Effectiveness of nurse-home visiting in improving child and maternal outcomes prenatally to age two years: a randomised controlled trial (British Columbia Healthy Connections Project).

BACKGROUND: We investigated the effectiveness of Nurse-Family Partnership (NFP), a prenatal-to-age-two-years home-visiting programme, in British Columbia (BC), Canada. METHODS: For this randomised controlled trial, we recruited participants from 26 public health settings who were: <25 years, nulliparous, <28 weeks gestation and experiencing socioeconomic disadvantage. We randomly allocated participants (one-to-one; computer-generated) to intervention (NFP plus existing services) or comparison (existing services) groups. Prespecified outcomes were prenatal substance exposure (reported previously); child injuries (primary), language, cognition and mental health (problem behaviour) by age two years; and subsequent pregnancies by 24 months postpartum. Research interviewers were masked. We used intention-to-treat analyses. (ClinicalTrials.gov, NCT01672060.) RESULTS: From 2013 to 2016 we enrolled 739 participants (368 NFP, 371 comparison) who had 737 children. Counts for child injury healthcare encounters [rate per 1,000 person-years or RPY] were similar for NFP (223 [RPY 316.17]) and comparison (223 [RPY 305.43]; rate difference 10.74, 95% CI -46.96, 68.44; rate ratio 1.03, 95% CI 0.78, 1.38). Maternal-reported language scores (mean, M [SD]) were statistically significantly higher for NFP (313.46 [195.96]) than comparison (282.77 [188.15]; mean difference [MD] 31.33, 95% CI 0.96, 61.71). Maternal-reported problem-behaviour scores (M [SD]) were statistically significantly lower for NFP (52.18 [9.19]) than comparison (54.42 [9.02]; MD -2.19, 95% CI -3.62, -0.75). Subsequent pregnancy counts were similar (NFP 115 [RPY 230.69] and comparison 117 [RPY 227.29]; rate difference 3.40, 95% CI -55.54, 62.34; hazard ratio 1.01, 95% CI 0.79, 1.29). We observed no unanticipated adverse events. CONCLUSIONS: NFP did not reduce child injuries or subsequent maternal pregnancies but did improve maternal-reported child language and mental health (problem behaviour) at age two years. Follow-up of long-term outcomes is warranted given that further benefits may emerge across childhood and adolescence.

Development of EPAT: An assessment tool for pediatric hematology/oncology training programs.

PURPOSE: In the absence of a standardized tool to assess the quality of pediatric hematology/oncology training programs, the Education Program Assessment Tool (EPAT) was conceptualized as a user-friendly and adaptable tool to evaluate and identify areas of opportunity, pinpoint needed modifications, and monitor progress for training programs around the world. METHODS: The development of EPAT consisted of three main phases: operationalization, consensus, and piloting. After each phase, the tool was iteratively modified based on feedback to improve its relevance, usability, and clarity. RESULTS: The operationalization process led to the development of 10 domains with associated assessment questions. The two-step consensus phase included an internal consensus phase to validate the domains and a subsequent external consensus phase to refine the domains and overall function of the tool. EPAT domains for programmatic evaluation are hospital infrastructure, patient care, education infrastructure, program basics, clinical exposure, theory, research, evaluation, educational culture, and graduate impact. EPAT was piloted in five training programs in five countries, representing diverse medical training and patient care contexts for proper validation of the tool. Face validity was confirmed by a correlation between the perceived and calculated scores for each domain (r = 0.78, p < .0001). CONCLUSIONS: EPAT was developed following a systematic approach, ultimately leading to a relevant tool to evaluate the different core elements of pediatric hematology/oncology training programs across the world. With EPAT, programs will have a tool to quantitatively evaluate their training, allowing for benchmarking with centers at the local, regional, and international level.

Translation and cultural adaptation of the Health Utilities Preschool to Brazilian Portuguese.

