Impact of hospitalization for vaso-occlusive crisis on health-related quality of life in children with sickle cell disease.

BACKGROUND: Sickle cell disease (SCD) is characterized by vaso-occlusive crises (VOCs) that impair the health-related quality of life (HRQoL). The aim of this study is to evaluate the impact of hospitalization for VOCs on HRQoL in children with SCD over time. METHODS: In this longitudinal cohort study, children aged 8-18 years diagnosed with SCD at the Amsterdam UMC were included between 2012 and 2021. HRQoL was annually measured as part of standard care using the Pediatric Quality of Life Inventory. The impact of hospitalization for VOC on HRQoL was evaluated using linear mixed models 3, 6, 9, and 12 months after hospitalization. The effect of frequency of hospitalization for VOC on HRQoL was evaluated over the last 12 months. RESULTS: In total, 94 children with SCD were included with a median age of 11.8 years (interquartile range [IQR]: 9-14). Thirty-seven patients (39%) had been hospitalized for a VOC. Hospitalization for VOC led to a decrease of 3.2-4.8 points in total HRQoL compared to patients without hospitalization, most pronounced 3 months after hospitalization. Recurrent admission for VOC in the last 12 months was associated with a decrease of 2.3 points in total HRQoL (p = .04). The most affected subscale was physical functioning. CONCLUSION: The adverse effects of hospitalization for VOC in children with SCD persist up to 12 months after hospitalization. After hospitalization for VOC, extra attention and support for its negative impact on HRQoL are recommended. This study also underlines the importance of systematically measuring HRQoL, allowing clinicians to intervene accordingly.

Understanding the impact of tuberous sclerosis complex: development and validation of the TSC-PROM.

BACKGROUND: Tuberous sclerosis complex (TSC) is a rare and complex genetic disorder, associated with tumor growth in various organ systems, epilepsy, and a range of neuropsychiatric manifestations including intellectual disability. With improving patient-centered care and targeted therapies, patient-reported outcome measures (PROMs) are needed to measure the impact of TSC manifestations on daily functioning. The aim of this study was to develop a TSC-specific PROM for adults that captures the impact of TSC on physical functions, mental functions, activity and participation, and the social support individuals with TSC receive, called the TSC-PROM. METHODS: COSMIN methodology was used to develop a self-reported and proxy-reported version. Development and validation consisted of the following studies: PROM development, content validity, structural validity, internal consistency, and construct validity. The International Classification of Functioning and Disability was used as a framework. Content validity was examined by a multidisciplinary expert group and cognitive interview study. Structural and construct validity, and internal consistency were examined in a large cohort, using confirmatory factor analysis, hypotheses testing, and Cronbach's alpha. RESULTS: The study resulted in an 82-item self version and 75-item proxy version of the TSC-PROM with four subscales (physical functions 18 and 19 items, mental functions 37 and 28 items, activities and participation 13 and 14 items, social support 13 items, for self version and proxy version respectively). Sufficient results were found for structural validity with sufficient unidimensionality for each subscale. With regard to construct validity, 82% of the hypotheses were met for the self version and 59% for the proxy version. The PROM showed good internal consistency (Cronbach's alpha 0.78-0.97). CONCLUSIONS: We developed a PROM for adults with TSC, named TSC-PROM, showing sufficient evidence for reliability and validity that can be used in clinical and research settings to systematically gain insight into their experiences. It is the first PROM in TSC that addresses the impact of specific TSC manifestations on functioning, providing a valuable, patient-centered addition to the current clinical outcomes.

Pediatric patient engagement in clinical care, research and intervention development: a scoping review.

