Veronica Dussel1, Liliana Orellana2, Natalie Soto3, Kun Chen4, Christina Ullrich5, Tammy I Kang6, Jeffrey R Geyer7, Chris Feudtner6, Joanne Wolfe8. 1. Department of Psychosocial Oncology and Palliative Care, Dana-Farber Cancer Institute, Boston, Massachusetts, USA; Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina. 2. Biostatistics Unit Faculty of Health, Deakin University, Victoria, Australia. 3. Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina. 4. Center for Outcomes and Policy Research, Dana-Farber Cancer Institute, Boston, Massachusetts, USA. 5. Department of Psychosocial Oncology and Palliative Care, Dana-Farber Cancer Institute, Boston, Massachusetts, USA; Center for Outcomes and Policy Research, Dana-Farber Cancer Institute, Boston, Massachusetts, USA; Department of Medicine, Boston Children's Hospital, Boston, Massachusetts, USA; Harvard Medical School, Boston, Massachusetts, USA. 6. Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA; Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA. 7. Division of Pediatric Hematology/Oncology, Seattle Children's Hospital, Seattle, Washington, USA; Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; University of Washington, Seattle, Washington, USA. 8. Department of Psychosocial Oncology and Palliative Care, Dana-Farber Cancer Institute, Boston, Massachusetts, USA; Center for Outcomes and Policy Research, Dana-Farber Cancer Institute, Boston, Massachusetts, USA; Department of Medicine, Boston Children's Hospital, Boston, Massachusetts, USA; Harvard Medical School, Boston, Massachusetts, USA. Electronic address: joanne_wolfe@dfci.harvard.edu.
Abstract
CONTEXT: Pediatric palliative care randomized controlled trials (PPC-RCTs) are uncommon. OBJECTIVES: To evaluate the feasibility of conducting a PPC-RCT in pediatric cancer patients. METHODS: This was a cohort study embedded in the Pediatric Quality of Life and Evaluation of Symptoms Technology Study (NCT01838564). This multicenter PPC-RCT evaluated an electronic patient-reported outcomes system. Children aged two years and older, with advanced cancer, and potentially eligible for the study were included. Outcomes included: pre-inclusion attrition (patients not approached, refusals); post-inclusion attrition (drop-out, elimination, death, and intermittent attrition (IA; missing surveys) over nine months of follow-up); child/teenager self-report rates; and, reasons to enroll/participate. RESULTS: Over five years, of the 339 identified patients, 231 were eligible (in 22, we could not verify eligibility); 84 eligible patients were not approached and 43 declined participation. Patients not approached were more likely to die or have brain tumors. We enrolled 104 patients. Average enrollment rate was one patient per site per month; shortening follow-up from nine to three months (with optional re-enrollment) increased recruitment by 20%. A total of 87 patients completed the study (24 died) and 17 dropped out. Median IA was 41% in the first 20 weeks of follow-up and more than 60% in the eight weeks preceding death. Child/teenager self-report was 94%. Helping others, low burden procedures, incentives, and staff attitude were frequent reasons to enroll/participate. CONCLUSION: A PPC-RCT in children with advanced cancer was feasible, post-inclusion retention adequate; many families participated for altruistic reasons. Strategies that may further PPC-RCT feasibility include: increasing target population through large multicenter studies, approaching sicker patients, preventing exclusion of certain patient groups, and improving data collection at end of life.
RCT Entities:
CONTEXT: Pediatric palliative care randomized controlled trials (PPC-RCTs) are uncommon. OBJECTIVES: To evaluate the feasibility of conducting a PPC-RCT in pediatric cancerpatients. METHODS: This was a cohort study embedded in the Pediatric Quality of Life and Evaluation of Symptoms Technology Study (NCT01838564). This multicenter PPC-RCT evaluated an electronic patient-reported outcomes system. Children aged two years and older, with advanced cancer, and potentially eligible for the study were included. Outcomes included: pre-inclusion attrition (patients not approached, refusals); post-inclusion attrition (drop-out, elimination, death, and intermittent attrition (IA; missing surveys) over nine months of follow-up); child/teenager self-report rates; and, reasons to enroll/participate. RESULTS: Over five years, of the 339 identified patients, 231 were eligible (in 22, we could not verify eligibility); 84 eligible patients were not approached and 43 declined participation. Patients not approached were more likely to die or have brain tumors. We enrolled 104 patients. Average enrollment rate was one patient per site per month; shortening follow-up from nine to three months (with optional re-enrollment) increased recruitment by 20%. A total of 87 patients completed the study (24 died) and 17 dropped out. Median IA was 41% in the first 20 weeks of follow-up and more than 60% in the eight weeks preceding death. Child/teenager self-report was 94%. Helping others, low burden procedures, incentives, and staff attitude were frequent reasons to enroll/participate. CONCLUSION: A PPC-RCT in children with advanced cancer was feasible, post-inclusion retention adequate; many families participated for altruistic reasons. Strategies that may further PPC-RCT feasibility include: increasing target population through large multicenter studies, approaching sicker patients, preventing exclusion of certain patient groups, and improving data collection at end of life.
Authors: Joanne Wolfe; Liliana Orellana; E Francis Cook; Christina Ullrich; Tammy Kang; Jeffrey Russell Geyer; Chris Feudtner; Jane C Weeks; Veronica Dussel Journal: J Clin Oncol Date: 2014-03-10 Impact factor: 44.544
Authors: Kiri Walsh; Louise Jones; Adrian Tookman; Christina Mason; Joanne McLoughlin; Robert Blizard; Michael King Journal: Br J Psychiatry Date: 2007-02 Impact factor: 9.319
Authors: M E Bensink; N R Armfield; R Pinkerton; H Irving; A R Hallahan; D G Theodoros; T Russell; A G Barnett; P A Scuffham; R Wootton Journal: Palliat Med Date: 2008-12-10 Impact factor: 4.762
Authors: Christina K Ullrich; Veronica Dussel; Liliana Orellana; Tammy I Kang; Abby R Rosenberg; Chris Feudtner; Joanne Wolfe Journal: Cancer Date: 2018-10-06 Impact factor: 6.860
Authors: Katherine E Nelson; James A Feinstein; Cynthia A Gerhardt; Abby R Rosenberg; Kimberley Widger; Jennifer A Faerber; Chris Feudtner Journal: Children (Basel) Date: 2018-02-26
Authors: Emily Morell; Mary Katherine Miller; Minmin Lu; Kevin G Friedman; Roger E Breitbart; Jeffrey R Reichman; Julie McDermott; Lynn A Sleeper; Elizabeth D Blume Journal: J Am Heart Assoc Date: 2021-01-14 Impact factor: 5.501