INTRODUCTION: Health research is particularly important in low- and middle-income countries (LMICs), where improvements must be achieved with limited resources, and where the great majority of the world's population, especially children, live. Improvements in public health detection in Brazil have resulted in cancer becoming the most prevalent cause of death by disease in the group aged 1 to 19 years, hence, delivering cost-effective care to the group is a priority. Preference-based measures of health status and health-related quality of life (HRQL) integrate morbidity and mortality and provide utility scores for the estimation of quality-adjusted life years to be used in cost-effectiveness analyses and economic evaluation. The generic preference-based instrument Health Utilities - Preschool (HuPS) measures the health status of young children and is applicable to the age group 2 to 5 years, who carry the highest incidence of cancer in childhood. METHODS: The translation of the HuPS classification system followed recommended protocols from published guidelines. Forward and backward translations were performed by a team of six qualified professionals and linguistic validation was undertaken with a sample of parents of preschool children. MAIN RESULTS: Initial disagreements on individual words occurring in 0.5-1.5% were resolved by consensus. A final version of the instrument was validated by the sample of parents. CONCLUSIONS: The translation and cultural adaptation of the HuPS into Brazilian Portuguese were accomplished as the first step in the validation of the HuPS instrument in Brazil.

Addition of arm anthropometry to body mass index for age, but not serum albumin, improves the accuracy of the nutritional assessment in severely and moderately malnourished children with cancer.

Arm anthropometry is a more sensitive measure of nutritional status than body mass index for age (BMI) in children with cancer, but the added utility of serum albumin remains uncertain. Concordance was determined among four forms of classifying nutritional status in a cohort of undernourished children with cancer: method 1: BMI-for-age Z score; method 2: method 1 + mid-upper arm circumference (MUAC) percentile; method 3: method 2 + triceps skinfold thickness (TSFT) percentile; and method 4: method 3 + serum albumin. Concordance was highest between methods 2 and 3, followed closely by 3 and 4, indicating that addition of arm anthropometry, but not serum albumin, to BMI increased the sensitivity of baseline nutritional assessment.

Parent-reported health status of preterm survivors in a Canadian cohort.

OBJECTIVES: Health status (HS)/ health-related quality of life measures, completed by self or proxy, are important outcome indicators. Most HS literature on children born preterm includes adolescents and adults with limited data at preschool age. This study aimed to describe parent-reported HS in a large national cohort of extreme preterm children at preschool age and to identify clinical and sociodemographic variables associated with HS. METHODS: Infants born before 29 weeks' gestation between 2009 and 2011 were enrolled in a prospective longitudinal national cohort study through the Canadian Neonatal Network (CNN) and the Canadian Neonatal Follow-Up Network (CNFUN). HS, at 36 months' corrected age (CA), was measured with the Health Status Classification System for Pre-School Children tool completed by parents. Information about HS predictors was extracted from the CNN and CNFUN databases. RESULTS: Of 811 children included, there were 79, 309 and 423 participants in 23-24, 25-26 and 27-28 weeks' gestational age groups, respectively. At 36 months' CA, 78% had a parent-reported health concern, mild in >50% and severe in 7%. Most affected HS attributes were speech (52.1%) and self-care (41.4%). Independent predictors of HS included substance use during pregnancy, infant male sex, Score for Neonatal Acute Physiology-II, bronchopulmonary dysplasia, severe retinopathy of prematurity, caregiver employment and single caregiver. CONCLUSION: Most parents expressed no or mild health concerns for their children at 36 months' CA. Factors associated with health concerns included initial severity of illness, complications of prematurity and social factors.

Reference centile curves for mid-upper arm circumference for assessment of under- and overnutrition in school-aged Indian children and adolescents.