BACKGROUND: In the last decades, pediatric patient engagement has received growing attention and its importance is increasingly acknowledged. Pediatric patient engagement in health care can be defined as the involvement of children and adolescents in the decision-making of daily clinical care, research and intervention development. Although more attention is paid to pediatric patient engagement, a comprehensive overview of the activities that have been done regarding pediatric patient engagement and the changes over time is lacking. Therefore, the aim of this study is to provide an overview of the literature about pediatric patient engagement. METHODS: The methodological framework of Arksey & O'Malley was used to conduct this scoping review. The bibliographic databases Medline, Embase, and PsycINFO were searched for eligible articles. All retrieved articles were screened by at least two researchers in two steps. Articles were included if they focused on pediatric patient engagement, were carried out in the context of clinical care in pediatrics, and were published as full text original article in English or Dutch. Data (year of publication, country in which the study was conducted, disease group of the participants, setting of pediatric patient engagement, used methods, and age of participants) were extracted, synthesized, and tabulated. RESULTS: A total of 288 articles out of the 10,714 initial hits met the inclusion criteria. Over the years, there has been an increase in the number of studies that engage pediatric patients. Pediatric patients, especially patients with multiple conditions or oncology patients, were most involved in studies in the United States, United Kingdom, and Canada. Pediatric patients were most often asked to express their views on questions from daily clinical care and the individual interview was the most used method. In general, the extent to which pediatric patients are engaged in health care increases with age. DISCUSSION: This scoping review shows that there is an increasing interest in pediatric patient engagement. However, lack of uniformity about the definition of pediatric patient engagement and clear information for clinicians hinders engagement. This overview can inform clinicians and researchers about the different ways in which pediatric patient engagement can be shaped and can guide them to engage pediatric patients meaningfully in their projects.

Physical, Mental, and Social Health of Adult Patients with Sickle Cell Disease after Allogeneic Hematopoietic Stem Cell Transplantation: A Mixed-Methods Study.

Patients with sickle cell disease (SCD) experience a considerable physical and psychosocial disease burden. In recent years, the application of allogeneic hematopoietic stem cell transplantation (HSCT) to treat adults with SCD has increased. A thorough understanding of patients' physical, mental, and social health before and after cure is needed to meet the needs of this growing group of patients. We aimed to explore the perspectives of adult SCD patients on the changes in their experienced health and personal life goals after being cured. A mixed-methods approach was used, comprising a semistructured interview and a set of 9 Patient Reported Outcomes Measurement Information System (PROMIS) measures. Adult SCD patients who underwent HSCT at least 1 year earlier were eligible to participate in the study. Interviews were thematically analyzed using MAXQDA software. PROMIS T scores were compared with reference scores of the general population using SPSS Statistics. Ten patients participated in the study; their median age was 29.5 years (range, 19 to 49 years), and their median time since HSCT was 2.7 years (range, 1.0 to 3.5 years). Themes from the interviews were (1) pain/living pain free, (2) physical well-being, (3) mental well-being, (4) perspective/ outlook, (5) education/work, (6) family/friends, and (7) activities/participation. Following the PROMIS framework, we described these themes in a narrative synthesis according to health domain and categorized in 4 chronological time phases: before HSCT, first year post-transplantation, current situation, and future expectations. Physical health improved greatly, but transplantation-related toxicity, ongoing pain from avascular osteonecrosis, and fatigue negatively impacted quality of life in some patients. Furthermore, emotional struggles during the post-transplantation period were common, and patients expressed a need for psychological help. Patients reported improvements in social health and the ability to pursue personal life goals. The mean T scores of all PROMIS measures fell within the normal symptom limits compared with reference data of the general population, although, large variations were observed among the participants, matching our qualitative findings. In general, adult SCD patients experienced improved physical, mental, and social health after cure by HSCT and were able to pursue personal life goals. Yet they found themselves confronted with a new and unfamiliar reality that brought different challenges. Pain due to irreversible avascular osteonecrosis continued to have a negative impact. Clinicians should aim to help patients have realistic expectations before transplantation and offer timely psychological care.

Late Effects in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation for Nonmalignant Diseases: Proxy- and Patient-Reported Outcomes.

