Society.
CI  - (c) 2017 American Cancer Society.
FAU - Hinds, Pamela S
AU  - Hinds PS
AUID- ORCID: 0000-0001-6491-6649
AD  - Department of Nursing Research and Quality Outcomes, Children's National Health 
      System, Washington, DC.
AD  - Department of Pediatrics, George Washington University, Washington, DC.
FAU - Wang, Jichuan
AU  - Wang J
AD  - Department of Pediatrics, George Washington University, Washington, DC.
AD  - Division of Biostatistics and Study Methodology, Children's National Health 
      System, Washington, DC.
FAU - Stern, Emily Dunn
AU  - Stern ED
AD  - Division of Hematology/Oncology, Children's National Health System, Washington, 
      DC.
FAU - Macpherson, Catherine Fiona
AU  - Macpherson CF
AD  - Nursing, Seattle Children's Hospital, Seattle, Washington.
FAU - Wharton, Claire M
AU  - Wharton CM
AD  - Division of Oncology, Seattle Children's Hospital, Seattle, Washington.
FAU - Okorosobo, Ruthanna
AU  - Okorosobo R
AD  - The George Washington University, Washington, DC.
FAU - Cheng, Yao Iris
AU  - Cheng YI
AD  - Division of Biostatistics and Study Methodology, Children's National Health 
      System, Washington, DC.
FAU - Gross, Heather E
AU  - Gross HE
AD  - University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
FAU - Meany, Holly J
AU  - Meany HJ
AD  - Department of Pediatrics, George Washington University, Washington, DC.
AD  - Division of Hematology/Oncology, Children's National Health System, Washington, 
      DC.
FAU - Jacobs, Shana
AU  - Jacobs S
AD  - Department of Pediatrics, George Washington University, Washington, DC.
AD  - Division of Hematology/Oncology, Children's National Health System, Washington, 
      DC.
LA  - eng
GR  - R21 NR012716/NR/NINR NIH HHS/United States
GR  - U54 HD090257/HD/NICHD NIH HHS/United States
PT  - Journal Article
PT  - Validation Study
DEP - 20170605
PL  - United States
TA  - Cancer
JT  - Cancer
JID - 0374236
RN  - 0 (Antineoplastic Agents)
SB  - IM
MH  - Adolescent
MH  - Antineoplastic Agents/*adverse effects
MH  - Child
MH  - *Clinical Trials, Phase I as Topic
MH  - *Clinical Trials, Phase II as Topic
MH  - Family
MH  - Fatigue/*chemically induced
MH  - Feasibility Studies
MH  - Female
MH  - Humans
MH  - Longitudinal Studies
MH  - Male
MH  - *Mobility Limitation
MH  - Neoplasms/drug therapy
MH  - *Patient Reported Outcome Measures
MH  - *Quality of Life
MH  - Reproducibility of Results
MH  - Surveys and Questionnaires
MH  - Symptom Assessment/*methods
PMC - PMC5610606
MID - NIHMS872158
OTO - NOTNLM
OT  - pediatric oncology
OT  - pediatric patient self-reports
OT  - phase 1 clinical trial
OT  - symptoms
COIS- Disclosure Statements: There are no conflicts of interest to disclose for any of 
      the co-authors.
EDAT- 2017/06/06 06:00
MHDA- 2017/09/28 06:00
PMCR- 2018/10/01
CRDT- 2017/06/06 06:00
PHST- 2016/12/21 00:00 [received]
PHST- 2017/04/18 00:00 [revised]
PHST- 2017/04/19 00:00 [accepted]
PHST- 2017/06/06 06:00 [pubmed]
PHST- 2017/09/28 06:00 [medline]
PHST- 2017/06/06 06:00 [entrez]
PHST- 2018/10/01 00:00 [pmc-release]
AID - 10.1002/cncr.30782 [doi]
PST - ppublish
SO  - Cancer. 2017 Oct 1;123(19):3799-3806. doi: 10.1002/cncr.30782. Epub 2017 Jun 5.