OBJECTIVES: Malnutrition is common in developing countries and is not restricted to young children. It has been suggested that measuring mid-upper arm circumference (MUAC) is an easy, accurate, and low-cost method of identifying malnutrition in the early stages. The aims of this study were to construct age- and sex-specific MUAC reference centiles, and to define and validate cutoffs for assessment of under- and overnutrition in Indian children 5 to 17 y of age. METHODS: This was a cross-sectional, multicentric, observational study conducted in seven schools in seven states from June 2018 to November 2019. The study included 6680 healthy 5- to17-y-old children. MUAC was measured using non-stretch tapes (UNICEF). Sex-specific MUAC percentiles were computed for age and height. Cutoffs for MUAC z-scores for thinness and overnutrition were defined and validated for healthy school children (n = 726) and children with cancer (n = 500). RESULTS: Reference centiles for MUAC for age (and height) for boys and girls are presented. Cutoffs defined for thinness and for obesity were -0.7 and +1.5 z-score, respectively (corresponding to 25th and 95th percentiles of the MUAC for age/height). For ease of use, rounded cutoffs for thinness were 16 and 18.5 cm from 5 to 9 and 10 to 14 y of age, respectively, in both sexes, and a cutoff of 22 cm in boys and 20 cm in girl from 15 to 17 y of age. For obesity, 20 and 25.5 cm from 5 to 9 and 10 to 14 y of age, respectively, in both girls and boys and a rounded cutoff of 29 cm in boys and 27 cm in girls from 15 to17 y are proposed. CONCLUSIONS: We presented MUAC percentiles and cutoffs for screening for thinness and overnutrition in Indian children from 5 to 17 y of age. These data may also be used in children with cancer and other chronic disorders with growth failure.

Bone health in long-term survivors of pediatric acute lymphoblastic leukemia. An assessment by peripheral quantitative computed tomography.

BACKGROUND: Loss of bone mineral is a common concomitant of the treatment of acute lymphoblastic leukemia (ALL) due mainly to chemotherapy, especially with corticosteroids. Osteopenia/osteoporosis may be encountered long into survivorship. Measurement of bone mineral density (BMD) by dual-energy X-ray absorptiometry is limited to two-dimensionality and cannot distinguish trabecular from cortical bone. METHODS: A sample of 74 subjects, ages 13.5-38.3 years more than 10 years from diagnosis, underwent peripheral quantitative computed tomography (pQCT) at metaphyseal (trabecular bone) and diaphyseal (cortical bone) sites in the radius and tibia. pQCT provides three-dimensional assessment of bone geometry, density, and architecture. RESULTS: Average values in multiple metrics were similar to those in healthy individuals, but deficits in both trabecular and cortical bones were revealed by lower Z scores using an ethnically comparable sample of healthy individuals. Connectivity, a measure of bone architecture and a surrogate measure of bone strength, was lower in females than males. Survivors of standard-risk ALL had greater connectivity in and more compact trabecular bone than high-risk survivors who had received more intensive osteotoxic chemotherapy. There were no statistically significant differences in any of the metrics at any of the sites between subjects who had or had not a history of fracture, cranial irradiation, or use of a bisphosphonate. CONCLUSIONS: These long-term survivors of ALL have somewhat compromised bone health, but data in comparable healthy populations are limited. Longitudinal studies in larger and more ethnically diverse cohorts will provide greater insight into bone health in this vulnerable population.

Shortages and price variability of essential cytotoxic medicines for treating children with cancers.

INTRODUCTION: Low-income and middle-income countries (LMICs) face the largest burden of mortality from childhood cancers with limited access to curative therapies. Few comparative analyses across all income groups and world regions have examined the availability and acquisition costs of essential medicines for treating cancers in children. METHODS: A cross-sectional survey involved countries in five income groups-low-income (LIC), lower-middle-income (LMC), upper-middle-income (UMC), two high-income country groups (HIC1, HIC2). Physicians and pharmacists reported institutional use, availability, stock outs and prices (brand and generic products) of 34 essential medicines. Price comparisons used US$, applying foreign exchange rates (XR) and purchasing power parity (PPP) adjustments. Medicine costs for treating acute lymphoblastic leukaemia (ALL), Burkitt lymphoma (BL) and Wilms tumour (WT) were calculated (child 29 kg, body surface area 1 m2). Comparisons were conducted using non-parametric Kruskal-Wallis tests. RESULTS: Fifty-eight respondents (50 countries) provided information on medicine use, availability and stock outs, with usable price data from 42 facilities (37 countries). The extent of use of International Society of Paediatric Oncology core and ancillary medicines varied across income groups (p<0.0001 and p=0.0002 respectively). LMC and LIC facilities used fewer medicines than UMC and HIC facilities. UMC and LMC facilities were more likely to report medicines not available or stockouts.Medicine prices varied widely within and between income bands; generic products were not always cheaper than brand equivalents. PPP adjustment showed relatively higher prices in UMC and LMC facilities for some medicines. Medicine costs were highest in HICs for ALL (p=0.0075 XR; p=0.0178 PPP-adjusted analyses) and WT (p =<0.0001 XR; p=0.0007 PPP-adjusted). Medicine costs for BL were not significantly different. CONCLUSION: Problems with the availability of essential medicines, dependable supply chains, confidential medicine prices and wide variability in treatment costs contribute to persistent challenges in the care of children with treatable cancers, especially in LMICs.