Survival rates in pediatric hematopoietic stem cell transplantation (HSCT) for nonmalignant diseases have improved due to advances in conditioning regimens, donor selection, and prophylaxis and treatment of infections and graft-versus-host disease. Insight into the long-term patient-reported outcomes (PROs) after pediatric HSCT for nonmalignant disease is lacking but essential for optimal shared decision making, counseling, and quality of care. The purpose of this research was to determine long-term patient-reported outcomes in allogeneic pediatric HSCT for nonmalignant diseases and to compare these results with Dutch reference data. This single-center cohort study evaluated PROs (PedsQL 4.0, PROMIS item banks), self- or proxy-reported, among patients at ≥2 years after pediatric allogeneic HSCT for nonmalignant disease. Mean scores were compared with those of the Dutch general population. Of 171 eligible patients, 119 participated, for a 70% response rate. The median patient age was 15.8 years (range, 2 to 49 years), and the median duration of follow-up was 8.7 years (range, 2 to 34 years). Indications for HSCT included inborn errors of immunity (n = 41), hemoglobinopathies (n = 37), and bone marrow failure (n = 41). Compared with reference data, significantly lower scores were found in adolescents (age 13 to 17 years) on the Total, Physical Health, and School Functioning PedsQL subscales. Significantly more Sleep Disturbance was reported in children (age 8 to 18 years). On the other hand, significantly better scores were seen on PROMIS Fatigue (age 5 to 7 years) and Pain Interference (age 8 to 18 years) and, in adults (age 19 to 30 years), on Depressive Symptoms and Sleep Disturbance. This study showed better or comparable very long-term PROs in patients after pediatric HSCT for nonmalignant diseases compared with the reference population. Children and adolescents seem to be the most affected, indicating the need for supportive care to prevent impaired quality of life and, more importantly, to amplify their long-term well-being.

Age-specific and family-centered information modalities to prepare children at home for day-care surgery.

BACKGROUND: Surgery induced stress and anxiety in children and parents can be reduced by providing preoperative information adapted to their needs. Aim of this study was to evaluate the effects of three different information modalities (coloring page, mobile application and videos) to prepare children and their parents for day-care surgery on preoperative anxiety and postoperative pain experienced by the child, and preoperative anxiety and satisfaction of parents. METHODS: Prospective observational study including children and their parents that were offered specifically developed information modalities to prepare for day-care surgery. Results were compared between children and their parents that used none (i.e., control group) versus one or more information modalities (i.e., intervention group). Primary outcomes were preoperative anxiety measured using PROMIS v2.0 Anxiety and postoperative pain in children. Secondary outcomes were preoperative parental anxiety (STAI questionnaire) and family satisfaction with information and communication (modified PedsQL Healthcare Satisfaction questionnaire). Subgroup analyses were performed between preschoolers (0-5 years) and school-aged (≥5) children. RESULTS: 93 patients (male 53%) were included in the intervention (n=56) and control group (n=37). Levels of children's preoperative anxiety and postoperative pain, and parental anxiety did not differ between both groups. Families of prepared children were more satisfied with information and communication about preoperative surgical information (8 vs. 6.6, p=0.004) and satisfaction with how parents (7 vs 8, p=0.019) and children (8 vs 6, p=0.018) were prepared for surgery. CONCLUSIONS: Preoperative anxiety did not differ between prepared and unprepared children. The use of specifically developed family-centered and age-appropriate information modalities to prepare children for day-care surgery at home results in superior family satisfaction. LEVEL OF EVIDENCE: III.

Views of patients and parents of children with genetic disorders on population-based expanded carrier screening.

OBJECTIVE: Faster and cheaper next generation sequencing technologies have enabled expansion of carrier screening for recessive disorders, potentially facilitating population-based implementation regardless of ancestry or family history. Little is known, however, about the attitudes regarding population-based carrier screening among families with genetic disorders. This study assessed views among parents and patients with a recessive disorder and parents of children with Down syndrome (DS) on expanded carrier screening (ECS). METHOD: In total, 85 patients with various recessive disorders, 110 parents of a child with a recessive disorder and 89 parents of a child with DS participated in an online survey in the Netherlands. Severity of recessive disorders was classified as mild/moderate or severe/profound. RESULTS: The majority of the (parents of) patients with a recessive disorder had a positive attitude towards population-based ECS, including screening for their own or their child's disorder. DS parents were significantly less positive towards ECS. Subgroup analyses showed that the severity of the disorder, rather than being a patient or parent, influences the attitudes, beliefs and intention to participate in ECS. CONCLUSION: Our findings have important implications for future implementation initiatives as they demonstrate the different perspectives from people with experiential knowledge with genetic disorders.

