Understanding and Utilizing the Unmet Needs of Teenagers and Young Adults with Cancer to Determine Priorities for Service Development: The Macmillan On Target Programme.

Purpose: A systematic attempt to identify and address unmet needs among patients in a large regional teenagers and young adults (TYA) cancer service in the United Kingdom, including perspectives obtained from patients themselves, their families/supporters, and professionals. Methods: Questionnaires, focus groups, and interviews were undertaken with the following: patients (diagnosed ≥16 years, and aged 16-24 years at the time of study)-participation was 42 for questionnaire, 7 for focus group, and 6 for interview; family members/others in patients' lives ("networkers") (participation: 28, 0, and 4); and professionals (participation: 54, 0, and 97). Requirement management methodology was used to specify components for potential service interventions, which were then scored and prioritized. Co-creation was utilized to incorporate a deeper understanding of patient experience. Results: 42/108(39%) patients, 28/177(24%) networkers, and 122/322(38%) professionals participated. For patients, seven themes that "mattered most" (identified by >60% responders) were defined. For many, support was provided both to a lesser extent than needed and was sometimes unsatisfactory. For networkers, results identified the significant support offered by those around the patient and the impact on their own lives. For professionals, consensus was reached on interventions that could be utilized in clinical encounters with TYA to enhance care. A list of prioritized "requirements" was created to drive future service improvement. Conclusions: Areas identified for development included three specific initiatives applicable to other TYA services: a support website (www.tyahelp.co.uk); an electronic, age/developmentally specific, holistic needs assessment tool (the Integrated Assessment Map www.tyaiam.co.uk); and a portal linking use of the IAM to resources within the Help website (video illustration available at: https://vimeo.com/191019826).

Conservative strategy in infantile fibrosarcoma is possible: The European paediatric Soft tissue sarcoma Study Group experience.

BACKGROUND: Infantile fibrosarcoma (IFS) is a very rare disease occurring in young infants characterised by a high local aggressiveness but overall with a favourable survival. To try to reduce the total burden of therapy, the European pediatric Soft tissue sarcoma Study Group has developed conservative therapeutic recommendations according to initial resectability. MATERIAL AND METHODS: Between 2005 and 2012, children with localised IFS were prospectively registered. Initial surgery was suggested only if possible without mutilation. Patients with initial complete (IRS-group I/R0) or microscopic incomplete (group II/R1) resection had no further therapy. Patients with initial inoperable tumour (group III/R2) received first-line vincristine-actinomycin-D chemotherapy (VA). Delayed conservative surgery was planned after tumour reduction. Aggressive local therapy (mutilating surgery or external radiotherapy) was discouraged. RESULTS: A total of 50 infants (median age 1.4 months), were included in the study. ETV6-NTRK3 transcript was present in 87.2% of patients where investigation was performed. According to initial surgery, 11 patients were classified as group I, 8 as group II and 31 as group III. VA chemotherapy was first delivered to 25 children with IRS-III/R2 and one with IRS-II/R1 disease. Response rate to VA was 68.0%. Mutilating surgery was only performed in three cases. After a median follow-up of 4.7 years (range 1.9-9.0), 3-year event-free survival and overall survival were respectively 84.0% (95% confidence interval [CI] 70.5-91.7) and 94.0% (95% CI 82.5-98.0). CONCLUSIONS: Conservative therapy is possible in IFS as only three children required mutilating surgery, and alkylating or anthracycline based chemotherapy was avoided in 71.0% of patients needing chemotherapy. VA regimen should be first line therapy in order to reduce long term effects.

Recruitment of childhood leukaemia patients to clinical trials in Great Britain during 1980-2007: variation by birth weight, congenital malformation, socioeconomic status and ethnicity.

OBJECTIVE: To assess recruitment of children to national clinical trials for acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML) in Great Britain during 1980-2007 and describe variation by some factors that might influence trial entry. DESIGN AND SETTING: Records of leukaemia patients aged 0-14 years at diagnosis were identified in the National Registry of Childhood Tumours and linked to birth registrations, Children's Cancer and Leukaemia Group records, Hospital Episode Statistics and Medical Research Council clinical trial registers. Trial entry rates were compared between categories of birth weight, congenital malformation, socioeconomic status and ethnicity. RESULTS: 9147 ALL and 1466 AML patients were eligible for national clinical trials during 1980-2007. Overall recruitment rates were 81% and 60% respectively. For ALL, rates varied significantly with congenital malformation (Down syndrome 61%, other malformations 80%, none 82%; p<0.001) and ethnicity (South Asian 78%, other minority groups 80%, white 85%; p<0.001). For AML, rates varied with birth weight (< 2500 g 48%, 2500-4000 g 69%, >4000 g 67%; p=0.001) and congenital malformation (Down syndrome 28%, other malformations 56%, none 63%; p<0.001). CONCLUSIONS: Although recruitment rates to clinical trials for childhood leukaemia are high, future trials should monitor possible variation by birth weight, ethnicity and presence of congenital malformations.