Sustainable care for children with cancer: a Lancet Oncology Commission.

We estimate that there will be 13·7 million new cases of childhood cancer globally between 2020 and 2050. At current levels of health system performance (including access and referral), 6·1 million (44·9%) of these children will be undiagnosed. Between 2020 and 2050, 11·1 million children will die from cancer if no additional investments are made to improve access to health-care services or childhood cancer treatment. Of this total, 9·3 million children (84·1%) will be in low-income and lower-middle-income countries. This burden could be vastly reduced with new funding to scale up cost-effective interventions. Simultaneous comprehensive scale-up of interventions could avert 6·2 million deaths in children with cancer in this period, more than half (56·1%) of the total number of deaths otherwise projected. Taking excess mortality risk into consideration, this reduction in the number of deaths is projected to produce a gain of 318 million life-years. In addition, the global lifetime productivity gains of US$2580 billion in 2020-50 would be four times greater than the cumulative treatment costs of $594 billion, producing a net benefit of $1986 billion on the global investment: a net return of $3 for every $1 invested. In sum, the burden of childhood cancer, which has been grossly underestimated in the past, can be effectively diminished to realise massive health and economic benefits and to avert millions of needless deaths.

Development of System Performance Indicators for Adolescent and Young Adult Cancer Care and Control in Canada.

OBJECTIVES: To develop an expert-group, consensus-based list of system performance indicators to be used for monitoring, evaluating, and benchmarking progress for cancer care and control in adolescents and young adults (AYAs) in Canada. METHODS: A national multidisciplinary panel of AYA oncology experts was convened; they prepared a literature review and undertook a brainstorming exercise to create a comprehensive list of indicators based on a previously defined framework for AYA cancer care and control in Canada. A modified Delphi process was then undertaken to cull the list based on 3 quick screen criteria. Three rounds of ranking were required. The fourth stage employed a face-to-face meeting, and the final stage utilized a survey to rank the indicators on the basis of importance and feasibility. RESULTS: Nineteen participants contributed to the 5-stage process. From an initial list of 114 indicators, 14 were ultimately endorsed, representing 5 themes: active care, survivorship, psychosocial issues, palliative care, and research. The 5 highest ranked indicators were assessed as very to moderately feasible, with only a single indicator (clinical trial enrollment) in the top 5 assigned a least feasible ranking. CONCLUSION: The 14 indicators provide a starting point for the development of a standard set of metrics for AYA cancer care and control in Canada and have potential for international utility.

Shifting Priorities for the Survival of My Child: Managing Expenses, Increasing Debt, and Tapping Into Available Resources to Maintain the Financial Stability of the Family.