Including the voice of paediatric patients: Cocreation of an engagement game.

BACKGROUND: Engaging patients in health care, research and policy is essential to improving patient-important health outcomes and the quality of care. Although the importance of patient engagement is increasingly acknowledged, clinicians and researchers still find it difficult to engage patients, especially paediatric patients. To facilitate the engagement of children and adolescents in health care, the aim of this project is to develop an engagement game. METHODS: A user-centred design was used to develop a patient engagement game in three steps: (1) identification of important themes for adolescents regarding their illness, treatment and hospital care, (2) evaluation of the draft version of the game and (3) testing usability in clinical practice. Adolescents (12-18 years) were engaged in all steps of the development process through focus groups, interviews or a workshop. These were audio-recorded, transcribed verbatim and analysed in MAXQDA. RESULTS: (1) The important themes for adolescents (N = 15) were included: visiting the hospital, participating, disease and treatment, social environment, feelings, dealing with staff, acceptation, autonomy, disclosure and chronically ill peers. (2) Then, based on these themes, the engagement game was developed and the draft version was evaluated by 13 adolescents. Based on their feedback, changes were made to the game (e.g., adjusting the images and changing the game rules). (3) Regarding usability, the pilot version was evaluated positively. The game helped adolescents to give their opinion. Based on the feedback of adolescents, some last adjustments (e.g., changing colours and adding a game board) were made, which led to the final version of the game, All Voices Count. CONCLUSIONS: Working together with adolescents, All Voices Count, a patient engagement game was developed. This game provides clinicians with a tool that supports shared decision-making to address adolescents' wishes and needs. PATIENT OR PUBLIC CONTRIBUTION: Paediatric patients, clinicians, researchers, youth panel of Fonds NutsOhra and patient associations (Patient Alliance for Rare and Genetic Diseases, Dutch Childhood Cancer Organization) were involved in all phases of the development of the patient engagement game-from writing the project plan to the final version of the game.

One-Stop Surgery: An Innovation to Limit Hospital Visits in Children.

INTRODUCTION: One-stop surgery (OSS) allows for same-day outpatient clinic visit, preoperative assessment, and surgical repair. This study aims to determine the efficiency, (cost-)effectiveness, and family satisfaction of one-stop inguinal hernia surgery compared with usual care. MATERIAL AND METHODS: Children (≥ 3 months) with inguinal hernia and American Society of Anesthesiologists (ASA) grades I-II, scheduled for OSS (intervention) or regular treatment (control) between March 1, 2017, and December 1, 2018, were eligible for inclusion. Exclusion criteria consisted of age less than 3 months and ASA grades III-IV. The primary outcome measure was treatment efficiency (i.e., total number of hospital visits and waiting time [days] between referral and surgery). Secondary outcome measures were the effectiveness in terms of complication and recurrence rate, and parent-reported satisfaction and cost-effectiveness using the Dutch Pediatric Quality of Life Healthcare Satisfaction and Institute for Medical Technology Assessment Productivity Cost Questionnaire. RESULTS: Ninety-one (intervention: 54; control: 37) patients (56% boys) were included. Median (interquartile range) number of hospital visits was lower in the intervention group (1 vs 3; p < 0.001). All but one of the OSS patients (98%) were discharged home on the day of surgery. Postoperative complication (1.9% vs 2.7%; p = 0.787) and recurrence rates (0% vs 2.7%; p = 0.407) did not differ between the intervention and control patients. "General satisfaction," "satisfaction with communication," and "inclusion of family" were higher after OSS, while satisfaction about "information," "technical skills," and "emotional needs" were similar. Median (range) follow-up was 28 (15-36) months. CONCLUSIONS: Pediatric one-stop inguinal hernia repair seems to be an effective treatment strategy that limits the number of hospital visits and provides enhanced family satisfaction without compromising the quality of care.