Risk of childhood cancer with symptoms in primary care: a population-based case-control study.

BACKGROUND: Guidelines describing symptoms in children that should alert GPs to consider cancer have been developed, but without any supporting primary-care research. AIM: To identify symptoms and signs in primary care that strongly increase the likelihood of childhood cancer, to assist GPs in selection of children for investigation. DESIGN AND SETTING: A population-based case-control study in UK general practice. METHOD: Using electronic primary care records from the UK General Practice Research Database, 1267 children aged 0-14 years diagnosed with childhood cancer were matched to 15 318 controls. Clinical features associated with subsequent diagnosis of cancer were identified using conditional logistic regression, and likelihood ratios and positive predictive values (PPVs) were estimated for each. RESULTS: Twelve symptoms were associated with PPVs of ≥0.04%, which represents a greater than tenfold increase in prior probability. The six symptoms with the highest PPVs were pallor (odds ratio, OR = 84; PPV = 0.41% (95% confidence interval [CI] = 0.12% to 1.34%), head and neck masses (OR = 17; PPV = 0.30%; 95% CI = 0.10% to 0.84%), masses elsewhere (OR = 22; PPV = 0.11%; 95% CI = 0.06% to 0.20%), lymphadenopathy (OR = 10; PPV = 0.09%; 95% CI = 0.06% to 0.13%), symptoms/signs of abnormal movement (OR = 16; PPV = 0.08%; 95% CI = 0.04% to 0.14%), and bruising (OR = 12; PPV = 0·08%; 95% CI = 0.05% to 0.13%). When each of these 12 symptoms was combined singly with at least three consultations in a 3-month period, the probability of cancer was between 11 and 76 in 10 000. CONCLUSION: Twelve features of childhood cancers were identified, each of which increased the risk of cancer at least tenfold. These symptoms, particularly when combined with multiple consultations, warrant careful evaluation in general practice.

A detailed prospective longitudinal assessment of health status in children with brain tumors in the first year after diagnosis.

PURPOSE: To compare health status (HS) in children with brain tumors at 1 (t1), 6 (t6), and 12 (t12) months after diagnosis with "normal" controls. To assess the relationship between parent-report and self-report HS for patients at t12. METHODS: HS was assessed using the Health Utilities Index Mark III parent-report at all time points and self-report at t12. Twenty-nine patients and 32 controls were included in analysis of parent-report, and 21 patients and 22 controls in self-report HS at t12. Nonparametric analyses were used. RESULTS: Patients scored significantly lower than controls for global overall HS at all time points for parent-report and at t12 for self-report (Pmax=0.009). For parent-report, patients scored significantly lower than controls in the attributes of emotion, cognition, and pain at t1 and t6, in ambulation at t1 and in dexterity at t6. At t12, the difference was statistically significant for parent-report cognition only (all P<0.01). No attributes reached significance for self-report at t12. For patients, correlations between parent-report and self-report were good (rs>0.73) for all Health Utilities Index Mark 3 scores with the exception of emotion and pain. CONCLUSION: HS is significantly compromised in children with brain tumors over the first year after diagnosis, but improves with time. Parent-report and self-report differ, and both should be considered in assessing outcomes or defining interventions.

Treatment for childhood rhabdomyosarcoma: the cost of cure.

Survival of children and young people with rhabdomyosarcoma has improved substantially during the past 30 years. The improvement can be attributed to the introduction of coordinated multimodality therapy through the efforts of collaborative clinical-trial groups in the USA and Europe. As survival has improved, important late sequelae of treatment have been characterised, and many relate to the local therapy used. Efforts have since been made by some groups to explore ways in which local treatment, particularly radiotherapy, can be omitted for some patients in an attempt to reduce the risk of late sequelae without compromising prospects for cure. An important issue is the overall cost of cure in relation to the treatment received and the need for better selection of patients most likely to benefit from this approach to therapy.