BACKGROUND: Parents are confronted with a range of direct costs and intense caregiving demands following their child's cancer diagnosis, which may potentially threaten the financial stability of the family. OBJECTIVE: The aims of this study were to explore the financial impact of a new childhood cancer diagnosis on families and understand the strategies families use to manage these financial impacts. METHODS: As part of the mixed-methods Childhood Cancer Cost Study, a descriptive qualitative design was conducted. Parents discussed costs and their impact in a semistructured, audio-taped interview. A qualitative content analysis was used to analyze the transcribed data. RESULTS: Seventy-eight parents participated. Parents used several strategies to maintain financial stability. These strategies consisted of managing expenses, which entailed reducing living expenses and cutting unexpected cancer costs. Efforts to absorb these expenses required families to increase their debt while seeking ways to tap into available resources, including relying on their savings and leveraging their benefits and assets, increasing their paid work hours, relying on their support networks, and seeking help from philanthropy and government agencies for financial help. CONCLUSION: Parents used several strategies to manage the increased out-of-pocket expenses and reduced household income. IMPLICATIONS FOR PRACTICE: Our findings of the financial impact of cancer costs on families provide insight into needed practice and policy changes aimed at lessening the economic impact of a childhood cancer diagnosis on the family and allow healthcare professionals and researchers to pursue more in-depth cost assessments in the future.

Health-related quality of life in adolescents and young adults with cancer - Including a focus on economic evaluation.

Health-related quality of life (HRQL) is an amalgam of three elements - the opportunities that a person's health status affords, the constraints that it places upon the person and the value that a person places on his/her health status. HRQL measures are specific, for example for a disease, or generic with broad applicability. The latter include preference-based measures that can be used to generate quality-adjusted life years and so contribute to economic evaluation. Measures of HRQL in adolescents and young adults (AYAs) with cancer may fail to capture some important dimensions, for example sexual health. However, the use of HRQL measures in this population has identified burdens of morbidity according to disease, treatment status and duration of follow-up. There are few economic evaluations of the treatment of cancer in AYAs but preliminary evidence suggests that this is a cost-effective undertaking. Opportunities abound to include measurement of HRQL in routine clinical care.

Understanding, measuring, and addressing the financial impact of cancer on adolescents and young adults.

The financial impact of cancer treatment among adolescents and young adults (AYAs, 15-39 years) is deep and long lasting. Compared with other age groups, because of their life stage, AYAs are particularly vulnerable to the adverse economic effects of cancer treatment, also known as financial toxicity. Clinical manifestations of cancer-related financial toxicity include interrupted work and income loss, accumulated debt, treatment nonadherence, avoidance of medical care, and social isolation. Effective clinical interventions should include efforts to increase financial self-efficacy as well as direct support. Measures that are valid, reliable, multidimensional, and age-appropriate are needed to study and address financial toxicity in the AYA population.

Nutritional status at diagnosis of cancer in children and adolescents in Guatemala and its relationship to socioeconomic disadvantage: A retrospective cohort study.

BACKGROUND: At least 80% of children with cancer live in low- and middle-income countries where the prevalence of malnutrition and socioeconomic disadvantage is high. We examined the relationship between nutritional status (NS), assessed by arm anthropometry, and socioeconomic status (SES) in children diagnosed with cancer at Unidad Nacional de Oncologia Pediatrica (UNOP) in Guatemala over a three-year period. METHOD: Patients aged 0 to 18 years of age diagnosed between January 2015 and December 2017 were included. NS was evaluated by mid-upper arm circumference, triceps skin fold thickness, and serum albumin level, and subjects were classified as adequately nourished, moderately depleted, and severely depleted nutritionally. SES was measured by a 15-item instrument developed at UNOP. RESULTS: Of 1365 patients diagnosed in the study period, 1060 (78%) fulfilled the eligibility criteria. Only 6% of patients were classified as medium to high, the remainder as medium-low to extremely low SES. Almost 47% were severely depleted at diagnosis, 19% moderately depleted, and 34% adequately nourished. SES was shown to be a determinant of NS; with progressively lower SES, the probability of a decline in NS increased by a factor of 1.04 points (P < 0.0001). Leukemia and lymphoma were also important predictors of nutritional depletion with odds ratios of 6.08 (95% CI, 1.74-28.28; P = 0.008) for leukemias and 4.83 (95% CI, 1.33-23.03; P = 0.03) for lymphomas. CONCLUSION: Both low SES and a diagnosis of leukemia or lymphoma are strong predictors of poor NS at diagnosis in children with cancer in Guatemala.