Health-related quality of life in infants, toddlers and young children with sickle cell disease.

BACKGROUND: Little is known about health-related quality of life (HRQoL) in young children with sickle cell disease living in a European country. METHODS: A retrospective cross-sectional evaluation of TNO-AZL Preschool Children Quality of Life questionnaire (TAPQOL, 0-1 year) and Pediatric Quality of Life Inventory (PedsQL, 2-7 years) data was conducted. Study participants included caregivers of children with sickle cell disease aged 0-7 years attending the sickle cell centre at the Erasmus Medical Center or the Amsterdam University Medical Centers between April 2012 and October 2020. Comparisons were made with normative data on HRQoL in the general paediatric population. RESULTS: The study enrolled 136 caregivers of 136 children. In children aged 0-5 years, no significant differences emerged between children with sickle cell disease and the general population. However, in children aged 5-7 years, children with sickle cell disease scored significantly lower on all subscales except for emotional functioning. Multiple regression models showed a negative association between age and HRQoL. No association was found between HRQoL and disease severity or sociodemographic characteristics. CONCLUSIONS: This study demonstrates that HRQoL is negatively correlated with age in young children with sickle cell disease with a significantly lower HRQoL in 5- to 7-year-olds when compared to the general population. Our study underlines the importance of measuring HRQoL in young children to identify patients with impaired HRQoL early in life in order to be able to intervene accordingly. Future research should focus on deepening the knowledge of factors influencing HRQoL in children with sickle cell disease.

Preconception expanded carrier screening: Impact of information presented by text or video on genetic knowledge and attitudes.

Preconception expanded carrier screening (ECS) aims to identify couples with an increased risk of having a child with an autosomal recessive (AR) disorder before pregnancy, thereby enabling reproductive choices. Genetic knowledge and experiential knowledge both influence the uptake of ECS. As people in the general public often lack such knowledge, it is essential to provide appropriate and understandable information when offering ECS. This study investigated the effect of an educational video, compared to an educational text, on the knowledge and attitudes toward preconception ECS in the general population. Both the text and video consisted of a brief educational summary on AR inheritance and on the type of disorders included in ECS, with the progressive neurodegenerative condition mucopolysaccharidosis type III (MPS III) as an example. Participants in the reproductive age were invited in collaboration with a research agency. Respondents (N = 789) were offered an educational video prior to completing an online questionnaire that examined genetic knowledge, the perceived severity of MPS III, perceived risk, and attitudes toward ECS. Outcomes were compared to reference data collected previously in which respondents had been offered an educational text (N = 781). We first again studied the attitudes toward ECS in a smaller educational text group (N = 266) in order to assess whether attitudes had changed over time due to increased media coverage on ECS, which did not reveal any significant changes. Respondents who were offered the video had a better genetic knowledge, perceived MPS III as more severe, perceived their risks higher and were more likely to participate in ECS compared to those who were offered text. Online video may well be used as supportive tool to the genetic counseling process, creating more knowledge on ECS and severe genetic disorders included in preconception screening panels.

Using an implementation science approach to implement and evaluate patient-reported outcome measures (PROM) initiatives in routine care settings.

