To maximize the quality of pediatric cancer care delivered, the creation and implementation of supportive care clinical practice guidelines are needed.
Cure rates for children with cancer diagnosed in the United States and Canada are greater than 82%.1,2 This remarkable improvement from an almost universally fatal disease to one in which the majority of children with cancer are cured has been attributed primarily to the conduct of successive randomized controlled trials. These trials have led to the identification of better treatment strategies and the standardization of therapy according to treatment protocols, even for children not enrolled onto a trial.3 Consequently, most children in the United States and Canada are treated on, or according to, multi-institutional therapeutic protocols, resulting in uniformity in treatment approach by diagnosis.
Nonetheless, despite these great gains, gaps in care remain. Challenges arise because pediatric cancer care is complex. First, we need to focus on accurate and timely diagnosis and risk stratification, particularly with the increasing number of molecular diagnostic tests. Second, pediatric cancer treatments are intense and therefore, many treatment approaches are associated with a high burden of acute symptoms4–6 and nonprogressive disease-related mortality. Third, there are challenges with obtaining patient-reported outcomes (PROs), and little is known about the best ways to elicit symptoms directly from children, particularly for the youngest patients. Fourth, there are gaps in the provision of effective care for adolescents and young adults and challenges with transition to adult facilities. Finally, there continues to be growing recognition of the long-term consequences of therapy for survivors of childhood cancer.7 This article will focus on quality care related to symptom control and PRO elicitation.
Symptom control and elicitation of PROs are important considerations in the provision of quality care. Quality indicators in pediatric oncology care were recently identified by the Pediatric Oncology Group of Ontario.8 Using a systematic review, a targeted gray literature search, and focus groups, 20 quality indicators were identified, three of which specifically focus on supportive care and PROs, namely, treatment-related mortality, supportive care guidelines, and access to expert management of pain control. From the perspective of supportive care, the urgent to-do list includes the following: First is the development and implementation of supportive care clinical practice guidelines (CPGs). Second are strategies to measure symptoms from the patient perspective. Finally, we need to measure the effectiveness of interventions in real-life practice outside the carefully controlled setting of clinical trials.
DEVELOPMENT AND IMPLEMENTATION OF SUPPORTIVE CARE CPGS
We need to create and implement rigorously developed supportive care CPGs. Children with cancer experience a high symptom burden during treatment, and in particular, children experience pain, nausea, fatigue, and mucositis along with psychosocial symptoms including sadness, frustration, fear and worry. Symptoms and toxicity result in suffering, poor quality of life, morbidity, and sometimes nonprogressive disease mortality. Symptoms may also result in longer term psychosocial consequences.9 For the prevention and treatment of toxicities, effective therapies are not being given, and unnecessarily treatments are being administered.10,11 The failure to maximize symptom control is, in part, related to the lack of standardization and lack of adherence to CPGs.12 Thus, there is a need to develop and implement good CPGs in pediatric oncology.
CPGs are systematically developed statements designed to assist in making health care–related decisions.13 It is important to highlight that only rigorously developed or trustworthy guidelines should be used in clinical practice.14,15 Several pediatric cancer groups are focusing on the development of trustworthy pediatric cancer supportive care guidelines, including the Pediatric Oncology Group of Ontario, the C17 Research Network, The American Society of Pediatric Hematology and Oncology, the Dutch Children's Oncology Group, and the Children's Cancer Leukemia Group.
The need for CPGs in pediatric cancer is mainly related to supportive care and not cancer treatment, in part because of the wide-spread adoption of clinical trials as the standard approach for oncologic treatment. Almost all children with cancer in North America are enrolled onto a clinical trial or treated “as per” the standard arm of a trial, at least in the new diagnosis setting. This approach results in remarkable uniformity in treatment protocols used for children with cancer. In sharp contrast, we know there is remarkable heterogeneity in approaches for supportive care, even within institutions. This lack of standardization in supportive care was recently identified as an important impediment to the provision of high-quality care in pediatric cancer.12
It is not sufficient to simply create supportive care CPGs; they need to be implemented in routine clinical practice. We have begun implementation efforts within the Children's Oncology Group (COG). COG is the largest federally funded pediatric cancer clinical trials network worldwide, and its mission is to improve the outcome for all children with cancer.16 Recognizing the need for uniform supportive care recommendations for patients enrolled onto trials, COG has created a formal process to identify and disseminate recommendations from CPGs. The need for a supportive care guideline is solicited from the membership, a search is conducted to identify all available CPGs on that topic, and the CPGs are evaluated using the Appraisal of Guidelines for Research & Evaluation II tool.17 A supportive care CPG is then selected. The recommendations are currently available through a link in each therapeutic protocol. Future work planned by COG includes understanding barriers and facilitators of CPG implementation at sites; identifying effective interventions to improve CPG adherence; and, finally, testing interventions in cluster randomized trials.
STRATEGIES TO MEASURE SYMPTOMS FROM THE PATIENT PERSPECTIVE
We need to develop strategies to measure symptoms from the patient perspective. Active screening and ongoing assessment of symptoms are important. Children undergoing cancer treatment may accept symptoms as an inevitable consequence of chemotherapy and often seek help only when the symptoms become severe.18,19 It is crucial to emphasize the importance of self-reported symptoms by children as opposed to proxy report by guardians or health care providers. Identification of symptoms by the child, most especially subjective symptoms, ensures that the perspectives and experiences of the child are being captured and that the attention of clinicians and parents is focused on the symptoms most important to that child.
We conducted a systematic review of symptom assessment scales used in pediatric oncology to determine if one was suitable as a symptom screening measure.20 When no suitable scale was identified,21 we began construction of a new pediatric cancer–specific self-report symptom screening and assessment tool. Item generation was accomplished through a nominal group technique,21 and an initial draft of an instrument was developed and named the Symptom Screening in Pediatrics Tool. With the input of children with cancer and parents, the tool was then refined, and its psychometrics will now be evaluated in a multicenter trial.22
MEASURING THE EFFECTIVENESS OF INTERVENTIONS IN REAL-WORLD PRACTICE
The final challenge is to begin to measure the effectiveness of interventions in all children with cancer, including those not registered on trials. Patients enrolled onto clinical trials are not representative of the general population.23 Those who fail to meet eligibility criteria for trials often have poor performance status, comorbidities, or diagnostic complexities. Language may also be a barrier to enrollment. Further, those who decline are expected to be systematically different than families who accept trials, particularly those with a randomized design. In order to identify the effectiveness of interventions in all patients, we need population-based data.
A unique initiative in Canada has been the development of a population-based data set funded by the Public Health Agency of Canada named Cancer in Young People–Canada. This data set includes all children with cancer diagnosed from 2001 forward and contains standard diagnostic and treatment information. However, in addition, times to diagnosis and treatment, cumulative doses of specific agents, and complications are captured. This resource will permit us to begin to address effectiveness questions, for both therapeutic and supportive care issues.
In conclusion, remarkable gains have been made in pediatric cancer. However, several challenges remain. In order to maximize the quality of care delivered, we need to create and implement supportive care CPGs. We need to develop mechanisms to elicit PROs from children themselves and ensure clinicians make decisions based on those PRO measures. Finally, we need to understand the effectiveness of interventions in all children with cancer, including those not enrolled onto clinical trials.
Copyright © 2015 by American Society of Clinical Oncology
AUTHOR'S DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST
Disclosures provided by the authors are available with this article at jop.ascopubs.org.
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AUTHOR'S DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST
Priorities for Quality Care in Pediatric Oncology Supportive Care
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No relationship to disclose