Models of Care-There Is More Than One Way to Deliver.

Adolescents and young adults (AYAs) with cancer constitute a particular group of patients with unique features, whose needs during and after treatment are poorly met. A standardized model of care for them has yet to be established, as neither the pediatric nor the adult oncology systems seamlessly fit their needs. Regardless of the setting in which they are treated, their health care providers should be aware of the impact that the disease and its treatments have on these especially vulnerable patients. Simple ways of improving the AYA experience should be considered: reducing isolation through connections with peers, adapting the staff's approach to the emotional and developmental needs of this age group, and modifying the hospital environment making it more age appropriate. Commitment of national governments is valuable; building and sharing international experience will accelerate advances in clinical care, education, and research. Further progress in the care of AYA cancer patients is still needed to improve their outcomes.

The Impact of a Childhood Cancer Diagnosis on the Children and Siblings' School Attendance, Performance, and Activities: A Qualitative Descriptive Study.

Families of children with cancer are confronted with unexpected out-of-pocket expenses and productivity costs associated with the diagnosis. One productivity cost that falls on children is the impact of cancer on children's school attendance, performance, and activities (eg, play, friendships, and socialization). Nested within the Childhood Cancer Cost Study, this qualitative descriptive study used convenience sampling to recruit and interview parents of children newly diagnosed with cancer. Content analysis techniques were used to inductively descriptive the semistructured interview data. Sixty-six parents of 65 children with cancer and of 73 siblings participated. The most commonly reported productivity loss in children with cancer was school absenteeism mainly due to cancer treatment. Children fell behind their classmates academically and lost important social time with peers. A few siblings also fell behind their peers primarily due to limited parental attention. Parents adopted various strategies to lessen the impact of the diagnosis on their children's school attendance, performance, and activities. Providing parents with additional resources and support may optimize their children's academic and social reintegration into school.

Lifetime Cost of Abusive Head Trauma at Ages 0-4, USA.

This paper aims to estimate lifetime costs resulting from abusive head trauma (AHT) in the USA and the break-even effectiveness for prevention. A mathematical model incorporated data from Vital Statistics, the Healthcare Cost and Utilization Project Kids' Inpatient Database, and previous studies. Unit costs were derived from published sources. From society's perspective, discounted lifetime cost of an AHT averages $5.7 million (95% CI $3.2-9.2 million) for a death. It averages $2.6 million (95% CI $1.0-2.9 million) for a surviving AHT victim including $224,500 for medical care and related direct costs (2010 USD). The estimated 4824 incident AHT cases in 2010 had an estimated lifetime cost of $13.5 billion (95% CI $5.5-16.2 billion) including $257 million for medical care, $552 million for special education, $322 million for child protective services/criminal justice, $2.0 billion for lost work, and $10.3 billion for lost quality of life. Government sources paid an estimated $1.3 billion. Out-of-pocket benefits of existing prevention programming would exceed its costs if it prevents 2% of cases. When a child survives AHT, providers and caregivers can anticipate a lifetime of potentially costly and life-threatening care needs. Better effectiveness estimates are needed for both broad prevention messaging and intensive prevention targeting high-risk caregivers.

Global assessment of cancer incidence and survival in adolescents and young adults.

In high-income countries, cancer remains the commonest cause of disease-related death in adolescents and young adults (AYAs) despite survival improvements. With more than 1,000,000 new diagnoses of cancer in AYAs annually worldwide, and their number of life-years affected by cancer being greatest of all ages, the global burden of cancer in AYAs exceeds that in all other ages. In low- and middle-income countries, where the great majority of the world's 3 billion AYAs reside, the needs of those with cancer have been identified and demand attention. Unique to the age group but universal, the psychosocial challenges they face are the utmost across life's spectrum. This lead-off article of a new series in Pediatric Blood and Cancer on AYA oncology attempts to assess the global status of this emerging discipline. The review includes the changing incidence and survival of the common cancers in AYAs-there is no other age group with a similar array of malignancies-and the specific challenges to quality and quantity of life that compromise their lives.