PURPOSE: Patient-reported outcome and experience measures (PROMs/PREMs) are well established in research for many health conditions, but barriers persist for implementing them in routine care. Implementation science (IS) offers a potential way forward, but its application has been limited for PROMs/PREMs. METHODS: We compare similarities and differences for widely used IS frameworks and their applicability for implementing PROMs/PREMs through case studies. Three case studies implemented PROMs: (1) pain clinics in Canada; (2) oncology clinics in Australia; and (3) pediatric/adult clinics for chronic conditions in the Netherlands. The fourth case study is planning PREMs implementation in Canadian primary care clinics. We compare case studies on barriers, enablers, implementation strategies, and evaluation. RESULTS: Case studies used IS frameworks to systematize barriers, to develop implementation strategies for clinics, and to evaluate implementation effectiveness. Across case studies, consistent PROM/PREM implementation barriers were technology, uncertainty about how or why to use PROMs/PREMs, and competing demands from established clinical workflows. Enabling factors in clinics were context specific. Implementation support strategies changed during pre-implementation, implementation, and post-implementation stages. Evaluation approaches were inconsistent across case studies, and thus, we present example evaluation metrics specific to PROMs/PREMs. CONCLUSION: Multilevel IS frameworks are necessary for PROM/PREM implementation given the complexity. In cross-study comparisons, barriers to PROM/PREM implementation were consistent across patient populations and care settings, but enablers were context specific, suggesting the need for tailored implementation strategies based on clinic resources. Theoretically guided studies are needed to clarify how, why, and in what circumstances IS principles lead to successful PROM/PREM integration and sustainability.

Parental distress 6 months after a pediatric cancer diagnosis in relation to family psychosocial risk at diagnosis.

BACKGROUND: This study was aimed at assessing fathers' and mothers' distress 6 months after a pediatric cancer diagnosis and at determining whether this is related to the level of family psychosocial risk 1 month after the diagnosis. METHODS: A sample of 192 families completed the electronic Psychosocial Assessment Tool (ePAT) 1 month after the diagnosis. At 6 months after the diagnosis, 119 mothers and 98 fathers completed the Distress Thermometer for Parents (DT-P; of which n=132 had also completed the ePAT at baseline). The DT-P consists of a thermometer score ranging from 0 to 10 (with a score ≥ 4 indicating clinical distress), problem domains (total, practical, social, emotional, physical, cognitive, and parenting for children < 2 years old and for children ≥ 2 years old), and a desire for a referral. The DT-P scores of mothers and fathers were compared with the scores of a reference group of 671 mothers and 463 fathers with healthy children. Within the pediatric cancer group, the DT-P scores of families with elevated total ePAT-scores were compared with the DT-P scores of parents with universal ePAT scores. RESULTS: Parents of children with cancer more often reported clinical distress on the DT-P than parents of healthy children (fathers, 59.2% vs 32.3%; P < .001; mothers, 63% vs 42.3%; P < .001) and reported more problems on all DT-P domains (P < .001 to P = .042) except for the parenting domain for children < 2 years old. Furthermore, the ePAT predicted parental distress 6 months after the diagnosis because parents with elevated ePAT scores reported more problems than parents with universal scores on the DT-P thermometer and most of the DT-P domains (P < .001 to P = 1.00). CONCLUSIONS: Initial ePAT risk scores at diagnosis are predictive of future mean levels of parental distress. Cancer 2018;124:381-90. © 2017 American Cancer Society.

Distress and everyday problems in Dutch mothers and fathers of young adolescents with Down syndrome.

BACKGROUND: To provide targeted support to parents of children with DS, knowledge of their distress and everyday problems is crucial. For this purpose, psychosocial screening instruments can be a valuable addition to routine clinical practice. AIMS: To determine differences on a psychosocial screener concerning distress and everyday problems in parents of young adolescents (YAs) with DS versus control parents and in mothers of YAs with DS versus fathers. METHODS AND PROCEDURES: We compared outcomes of the Distress Thermometer for Parents in 76 mothers and 44 fathers of 11-13-year-olds with DS versus 64 mothers and 52 fathers of age-matched children without DS (comparing mothers and fathers separately). Additionally, we compared mothers and fathers within 34 parent couples of YAs with DS. OUTCOMES AND RESULTS: Clinical distress was not more frequent than in control parents. Mothers further did not report more everyday problems and only differed from their controls on one problem domain and some problem items. Fathers, however, reported more problems than their controls across most domains and wished to talk to a professional about their situation more frequently. Outcomes in mothers and fathers within parent couples did not differ significantly. CONCLUSIONS AND IMPLICATIONS: This is one of few studies to report on the use of psychosocial screening instruments in parents of children with DS. Our results suggested that attention for fathers of YAs with DS is required. Psychosocial screening instruments that inquire about specific problems and the wish for referral can play an important role in achieving this.

