Determining Eligibility for Pediatric Cancer Clinical Trials Requires a Reassessment to Improve Inclusion and Patient Safety

A recent multicenter study published in the journal Cancer (online ahead of print) calls into question the validity of pediatric oncology providers' use of the Lansky Play-Performance Scale (LPPS) as an eligibility requirement for inclusion of children in cancer clinical trials.

The lead and corresponding author of the study was Scott H. Maurer, MD. Dr. Mauer is an associate professor of pediatrics at the University of Pittsburgh School of Medicine in the Department of Pediatrics, and currently serves as chief of the Division of Palliative Medicine and Supportive Care at UPMC Children's Hospital of Pittsburgh, and he serves as the medical director of UPMC Children's Supportive Care Program.

This study is the first of its kind to critically assess clinician-reported LPPS scores as a functional measure of patient status. While the LPPS has existed for a number of decades, it was never designed for or validated for use by clinicians for the kind of widespread use it now occupies in the field. 

In an accompanying editorial authored by Angela Steineck MD, MS, and Abby R. Rosenberg, MD, MS, MA, they point out that Dr. Maurer and colleague’s study is the first published analysis of any measure of pediatric functional status used as an indicator of one’s functional ability to safely participate in a clinical trial. 

Key Findings

Dr. Maurer and colleague’s assessment of the use of LPPS by clinicians as a proxy for clinical trial eligibility renders it a flawed approach based on their findings that parent or caregiver assessments using the measure deviated significantly from clinician’s use of the tool. 

Furthermore, the study also found that both caregiver and clinician LPPS scoring poorly correlated with the child’s self-report as measured by the Pediatric Patient-Reported Outcomes Measurement Information System® (PROMIS).

What this means, in general, is that children, caregivers, and clinicians differ significantly in their report of the child’s functional status, and these differences are sustained over various timepoints in therapy.

"Our findings show that we as clinicians, who are ultimately responsible for whether or not an individual patient can participate in a clinical trial, need to reassess our approach to evaluating and determining patient functional status and thus their ability to participate in trials. As we point out in our discussion, how the measures are currently being used, and how clinician use of LPPS stacks up against parent use of LPPS and patient-reported outcomes measures is discordant. This discordancy undoubtedly contributes to patients being excluded from trials who are good candidates and vice versa. This is an issue of access, potential efficacious outcomes, and safety,” says Dr. Maurer. “Moving forward, we need to study how best to incorporate recently validated tools for patient, parent, and clinician report of a child’s clinical status when determining what it means to be eligible for a clinical trial. In the meantime, clinical trialists and pediatric oncologists should be aware that parent and child perspectives should be included when considering the appropriateness of offering a clinical trial.”

Learn more about Dr. Maurer and his clinical practice and research.

To read the text of the full study and accompanying editorial, please see the references below, in addition to several other related studies published recently by Dr. Maurer et al.

References 

1. Maurer SH, Hinds PS, Reeve BB, Mack JW, McFatrich M, Lin L, Withycombe JS, Jacobs SS, Baker JN, Castellino SM, Freyer DR. Patients, Caregivers, and Clinicians Differ in Performance Status Ratings: Implications for Pediatric Cancer Clinical Trials. Cancer. 2021; 0:1-7. Epub Ahead of Print.

2. Steineck A, Rosenberg AR. Why Performance Status? A Case for Alternative Functional Assessments in Pediatric Oncology Clinical Trials. Cancer. 2021; Editorial.

Further Reading

Hinds PS, Weaver MS, Withycombe JS, Baker JN, Jacobs SS, Mack JW, Maurer SH, McFatrich M, Pinheiro LC, Reeve BB, Wang J. Subjective Toxicity Profiles of Children in Treatment for Cancer: A New Guide to Supportive Care? J Pain Symptom Manage. 2021; 61(6): 1188-1195.

Mack JW, McFatrich M, Withycombe JS, Maurer SH, Jacobs SS, Lin L, Lucas NR, Baker JN, Mann CM, Sung L, Tomlinson D, Hinds PS, Reeve BB. Agreement Between Child Self-Report and Caregiver-Proxy Report for Symptoms and Functioning of Children Undergoing Cancer Treatment. JAMA Pediatr. 2020 Nov 1;174(11):e202861.

McFatrich M, Brondon J, Lucas NR, Hinds PS, Maurer SH, Mack JW, Freyer DR, Jacobs SS, Baker JN, Mowbray C, Wang M, Castellino SM, Barz Leahy A, Reeve BB. Mapping Child and Adolescent Self-Reported Symptom Data to Clinician-Reported Adverse Event Grading to Improve Pediatric Oncology Care and Research. Cancer. 2020 Jan 1; 126(1): 140-147.