Real-world implementation of electronic patient-reported outcomes in outpatient pediatric cancer care.

OBJECTIVE: The KLIK method is an online tool that monitors and discusses electronic patient-reported outcomes (ePROs), which has been shown to enhance outcomes. This study aimed (1) to determine the fidelity (ie, extent to which used as intended) of the KLIK method as implemented in outpatient pediatric cancer care and (2) to study health care professional (HCP)-reported barriers and facilitators for implementation. METHODS: Two hundred five children with newly diagnosed cancer (enrollment rate 85%) participated. At 1 (T1), 3 (T2), and 6 (T3) months after diagnosis, patients (8-18 years) or parents (of patients 0-7 years) completed health-related quality of life (HRQoL) questionnaires, which were transformed into an ePROfile and discussed by their HCP during consultations. Fidelity was determined by the following: percentage of website registrations, HRQoL questionnaires completed, and ePROfiles discussed. Implementation determinants were assessed with HCPs after the final T3 with the Measurement Instrument for Determinants of Innovations. RESULTS: Depending on the time point (T1-T3), fidelity was 86% to 89% for website registration, 66-85% for completed HRQoL questionnaires, and 56% to 62% for ePROfile discussion. Barriers were mainly related to organizational issues (eg, organizational change) and less frequently to users (eg, motivation to comply) or the intervention (compatibility). Facilitators were related to the user (eg, positive outcome expectations) and intervention (simplicity) but not to the organization. CONCLUSIONS: When implementing ePROs in outpatient pediatric oncology practice, HCPs report determinants that influence ePRO integration. To improve implementation and outcomes, tailored organizational (eg, formal ratification by management and time) and specific local (eg, individualized assessments) strategies should be developed to achieve optimal ePRO discussion.

Healthcare Professionals' Preferences and Perceived Barriers for Routine Assessment of Patient-Reported Outcomes in Pediatric Oncology Practice: Moving Toward International Processes of Change.

BACKGROUND: Using patient-reported outcomes (PROs) in clinical practice has been shown to enhance detection of health-related quality of life problems and satisfaction with care in children with cancer. This study seeks to identify which PRO information healthcare professionals (HCPs) find useful and what the perceived barriers for routinely assessing PROs are. PROCEDURE: A total of 352 pediatric HCPs (43% male) from 52 countries completed a semistructured online 28-item questionnaire. Descriptive statistics (percentages) were used to identify highly important PRO information and perceived barriers. HCPs' perceived barriers were compared according to gender, years of work experience, and country using a Fishers exact test. RESULTS: The five highest ranked PRO topics relevant in routine assessment by HCPs were as follows: pain (98%), feeling sad or depressed (96%), overall physical symptoms (95%), problems with therapy adherence (94%), and overall emotional issues (93%). Five lowest ranked topics were as follows: difficulties praying (50%), other spiritual concerns (55, 56, and 60%), and feeling bored (60%). Barriers for assessing PROs included: time (58%), insufficient staff (49%), logistics (32%), and financial resources (26%). Providers from developing countries more often reported barriers concerning insufficient staff, logistics, and financial resources. CONCLUSIONS: HCPs strongly value the use of physical and psychosocial PROs within pediatric oncology practice, but mainly perceive organizational barriers for routine assessment. To successfully integrate PROs, efforts should be made to address HCP-perceived barriers, such that patient-reported problems can be detected and timely referrals made.

Training clinicians in how to use patient-reported outcome measures in routine clinical practice.

INTRODUCTION: Patient-reported outcome measures (PROs) were originally developed for comparing groups of people in clinical trials and population studies, and the results were used to support treatment recommendations or inform health policy, but there was not direct benefit for the participants providing PROs data. However, as the experience in using those measures increased, it became obvious the clinical value in using individual patient PROs profiles in daily practice to identify/monitor symptoms, evaluate treatment outcomes and support shared decision-making. A key issue limiting successful implementation is clinicians' lack of knowledge on how to effectively utilize PROs data in their clinical encounters. METHODS: Using a change management theoretical framework, this paper describes the development and implementation of three programs for training clinicians to effectively use PRO data in routine practice. The training programs are in three diverse clinical areas (adult oncology, lung transplant and paediatrics), in three countries with different healthcare systems, thus providing a rare opportunity to pull out common approaches whilst recognizing specific settings. For each program, we describe the clinical and organizational setting, the program planning and development, the content of the training session with supporting material, subsequent monitoring of PROs use and evidence of adoption. The common successful components and practical steps are identified, leading to discussion and future recommendations. RESULTS: The results of the three training programs are described as the implementation. In the oncology program, PRO data have been developed and are currently evaluated; in the lung transplant program, PRO data are used in daily practice and the integration with electronic patient records is under development; and in the paediatric program, PRO data are fully implemented with around 7,600 consultations since the start of the implementation. CONCLUSION: Adult learning programs teaching clinicians how to use and act on PROs in clinical practice are a key steps in supporting patient engagement and participation in shared decision-making. Researchers and clinicians from different clinical areas should collaborate to share ideas, develop guidelines and promote good practice in patient-centred care.

Development and validation of the distress thermometer for parents of a chronically ill child.

OBJECTIVE: To develop and validate a Distress Thermometer for Parents (DT-P) for chronically ill children and to determine a cutoff score for clinical distress. STUDY DESIGN: Parents of a chronically ill child (0-18 years) were recruited via announcements or were actively approached at the outpatient clinics of the Emma Children's Hospital/Academic Medical Center and Vrije Universiteit Medical Center. We modeled the development of the DT-P on the Distress Thermometer used in oncology medical care. The DT-P consists of a thermometer score from 0 (no distress) to 10 (extreme distress) and a problem list (practical, social, emotional, physical, cognitive, and parenting domains). The DT-P was validated with the Hospital Anxiety and Depression Scale (HADS) and the Parenting Stress Index. RESULTS: The mean thermometer score of the 706 participating parents was 3.7 (SD 3.0). The thermometer score and the scores in the practical, emotional, physical, and cognitive problem domains were strongly related to anxiety, depression, and the total score of the HADS (0.55 ≤ r ≤ 0.72). The thermometer score and all problem domain scores were moderately-to-strongly related to the Parenting Stress Index (0.38 ≤ r ≤ 0.63). A cutoff-score of 4 correctly identified 86% of "clinical HADS cases" (sensitivity) and 67% of "nonclinical HADS cases" (specificity). CONCLUSIONS: We developed the DT-P and examined its diagnostic utility in a large sample. The DT-P appeared to be a valid and useful short screening-tool for identifying parental distress.

Development and implementation of a patient reported outcome intervention (QLIC-ON PROfile) in clinical paediatric oncology practice.

OBJECTIVE: The use of patient reported outcomes (PRO) in routine clinical practice is becoming increasingly common, but there is limited knowledge about the development and implementation of PRO. The objective of the current paper is to provide a thorough description of the development and implementation of a PRO on health related quality of life (HRQOL)--the QLIC-ON PROfile--in clinical paediatric oncology practice. METHODS: The development of the QLIC-ON PROfile is explained by elucidating important choices: the HRQOL instrument, the professional that uses the QLIC-ON PROfile, the optimal form of HRQOL feedback and whether or not a clinically important difference is reported. The description of the implementation of the QLIC-ON PROfile focuses on the education and commitment of the professional that uses the QLIC-ON PROfile. Study design and outcome measures are also elaborated on. RESULTS: Important considerations regarding the development and implementation of PRO interventions are reported. These considerations have also resulted in educational material. CONCLUSION: Our study adds to current knowledge of PRO research. This paper can be used as a practical guide for researchers and other professionals, who are interested in setting up PRO interventions in any clinical setting.